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366 Study Matches

A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of the Safety and Pharmacodynamic Activity of Gene Therapy for Congenital Adrenal Hyperplasia through Administration of an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

This is a Phase 1/2, first-in-human, open-label, dose-escalation study designed to evaluate the safety, tolerability, and efficacy of BBP-631 administered to up to 25 adult participants diagnosed with classic congenital adrenal hyperplasia (CAH) (simple virilizing or salt-wasting, Group 1) or with classic salt-wasting CAH (Group 2) due to 21-hydroxylase deficiency (21-OHD) and who are monitored for 24 weeks posttreatment.

Kyriakie Sarafoglou
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04783181
STUDY00012144
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Key Inclusion Criteria
• Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
• Screening/baseline 17-OHP levels > 5-10 × ULN and < 40 × ULN (upper limit of normal)
• Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
• Naïve to prior gene therapy or AAV-mediated therapy Key Exclusion Criteria
• Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
• History of adrenalectomy and has no significant liver disease
Biological: AAV BBP-631
Congenital Adrenal Hyperplasia
CAH, Gene therapy, AAV, AAV5
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MT2020-21: A PHASE 1,OPEN-LABEL,DOSE ESCALATING STUDY EVALUATING THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND CLINICAL ACTIVITY OF THE COMBINATION OF CLBR001, AN ENGINEERED AUTOLOGOUS T-CELL PRODUCT, AND SWI019,AN ANTIBODY-BASED BIOLOGIC, IN PATIENTS WITH RELAPSED/REFRACTORY B-CELL MALIGNANCIES

This study is designed to assess the safety and tolerability of the combination of CLBR001 and SWI019 in patients with B cell malignancies who are refractory or unresponsive to salvage therapy or who cannot be considered for or have progressed after autologous hematopoietic cell transplantation.

Joseph Maakaron
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT04450069
STUDY00011917
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Inclusion Criteria:

• Patients with relapsed / refractory previously treated B cell malignancies (according to the World Health Organization classification; 2017)
• Patients must have received adequate prior therapy including at least two lines of prior therapies including anthracycline or bendamustine-containing chemotherapy, anti-CD20 (cluster of differentiation antigen 20) therapies and/or Brutton's tyrosine kinase (BTK) inhibitors
• Patients treated with prior CD19 targeted molecules (e.g., Blincyto) must have confirmed CD19+ disease
• Patients must be ineligible for allogeneic stem cell transplant (SCT)
• Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0 or 1
• Estimated life expectancy of ≥ 12 weeks from the first day of SWI019 dose administered
• Willing to undergo pre- and post-treatment core needle biopsy
• Adequate hematological, renal, pulmonary, cardiac, and liver function
• Resolved adverse events of any prior therapy to either baseline or CTCAE Grade ≤1
• Women of childbearing potential, a negative pregnancy test and must agree to practice effective birth control
• Men sexually active with female partners of child bearing potential must agree to practice effective contraception
• Willing and able to comply with scheduled visits, treatment plan, laboratory tests and other procedures
Exclusion Criteria:

• Patients diagnosed with certain disease histologies including pediatric lymphomas/leukemias, monoclonal gammopathy of undetermined significance (MGUS), T-cell histiocyte large B cell lymphoma
• Pregnant or lactating women
• Active bacterial, viral, and fungal infections
• History of allogeneic stem cell transplantation
• Treatment with any prior lentiviral or retroviral based CAR-T
• Patients receiving live (attenuated) vaccines within 4 weeks of screening visit or need for live vaccine on study
• Patients with known active central nervous system (CNS) disease. Patients with prior CNS disease that has been effectively treated may be eligible
• History of Class III or IV New York Heart Association (NYHA) heart failure, myocardial infarction, unstable angina or other significant cardiac disease within 6 months of screening
• Involvement of cardiac tissue by lymphoma
• Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP)
• HIV-1 and HIV-2 antibody positive patients
Combination Product: CLBR001 and SWI019
Relapsed/Refractory B-cell Lymphomas, Diffuse Large B Cell Lymphoma (DLBCL), Follicular Lymphoma (FL), Chronic Lymphocytic Leukemia (CLL), Marginal Zone Lymphoma (MZL), Mantle Cell Lymphoma, Small Lymphocytic Lymphoma (SLL), Primary Mediastinal Large B Cell Lymphoma, Transformed Follicular Lymphoma, Waldenstrom Macroglobulinemia, Lymphoplasmacytic Lymphoma, Burkitt Lymphoma, Cancer
Clinics and Surgery Center (CSC), CAR-T Cell Therapy, Switchable CAR-T Cell, Autologous Cell Therapy, CD19 Positive Disease, CD19 CAR-T Cell, Blood Cancer, Hematological malignancy, Neoplasms
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Phase 1/2 First-in-Human (FIH) Study of Leukocyte Immunoglobulin-Like Receptor B2 (LILRB2) Inhibitor Monoclonal Antibody (mAb) JTX-8064, as Monotherapy and in Combination with a Programmed Cell Death Receptor-1 (PD-1) Inhibitor, in Adult Subjects with Advanced Refractory Solid Tumor Malignancies

The purpose of this study is to find out: •What dose of JTX-8064 is safe and tolerable either when given by itself or in combination with JTX-4014 or pembrolizumab. •What effects, both good and bad, JTX-8064 may have when given alone or in combination with JTX-4014 or pembrolizumab. •How much JTX-8064 gets into the bloodstream and how long it takes for the body to get rid of it. •Whether the body generates an immune response against JTX-8064 that could possibly have a negative effect on its ability to work.

Deanna Teoh
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04669899
STUDY00013057
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• Able and willing to participate and comply with all study requirements and provide signed and dated informed consent prior to initiation of any study procedures;
• Histologically or cytologically confirmed advanced/metastatic extracranial solid tumor malignancy.
• Stages 1 and 2: Subject must have received, have been intolerant to, have been ineligible for, or have declined all treatment known to confer clinical benefit with the exception of subjects enrolled in combination cohorts with a PD-1i, where a PD-1i is approved by the local regulatory agencies
• Stage 3: This stage may enroll subjects with the following cancers: • 3L/4L PD-(L)1-naïve, platinum-resistant ovarian cancer
• Stage 4: This stage may enroll subjects with the following cancers:
• 2L/3L ccRCC. Subjects must have progressed on or after treatment with an anti-PD-(L)1 agent in their most recent prior line of therapy
• 2L-4L TNBC. Subjects must have progressed on or after treatment with a prior anti-PD-(L)1 therapy
• 1L, PD-(L)1-naïve, PD-L1+; combined positive score (CPS) ≥ 1% HNSCC
• 3L/4L, PD-(L)1-naïve, platinum-resistant ovarian cancer
• 2L/3L NSCLC; Subjects must have progressed on or after treatment with platinum-based chemotherapy and an anti-PD-(L)1-containing therapy. The anti-PD-(L)1 agent must have been a part of the most recent prior line of therapy. Subjects with EGFR mutations and ALK rearrangements will be excluded. Subjects with other targetable genomic aberrations for which FDA approved therapies exist must have received appropriate FDA-approved targeted therapy
• 2L/3L cSCC; Subjects must have progressed on or after treatment with an anti-PD-(L)1 agent in their most recent prior line of therapy
• 2L-4L PD-(L)1-naïve UPS and LPS
• 2L/3L HNSCC. Subjects must have progressed on or after treatment with platinum-based chemotherapy and an anti-PD-(L)1-containing therapy. The anti-PD-(L)1 agent must have been a part of the most recent prior line of therapy.
• 2/3L biliary tract cancer (BTC), including intra-and extra-hepatic biliary duct cancer and cancer of the gallbladder. Subjects must have progressed on or after gemcitabine/cisplatin (Gem/Cis) and an anti-PD-(L)1 therapy in the metastatic setting. Must have PD-(L)1 inhibitor resistance. Subjects with FGFR and IDH1 mutations must have progressed on or after targeted therapies for these mutations.
• Measurable disease, according to the RECIST version 1.1, that has objectively progressed since (or on) previous treatment as assessed by the Investigator;
• ≥ 18 years of age;
• Eastern Cooperative Oncology Group performance status 0 or 1;
• Predicted life expectancy of ≥ 3 months;
• Have specified laboratory values (obtained ≤ 28 days prior to first dose) in accordance with the study protocol;
• For women of childbearing potential (WOCBP): negative serum pregnancy test during the Screening period and a negative urine pregnancy test up to 24 hours in advance of C1D1
• WOCBP and males whose partners are WOCBP must agree to use a highly effective method of birth control throughout their participation and for 5 months following the last study drug administration.
Exclusion Criteria:

• Concurrent anticancer treatment, either FDA approved or investigational, for the cancer being evaluated in this study or for prior malignancies. A past history of other malignancies is allowed as long as the subject is not receiving treatment other than hormonal therapy and, in the judgment of the Investigator, is unlikely to have a recurrence. Of note, concurrent malignancies that do not require treatment and are clinically stable are allowed
• Prior infusion of JTX-8064, LILRB2, or ILT4-directed therapy;
• The therapies listed below within the specified timeframe:
• Immunotherapy or biologic therapy < 28 days prior to planned C1D1 or 5 half-lives, whichever is shorter
• Chemotherapy < 21 days prior to planned C1D1, or < 42 days for mitomycin or nitrosoureas or 5 half-lives, whichever is shorter
• Targeted small molecule therapy < 14 days or 5 half-lives, whichever is shorter, prior to planned C1D1
• Radiation therapy < 21 days prior to planned C1D1. Exception: Limited (e.g., pain palliation) radiation therapy is allowed prior to and during study drug administration as long as there are no acute toxicities, any AE due to prior radiation therapy has recovered to < Grade 2, and the radiation is not administered to a target lesion
• Symptomatic or uncontrolled brain metastases, leptomeningeal disease, or spinal cord compression not definitively treated with surgery or radiation (brain metastases that are stable and asymptomatic after prior treatment will be allowed);
• Women who are pregnant or breastfeeding or who plan to become pregnant/breastfeed while on study; men who plan to father children during the study
• Live vaccines ≤ 30 days of C1D1
Drug: JTX-8064, Drug: pimivalimab
Cancer
JTX-8064, LILRB2, ILT-4, PD-1, JTX-4014, Immunotherapy, Immuno-oncology, Solid tumor Malignancies, Dose escalation, INNATE, Monotherapy, Combination Therapy, Antineoplastic Agents, Immunological, Clinics and Surgery Center (CSC), Phase I Clinic
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MT2019-09: A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCR alpha beta +/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) PIDTC CSIDE Protocol (CSIDE)

To determine the incidence of humoral immune reconstitution by 2 years post-transplant in 2 SCID cohorts (IL2RG/JAK3, RAG1/RAG2) undergoing alternative donor HCT by randomized assignment to a busulfan preparative regimen targeted at cumulative area-under-the-curve (cAUC) exposure of 25-35 mg*h/L vs 55-65 mg*h/ L.

Christen Ebens
All
up to 2 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03619551
STUDY00006513
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Inclusion Criteria:

• Infants with SCID, either typical or leaky or Omenn syndrome.
• Typical SCID is defined as either of the following
• Absence or very low number of T cells (CD3+ T cells <300/microliter AND no or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin OR
• Presence of maternally derived T cells
• Leaky SCID is defined as the following • Absence of maternally derived T cells • AND either one or both of the following (i, ii): i) <50% of lower limit of normal T cell function as measured by response to PHA OR <30% of lower limit of normal T cell function as measured by response to CD3 ii) Absent or <10% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens (must document post vaccination or exposure for this criterion to apply) • AND at least two of the following (i through iii): i) CD3 T cells < 1500/microliter ii) >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of age) AND/OR are oligoclonal T iii) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower level of normal.
• Omenn syndrome • Generalized skin rash
• Maternal lymphocytes tested for and not detected.
• >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (<2 years of age)
• Absent or low (up to 30% lower limit of normal (LLN)) T cell proliferation to antigens (Candida, tetanus) to which the patient has been exposed IF: Proliferation to antigen was not performed, but at least 4 of the following 8 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the patient is eligible as Omenn Syndrome.
• Hepatomegaly
• Splenomegaly
• Lymphadenopathy
• Elevated IgE
• Elevated absolute eosinophil count
• *Oligoclonal T cells measured by CDR3 length or flow cytometry (upload report)
• *Proliferation to PHA is reduced to < 50% of lower limit of normal (LLN) or SI < 30
• *Low TRECs and/or percentage of CD4+/RA+ CD31+ or CD4+/RA+ CD62L+ cells below the lower level of normal
• Documented mutation in one of the following SCID-related genes a. Cytokine receptor defects (IL2RG, JAK3) b. T cell receptor rearrangement defects (RAG1, RAG2) 3. No available genotypically matched related donor (sibling) 4. Availability of a suitable donor and graft source
• Haploidentical related mobilized peripheral blood cells
• 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor mobilized peripheral blood cells 5. Age 0 to 2 years at enrollment Note: to ensure appropriate hepatic metabolism, age at time of busulfan start: For IL2RG/JAK3: 8 weeks For RAG1/RAG2: 12 weeks
• Adequate organ function defined as:
• Cardiac: Left ventricular ejection fraction (LVEF) at rest ≥ 40% or, shortening fraction (SF) ≥ 26% by echocardiogram.
• Hepatic: Total bilirubin < 3.0 x the upper limit of normal (ULN) for age (patients who have been diagnosed with Gilbert's Disease are allowed to exceed this limit) and AST and ALT < 5.0 x ULN for age.
• Renal: GFR estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2.
• Pulmonary No need for supplemental oxygen and O2 saturation > 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care).
Exclusion Criteria:

• Presence of any serious life-threatening or opportunistic infection at time of enrollment and prior to the initiation of the preparative regimen. Serious infections as defined below that occur after enrollment must be reported immediately to the Study Coordinating Center, and enrollment will be put on hold until the infection resolves. Ideally enrolled subjects will not have had any infection. If patients have experienced infections, these must have resolved by the following definitions: a. Bacterial i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site must be negative and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). ii. Tissue-based clinical infection (e.g. cellulitis): Complete resolution of clinical signs (e.g. erythema, tenderness, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). iii. Pneumonia, organism not identified by bronchoalveolar lavage: Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). If possible, radiographic resolution should also be demonstrated. b. Fungal i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site is negative and patient has completed appropriate course of antifungal therapy (typically at least 14 days). The patient may be continued on antifungal prophylaxis following completion of the treatment course. c. Pneumocystis i. Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of therapy (typically at least 21 days). If possible, radiographic resolution should also be demonstrated. The patient may be continued on prophylaxis following completion of the treatment course. d. Viral i. Viral PCRs from previously documented sites (blood, nasopharynx, CSF) must be re-tested and are negative. ii. If re-sampling a site is not clinically feasible (i.e. BAL fluid): Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.). If possible, radiographic resolution should also be demonstrated.
• Patients with HIV or HTLV I/II infection will be excluded.
Drug: Busulfan, Device: Cell processing for TCRαβ+/CD19+ depletion
SCID
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A Phase 2/3, Two-Part, Open-Label, Dose Escalation, Age De-escalation and Randomized, Observer-Blind, Placebo-Controlled Expansion Study to Evaluate the Safety, Tolerability, Reactogenicity, and Effectiveness of mRNA-1273 SARS-CoV-2 Vaccine in Healthy Children 6 months to < 12 Years of Age (mRNA-1273-P204) - COVID-19

The Sponsor of this study, ModernaTX, is studying the mRNA-1273 vaccine for the prevention of COVID-19 in children. This study is being conducted to learn about the safety, any side effects, and how your child’s body responds to the study vaccine (the “immune response”).

Shane McAllister
All
6 Months to 11 Years old
Phase 2/Phase 3
This study is also accepting healthy volunteers
NCT04796896
STUDY00012613
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Key
Inclusion Criteria:

• For participants with chronic diseases (such as, asthma, diabetes mellitus, cystic fibrosis, human immunodeficiency virus [HIV] infection), the disease should be stable, per investigator assessment.
• Investigator assessment that the parent(s)/legally acceptable representatives understand and are willing and physically able to comply with protocol mandated follow-up, including all procedures, written informed consent is provided, and participants provide assent.
• For children 2 years of age or older has a body mass index at or above the third percentile according to World Health Organization (WHO) Child Growth Standards at the Screening Visit.
• For children 6 months to <12 months of age: born at full-term with a minimum birth weight of 2.5 kilograms (kg).
• For female participants of childbearing potential: negative pregnancy test, adequate contraception or has abstained from all activities that could result in pregnancy for at least 28 days prior to the first injection, agreement to continue adequate contraception or abstinence through 3 months following the second injection (Day 29) and the third dose in Part 3 (Day 149/booster dose Day 1), and not currently breastfeeding. Key
Exclusion Criteria:

• Known history of SARS-CoV-2 infection within 2 weeks prior to administration of vaccine or known close contact with anyone with laboratory-confirmed SARS-CoV-2 infection or COVID-19 within 2 weeks prior to administration of vaccine.
• Prior administration of an investigational or approved CoV (such as, SARS-CoV-2, SARS CoV, Middle East Respiratory Syndrome CoV) vaccine.
• Treatment with investigational or approved agents for prophylaxis against COVID 19 (such as, receipt of SARS-CoV-2 monoclonal antibodies) within 6 months prior to enrollment.
• Known hypersensitivity to a component of the vaccine or its excipients.
• A medical or psychiatric condition that, according to the investigator's judgment, may pose additional risk as a result of participation, interfere with safety assessments, or interfere with interpretation of results.
• History of a diagnosis or condition that, in the judgment of the investigator, may affect study endpoint assessment or compromise participant safety.
• Received any non-study vaccine within 14 days before or after any dose of vaccine (except for seasonal influenza vaccine, which is not permitted within 14 days before or after any dose of vaccine)
• Received intravenous or subcutaneous blood products (red blood cells, platelets, immunoglobulins) within 3 months prior to Day 1
• Participated in an interventional clinical study within 28 days prior to Day 0 or plans to donate blood products while participating in this study.
Biological: mRNA-1273, Biological: Placebo, Biological: mRNA-1273.214
SARS-CoV-2
mRNA-1273, mRNA-1273 vaccine, SARS-CoV-2, SARS-CoV-2 Vaccine, Coronavirus, Virus Diseases, Messenger RNA, COVID-19, COVID-19 Vaccine, Moderna
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A two-cohort, open-label, single arm, multicenter study to evaluate efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and&#13;&#10;adults with macrophage activation syndrome (MAS) in Still s disease (including systemic juvenile idiopathic arthritis and Adult onset Still s disease) or with MAS in Systemic lupus erythematous (EMERALD)

The aim of this study is to investigate how safe, effective, and well-tolerated multiple infusions of the experimental study treatment, Emapalumab, are in controlling macrophage activation syndrome (MAS), as well as to check the concentrations of Emapalumab in the blood and the speed at which it leaves the body. Participants will have been treated with previous treatment for MAS and are presenting an unsatisfactory response to that treatment. The previous drugs should include at least the corticosteroids. Your study doctor will taper corticosteroid progressively when emapalumab treatment would be started.

Bryce Binstadt
All
6 Months to 80 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT05001737
STUDY00013755
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Inclusion criteria Run-in phase in all cohorts
• Informed consent provided by the subject or by the subject s' legally authorized representative(s) with the assent of subjects who are legally capable of providing it, as required by local law.
• Male and female subjects aged between 6 months and 80 years of age at the time of diagnosis of MAS.
• MAS defined as per the criteria defined below for each cohort and requiring treatment with GCs. Interventional phase in all cohorts
• Informed consent provided by the subject or by the subject's legally authorized representative(s) with the assent of subjects who are legally capable of providing it, as as required by local law.
• Male and female subjects aged between 6 months and 80 years of age at the time of diagnosis of active MAS.
• Subjects who have shown an inadequate response to high dose intravenous (i.v.) GCs administered for at least 3 days according to local standard clinical practice, including but not limited to pulses of 30 mg/kg PDN on 3 consecutive days. High i.v. GCs dose is recommended not to be lower than 2 mg/kg/ day PDN equivalent (or at least 60 mg/day in pediatric subjects of 30 kg or more, and at least 1g/day in adult MAS subjects). In case of rapid worsening of the subject's condition and/or laboratory parameters, as per Investigator judgment, inclusion may occur within less than 3 days from starting high dose GCs.
• Diagnosis of active MAS confirmed by the treating rheumatologist, having ascertained the followings: a. Febrile subjects presenting with ferritin > 684 ng/mL. b. and any 2 of: i. Platelet count ≤ 181 x109/L ii. AST-level > 48 U/L iii. Triglycerides > 156 mg/dL iv. Fibrinogen level ≤ 360 mg/dL
• Female subjects of child-bearing potential willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug. Specific inclusion criteria to Cohort 1 and Cohort 2
• Cohort 1:
• Confirmed sJIA diagnosis. For subjects presenting with MAS in the context of the onset of sJIA, high presumption of sJIA will suffice for eligibility.
• Confirmed diagnosis of AOSD as per Yamaguchi criteria.
• Cohort 2:
• Confirmed diagnosis of SLE as per SLICC'12 criteria. Exclusion criteria
• Primary HLH documented by either the presence of a known causative genetic mutation or abnormal perforin expression and CD107a degranulation assay as described with primary hemophagocytic lymphohistiocytosis or by the presence of family history.
• Confirmed malignancy. Note: subjects with a suspected malignancy should have mononuclear cells typed by flow cytometry and/or tissue biopsy, as applicable, to rule out malignancy.
• Treatment with canakinumab, JAK inhibitors, TNF inhibitors and tocilizumab at the time of emapalumab initiation.
• Ongoing treatment with anakinra at a dose above 4 mg/kg at time of emapalumab initiation.
• Subjects treated with etoposide for MAS in the last 1 month.
• Clinically active mycobacteria (typical and atypical), Histoplasma Capsulatum, or Salmonella infections.
• Evidence of leishmania infections.
• Evidence of latent tuberculosis.
• History of hypersensitivity or allergy to any component of the study drug.
• Receipt of a Bacillus Calmette-Guerin (BCG) vaccine within 12 weeks prior to screening.
• Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to screening.
• Pregnancy or lactating female subjects.
Drug: Emapalumab
Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS
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Role of Pharmacotherapy in Counteracting Weight Regain in Adolescents with Severe Obesity

In this study we want to find out more about weight loss and how diet and medications can affect weight loss. This study will last for up to 58 weeks. There are two phases to the study: - A weight loss phase with prescribe meals that lasts 6 weeks. - A study medication/placebo phase that lasts up 52 weeks. You will not know if you are receiving the medication or the placebo.

Aaron Kelly
All
12 Years to 18 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04298203
STUDY00008743
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Inclusion Criteria:

• Severe obesity (BMI >/= 120% of the 95th percentile or BMI >/= 35 kg/m2)
• Age 12 to < 18 years of age at enrollment (screening) and Tanner stage >/= 2 - Female participants who are sexually active with males and who are able to get pregnant must agree to use two forms of contraception throughout the trial
Exclusion Criteria:

• Diabetes (type 1 or 2)
• Current or recent (< six months prior to enrollment) use of anti-obesity medication(s) defined as orlistat, phentermine, topiramate, combination phentermine/topiramate, liraglutide, and/or combination naltrexone/bupropion (monotherapy use of naltrexone or bupropion is not an exclusion)
• Previous metabolic/bariatric surgery
• Current use of a stimulant medication
• History of glaucoma
• Current or recent (<14 days) use of monoamine oxidase inhibitor
• Known hypersensitivity to sympathomimetic amines
• Any history of treatment with growth hormone
• Any history of bulimia nervosa
• Major psychiatric disorder as determined by the local medical monitor
• Unstable and clinically-diagnosed (defined as documented in the medical record, if available) depression
• Any history of active suicide attempt
• History of suicidal ideation or self-harm within the previous 30 days
• PHQ-9 score >15
• Current pregnancy or plans to become pregnant during study participation
• Current tobacco use
• ALT or AST >/= 3 times the upper limit of normal
• Bicarbonate <18 mmol/L
• Creatinine > 1.2 mg/dL
• Any history of seizures
• Uncontrolled hypertension as determined by the local medical monitor
• History of structural heart defect or clinically significant arrhythmia
• Diagnosed monogenic obesity
• Any history of cholelithiasis
• Any history of nephrolithiasis
• Clinically diagnosed hyperthyroidism
• Untreated thyroid disorder
• Any disorder, unwillingness, or inability, not covered by any other exclusion criteria, which in the investigator's opinion, might jeopardize the subject's safety or compliance with the protocol
Drug: Phentermine-Topiramate, Dietary Supplement: Meal Replacement Therapy, Other: Placebo
Obesity
Childhood, Adolescent, Clinics and Surgery Center (CSC)
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Randomization of Cytarabine Monotherapy Versus Standard-of-Care Vinblastine/Prednisone For Frontline Treatment of Langerhans Cell Histiocytosis (TXCH LCH0115)

The purpose of this research study is to compare previously used vinblastine/prednisone to single therapy with cytarabine for LCH. We will evaluate the utility of an imaging study called a positron emission tomography (PET) scan to more accurately assess areas of LCH involvement not otherwise seen in other imaging studies as well as response to therapy. We also want to identify if genetic and other biomarkers (special proteins in patient's blood and in patient's cancer) relate to the response of patients LCH to study treatment.

Lucie Turcotte
All
up to 21 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT02670707
STUDY00008859
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Inclusion Criteria:

• Patient must have biopsy-confirmed diagnosis of Langerhans cell histiocytosis.
• Patient must be between 0-21 years of age.
• Patient must have a Karnofsky performance score ≥ 50% or Lansky performance score ≥ 50%.
Exclusion Criteria:

• Patient may not have received any prior systemic cytotoxic or other chemotherapies for LCH or any other malignant disorder prior to the initiation of protocol therapy on TXCH LCH0115 with the exception of: Steroid pretreatment: Systemic glucocorticosteroids (prednisone, methylprednisone, dexamethasone, etc.) for less than or equal to 120 hours (5 days) in the 7 days prior to initiating protocol therapy or for less than or equal to 336 hours (14 days) in the 28 days before the initiation of protocol therapy does not affect eligibility. The dose of steroid previously given does not affect eligibility. Patients who have only received surgical or radiation therapy, intralesional injection of steroids, inhalational steroids, systemic mineralocorticoids (hydrocortisone), or topical steroids may also be enrolled.
• Patient may not have disease limited to a single skin or bone site, with the following exceptions:
• Central Nervous System (CNS) risk lesions/special site disease: patients with single bone sites that are CNS-risk (sphenoid, mastoid, orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or neurodegenerative disease) or are "special sites" (odontoid peg, vertebral lesion with intraspinal soft tissue extension) require systemic therapy as standard of care and thus are eligible for the study.
• Functionally critical lesions: A single lesion not described above which may cause "functionally critical anatomic abnormality" wherein attempts at local therapy (such as surgical curettage or radiation) would cause unacceptable morbidity. These patients may be enrolled with written approval of the Coordinating Center PI or Vice-Chair and documentation of the rationale justifying systemic therapy.
• Asynchronous multisite LCH presentation: A patient may also have any single site of disease involvement at the time of enrollment if they previously had at least one other site of LCH disease in the past (which may have been treated with local therapy/surgery as described), as long as no systemic therapy was previously given per protocol guidelines.
• Patient may not have severe renal disease (creatinine greater than 3 times normal for age OR creatinine clearance < 50 ml/m2/1.73m^2).
• Patient may not have severe hepatic disease (direct bilirubin greater than 3 mg/dl OR aspartate aminotransferase (AST) greater than 500 IU/L), unless hepatic injury is due to LCH.
• Female patients may not be pregnant or breastfeeding.
• Patients of reproductive potential not willing to use an adequate method of birth control for the duration of the study.
• Patients who are HIV positive may not be enrolled. NOTE: Patients excluded for laboratory abnormalities or performance score only may be enrolled on the study with written approval from the Coordinating Center PI or Vice-Chair.
Drug: Cytarabine, Drug: Vinblastine/prednisone
Langerhans Cell Histiocytosis
vinblastine, prednisone, cytarabine, Langerhans Cell Histiocytosis (LCH)
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A Phase 1b/2 Study of Abemaciclib in Combination with Irinotecan and Temozolomide (Part A) and Abemaciclib in Combination with Temozolomide (Part B) in Pediatric and Young Adult Patients with Relapsed/Refractory Solid Tumors and Abemaciclib in Combination with Dinutuximab, GM-CSF, Irinotecan, and Temozolomide in Pediatric and Young Adult Patients with Relapsed/Refractory Neuroblastoma (Part C). Protocol Number: I3Y-MC-JPCS

The study's purpose is to see if the drug abemaciclib is safe and effective in combination with temozolomide and irinotecan (Part A) and abemaciclib in combination with temozolomide (Part B) in pediatric and young adult participants with relapsed/refractory solid tumors.

Emily Greengard
All
up to 21 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04238819
STUDY00013998
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Inclusion Criteria:

• Parts A and B only:
• Participants must be less than or equal to (≤)18 years of age.
• Body weight greater than or equal to (≥)10 kilograms and body surface area (BSA) ≥0.5 -- Participants with any relapsed/refractory malignant solid tumor (excluding lymphoma), including central nervous system tumors, that have progressed on standard therapies.
• For sites that are actively enrolling Parts B and C, participants with neuroblastoma who are eligible for Part C will be excluded from Part B unless approved by Lilly CRP/CRS.
• Part C only:
• Participants must be less than (<) 21 years of age.
• Participants have a BSA ≥0.3 m².
• Participants with first relapse/refractory neuroblastoma.
• All Parts
• Participants must have measurable or evaluable disease by RECIST v1.1 or RANO.
• A Lansky score ≥50 for participants <16 years of age or Karnofsky score ≥50 for participants ≥16 years of age.
• Participants must have discontinued all previous treatments for cancer or investigational agents and must have recovered from the acute effects to Grade ≤1 at the time of enrollment.
• Able to swallow.
• Adequate hematologic and organ function ≤2 weeks (14 days) prior to first dose of study drug.
• Females of reproductive potential must have negative urine or serum pregnancy test at baseline (within 7 days prior to starting treatment).
• Female participants of reproductive potential must agree to use highly effective contraceptive precautions during the trial. For abemaciclib, females should use contraception for at least 3 weeks following the last abemaciclib. For other study drugs, highly effective contraceptive precautions (and avoiding sperm donation) must be used according to their label.
• Life expectancy of at least 8 weeks and able to complete at least 1 cycle of treatment.
• Caregivers and participants willing to make themselves available for the duration of the trial.
Exclusion Criteria:

• Received allogenic bone marrow or solid organ transplant.
• Received live vaccination.
• Intolerability or hypersensitivity to any of the study treatments or its components.
• Diagnosed and/or treated additional malignancy within 3 years prior to enrollment that may affect the interpretation of results, with the exception of curatively treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, and/or curatively resected in situ cervical and/or breast cancers.
• Pregnant or breastfeeding.
• Active systemic infections or viral load.
• Serious and/or uncontrolled preexisting medical condition(s) that would preclude participation in this study.
• Parts A and C only: Have a bowel obstruction.
• Prior treatment with drugs known to be strong inhibitors or inducers of isoenzyme cytochrome P450 3A (CYP3A) or strong inhibitors of uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) if the treatment cannot be discontinued or switched to a different medication at least 5 half-lives prior to starting study drug.
• Received prior treatment with cyclin-dependent kinase (CDK) 4 & 6 inhibitor.
• Part C only: Received prior systemic therapy for relapsed/refractory neuroblastoma.
• Currently enrolled in any other clinical study involving an investigational product or non-approved use of a drug or device.
• Has received an experimental treatment in a clinical trial within the last 30 days or 5 half-lives, whichever is longer.
Drug: Abemaciclib, Drug: Irinotecan, Drug: Temozolomide, Drug: Dinutuximab, Drug: GM-CSF
Relapsed Solid Tumor, Refractory Solid Tumor
CDK4, CDK6, Ewing's sarcoma, Neuroblastoma, Malignant rhabdoid tumor, Rhabdomyosarcoma, Osteosarcoma, Brain tumor, Glioblastoma, Malignant glioma, Diffuse intrinsic pontine glioma, Medulloblastoma, Ependymoma, Solid tumor, High-grade glioma
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Vibrotactile stimulation of the larynx to treat unexplained chronic cough

This is a study of adults with unexplained chronic cough between 18-80 years old. This study is trying to determine whether a noninvasive vibrotactile stimulation device can help reduce cough symptoms.

Stephanie Misono
18 Years and over
Pilot
This study is NOT accepting healthy volunteers
ENT-2020-28903
STUDY00012174
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Inclusion Criteria:
- Adults aged 18-80 - >8 weeks of non-productive cough - Cough visual analogue scale (VAS): Rating of 40 or greater on a 0-100 cough severity scale - CXR or chest CT negative (collected as part of routine clinical care); no time limit on imaging (if available) - Clinical impression that untreated or inadequately treated gastroesophageal, pulmonary, and/or sinonasal source is not primary cough etiology - Ability to provide informed consent and independently complete questionnaires - Ability to read and speak English
Exclusion Criteria:
- Current or recent (quit < 3 months ago) smoking - Known currently infectious cause for cough (e.g., TB, pertussis, COVID) - History of known or suspected aspiration pneumonia - Neuromuscular impairment that may affect cough/laryngeal sensation and/or function (e.g., multiple system atrophy, Parkinson, CVA) - Untreated carotid disease - Electronic implants (e.g., pacemaker) - Specific medication use conditions, including: - Taking ACE-inhibitors - Around the clock use of benzodiazepines - Anticipate new medications targeting cough during the period of the study - If on neuromodulators and/or opioids, anticipate change in dose during the period of the study - Currently doing speech therapy for cough - BMI > 30 (for transmission of VTS through soft tissue) - Contraindications to safe VTS device use, including: - Allergy to adhesives - Unable to manipulate device - Recent intubation/neck surgery (within 8 weeks) - Drug/alcohol dependency or abuse - Pregnant - No regular access to wifi internet
Breathing, Lung & Sleep Health, Ear, Nose & Throat
Clinics and Surgery Center (CSC), chronic cough, cough, larynx, vibrotactile stimulation
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Vibrotactile stimulation of the larynx to treat unexplained chronic cough

This is a study of adults with unexplained chronic cough between 18-80 years old. This study is trying to determine whether a noninvasive vibrotactile stimulation device can help reduce cough symptoms.

Stephanie Misono
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT05273190
STUDY00012174
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Inclusion Criteria:
- Adults aged 18-80 - >8 weeks of non-productive cough - Cough visual analogue scale (VAS): Rating of 40 or greater on a 0-100 cough severity scale - CXR or chest CT negative (collected as part of routine clinical care); no time limit on imaging (if available) - Clinical impression that untreated or inadequately treated gastroesophageal, pulmonary, and/or sinonasal source is not primary cough etiology - Ability to provide informed consent and independently complete questionnaires - Ability to read and speak English
Exclusion Criteria:
- Current or recent (quit < 3 months ago) smoking - Known currently infectious cause for cough (e.g., TB, pertussis, COVID) - History of known or suspected aspiration pneumonia - Neuromuscular impairment that may affect cough/laryngeal sensation and/or function (e.g., multiple system atrophy, Parkinson, CVA) - Untreated carotid disease - Electronic implants (e.g., pacemaker) - Specific medication use conditions, including: - Taking ACE-inhibitors - Around the clock use of benzodiazepines - Anticipate new medications targeting cough during the period of the study - If on neuromodulators and/or opioids, anticipate change in dose during the period of the study - Currently doing speech therapy for cough - BMI > 30 (for transmission of VTS through soft tissue) - Contraindications to safe VTS device use, including: - Allergy to adhesives - Unable to manipulate device - Recent intubation/neck surgery (within 8 weeks) - Drug/alcohol dependency or abuse - Pregnant - No regular access to wifi internet
Device: Vibrotactile Stimulation
Breathing, Lung & Sleep Health, Ear, Nose & Throat, Cough
Chronic cough, Clinics and Surgery Center (CSC), chronic cough, cough, larynx, vibrotactile stimulation
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The Effect of Tirzepatide Versus Dulaglutide on Major Adverse Cardiovascular Events in Patients With Type 2 Diabetes (SURPASS-CVOT)

Study I8F-MC-GPGN (GPGN), also known as SURPASS-CVOT, is a Phase 3, event-driven, multicenter, international, randomized, double-blind, active comparator, parallel-group study. This study will assess the effect of QW tirzepatide (up to 15 mg) versus dulaglutide (1.5 mg) on CV outcomes when added to the standard of care in patients with T2DM with established cardiovascular disease and elevated risk for MACE.

Les Forgosh
18 Years and over
Phase III
This study is NOT accepting healthy volunteers
NCT04255433
STUDY00009070
Diabetes & Endocrine, Heart & Vascular, Type 2 Diabetes Mellitus
Diabetes Mellitus, Diabetes Mellitus, Type 2, Endocrine System Diseases, Gastric Inhibitory Polypeptide, Glucagon-Like Peptide 1, Glucose Metabolism Disorders, Heart Disease, Hormones, Hormones, Hormone Substitutes, and Hormone Antagonists, Hypoglycemic Agents, Incretins, Metabolic Diseases, Physiological Effects of Drugs, Tirzepatide, Type 2 Diabetes
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RCT01437: Proactive infliximab optimization using a pharmacokinetic dashboard versus standard of care in patients with Crohn s disease: The OPTIMIZE Trial

The purpose of this study is to find out if using a computer program (called iDose) to guide infliximab dosing is more effective and safer than using standard infliximab dosing over 52 weeks. All patients in this study will be receiving infliximab as part of their medical care, this study is only looking at two different methods of determining the dose and timing of administration.

Byron Vaughn
All
18 Years and over
Phase 4
This study is NOT accepting healthy volunteers
NCT04835506
STUDY00013632
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Inclusion Criteria:

• Males or nonpregnant, nonlactating females aged 16 to 80 years inclusive.
• Diagnosis of CD prior to screening using standard endoscopic, histologic, or radiologic criteria. Subjects with patchy colonic inflammation initially diagnosed as indeterminate colitis would meet inclusion criteria, if the investigator feels that the findings are consistent with CD.
• Moderately to severely active CD, defined by a total Crohn's Disease Activity Index (CDAI) score between 220 and 450 points, and at least 1 of the following:
• Elevated CRP > upper limit of normal )
• Elevated fecal calprotectin (FC) (> 250 μg/g)
• SES-CD > 6, or SES-CD > 3 for isolated ileal disease
• Physician intends to prescribe IFX as part of the usual care of the subject.
• No previous use of IFX prior to enrolment in the current study, unless the participant received 1 prior dose of IFX (within 2.5 weeks of enrolment) and met all eligibility criteria at the time of starting IFX and IFX was administered according to the requirements outlined in this protocol
• Able to participate fully in all aspects of this clinical trial.
• Written informed consent must be obtained and documented.
Exclusion Criteria:

• Subjects with any of the following CD-related complications:
• Abdominal or pelvic abscess, including perianal
• Presence of stoma or ostomy
• Isolated perianal disease
• Obstructive disease, such as obstructive stricture
• Short gut syndrome
• Toxic megacolon or any other complications that might require surgery, or any other manifestation that precludes or confounds the assessment of disease activity (CDAI or SES-CD)
• Total colectomy.
• History or current diagnosis of ulcerative colitis, indeterminate colitis, microscopic colitis, ischemic colitis, colonic mucosal dysplasia, or untreated bile acid malabsorption.
• Current bacterial or parasitic pathogenic enteric infection, according to SOC assessments, including: Clostridioides difficile; tuberculosis; known infection with hepatitis B or C virus; known infection with HIV; sepsis; abscesses. History of the following: opportunistic infection within 6 months prior to screening; any infection requiring antimicrobial therapy within 2 weeks prior to screening; more than 1 episode of herpes zoster or any episode of disseminated zoster; any other infection requiring hospitalization or intravenous antimicrobial therapy within 4 weeks prior to screening.
• Has any malignancy or lymphoproliferative disorder other than nonmelanoma cutaneous malignancies or cervical carcinoma in situ that has been treated with no evidence of recurrence within the last 5 years.
• Known primary or secondary immunodeficiency.
• PNR to adalimumab, defined as no objective evidence of clinical benefit after 14 weeks of therapy.
• Subjects with failure to a prior biologic, defined as PNR or SLR, will be excluded when a maximum of 40% of the planned enrollment (approximately 78 subjects) have failure to prior biologic exposure.
• Concomitant use of oral corticosteroid therapy exceeding prednisone 40 mg/day, budesonide 9 mg/day, or equivalent, unless a tapering schedule is initiated with a plan to be off CS by Week 14
• Presence of any medical condition or use of any medication that is a contraindication for IFX use, as outlined on the product label.
• A concurrent clinically significant, serious, unstable, or uncontrolled underlying cardiovascular, pulmonary, hepatic, renal, GI, genitourinary, hematological, coagulation, immunological, endocrine/metabolic, or other medical disorder that, in the opinion of the investigator, might confound the study results, pose additional risk to the subject, or interfere with the subject's ability to participate fully in the study.
• Pregnant or lactating women, to be excluded based on the physician's usual practice for determining pregnancy or lactation status.
• Known intolerance or hypersensitivity to IFX or other murine proteins.
Drug: Infliximab
Crohn Disease, Digestive & Liver Health
Clinics and Surgery Center (CSC)
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Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas (POPLAR-NF2)

This study will investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutations. This study is a parallel-group, two-staged, Phase 2/3, randomized, multi-center study with two cohorts: Cohort A followed by Cohort B. The purpose of Cohort A is to provide early data on efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas, and provide guidance for the correct dose, population, sample size, and endpoint for the confirmatory part of the study (Cohort B). Additional goals for Cohort A are to assess effects of food on drug absorption. The purpose of Cohort B of the study is to assess the efficacy and safety of REC-2282 compared with placebo in participants with progressive NF2 mutated meningiomas.

Christopher Moertel, MD
All
12 Years and over
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT05130866
STUDY00015488
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Inclusion Criteria:

• ≥12 years of age and weighing at least 40 kg
• Progressive meningioma that is amenable to volumetric analysis
• Has either 1) sporadic meningioma with confirmed NF2 mutation; or, 2) confirmed diagnosis of NF2 disease (revised Manchester criteria); or, 3) at least one NF2-related tumor (with pathogenic germline or proven mosaic NF2 variant)
• Adequate bone marrow function
• Has provided written informed consent/assent to participate in the study
Exclusion Criteria:

• Progressive disease associated with significant or disabling clinical symptoms likely to require surgery or radiation therapy within the next 3 months.
• Received prior surgery, radiosurgery, or laser interstitial thermal therapy in the target tumor, or immediately adjacent to the target tumor within 6 months prior to screening.
• Received an anti- tumor agent for meningioma within 3 months, or 5 half-lives (whichever is longer), prior to screening.
• History of an active malignancy within the previous 3 years except for localized cancers that are considered cured, and, in the opinion of the investigator, present a low risk of recurrence.
• Received another investigational drug within 30 days prior to screening
• Pregnant, lactating, or is planning to attempt to become pregnant or impregnate someone during this study or within 90 days after the last dose of IMP.
Drug: REC-2282, Drug: Placebo
Neurofibromatosis Type 2
Neurofibromatosis Type 2, Neurofibromatosis Type II
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A closed-loop assessment and treatment platform for unipolar depression and anxiety

This study is a validation study to evaluate the acceptability and feasibility of the closed-loop strategy employing the mobile mental health application with the target population to prepare for a large-scale efficacy trial in adults with MDD, depression and/or anxiety.

Gamze Camsari
All
18 Years to 60 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02948036
STUDY00004179
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Inclusion Criteria:

• Participant must be 18 to 60 years of age.
• Participant must score ≥ 9 on the Patient Health Questionnaire-9 (PHQ-9).
• Participant taking antidepressants or engaged in psychotherapy will not be excluded. If potential participants are currently prescribed psychotropic medication, they must be on a clinically stable medication regimen for ≥ 6 weeks prior to screening, based on self-report or as verified by medical health records, when available and authorized.
• Participant must be a fluent English speaker.
• Participant must have adequate sensorimotor capacity to perform the program, including visual capacity adequate to read from a computer screen at a normal viewing distance, auditory capacity adequate to understand normal speech, and motor capacity adequate to control and use a mobile device and/or computer as required to complete study activities.
• Participant must have access to wireless Internet connectivity.
• Participant must be willing to communicate with study staff via email.
Exclusion Criteria:

• Participant with unstable and/or untreated conditions that may affect cognition, including untreated substance abuse/dependence disorders, unmanaged cardiovascular disease, endocrine or neurologic disorder, epilepsy, brain injury, hospitalization within 6-weeks of enrollment, ongoing chemotherapy or other cancer treatment (e.g., radiation) .
• Participant with history or current DSM-5 diagnosis of psychosis, such as schizophrenia, schizoaffective disorder, delusion disorder, psychotic disorder NOS, bipolar disorder, substance abuse (<1 year), and/or mood congruent or mood incongruent psychotic features or disorders.
• Participant has a history or current diagnosis of dementia and/or scores less than a 14 (75%) on the UBACC.
• Participant with active suicidal ideations or behaviors within 2 months of screening.
• Participant that shows signs of intoxication due to current substance abuse (including alcohol and/or illegal drugs) during any in person visit. Such participants will have that visit re-scheduled; participants with this problem occurring more than once may be excluded and dropped at the discretion of the Principal Investigators.
• Participant has problems performing assessments or comprehending or following spoken instructions, or those with behaviors during screening or baseline visits that, in the judgment of the screening staff, are likely to present significant problems for the staff conducting assessments.
• Participant is enrolled in a concurrent clinical trial involving an investigational pharmaceutical, nutraceutical, medical device, or behavioral treatment that could affect the outcome of this study. However, participation in standard treatments (e.g., occupational therapy) or use of prescribed medications (e.g., anti-depressants) is allowable..
• Participant is using computer-based cognitive training programs or has used it within a month of the consent date.
Other: Mobile-device, plasticity-based adaptive cognitive treatment
Major Depressive Disorder
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ZEUS - Effects of ziltivekimab versus placebo on cardiovascular outcomes in patients with established atherosclerotic cardiovascular disease, chronic kidney disease and systemic inflammation

We are doing this study to see if ziltivekimab reduces the risk of having cardiovascular events (for example heart attack and stroke) in people with cardiovascular disease, chronic kidney disease and inflammation. Participants will either get ziltivekimab (active medicine) or placebo (a dummy medicine which has no effect on the body). This is known as the study medicine. Which treatment participants get is decided by chance. Participants chance of getting ziltivekimab or placebo is the same. Ziltivekimab is not yet approved in any country or region in the world. It is a new medicine doctors cannot prescribe. Participants will get the study medicine in a pre filled syringe. Participants will need to use the pre filled syringe to inject the study medicine into a skinfold once-monthly. The study is expected to last for 2.5 to 4 years. Participants will have up to 20 clinic visits. Participants will have blood and urine samples taken at most of the clinic visits. Participants will have their heart examined using sound waves (echocardiography) and electrodes (electrocardiogram). Women cannot take part if pregnant, breast-feeding or planning to get pregnant during the study period.

Daniel Duprez
Phase III
This study is NOT accepting healthy volunteers
NCT05021835
STUDY00013843
Cardiovascular Risk, Chronic Kidney Disease, Inflammation
Clinics and Surgery Center (CSC)
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Neural Correlates of the Shift in Social Buffering of Social Evaluative Threat

This study is one of three studies on an NIH-funded project addressing the effectiveness of parents in buffering children and adolescents from the physiological and brain responses to stress. This study uses MRI scanning to measure the brain response to social evaluative stress (giving a speech and doing math problems in front of a panel of judges) as well as the impact of the presence of various social partners (no one, researcher, or parent) in buffering the physiological and brain responses to social evaluative stress.

All
11 Years to 14 Years old
N/A
This study is also accepting healthy volunteers
NCT04211155
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Inclusion Criteria:

• sufficient vision to complete assent and study procedures
• sufficient hearing to complete assent and study procedures
• sufficient language skills to provide verbal and written assent
Exclusion Criteria:

• Premature birth (less than 37 weeks)
• congenital and/or chromosomal disorders (e.g. cerebral palsy, FAS, mental retardation, Turner Syndrome, Down Syndrome, Fragile X)
• Autism Spectrum Disorders
• history of serious medical illness (e.g., cancer, organ transplant)
• youth taking systemic glucocorticoids
• youth taking beta-adrenergic medications
• diagnoses of psychiatric illness, seizure disorder or other neurological disorders
• contraindications for MRI (implanted medical device; presence of non-removal metal in or on the body, including piercings, orthodontic braces or certain permanent retainers)
• known pregnancy
• tattoos
• history of significant claustrophobia
Other: Questionnaires, Other: MRI
Social Stress, Adolescent Behavior
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Measurement of Glucose Homeostasis in Human Brain by NMR: Effect of Recurrent Hypoglycemia on Type 1 Diabetes (Aim 1)

We are studying changes in the brain that result from hypoglycemia. People, who have Type 1 diabetes (for 2 to 30 years) and can identify hypoglycemia when it happens, may be eligible for the study. We will do brain scans during 2-hour hypoglycemic periods that we induce.

Elizabeth Seaquist
All
18 Years to 65 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03410277
STUDY00002192
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Inclusion Criteria:

• Type 1 diabetes diagnosed on clinical or laboratory grounds
• Diabetes duration 2 - 30 years
• Hemoglobin A1C <8.5%
Exclusion Criteria:

• Impaired awareness of hypoglycemia as determined by the Cox and Gold questionnaires
• Pregnant or plan to become pregnant during the study period
• Uncontrolled hypertension (blood pressure > 145/95 mmHg at screening)
• Evidence of autonomic neuropathy (presence of orthostatic hypotension or history of gastroparesis)
• Proliferative retinopathy
• Impaired kidney function (GFR < 45)
• History of myocardial infarction, stroke, seizures, neurosurgical procedures, major depression requiring hospitalization within the last 5 years, arrhythmias
• Current substance abuse
• Use of drugs that can alter glucose metabolism including but not limited to glucocorticoids and niacin, and excluding insulin and glucose lowering drugs used to treat diabetes, as determined by a clinician
• Inability to undergo MRI scanning, including but not limited to unable to remain still in an MRI scanner for more than 30 minutes, claustrophobia, presence of paramagnetic substances or pacemakers in body, weight over 300 lbs
Other: Experimental hypoglycemia
Diabetes Mellitus, Type 1
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Prefrontal Cortical Stimulation in Severe Treatment Resistant Depression

This study looks at the use of an implanted brain stimulator for people who have treatment resistant depression. The change in brain function by EEG and symptoms of depression will be examined. This study is open to people 22-55 years old with Medicare or Medicare Advantage insurance.

Ziad Nahas
All
22 Years to 55 Years old
N/A
This study is NOT accepting healthy volunteers
NCT04124341
STUDY00006945
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Inclusion Criteria:

• Participant has a diagnosis of chronic (greater than or equal to 2 years) depressive episode as defined by DSM V criteria
• Participant has not had an adequate response to three or more adequate antidepressant treatments from at least two different antidepressant treatment categories in the current depressive episode according to the Antidepressant Treatment History Form (ATHF)
• Participant must have had ECT or refused to undergo ECT if clinically indicated to them
• Participant must have HRSD greater than or equal to 20
• Participant must be able to complete the evaluations needed for this study including the functional imaging scans and the EEG Bayesian optimization sessions
• Participant must be under the care of a licensed psychiatrist, undergoing regular care evaluations, and inform study team of any change to care team during study participation
• Participant must agree to allow all forms of communication between investigators and study staff and any health care provider (current or having provided service within two years of enrollment)
• Participant must provide name and contact information for at least two people greater than or equal to 22 in age who reside within a 30 minute drive of the participant's residents and whom staff may contact as necessary during study participation
• Participant must be enrolled in a Medicare program
Exclusion Criteria:

• The EpCS would (in the investigator's judgment) pose an unacceptable surgical or medical risk for the participant
• Participant is expected to require full body magnetic resonance imaging (MRI) during the clinical study
• Participant is judged by the investigator to be acutely suicidal (e.g. within the 30 days prior to the EpCS implant the participant has made a suicide attempt or gesture or has made specific plans or preparation to commit suicide or scores 14 or higher on the SSI)
• In addition to the acute suicidal risks mentioned above, participant meets any of the following:
• Has made a suicide attempt within the previous 12 months that required medical treatment
• Has made greater than or equal to two suicide attempts in the past 12 months
• Has a clear-cut plan for suicide that states that she/he cannot guarantee that she/he will call her/his regular psychiatrist or the Investigator if the impulse to implement the plan becomes substantial during the study
• Is likely to attempt suicide within the next six months, in the Investigator's opinion
• Participant has a history of schizophrenia, schizoaffective disorder, or other psychotic disorder, or a current major depressive episode that includes psychotic features (commonly referred to as psychotic depression) according to the DSM V criteria
• Participant with a diagnosis of dementia with a Mini-Mental State Exam (MMSE) less than or equal to 23
• Participant with a positive urine pregnancy test
• Participant with a positive urine drug screen
• Participant with DBS
• Participant with VNS if the device was active in the last 6 months prior to study enrollment
• Participant with history of seizures
Device: Prefrontal Cortical Stimulation (PCS)
Treatment Resistant Depression
Clinics and Surgery Center (CSC)
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A randomized double-blind, placebo-controlled, multicenter;trial assessing the impact of lipoprotein (a) lowering with;TQJ230 on major cardiovascular events in patients with;established cardiovascular disease (HORIZON)

This is a pivotal phase 3 study designed to support an indication for the reduction of cardiovascular risk in patients with established CVD and elevated Lp(a). The primary objectives of this study is to demonstrate the superiority of TQJ230 compared to placebo in reducing the risk of expanded MACE (cardiovascular death, non-fatal MI, non-fatal stroke and urgent coronary re-vascularization requiring hospitalization) in 1) the overall study population with established CVD (Lp(a) ≥ 70 mg/dL) and/or 2) in a sub-population with established CVD and Lp(a) ≥ 90 mg/dL.

Daniel Duprez
18 Years and over
Phase III
This study is NOT accepting healthy volunteers
NCT04023552
STUDY00011204
Cardiovascular Disease and Lipoprotein(a)
Clinics and Surgery Center (CSC), Lipoprotein(a), Lp(a),CVD, myocardial infarction, PAD,Stroke, Pelacarsen
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A multiple ascending dose trial investigating safety, tolerability, and pharmacokinetics of NNC0361-0041 administered subcutaneously to patients with type 1 diabetes mellitus (TOPPLE T1D)

This study is looking at 48 adult patients that have been diagnosed with type 1 diabetes within the past 4 years and giving them subcutaneous injections weekly of NNC0361-0041 plasmid to assess the safety and tolerability. This is a phase1 study that will enrolled over a 28 week period.

Antoinette Moran
All
18 Years to 45 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04279613
STUDY00011044
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Inclusion Criteria:

• Willing to provide Informed Consent
• Participants must live in a location with rapid access to emergency medical services
• Age 18-45 years (both inclusive) at the time of signing informed consent
• Must have a diagnosis of T1D for less than 48 months at randomization
• Must have at least one diabetes-related autoantibody present (GAD65A; mIAA, if obtained within 10 days of the onset of insulin therapy; IA-2A; ICA; or ZnT8A)
• Must have stimulated C-peptide levels greater than or equal to 0.2 pmol/ml measured during an MMTT conducted at least 21 days from diagnosis of diabetes and within one month (37 days) of randomization
• Be willing to comply with intensive diabetes management
• HbA1c ≤8.5% at screening
• Subjects who are CMV and/or EBV seronegative at screening must be CMV and/or EBV PCR negative within 37 days of randomization and may not have had signs or symptoms of a CMV and/or EBV compatible illness lasting longer than 7 days within 37 days of randomization
• Be up to date on recommended immunizations
• Be at least 6 weeks from last live immunization
• Be at least 4 weeks from killed vaccine other than flu vaccine
• Participants are required to receive killed influenza vaccination at least 2 weeks prior to randomization when vaccine for the current or upcoming flu season is available
• Be willing and medically acceptable to postpone live vaccines during the treatment period and for 3 months following last dose of study drug
• If participant is female with reproductive potential, she must have a negative pregnancy test at screening and be willing to avoid pregnancy using a highly effective contraceptive method for the 12 months of the study
• Males of reproductive age must use adequate contraceptive method during the treatment phase and for 3 months following last dose of study drug
• Participants are required to receive an authorized non-live COVID-19 vaccination and be fully vaccinated, including eligible boosters as indicated, at least two weeks prior to randomization.
Exclusion Criteria:
Potential participants must not meet any of the following exclusion criteria:
• One or more screening laboratory values as stated
• Leukocytes < 3,000/μL
• Neutrophils <1,500 /μL
• Lymphocytes <800 /μL
• Platelets <100,000 /μL
• Haemoglobin <6.2 mmol/L (10.0 g/dL)
• Potassium >5.5 mmol/L or <3.0 mmol/L
• Sodium >150mmol/L or < 130mmol/L
• AST or ALT ≥2.5 times the upper limits of normal
• Bilirubin ≥ 1.5 times upper limit of normal
• Glomerular Filtration Rate (eGFR) value of eGFR < 60 ml/min/1.73 m2 as defined by KDIGO 2012 (43)
• Any other laboratory abnormality that might, in the judgment of the investigator, place the subject at unacceptable risk for participation in this trial
• Current or ongoing use of non-insulin pharmaceuticals that affect glycemic control within prior 7 days of screening
• Use of other immunosuppressive agents including chronic use of systemic steroids. Topical products are acceptable (nasal, conjunctival, skin)
• Have active signs or symptoms of acute infection at the time of randomization
• Have current, confirmed COVID-19 infection
• Chronic active infection other than localized skin infections
• Have evidence of prior or current tuberculosis infection as assessed by PPD, interferon gamma release assay or by history
• Have evidence of current or past HIV, Hepatitis B infection
• Have evidence of active Hepatitis C infection
• Vaccination with a live virus within the last 6 weeks and killed vaccine within 4 weeks (except 2 weeks for flu vaccine)
• Be currently pregnant or lactating, or anticipate getting pregnant within the one-year study period.
• Have severe obesity: adults BMI ≥ 40
• Have a history of malignancies
• Untreated hypothyroidism or active Graves' disease
• History of severe reaction to prior vaccination
• Participation in any clinical trial of an approved or non-approved investigational medicinal product within 30 days after last blood draw (or 5 half-lives of investigational drug, whichever is greater) before screening, or currently enrolled in any other clinical trial
• Subject is the investigator or any sub-investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the trial
• Supine blood pressure at screening outside the range of 90-139 mmHg for systolic or 50-89 mmHg for diastolic. To exclude white-coat nervousness a single repeat measurement is allowed
• Have any complicating medical issues or abnormal clinical laboratory results that may interfere with study conduct, or cause increased risk
• Any condition that in the investigator's opinion may adversely affect study participation or may compromise the study results
Drug: NNC0361-0041, Other: Placebo
Type I Diabetes
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Transcranial Magnetic Stimulation to Augment Behavior Therapy for Tics

The study will examine whether combining Comprehensive Behavioral Intervention for Tics (CBIT) with inhibition of the supplementary motor area (SMA) using transcranial magnetic stimulation (TMS) normalizes activity in the SMA-connected circuits, improves tic suppression ability, and enhances CBIT outcomes in young people with tic disorder. The study will also examine different TMS dosing strategies.

Christine Conelea
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT04578912
STUDY00010519
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Inclusion Criteria:
-- 12-21 years old -- right-handed -- able to undergo MRI -- currently experiencing chronic motor and/or vocal tics
Exclusion Criteria:
-- currently receiving therapy focused on tics -- currently taking neuroleptic/antipsychotic medications
Behavioral: Comprehensive Behavioral Intervention for Tics (CBIT), Device: Repetitive Transcranial Magnetic Stimulation (rTMS), Device: Continuous Theta Burst Stimulation (cTBS)
Brain & Nervous System, Children's Health, Mental Health & Addiction, Tic Disorders, Tics, Tic, Motor, Tic Disorder, Childhood, Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence
CBIT, TMS, Tourette's, neurodevelopmental disorders, tics, transcranial magnetic stimulation
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Neural mechanisms of early visual dysfunction in psychosis

Michael-Paul Schallmo
18 Years and over
NA
This study is also accepting healthy volunteers
PSYCH-2019-28336
STUDY00007958
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Inclusion Criteria:
18-60 years old; Normal or corrected-to-normal vision; Current diagnosis of schizophrenia or schizoaffective disorder
Exclusion Criteria:
Claustrophobia; Current substance dependence (besides nicotine); Any vision anomaly (e.g. strabismus/crossed eyes, lazy eye, color blindness); Current or past diagnosis of bipolar I disorder
Brain & Nervous System, Vision & Eyes
EEG, MRI, neuroscience, psychosis, vision
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COG AALL1732: A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy

This phase III trial studies whether inotuzumab ozogamicin added to post-induction chemotherapy for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL) improves outcomes. This trial also studies the outcomes of patients with mixed phenotype acute leukemia (MPAL), and B-lymphoblastic lymphoma (B-LLy) when treated with ALL therapy without inotuzumab ozogamicin. Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a type of chemotherapy called calicheamicin. Inotuzumab attaches to cancer cells in a targeted way and delivers calicheamicin to kill them. The overall goal of this study is to understand if adding inotuzumab ozogamicin to standard of care chemotherapy maintains or improves outcomes in High Risk B-cell Acute Lymphoblastic Leukemia (HR B-ALL). The first part of the study includes the first two phases of therapy: Induction and Consolidation. This part will collect information on the leukemia, as well as the effects of the initial treatment, in order to classify patients into post-consolidation treatment groups. On the second part of this study, patients will receive the remainder of the chemotherapy cycles (interim maintenance I, delayed intensification, interim maintenance II, maintenance), with some patients randomized to receive inotuzumab. Other aims of this study include investigating whether treating both males and females with the same duration of chemotherapy maintains outcomes for males who have previously been treated for an additional year compared to girls, as well as to evaluate the best ways to help patients adhere to oral chemotherapy regimens. Finally, this study will be the first to track the outcomes of subjects with disseminated B-cell Lymphoblastic Leukemia (B LLy) or Mixed Phenotype Acute Leukemia (MPAL) when treated with B-ALL chemotherapy.

Peter Gordon
All
1 Year to 24 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03959085
STUDY00008473
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Inclusion Criteria:

• B-ALL and MPAL patients must be enrolled on APEC14B1 and consented to eligibility studies (Part A) prior to treatment and enrollment on AALL1732. Note that central confirmation of MPAL diagnosis must occur within 7 business days after enrollment for MPAL patients. If not performed within this time frame, patients will be taken off protocol.
• APEC14B1 is not a requirement for B-LLy patients but for institutional compliance every patient should be offered participation in APEC14B1. B-LLy patients may directly enroll on AALL1732.
• White blood cell count (WBC) criteria for patients with B-ALL (within 7 days prior to the start of protocol-directed systemic therapy):
• Age 1-9.99 years: WBC >= 50,000/uL
• Age 10-24.99 years: Any WBC
• Age 1-9.99 years: WBC < 50,000/uL with:
• Testicular leukemia
• CNS leukemia (CNS3)
• Steroid pretreatment.
• White blood cell count (WBC) criteria for patients with MPAL (within 7 days prior to the start of protocol-directed systemic therapy):
• Age 1-24.99 years: any WBC.
• Patient has newly diagnosed B-ALL or MPAL (by World Health Organization [WHO] 2016 criteria) with > 25% blasts on a bone marrow (BM) aspirate;
• OR If a BM aspirate is not obtained or is not diagnostic of acute leukemia, the diagnosis can be established by a pathologic diagnosis of acute leukemia on a BM biopsy;
• OR A complete blood count (CBC) documenting the presence of at least 1,000/uL circulating leukemic cells if a bone marrow aspirate or biopsy cannot be performed.
• Patient has newly diagnosed B-LLy Murphy stages III or IV.
• Patient has newly diagnosed B-LLy Murphy stages I or II with steroid pretreatment.
• Note: For B-LLy patients with tissue available for flow cytometry, the criterion for diagnosis should be analogous to B-ALL. For tissue processed by other means (i.e., paraffin blocks), the methodology and criteria for immunophenotypic analysis to establish the diagnosis of B-LLy defined by the submitting institution will be accepted.
• All patients and/or their parents or legal guardians must sign a written informed consent.
• All institutional, Food and Drug Administration (FDA), and NCI requirements for human studies must be met.
Exclusion Criteria:

• Patients with Down syndrome are not eligible (patients with Down syndrome and B-ALL are eligible for AALL1731, regardless of NCI risk group).
• With the exception of steroid pretreatment or the administration of intrathecal cytarabine, patients must not have received any prior cytotoxic chemotherapy for the current diagnosis of B-ALL, MPAL, or B-LLy or for any cancer diagnosed prior to initiation of protocol therapy on AALL1732.
• Patients who have received > 72 hours of hydroxyurea within one week prior to start of systemic protocol therapy.
• Patients with B-ALL or MPAL who do not have sufficient diagnostic bone marrow submitted for APEC14B1 testing and who do not have a peripheral blood sample submitted containing > 1,000/uL circulating leukemia cells.
• Patients with acute undifferentiated leukemia (AUL) are not eligible.
• For Murphy stage III/IV B-LLy patients, or stage I/II patients with steroid pretreatment, the following additional exclusion criteria apply:
• T-lymphoblastic lymphoma.
• Morphologically unclassifiable lymphoma.
• Absence of both B-cell and T-cell phenotype markers in a case submitted as lymphoblastic lymphoma.
• Patients with known Charcot-Marie-Tooth disease.
• Patients with known MYC translocation associated with mature (Burkitt) B-cell ALL, regardless of blast immunophenotype.
• Patients requiring radiation at diagnosis.
• Female patients who are pregnant, since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential.
• Lactating women who plan to breastfeed their infants while on study and for 2 months after the last dose of inotuzumab ozogamicin.
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of study participation. For those patients randomized to inotuzumab ozogamicin, there is a minimum of 8 months after the last dose of inotuzumab ozogamicin for females and 5 months after the last dose of inotuzumab ozogamicin for males.
Drug: Calaspargase Pegol-mknl, Drug: Cyclophosphamide, Drug: Cytarabine, Drug: Daunorubicin Hydrochloride, Drug: Dexamethasone, Drug: Doxorubicin Hydrochloride, Biological: Inotuzumab Ozogamicin, Drug: Leucovorin Calcium, Drug: Mercaptopurine, Drug: Methotrexate, Drug: Pegaspargase, Drug: Prednisolone, Other: Questionnaire Administration, Radiation: Radiation Therapy, Radiation: Radiation Therapy, Drug: Thioguanine, Drug: Vincristine Sulfate
B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Central Nervous System Leukemia, Mixed Phenotype Acute Leukemia, Testicular Leukemia
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Smart Use of Medication for the Treatment of Adolescent Severe Obesity (SMART)

We are studying the best time to add weight loss medication to diet and exercise for helping adolescents who carry extra weight. All participants start with a lifestyle modification program and some participants may also receive study medication. Participants must be 12-17 years of age and carry extra weight. The program will last for 48 weeks.

Claudia Fox
All
up to 18 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04007393
STUDY00006824
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Inclusion Criteria:

• Provision of signed and dated informed assent form;
• Provision of signed and dated informed parental consent form from at least 1 legal parent/guardian;
• Stated willingness to comply with all study procedures and availability for the duration of the study;
• BMI >/= 1.2 times the 95th percentile or BMI >/= 35 Kg/m2, whichever is lower;
• Tanner stage >/= 2;
• Male or female, aged 12-17 at time of consenting;
• For females of reproductive potential: when sexually active, agreement to use highly effective contraception (oral contraceptive pill, intra-uterine device (IUD), or implant) during study participation;
• For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner.
Exclusion Criteria:

• Contraindications to phentermine or topiramate use according to package inserts, including: history of glaucoma; current or recent (< 14 days) use of monoamine oxidase inhibitor; known hypersensitivity to sympathomimetic amines; current pregnancy, plans to become pregnant, or if sexually active refusal to use 2 forms of birth control; history of cardiac disease including coronary artery disease; clinically significant cardiac arrhythmias; heart failure or uncontrolled hypertension;
• Diabetes (type 1 or 2);
• Presence of cardiac pacemaker;
• Current or recent (<6 months prior to enrollment) use of weight loss medication(s);
• Current use of weight-altering medication(s) (e.g., atypical antipsychotic, metformin) unless dose has been stable for past 6 months;
• Current use of other sympathomimetic amine such as attention-deficit hyperactivity disorder (ADHD) stimulants;
• Seizure disorder (other than infantile febrile seizure);
• Previous bariatric surgery;
• Recent initiation of change in dose (< 3 months prior to enrollment) of anti-hypertensive or lipid medication(s);
• Tobacco use
• History of or current diagnosis of schizophrenia, psychosis, mania, chemical dependency;
• Unstable depression or anxiety that has required hospitalization in the past year;
• Any history of suicide attempt;
• Suicidal ideation or self-harm within 12 months prior to enrollment;
• Bicarbonate < 18 mmol/L;
• Creatinine > 1.2 mg/dL;
• History of cholelithiasis;
• History of nephrolithiasis;
• Untreated thyroid disorder;
• Hyperthyroidism;
• Breastfeeding
Behavioral: Lifestyle Modification Therapy (LSMT), Drug: Phentermine Pill, Drug: Topiramate Pill
Children's Health, Diabetes & Endocrine, Adolescent Obesity
Weight management, Clinics and Surgery Center (CSC)
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A Pilot Trial of UrApp, a Novel Mobile Application for Childhood Nephrotic Syndrome Management

We are studying a new phone app, UrApp, for parents (or caregivers) to use when managing the care of children who have been diagnosed with nephrotic syndrome in the last six weeks. We will look at medication and urine monitoring with two groups; one will use the app, the other will have usual care. Study participation will last for one year.

Michelle Rheault
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04075656
STUDY00006828
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Inclusion Criteria:

• Caregivers of patients ages 1-17 with steroid sensitive nephrotic syndrome (clinical diagnosis with edema, nephrotic range proteinuria [urine protein to creatinine ratio >2 mg/mg, or ≥ 300 mg/dL or ≥ 3+ protein on urine dipstick], and hypoalbuminemia ≤ 2.5 g/dL; resolution of proteinuria [negative/trace protein on urine dipstick] within 4 weeks of corticosteroid treatment)
• Caregivers of pediatric patients with steroid sensitive nephrotic syndrome diagnosed within 42 days at the time of enrollment
• Access to internet/wireless fidelity (Wi-Fi) in the home
• Caregiver proficiency with the English language
Exclusion Criteria:

• Caregivers of pediatric patients with end-stage kidney disease
• Caregivers of pediatric patients with renal transplantation
• Caregivers of pediatric patients with clinical or histologic evidence of secondary nephrotic syndrome (e.g., systemic lupus erythematosus)
Behavioral: Standard of Care, Behavioral: UrApp
Idiopathic Nephrotic Syndrome
Pediatrics, mHealth, Behavioral intervention
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MT2020-27: Phase I/II Trial Using E7777 to Enhance Regulatory T-Cell Depletion Prior to Tisagenlecleucel (Kymriah) Therapy for Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL)

This purpose of this study is to identify a safe dose level for the study drug, E7777, when given with standard tisagenlecleucel therapy (also known by its brand name, Kymriah, is an immunotherapy that is made from the participants own blood cells) in participants with Diffuse Large B-Cell Lymphoma (DLBCL). Up to three dose levels of E7777 will be tested.

Veronika Bachanova, MD
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04855253
STUDY00011895
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Inclusion Criteria:

• Diagnosis of a relapse or refractory (r/r) large B cell lymphoma, for which treatment with Kymriah is planned, including:
• diffuse large B-cell lymphoma (DLBCL) not otherwise specified,
• high grade B-cell lymphoma
• DLBCL arising from follicular lymphoma
• Considered at high risk for progression after CAR-T therapy by meeting one or more of the following factors:
• refractory to last line of therapy
• myc over expression >40% in any prior biopsy
• ≥2 sites of extranodal disease
• Received two or more lines of systemic therapy
• Has secured insurance coverage for Kymriah administration either in the outpatient or inpatient setting.
• Age 18 years or older at the time of signing consent.
• ECOG performance status of 0, 1, or 2
• Adequate bone marrow reserve defined as:
• Absolute neutrophil count (ANC) > 1,000/mm^3
• Platelets ≥ 50,000/mm^3 (transfusion support can be provided)
• Hemoglobin >8.0 mg/dl (transfusion support can be provided) Bone marrow involvement at disease assessment is an exclusion as these patients are at an increased risk of severe CRS and/or neurotoxicity
• Adequate organ function at enrollment and within 14 days of planned E7777 treatment including:
• renal function: eGFR ≥ 50 mL/min/1.73 m^2
• liver function: ALT ≤ 3 times the upper limit of normal (ULN) for age, AST ≤ 3 times the ULN, total bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN (if liver is involved by lymphoma, the exception are allowed upon approval of PI)
• albumin ≥ 3.0 g/dl
• Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea (CTCAE v5) and pulse oxygenation SpO2 > 91% on room air. Pulmonary function tests within 28 days of enrollment: >50% corrected DLCO and FEV1
• Hemodynamically stable and LVEF ≥ 50% confirmed by echocardiogram or MUGA
• Life expectancy ≥12 weeks in the opinion of the enrolling investigator as documented in the medical record
• Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use birth control for at least 30 days after study treatment or at least at least 4 months after the final dose of CY, whichever is longer Female participants: Two forms of birth control, one of which must be a barrier method, for example: use of intrauterine device (IUD) or oral contraceptives, plus a barrier method such as a condom, diaphragm or cervical cap Male participants: If possible to father a child (unless a successful vasectomy with confirmed azoospermia) participant and female partner, must use adequate contraception
• Written voluntary consent prior to the performance of any research related tests or procedures
Exclusion Criteria:

• Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to study enrollment to rule out pregnancy. All females will be considered to be of childbearing potential unless they are postmenopausal (amenorrheic for at least 12 consecutive months, in the appropriate age group, and without other known or suspected cause) or have been sterilized surgically (i.e., bilateral tubal ligation, total hysterectomy, or bilateral oophorectomy, all with surgery at least 1 month before dosing)
• Known bone marrow involvement, if history of bone marrow involvement must have a BM biopsy to rule-out current involvement
• Prior allogeneic transplant
• Ocular disease or complaints visual acuity impairment, color or shape distortion, or blurred vision - potential participants are required to have an ophthalmological examine as part of screening
• Known CNS involvement by malignancy - if clinically suspicious, must be ruled-out by examination of cerebrospinal fluid (CSF) by flow cytometry
• Uncontrolled active hepatitis B or hepatitis C
• Active or inactive HIV infection
• Untreated active bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to enrollment)
• History of heart failure or pulmonary edema, evidence of pleural effusion or active lower extremity edema
• Uncontrolled unstable angina and/or myocardial infarction within 3 months of enrollment
• Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
Drug: E7777
DLBCL, Diffuse Large B Cell Lymphoma, High-grade B-cell Lymphoma, DLBCL Arising From Follicular Lymphoma
Clinics and Surgery Center (CSC)
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Autonomic regulation of blood pressure in premature and early menopausal women

The goal of this study is to learn more about the effects of menopause on women's blood pressure and heart health. We are looking for women between the ages of 35 and 70 years to participate in the study. Participants may be pre- or postmenopausal; we are specifically interested in evaluating the influence of premature (< age 40 years) and early (< age 46 years) menopause.

Manda Keller-Ross
Female
35 Years to 70 Years old
This study is also accepting healthy volunteers
NCT04439370
STUDY00004979
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Inclusion Criteria:

• Aged 35-49 or 50-70 years of age who experienced premature (<40) or early (≤45) menopause
• Premenopausal 35-49 years of age
• Typical-age menopause (i.e., after 45 years of age), who are between 50-70 years old
• Menopause will be confirmed by subject report of amenorrhea for 12 months and serum FSH of >30 mIU/mL
Exclusion Criteria:

• Current nicotine/tobacco use within the past six months
• Are diabetic or asthmatic
• Have diagnosed significant carotid stenosis
• Have a history of significant autonomic dysfunction, heart disease, respiratory disease or a severe neurologic condition such as stroke or traumatic brain injury.
• Have existing metabolic or endocrine abnormities
• Take any heart/blood pressure medications that are determined to interfere with study outcomes
• IF the participant is premenopausal AND currently taking OC or other exogenous steroids that are determined to interfere with study outcomes
• Females who classify as having early or premature menopause AND are not willing to discontinue OC or MHT in order to complete the study
• Are pregnant or breastfeeding
Diagnostic Test: Microneurography to measure muscle sympathetic nerve activity (MSNA), Diagnostic Test: Baroreflex sensitivity testing, Diagnostic Test: Sympathoexcitatory Maneuvers, Diagnostic Test: Blood tests
Hypertension, Menopause, Premature, Menopause, Blood Pressure
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Care Improving Cognition for ADolescents on the Autism Spectrum (CICADAS)

This is a validation study to evaluate the acceptability, feasibility and impact of CICADAS (Care Improving Cognition for ADolescents on the Autism Spectrum), a clinician-assisted, mobile application that aims to prime the brain to engage in flexible, adaptive long-term learning about social-emotional events through closed-loop technology. The goal of this study is to evaluate the CICADAS app in adolescents with Autism Spectrum Disorder (ASD).

Suma Jacob
All
11 Years to 18 Years old
N/A
This study is NOT accepting healthy volunteers
NCT04562688
STUDY00010997
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Inclusion Criteria:

• Potential participant is between the age of 11 and 18 (inclusive) at the time of consent.
• Potential participant has a clinical diagnosis of Autism Spectrum Disorder (ASD), as confirmed by medical/clinical records or standardized assessments/interviews (e.g., Autism Diagnostic Observation Schedule, 2nd Edition (ADOS-2) or Autism Diagnostic Interview - Revised (ADI-R)).
• Potential participant has an IQ Score > 70 on the Wechsler Abbreviated Scale of Intelligence (WASI-II) or a comparable measure in medical/clinical records.
• Potential participant has normal or corrected to normal vision (20/20 or better; self/parent-reported.
• Potential participant has normal hearing (self/parent-reported).
• Potential participant is a fluent English speaker, based on participant and/or parent/legal guardian self-report and as determined by the screening clinician, to ensure reasonable neuropsychological results on key assessments.
• Potential participant has adequate sensorimotor capacity to perform the intervention and study activities, including visual capacity adequate to read from a computer screen or mobile device at a normal viewing distance, auditory capacity adequate to understand normal speech, and motor capacity adequate to control and use a mobile device and/or computer, based on participant and/or parent/legal guardian self-report and as determined by the screening clinician and/or study team.
• Potential participant must be clinically stable as a result of therapy or medication regimen for 4 weeks prior to enrolling into the study.
• Potential participant has reliable access to the internet.
Exclusion Criteria:

• Potential participant has history of psychotic disorders and/or seizure disorder and/or seizure episodes within the last 2 years.
• Potential participant has a motor/perceptual handicap that prevents digital device use, as determined by the screening clinician and/or study team.
• Potential participant has problems in performing assessments or comprehending or following spoken instructions, as determined by the screening clinician and/or study team.
• Potential participant has medical illnesses/genetic syndromes deemed to interfere with participation in study activities and/or unstable and/or untreated conditions that may affect cognition, including substance abuse/dependence disorders, ongoing chemotherapy or other cancer treatment.
• Potential participant has a history of head trauma, traumatic brain injury, or other neurological disorder that impairs cognition
• Potential adult participant scores less than a 14 (75%) on the University of California, San Diego Brief Assessment of Capacity to Consent (UBACC). Please note, this criteria applies only to adult participants, age 18, at the time of screening.
Other: CICADAS and then PEERS, Other: PEERS + CICADAS and then no-contact, Other: PEERS + Active Comparator and then no-contact
Autism Spectrum Disorder
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Stress Response and Opioid Dysfunction in Nicotine Dependence

This study includes healthy adults between 18-70 years old who are either non-smokers or cigarette smokers interested in quitting. The purpose of this study is to learn more about how people respond to stress and to taking one dose of a widely and safely used drug called naltrexone as well as to learn about how these responses relate to whether or not someone smokes, smoking cessation, and smoking relapse.

Mustafa al'Absi
18 Years and over
NA
This study is NOT accepting healthy volunteers
DMED-2019-28512
STUDY00008687
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Inclusion Criteria:

• Live in Minnesota.
• Between 18-70 years old.
• Generally healthy.
• Want to quit using tobacco and nicotine.
• Are willing to attend up to 11 online (videoconference) study visits over a period of approximately 4 months (though you may be asked to complete the last visits over a period of up to 1 year).
Exclusion Criteria:

• Do not live in Minnesota.
• Not between 18-70 years old.
• Not willing to attend to up to 11 online (videoconference) study visits over a period of approximately 4 months.
Heart & Vascular, Mental Health & Addiction, Prevention & Wellness
cigarette, nicotine, quit smoking, smoker, smoking, smoking cessation, stress, tobacco
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