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A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
The main purpose of this study is to check how safe the study drug is and how well your body handles taking it. The study will also check: • if the study drug works to improve your kidney function • whether the study drug has an impact on your daily life • the amount of the study drug in your blood over a period of time (called pharmacokinetics)
Michelle Rheault
STUDY00015869
Inclusion Criteria:
All Patients:
Patient is able to communicate well with the investigator, understands and is willing to comply with all requirements of the study, and understands and signs the written informed consent form (ICF).
For children to be eligible, one or both parents/legal guardians must sign a parental permission form which provides information contained in the ICF. Children capable of assent must express their willingness to participate by signing an assent form.
If patient has received a COVID vaccination, the baseline visit must occur at least one week or more after the second/booster vaccination.
Patients who have had active symptoms of COVID within 3 months prior to screening and are now asymptomatic for the last 2 weeks but have tested COVID PCR positive. If a patient is asymptomatic at screening but is COVID positive, then rescreening can occur after a minimum of two weeks.
Both female patients, as well as, female partners of male patients who are of child-bearing potential must be willing to not become pregnant for the complete duration of the study (>180 days) (90 days after the last dose of study medication).
Males (including sterilized subjects) whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 90 days after the last dose of study drug. They must agree to immediately inform the investigator if their partner becomes pregnant during the study.
Alport Syndrome Patients Inclusion Criteria (in addition):
Males and females with X-Linked AS and males and females with autosomal inherited AS.
For countries that are enrolling pediatric patients: patients from age 12 years and older.
For countries that are not enrolling pediatric patients: patients from age 18 years and older.
Confirmed diagnosis of AS by genetic testing and /or kidney biopsy. For patients enrolled in the US who meet all inclusion and exclusion criteria but have not had their diagnosis confirmed by genetic testing or kidney biopsy, the Sponsor will provide for patient's genetic testing.
UPCR ≥1.0 g/g.
eGFR ≥ 45 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
ACEi/ARB therapy at maximum tolerated dose stable for at least 4 weeks prior to screening. ACEi/ARB dose should remain stable over the course of the study.
Focal Segmental Glomerulosclerosis Patients Inclusion Criteria (in addition):
Male or female patients,
For countries that are enrolling pediatric patients: 12 to 75 years old at the time of signing the informed consent
For countries that are not enrolling pediatric patients: 18 to 75 years old at the time of signing the informed consent
Primary FSGS, (without any identifiable cause, and where the FSGS is confirmed by renal biopsy) or FSGS where there is documentation of a genetic mutation in a podocyte protein associated with FSGS.
Steroid-resistance defined as failure to achieve partial or complete remission, or experienced adverse events without acceptable clinical benefit after at least 8 weeks of adequate corticosteroid therapy for children and 12 weeks for adults.
UPCR between 3.5g/g and 12.0g/g.
eGFR > 45 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
If taking concomitant ACEi and/or ARB treatment, it should remain at a stable dose for a minimum of 28 days prior to enrollment and during the course of the study.
Exclusion Criteria:
All Patients:
Uncontrolled diabetes mellitus as evidenced by an HbA1c ≥ 11%.
Uncontrolled hypertension
Adults: (SBP ≥ 180mmHg and/or DBP ≥ 100mmHg).
Children: ≥ 95th percentile or ≥ 130/80 mm Hg, whichever is lower
Moderate or severe hepatic impairment (Child-Pugh B or C), except if (a) decreased serum albumin is directly related to the renal disease (resulting in a Child Pugh score of 7), and (b) no other Child-Pugh Score parameters are increased and (c) patient has no liver pathology in medical history.
Presence of any active (i.e., with symptoms) and/or uncontrolled infection (including COVID).
Presence of Human immunodeficiency virus (HIV).
BMI > 40.
History of malignancy other than treated basal cell or squamous cell skin cancer within the past 5 years.
History of alcohol abuse in the last 5 years or currently drinks in excess of 21 and 14 units per week for males and females, respectively.
Received an investigational agent within 30 days or 5 half-lives prior to screening (whichever is longer).
History of non-compliance such that patient is unlikely to be compliant with study visits, procedures or drug administration.
Patient has had an organ transplant, is currently on an organ transplant waiting list or there is a reasonable possibility that the patient will have an organ transplant in the 6 months after screening.
Participation in an interventional trial within the previous 3 months prior to screening or concurrent participation in a research trial.
Patient is not suitable to participate in the study for any reason (including, but not limited to co-morbidities, history of non-compliance with study visits, procedures, or drug administration) in the opinion of the investigator.
Females of childbearing potential (those who are not surgically sterilized or post-menopausal for at least 1 year) are excluded from participation in the study unless they agree to use highly effective contraception.
Females that are lactating.
History of hypersensitivity to study drug and/or any of its excipients.
Patients with hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption.
Required concomitant use of bardoxolone, rituximab, cyclo-phosphamide, abatacept, or sparsentan
Alport Syndrome Patients Exclusion Criteria (in addition):
Kidney disease apart from AS, e.g. diabetic nephropathy or lupus nephritis.
Use of Bardoxolone or sparsentan treatment in the 30 days prior to screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for at least 3 months prior to screening.
Focal Segmental Glomerulosclerosis Patients Exclusion Criteria (in addition):
Patient has collapsing variant of FSGS on renal biopsy.
Patient has FSGS secondary to another condition (e.g. obesity, cardiovascular, infectious, or autoimmune disorder).
Use of Rituximab, cyclophosphamide or abatacept treatment in the 120 days prior to screening. If taking other chronic immune-modulatory medications that are small molecules, the dosage must be stable for 4 weeks prior to screening.
If previous Rituximab treatment is greater than 120 days from screening, CD20 cell count should be within normal limits.
If previous other antibody treatment on a stable dose is greater than 120 days from screening, the investigator must deem administration of study drug to be safe.
Use of sparsentan in the 30 days prior to screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for at least 3 months prior to screening. Clinics and Surgery Center (CSC)
A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects with Propionic or Methylmalonic Acidemia Followed by a 6-Month, Randomized, Double-blind, Placebo-controlled, 2-period Crossover and an Open-label, Long-term Extension (HERO)
Sue Berry
STUDY00010175
Inclusion Criteria:
• 2 years and older
• confirmed diagnosis of symptomatic PA or MMA (Mutase) . inadequate metabolic control while receiving standard of care
• plasma MCA concentration more than 3 times upper limit of normal
• stable supplementation dose of carnitine for at least 1 week
Exclusion Criteria:
• moderate-to-severely impaired cardiac function
• clinically significant heart arrhythmia
• moderate to severe chronic kidney disease
• exposure to any investigational therapy, apart for a COVID-19 vaccine, within the past 6 months
• exposure to gene therapy for PA or MMA at any time
• history of organ transplantation
Children's Health, Rare Diseases
Clinics and Surgery Center (CSC)
A Phase 1b Open-Label Multicenter Study of OP-1250 in Combination with the CDK4/6 Inhibitor Ribociclib or with the PI3K Inhibitor Alpelisib in Adult Subjects with Advanced and/or Metastatic HR Positive, HER2 Negative Breast Cancer
The main purpose of this study is to look at how safe and well tolerated the study drug is in combination with ribociclib (Group 1) or alpelisib (Group 2), the levels of the study drug and ribociclib or alpelisib in your blood, and how your body and your cancer respond.
David Potter
STUDY00019201
Inclusion Criteria:
• at least 18 years old
• diagnosis of advanced and/or Metastatic HR Positive, HER2 Negative Breast Cancer
• received no more than 2 prior hormonal regimens for advanced or metastatic disease
• received no more than 1 prior chemotherapy for locally advanced or metastatic breast cancer
Exclusion Criteria:
• significant heart disease
• cerebral vascular disease within 6 months
• pulmonary embolism, or deep venous thrombosis within the last 6 months
• pneumonitis or interstitial lung disease
• history or ongoing gastrointestinal disorders that result in poor absorption of medications
• history of significant liver disease
• study staff will review medical history
Cancer
Clinics and Surgery Center (CSC), alpelisib, Breast Cancer, HER2-neg breast cancer, HR-positive Breast Cancer, Metastatic Breast cancer, ribociclib
A Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety and Tolerability of RO7017773 in Participants Aged 15 to 45 Years with Autism Spectrum Disorder (ASD)
Suma Jacob
STUDY00010423
Inclusion Criteria:
• diagnosis of Autism Spectrum Disorder (ASD)
• Body mass index between 18.5 to 40 kg/m2
• women willing to abstain from sexual activity or use highly effective birth control for the duration of the study and 28 days after the last dose of the study drug
• hearing, vision, and language skills are compatible with the study requirements
• current treatment regimens stable for 8 weeks prior to study
Exclusion Criteria:
• women who are pregnant or breast feeding or with to become pregnant
• significant psychiatric and/or neurological disorder including alcohol or substance abuse disorder
• history of cancer (except non-melanoma skin cancer or cervical cancer in situ)
• donation or loss of blood over 500 mL in adults and 250 mL in adolescents in past 3 months
• other exclusions apply (study staff will review)
Mental Health & Addiction
ASD, Autism, Autism Spectrum Disorder
A Phase III, adjudicator-blinded, randomised study to evaluate the efficacy and safety of treatment with olorofim versus treatment with AmBisome? followed by standard of care (SOC) in patients with invasive fungal disease (IFD) caused by Aspergillus species (OASIS)
This is a Phase III, randomised, adjudicator-blinded, multicentre, active comparator, parallel, two-arm study to evaluate the efficacy and safety of treatment with olorofim versus treatment with AmBisome® followed by SOC in ITT patients with proven IA at any site or probable lower respiratory tract disease (LRTD) IA where: (i) Therapy with a mould-active azole is inappropriate OR a non-azole antifungal is required, AND (ii) For whom the site Investigator agrees that therapy with AmBisome® at 3 mg/kg/day followed by SOC would be an appropriate treatment. Approximately 225 patients (150 to receive olorofim and 75 to receive AmBisome®) will be enrolled at approximately 100 centres globally over a period of approximately 30 months. The study is comprised of a screening period, a treatment period of up to 84 days (± 7 days), and a safety follow-up (FU) period of 4 weeks ± 7 days. The maximum duration of the study for any individual patient is approximately 18 weeks
Jo-Anne Young, MD
STUDY00019092
Inclusion Criteria:
• over 18 years old
• weigh more than 40 kg (88 pounds)
• Invasive Aspergillosis (IA) at any site
• require therapy with an antifungal agent other than a mold-active azole
Exclusion Criteria:
• women who are pregnant or breastfeeding
• known history of allergy, hypersensitivity, or any serious reaction to any component of the study drug
• people with chronic aspergillosis, aspergilloma, or allergic bronchopulmonary aspergillosis
• human immunodeficiency virus (HIV) infection but not currently receiving antiretroviral therapy
• certain heart and liver conditions (study staff will review)
Infectious Diseases, Respiratory System
Aspergillosis, IA, Invasive Aspergillosis (IA), Lung Infection
HM2021-31: A Phase 1b Open-Label Study to Evaluate the Safety and Anti-cancer Activity of Loncastuximab Tesirine in Combination with Other Anti-cancer Agents in Patients with Relapsed or Refractory B-cell Non-Hodgkin Lymphoma (LOTIS-7)
This protocol aims to characterize the safety and tolerability of loncastuximab tesirine in combination with gemcitabine, lenalidomide, polatuzumab vedotin, or umbralisib, and to identify the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) for any of the combinations in subjects with relapsed or refractory B-cell Non-Hodgkin Lymphoma. This project aims to address the resistance mechanisms to single agent therapies and enhance efficacy by engaging different targets, in synergistic or additive manner.
Marie Hu
STUDY00015805
Inclusion Criteria:
Male or female participant aged 18 years or older
Pathologic diagnosis of relapsed (disease that has recurred following a response) or refractory (disease that failed to respond to prior therapy) B-NHL (2016 World Health Organization classification) who have failed, or been intolerant to any approved therapy and had received at least two systemic treatment regimens in dose-escalation part; and at least one systemic treatment regimen in dose-expansion part
DLBCL (including transformed diseases, but for Arms E and F, including transformed FL only)
HGBCL
FL
MZL
MCL (for Arm C only)
BL (for Arm C only)
Life expectancy of at least 24 weeks according to Investigator's judgement
Need of systemic treatment for any of the listed indications as assessed by the investigator, including indolent B-NHLs (e.g. FL and MZL)
Measurable disease as defined by the 2014 Lugano Classification
Availability of formalin-fixed paraffin-embedded tumor tissue block
ECOG performance status 0 to 2
Adequate organ function
Women of childbearing potential (WOCBP) must agree to use a highly effective method of contraception from the time of giving informed consent until at least 10 months after the last dose of loncastuximab tesirine. Men with female partners who are of childbearing potential must agree to use a condom when sexually active or practice total abstinence from the time of giving informed consent the first dose until at least 7 months after the last dose of loncastuximab tesirine. Men must refrain from donating sperm during this same period. For the arm that includes glofitamab, WOCBP must agree to use contraceptive methods that result in a failure of <1% per year or remain abstinent (refrain from heterosexual intercourse) during the treatment period and for at least 18 months after pretreatment with obinutuzumab. For the arm that includes mosunetuzumab, WOCBP must agree to use contraceptive methods that result in a failure of <1% per year or remain abstinent (refrain from heterosexual intercourse) during the treatment period and for at least 3 months after the final dose of mosunetuzumab and tocilizumab (if applicable).
Exclusion Criteria:
Known history of hypersensitivity resulting in treatment discontinuation to or positive serum human ADA to a CD19 antibody
Previous therapy with loncastuximab tesirine
Previous treatment with polatuzumab vedotin, glofitamab or mosunetuzumab (applied to relevant arm and/or cohort of the specific drug administered)
Participants who received previous treatment of polatuzumab vedotin containing regimen will be excluded from Arm C
Participants who received previous treatment of glofitamab containing regimen will be excluded from Arm E
Participants who received previous treatment of mosunetuzumab containing regimen will be excluded from Arm F
Allogenic or autologous stem cell transplant within 60 days prior to start of study drug (C1 D1)
Human immunodeficiency virus (HIV) seropositive
Serologic evidence of chronic hepatitis B virus (HBV) infection and unable or unwilling to receive standard prophylactic antiviral therapy or with detectable HBV viral load
Serologic evidence of hepatitis C virus (HCV) infection without completion of curative treatment or with detectable HCV viral load
History of confirmed progressive multifocal leukoencephalopathy
History of Stevens-Johnson syndrome, toxic epidermal necrolysis, or macrophage activation syndrome (MAS)/hemophagocytic lymphohistiocytosis (HLH)
Lymphoma with active central nervous system (CNS) involvement at the time of screening, including leptomeningeal disease
Clinically significant third space fluid accumulation (i.e., ascites requiring drainage or pleural effusion that is either requiring drainage or associated with shortness of breath)
Breastfeeding or pregnant
Significant medical comorbidities
Major surgery, radiotherapy, chemotherapy, or other anti-neoplastic therapy, within 14 days prior to start of study drugs (C1 D1), except shorter if approved by the Sponsor
Live vaccine within 4 weeks prior to C1D1
Failure to recover to Grade ≤1 (Common Terminology Criteria for Adverse Events [CTCAE] version 5.0) from acute non-hematologic toxicity (Grade ≤2 alopecia) due to previous therapy prior to screening
Active second primary malignancy other than non-melanoma skin cancers, non-metastatic prostate cancer, in situ cervical cancer, ductal or lobular carcinoma in situ of the breast, or other malignancy that the Sponsor's medical monitor and Investigator agree and document should not be exclusionary
Extra Exclusion Criteria for Arms E (includes glofitamab) and F (includes mosunetuzumab) Note: as applicable, the arm-specific exclusion criteria may supersede the general ones, such as stem cell transplant.
Prior allogeneic stem cell transplant and solid organ transplant
Autologous stem cell transplant within 100 days prior to C1D1
History of CNS lymphoma or leptomeningeal infiltration
Current or history of CNS disease, such as stroke, epilepsy, CNS vasculitis, or neurodegenerative disease
Known active infection, reactivation of a latent infection, whether bacterial, viral, fungal, mycobacterial, or other pathogens (excluding fungal infections of nail beds), or any major episode of infection requiring hospitalization or treatment with intravenous (IV) antibiotics within four weeks prior to C1D1
Active or history of autoimmune disease or immune deficiency, including but not limited to, myasthenia gravis, myositis, autoimmune hepatitis, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, antiphospholipid antibody syndrome, Wegener granulomatosis, Sjögren syndrome, Guillain Barré syndrome, or multiple sclerosis, with certain exceptions
Prior treatment with anti-cancer/lymphoma targeted therapies (e.g., tyrosine kinase inhibitors, systemic immunotherapeutic/immunostimulating agents, including, but not limited to, cluster of differentiation 137 agonists or immune checkpoint blockade therapies, including anti-cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4), anti-programmed cell death protein 1 (PD1), and anti-programmed death ligand 1 (PDL1) therapeutic antibodies, radio-immunoconjugates, ADCs, immune/cytokines and monoclonal antibodies) or treatment with systemic immunosuppressive medication (including, but not limited to, cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor agents) within 4 weeks or five half-lives of the drug, whichever is shorter, prior to C1D1, or anticipation of need for systemic immunosuppressive medication during study treatment, with certain exceptions
Prior treatment with chimeric antigen receptor T-cell therapy within 30 days prior to C1D1
Toxicities from prior anti-cancer therapy including immunotherapy that did not resolve to ≤ Grade 1 with the exception of alopecia, endocrinopathy managed with replacement therapy and stable vitiligo
Any history of immune-related Grade ≥3 AE with the exception of endocrinopathy managed with replacement therapy
Ongoing corticosteroid use >25 mg/day of prednisone or equivalent within 4 weeks prior and during study treatment
Administration of a live attenuated vaccine within 4 weeks prior to the first dose of study treatment or anticipation that such a live attenuated vaccine will be required during the study or within 5 months after last dose of study treatment
Extra Exclusion Criteria for Arm E (includes glofitamab) only.
• Known history of hypersensitivity to obinutuzumab Clinics and Surgery Center (CSC)
NRG-GY026: A Phase II/III Study of Paclitaxel/Carboplatin Alone or Combined with either Trastuzumab and Hyaluronidase-Oysk (Herceptin Hylecta) or Pertuzumab, Trastuzumab, and Hyaluronidase-Zzxf (Phesgo) in HER2 Positive, Stage I-IV Endometrial Serous Carcinoma or Carcinosarcoma
Britt Erickson
STUDY00017234
Inclusion Criteria:
• HER2+ tumor
• have not had chemotherapy for treatment of this cancer
• surgery (or biopsy if surgery isn't being done) no more than 8 weeks prior
Exclusion Criteria:
• radiation therapy used to treat the tumor
• history of serious heart or lung disease
• breast feeding and unwilling to stop
Cancer, Women's Health
endometrial carcinosarcoma, Endometrial Serous Adenocarcinoma, Clinics and Surgery Center (CSC)
A Phase 1/2 Study of the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Relatlimab Plus Nivolumab in Pediatric and Young Adult Participants with Recurrent or Refractory Classical Hodgkin Lymphoma and Non-Hodgkin Lymphoma Protocol Number: CA224069 (RELATIVITY-069)
CA224069 is an open-label, Phase 1/2 clinical trial of relatlimab + nivolumab in children, adolescents and young adults with Recurrent or Refractory Classical Hodgkin Lymphoma (R/R cHL) and Non-Hodgkin Lymphoma (NHL). Part A will encompass safety and dose determination of relatlimab + nivolumab. Part B will be composed of an expansion cohort of cHL (Cohort 1) and an exploratory assessment in NHL (Cohort 2).
Peter Gordon
STUDY00015731
Inclusion Criteria:
Pathologically confirmed high-risk recurrent/relapsed or refractory (R/R) classical Hodgkin lymphoma (cHL), after non-response to or failure of first-line standard therapy prior to a definitive therapy e.g.high-dose chemotherapy/autologous stem cell transplant (HDCT/ASCT)
Participants with pathologically confirmed R/R NHL after failure or non-response to second line therapy, including but not limited to primary mediastinal B-cell lymphoma, diffuse large B-cell lymphoma (DLBCL), mediastinal gray zone lymphoma (MGZL), anaplastic large cell lymphoma (ALCL), or peripheral T-cell lymphoma (PTCL).
Participants must have measurable PET positive disease in both cHL and NHL cohorts.
Exclusion Criteria:
Aggressive B-cell lymphomas subtypes including Burkitt lymphoma (BL), lymphoblastic lymphoma, and NK/T-cell lymphoma/leukemia.
Primary CNS lymphoma of the brain or spinal cord, and secondary CNS lymphoma (ie, from systemic non-Hodgkin lymphoma) involving the brain, spinal cord, or with leptomeningeal seeding.
Prior treatment with an anti-cytotoxic T-lymphocyte-associated protein 4 (anti-CTLA-4) antibody, or any other antibody or drug specifically targeting T-cell co-stimulation or checkpoint pathways, with the exception of anti-PD(L)-1 targeted therapies
Prior treatment with lymphocyte activation gene-3 (LAG-3)-targeted agents
Prior autologous stem cell transplantation (HDCT/ASCT)
History of allogeneic bone marrow transplantation.
Other protocol-defined inclusion/exclusion criteria applyVX21-522-001: A Phase 1 Single Dose Escalation Study Evaluating the Safety and Tolerability of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
This is a clinical research study exploring the safety and tolerability of a single dose of VX-522 for people with cystic fibrosis (CF) who are not expected to benefit from CFTR modulators.
Joanne Billings
STUDY00016034
Inclusion Criteria:
• 18 to 65 years old
• Stable cystic fibrosis disease
• FEV1 at least 40%
• Specific CFTR gene mutations
Exclusion Criteria:
• Uncontrolled asthma in the last year
• Oxygen saturation without oxygen therapy is >94%
• Severe liver disease
Rare Diseases, Breathing, Lung & Sleep Health
Cystic Fibrosis, CF
MT2022-41 A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises (BEACON Trial) (BEACON)
To determine the safety and clinical efficacy of a single dose of autologous gene--edited CD34+ hematopoietic stem cell and progenitor cells (BEAM-101) in patients with severe SCD
Ashish Gupta
STUDY00017341
Key Inclusion Criteria Include:
Age ≥18 years to ≤35 years for the initial sentinel cohort; for subsequent enrollment patients from ≥12 years up to ≤35 years may be enrolled only upon approval by FDA.
Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures
Key Exclusion Criteria Include:
HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
Available and willing matched sibling donor
Definitive diagnosis of moyamoya syndrome based on screening brain MRA
History of overt stroke
Rare Diseases
Clinics and Surgery Center (CSC)
MT2020-08 A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate the Safety and Clinical Activity of PBCAR0191 in subjects with Relapsed/Refractory Non-Hodgkin Lymphoma and r/r B-cell Acute Lymphoblastic Leukemia
To evaluate the safety and tolerability of PBCAR0191 in subjects with r/r B-ALL and r/r NHL and find an appropriate dose to optimize safety and efficacy. To evaluate the clinical benefit of PBCAR0191 in subjects with r/r B-ALL and r/r NHL. To evaluate the clinical activity of PBCAR0191 in subjects with r/r B-ALL and r/r NHL.
Joseph Maakaron
STUDY00009953
Key Inclusion Criteria*
Criteria for B-ALL:
Subject has unequivocal r/r CD19+ B-ALL that has been confirmed by morphology, flow cytometry, or a validated minimal residual disease assay.
Subjects with Philadelphia chromosome positive disease can be eligible if they are intolerant to tyrosine kinase inhibitor therapy or if they have r/r disease.
Criteria for NHL:
Subject has unequivocal aggressive CD19+ r/r B-cell NHL that is confirmed by archived tumor biopsy tissue from last relapse after CD19-directed therapy and corresponding pathology report. Alternatively, if at least 1 tumor involved site is accessible at time of Screening, the subject's diagnosis is confirmed by pretreatment biopsy (excisional when possible) or by flow cytometry of fine needle aspirate. If a subject never had a CR, a sample from the most recent biopsy is acceptable. NHL subtypes included but are not limited to:
Diffuse large B-cell lymphoma (DLBCL) including Richter's transformation
FL including Grade 3 or transformed FL
High-grade B-cell lymphoma
Primary mediastinal lymphoma
Subject has measurable or detectable (for example positron emission tomography-positive) disease according to the Lugano Classification.
Subject must have received at least 2 prior chemotherapy-containing regimens, consistent with standard of care treatment guidance (e.g., NCCN), unless no second line therapy of known benefit exists for a given subject. Other than those specifically prohibited, other therapies are allowed until 7 days prior to initiation of LD. In that case, all Screening safety laboratories and disease assessments must be performed after the last dose of prior therapy. For Richter's transformation, only 1 prior line of therapy is required for the DLBCL component.
Subject has received no more than 7 systemic lines of anti-cancer therapy for the disease under study.
Subjects previously treated with CD19-directed autologous CAR T therapies have received no more than 2 lines of therapy after administration of their previous CAR T product.
Expansion cohort only: Subjects must have received autologous CD19-directed CAR T therapy and demonstrated clinical response to the treatment at Day 28 or later, followed by relapse.
Criteria for both B-ALL and NHL:
Eastern Cooperative Oncology Group performance status score of 0 or 1.
An estimated life expectancy of at least 12 weeks according to the investigator's judgment.
Seronegative for human immunodeficiency virus antibody (i.e., intact immune function).
Subject has adequate bone marrow, renal, hepatic, pulmonary, and cardiac function defined as:
Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 (calculated using the CKD-EPI equation [Levey et al, 2009]). If there is a concern that eGFR calculation is not an accurate reflection of renal function, a 24-hour urine collection for creatinine clearance may be used at the investigator's discretion.
Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels both ≤3 times of upper limit of normal (ULN), unless there is suspected disease in the liver.
Total bilirubin <2.0 mg/dL, except in subjects with Gilbert's syndrome.
Platelet count ≥50,000/μL and absolute neutrophil count of ≥1000/ μL. Platelet transfusions within 14 days of screening are not allowed except for subjects in B-ALL disease cohort with extensive bone marrow disease burden, in which case adequate bone marrow recovery after prior treatment is required to be documented.
C-reactive protein (CRP) <2x ULN; subjects with CRP elevation within 2x ULN, ruling out infectious cause will be required.
Left ventricular ejection fraction >45% as assessed by echocardiogram (ECHO) or multiple gated acquisition scan performed within 1 month before starting lymphodepleting chemotherapy. ECHO results performed within 6 months before Screening and at least 28 days after the last cancer treatment may be acceptable if the subject has not received any treatment with cardiotoxicity risks.
No clinically significant evidence of pericardial effusion or pleural effusion causing clinical symptoms and needing immediate intervention, based on the investigator's opinion. Any known effusion must be stable without need for drainage within 2 weeks of enrollment.
No clinically significant renal/pulmonary comorbidities.
Baseline oxygen saturation >92% on room air.
Key Exclusion Criteria*
Criteria for B-ALL:
Burkitt cell (L3 ALL) or mixed-lineage acute leukemia.
No active central nervous system (CNS) disease. Subjects with a history of CNS involvement must have a documented CR on at least 2 imaging studies at least 3 months apart (with no masses in parenchyma and no ocular involvement) and a negative cerebrospinal fluid cytology on at least 2 evaluations (one evaluation may be during the Screening Period and the other must be at least 3 months prior).
Criteria for NHL:
No prior or active CNS disease.
Requirement for urgent therapy due to tumor mass effects such as bowel obstruction or blood vessel compression.
Active hemolytic anemia.
Criteria for B-ALL and NHL:
Subject has had a malignancy, besides the malignancies of inclusion (B-ALL or NHL), that in the investigator's opinion, has a high risk of relapse in the next 2 years. In the case of Richter's transformation, subjects may be enrolled with ongoing chronic lymphocytic leukemia/small lymphocytic lymphoma.
Uncontrolled and serious fungal, bacterial, viral, protozoal, or other infection that has not resolved and does require therapeutic anti-microbial medications at least 7 days prior to LD. Subjects with elevated CRP must undergo infectious disease workup and the recommendations discussed with medical monitor to be considered on an individual basis. The CRP must be trending toward the normal range for the laboratory with the exception when it's deemed related to the underlying malignancy.
Any form of primary immunodeficiency (e.g., severe combined immunodeficiency disease).
Active hepatitis B or hepatitis C confirmed by PCR. Subject positive for inactive hepatitis B is allowed to enroll if on prophylactic treatment.
Subject is seropositive for hepatitis B antigen with confirmation. If confirmatory tests are negative, the subject can be enrolled.
Subject is seropositive for hepatitis C antibody unless antigen negative. If hepatitis C antibody test is positive, the subject must be tested for the presence of RNA by reverse transcription PCR and be hepatitis C virus-RNA negative.
Any known uncontrolled cardiovascular disease at the time of Screening that, in the investigator's opinion, renders the subject ineligible, including but not limited to:
Active ventricular or atrial dysrhythmia ≥ Grade 2, bradycardia ≥ Grade 2.
Myocardial infarction within 6 months before Screening.
Pulmonary embolism, deep vein thrombosis, or any other significant coagulopathy including disseminated intravascular coagulation.
History of hypertension crisis or hypertensive encephalopathy within 3 months prior to Screening. In case of hypertensive crisis caused by omission of well-established treatment regimen, transient and promptly stabilized, enrollment must be discussed and agreed upon with sponsor and medical monitor.
History of severe immediate hypersensitivity reaction to any of the agents used in this study.
Presence of a CNS disorder that, in the opinion of the investigator, renders the subject ineligible for treatment.
Abnormal findings during the Screening Period or any other medical condition(s) or laboratory findings that, in the opinion of the investigator, might jeopardize the subject's safety.
History of concomitant genetic syndrome such as Fanconi anemia, Kostmann syndrome, Shwachman-Diamond syndrome, or any other known bone marrow failure syndrome.
Active uncontrolled autoimmune disease requiring active immunosuppression at the time of Screening (excluding subjects needing steroids for physiologic replacement).
Subject has received stem cell transplant within 90 days before Screening.
Subject has active GvHD symptoms.
Subject has received systemic biologic agent for treatment of disease under study within 28 days of LD or other systemic anti-cancer therapy within 10 days of LD Note: this criterion does not apply if the subject has clear evidence of disease progression after such an agent has been administered and all AEs have resolved to a Grade 2 or less in severity. This should be discussed with the medical monitor for confirmation.
Participation in noninterventional registries or epidemiological studies is not excluded.
Radiotherapy within 4 weeks before Screening should be discussed with monitor and determined on a case-by-case basis.
Presence of pleural/peritoneal/pericardial catheter, as well as biliary and ureteral stents (does not apply to intravenous lines).
Subject has received live vaccine within 4 weeks before Screening. Non-live virus vaccines are not excluded.
Subject has received CD19-directed therapy other than autologous CD19-directed CAR T therapy within 90 days of the anticipated start date of LD.
Additional criteria apply
COG ACNS1833 - A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
The overall goal of this phase 3 non-inferiority study is to assess if selumetinib works as well as the standard treatment using carboplatin and vincristine (called CV) for subjects with low-grade glioma (LGG).
Christopher Moertel, MD
STUDY00009277
Inclusion Criteria:
Patients must be >= 2 years and =< 21 years at the time of enrollment
Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
Patients must have non-neurofibromatosis type 1 (non-NF1) low-grade glioma (LGG) without a BRAFV600E mutation as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1 (NCT02402244) and that has not been treated with any modality besides surgery. Note: Patients may be newly-diagnosed OR previously diagnosed, and there is no required time frame between biopsy/surgery and treatment initiation.
Patients with residual tumor after resection or progressive tumor after initial diagnosis (with or without surgery) who have not received treatment (chemotherapy and/or radiation) are eligible
Patients must have two-dimensional measurable tumor >= 1 cm^2 to be eligible
Eligible histologies will include all tumors considered low-grade glioma or low-grade astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
Patients with metastatic disease or multiple independent primary LGG are eligible
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender as follows (performed within 7 days prior to enrollment):
Age: Maximum Serum Creatinine (mg/dL)
2 to < 6 years: 0.8 mg/dL (male); 0.8 mg/dL (female)
6 to < 10 years: 1 mg/dL (male); 1 mg/dL (female)
10 to < 13 years: 1.2 mg/dL (male); 1.2 mg/dL (female)
13 to < 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)
>= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (performed within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect [unconjugated] bilirubin levels as long as their direct [conjugated] bilirubin is < 3.1 mg/dL)
Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (performed within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
Albumin >= 2 g/dL (performed within 7 days prior to enrollment)
Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (performed within 4 weeks prior to enrollment)
Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (performed within 4 weeks prior to enrollment)
Absolute neutrophil count >= 1,000/uL (unsupported) (performed within 7 days prior to enrollment)
Platelets >= 100,000/uL (unsupported) (performed within 7 days prior to enrollment)
Hemoglobin >= 8 g/dL (may be supported) (performed within 7 days prior to enrollment)
Patients with a known seizure disorder should be stable and should not have experienced a significant increase in seizure frequency within 2 weeks prior to enrollment
Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for age, height, and gender at the time of enrollment (with or without the use of anti-hypertensive medications)
Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of anti-hypertensive medications)
Note for patients of all ages: Adequate blood pressure can be achieved using medication for the treatment of hypertension
All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment
For all patients, a magnetic resonance imaging (MRI) of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 4 weeks prior to enrollment
Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
Patients must have the ability to swallow whole capsules
All patients have signed an appropriate consent form and Health Insurance Portability and Accountability Act (HIPAA) authorization form (if applicable)
All patients and/or their parents or legal guardians must sign a written informed consent
All patients have been consented and enrolled on APEC14B1 (NCT02402244) followed by enrollment on the ACNS1833 Pre-Enrollment Eligibility Screening (Step 0) on the same day to complete the Rapid Central Review
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted
Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible
Patients with diffuse intrinsic pontine tumors as seen on MRI (> 2/3 of pons involvement on imaging) are not eligible even if biopsy reveals grade I/II histology
Patients may not be receiving any other investigational agents
Patients with any serious medical or psychiatric illness/condition, including substance use disorders or ophthalmological conditions, likely in the judgment of the investigator to interfere or limit compliance with study requirements/treatment
Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible
Female patients who are pregnant are not eligible 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 females who plan to breastfeed their infants are not eligible
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 12 weeks after stopping study therapy are not eligible.
Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo
Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented
Symptomatic heart failure
New York Health Association (NYHA) class II-IV prior or current cardiomyopathy
Severe valvular heart disease
History of atrial fibrillation
Current or past history of central serous retinopathy
Current or past history of retinal vein occlusion or retinal detachment
Patients with uncontrolled glaucoma
If checking pressure is clinically indicated, patients with intraocular pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible
Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E
Surgery within 2 weeks prior to enrollment, with the exception of surgical biopsy, placement of a vascular access device or cerebral spinal fluid (CSF) diverting procedure such as endoscopic third ventriculostomy (ETV) and ventriculoperitoneal (VP) shunt.
Note: Patients must have healed from any prior surgery
Patients who have an uncontrolled infection are not eligibleA 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 study designed to evaluate the safety, tolerability, and efficacy of a one-time gene therapy (BBP-631) for adult patients diagnosed with classic congenital adrenal hyperplasia (CAH). The goal of gene therapy for CAH is to give the body a functioning CYP21A2 gene using a vector (an agent used to deliver a gene into the body). Having a functioning CYP21A2 gene in the adrenal gland may allow the body to naturally produce its own cortisol and aldosterone. The study treatment and follow-up lasts 1 year with a long-term follow-up of 4 more years.
Kyriakie Sarafoglou
STUDY00012144
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/or significant liver disease
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
STUDY00008743
Inclusion Criteria:
• severe obesity (BMI >/= 120% of the 95th percentile or BMI >/= 35 kg/m2)
• 12 to less than 18 years of age at enrollment
• 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) (use of naltrexone or bupropion alone 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
• history of treatment with growth hormone
• history of bulimia nervosa
• major psychiatric disorder
• any history of active suicide attempt
• history of suicidal ideation or self-harm within the previous 30 days
• current pregnancy or plans to become pregnant during study participation
• current tobacco use
• history of cardiac, endocrine, kidney disease (study staff will review)
Children's Health, Diabetes & Endocrine
Clinics and Surgery Center (CSC), Obesity, overweight, weight loss
A Phase II, Randomized, Double-Blind, Placebo-Controlled Dose-Ranging, Parallel and;Adaptive Study to Evaluate the Efficacy and Safety of Enpatoran in Systemic Lupus Erythematosus and in Cutaneous Lupus Erythematosus (Subacute&Cutaneous Lupus Erythematosus and/or Discoid Lupus Erythematosus) Participants Receiving Standard of Care
This Phase 2 study involves the use of study drug (enpatoran) which is designed to block specific cell pathways that may contribute to an over-reaction of the immune system in people with SLE and CLE.
David Pearson
STUDY00017482
Inclusion Criteria:
• adults 18-75 years of age
• disease duration at least 6 months of either active discoid or subacute cutaneous lupus OR active systemic lupus
• on stable dose(s) of standard-of-care therapies for lupus
• willing to use contraception for the study period
Exclusion Criteria:
• Drug-induced lupus, within 3 months of induction therapy for lupus nephritis, or active CNS lupus
• history of epilepsy, significant cardiovascular events including arrhythmia, solid organ transplantation, or malignancy
• active infection including HIV, HBV, HCV, or tuberculosis
• there are specified wait times for people taking certain prior drugs (study staff will review)
Arthritis & Rheumatic Diseases, Dermatology (Skin, Hair & Nails), Rare Diseases
lupus, discoid, SLE, CLE, DLE, SCLE, WILLOW, enpatoran, M5049, Merck, EMD Serono, Pearson
Study to Evaluate the Efficacy (Effect on Disease) and Safety of Finerenone on Morbidity (Events Indicating Disease Worsening) and Mortality (Death Rate) in Participants With Heart Failure and Left Ventricular Ejection Fraction (Proportion of Blood Expelled Per Heart Stroke) Greater or Equal to 40% (FINEARTS-HF) (FINEARTS-HF)
This study is a randomized, double-blind, parallel-group, placebo-controlled, multicenter, event-driven Phase 3 study with independently adjudicated clinical outcome assessments.
Tamas Alexy
STUDY00010807
Inclusion Criteria:
Participant (male or female) must be aged 40 years and older.
Diagnosis of heart failure with New York Heart Association(NYHA) class II-IV, ambulatory or hospitalized primarily for heart failure.
On diuretic treatment for at least 30 days prior to randomization.
Documented left ventricular ejection fraction (LVEF) of ≥40% measured by any modality within the last 12 months.
Structural heart abnormalities based on any local imaging measurement within the last 12 months, defined by at least one of the following findings: left atrial diameter (LAD) ≥3.8cm, left atrial area (LAA) ≥20cm2, left atrial volume index (LAVI) >30 mL/m2, left ventricular mass index (LVMI) ≥115 g/m2 (♂)/ 95 g/m2 (♀), septal thickness or posterior wall thickness ≥1.1 cm
n-terminal prohormone B-type natriuretic peptide (NT-proBNP) ≥300 pg/mL (BNP ≥100 pg/mL) in sinus rhythm and patient does not have an ongoing diagnosis of paroxysmal atrial fibrillation or NT-proBNP ≥900 pg/mL (BNP ≥300 pg/mL) in atrial fibrillation (or if atrial fibrillation status is unknown or if patient has an ongoing diagnosis of paroxysmal atrial fibrillation) for participants obtained at the following time:
Within 90 days prior to randomization if patient had been hospitalized for heart failure (HF) requiring initiation or change in HF therapy or if patient had an urgent visit for HF requiring intravenous (IV) diuretic therapy, both within 90 days prior to randomization OR
Within 30 days prior to randomization if patient has not been hospitalized for HF nor had an urgent HF visit within the past 90 days.
Women of childbearing potential can only be included in the study if a pregnancy test is negative at screening and baseline and if they agree to use adequate contraception which is consistent with local regulations regarding the methods for contraception for those participating in clinical trials.
Exclusion Criteria:
Estimated glomerular filtration rate (eGFR) <25 mL/min/1.73 m² at either screening or randomization visit.
Serum/plasma potassium >5.0 mmol/L at either screening or randomization visit.
Acute inflammatory heart disease, e.g. acute myocarditis, within 90 days prior to randomization
Myocardial infarction or any event which could have reduced the ejection fraction within 90 days prior to randomization
Coronary artery bypass graft surgery in the 90 days prior to randomization
Percutaneous coronary intervention in the 30 days prior to randomization
Stroke or transient ischemic cerebral attack within 90 days prior to randomization
Probable alternative cause of participants' HF symptoms that in the opinion of the investigator primarily accounts for patient's dyspnea such as significant pulmonary disease, anemia or obesity. Specifically, patients with the below are excluded: Severe pulmonary disease requiring home oxygen, or chronic oral steroid therapy, History of primary pulmonary arterial hypertension, Hemoglobin <10 g/dl, Valvular heart disease considered by the investigator to be clinically significant, Body Mass Index (BMI) >50 kg/m2 at screening
Systolic blood pressure(SBP) ≥160 mmHg if not on treatment with ≥3 blood pressure lowering medications or ≥180 mmHg irrespective of treatments, on 2 consecutive measurements at least 2-minute apart, at screening or at randomization.
Concomitant systemic therapy with potent cytochrome P450 isoenzyme 3A4 (CYP3A4) inhibitors (e.g. itraconazole, ritonavir, indinavir, cobicistat, clarithromycin) or moderate or potent CYP3A4 inducers, that cannot be discontinued 7 days prior to randomization and for the duration of the treatment period. Clinics and Surgery Center (CSC)
JAK Inhibitors to Preserve C-Peptide Production in New Onset T1D: A Phase 2 Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subtype-Selective JAK Inhibitors for Preservation of Pancreatic Cell Function in Newly Diagnosed Type 1 Diabetes Mellitus
This is a new type 1 diabetes onset study for ages 12-35 years old. We are looking at JAK inhibitor drugs to see if they can preserve beta cell function.
Antoinette Moran
STUDY00019323
Inclusion Criteria:
• age 12-35 years (inclusive)
• diagnosis of T1D within 100 days of first study visit
• positive for at least one islet cell autoantibody
• HbA1c no more than 10 %
• body weight at least 35kg (77 pounds)
• willing to comply with intensive diabetes management and wear a Continuous Glucose Monitoring Device (CGM)
• up to date on recommended immunizations (including flu and COVID-19)
• willing to use highly effective contraception for 3 months after the last dose of study medication
Exclusion Criteria:
• current use of a medication that affects glucose control
• treatment with other immunosuppressive agents (including biologics or steroids), other than inhaled or topical glucocorticoids
• current history of infection (HIV, Hepatitis B, TB, herpes etc.)
• current or past use of tobacco or nicotine containing products more than the equivalent of 5 cigarettes per day
• current or past medical or mental health conditions (study staff will review)
• women who are pregnant, breast feeding, or planning to become pregnant
Children's Health, Diabetes & Endocrine
T1D, new onset, type 1 diabetes,
EFC17574: A Phase 3, single-arm, multicenter, multinational, open label, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged >= 12 years with severe hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX (ATLAS-NEO)
A study to test a medicine (fitusiran) injected under the skin for preventing bleeding episodes in male adolescent or adult participants with severe Hemophilia.
Jacob Cogan
STUDY00017896
Inclusion Criteria:
Diagnosis of severe congenital hemophilia A or B (FVIII <1% or FIX level ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
For participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) treatment within the last 6 months prior to screening.
Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of participants under the age of legal consent, per local and national requirements
Exclusion Criteria:
Known co-existing bleeding disorders other than congenital hemophilia A or B
History of arterial or venous thromboembolism, not associated with an indwelling venous access
History of intolerance to SC injection(s).
Current participation in immune tolerance induction therapy (ITI)
Prior gene therapy
Current or prior participation in a fitusiran trial
Current or prior participation in a gene therapy trial
Received an investigational drug or device within 30 days prior to the screening visit or within 5 half-lives of the investigational drug (or device) prior to the screening visit, whichever is longer
Presence of clinically significant liver disease AT activity <60% at Screening
Co-existing thrombophilic disorder
Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
Presence of acute or chronic hepatitis B infection
Known to be HIV positive with CD4 count <200 cells/μL.
Reduced renal function The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.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
STUDY00012174
Inclusion Criteria:
• adults aged 18-80
• more than 8 weeks of non-productive cough
• chest x-ray 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 sinus or nasal issue is not the reason for the cough
• able to read and speak English
Exclusion Criteria:
• current smoker or quit less than 3 months ago
• infectious cause for cough (e.g., TB, pertussis, COVID)
• history of known or suspected aspiration pneumonia
• recent intubation/neck surgery (within 8 weeks)
• neuromuscular impairment that may affect cough/laryngeal sensation and/or function (e.g., multiple system atrophy, Parkinson, CVA)
• untreated carotid artery disease
• electronic implants (e.g., pacemaker)
• specific medications (study staff will discuss)
• anticipate use of new medications to treat the cough during the period of the study
• currently having speech therapy for cough
• BMI > 40 (for transmission of VTS through soft tissue)
• allergy to adhesives
• drug/alcohol dependency or abuse
• pregnant
• without regular access to wifi and internet
Breathing, Lung & Sleep Health, Ear, Nose & Throat
chronic cough, cough, larynx, vibrotactile stimulation, Clinics and Surgery Center (CSC)
First Carpometacarpal Osteoarthritis Evaluation: Determining the Concurrent Validity and Test-Retest Reliability of the Thumb Disability Index (TDX) and Test-retest Reliability of Thumb Position Sense-Error using the Intermetacarpal Distance (IMD) Method
We are studying different questionnaires used to measure symptoms and activity limitations that are linked to thumb arthritis. We are also studying ways to measure thumb position sense in persons with thumb arthritis.
Corey McGee, PhD, MS, OTR/L, CHT
STUDY00006741
Inclusion Criteria:
• at least 18 years old
• osteoarthritis of the joint where the bones of the wrist meet the hand (CMC)
Exclusion Criteria:
• received steroid injection treatment in the past 3 months
• history of CMC joint replacement -nerve problems in the wrist or hand
• women who are pregnant
• unable to speak English
Arthritis & Rheumatic Diseases
Arthritis, CMC, CMC/Carpometacarpal, Osteoarthritis
RCT01437: Proactive infliximab optimization using a pharmacokinetic dashboard versus standard of care in patients with inflammatory bowel 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.
Eugenia Shmidt
STUDY00013632
Inclusion Criteria:
• 16 to 80 years of age
• diagnosis of moderate to severe Crohn's disease (CD) or Ulcerative colitis
• physician intends to prescribe infliximab for treatment
• have not previously taken infliximab
Exclusion Criteria:
• pregnant or breastfeeding
• complications of inflammatory bowel disease (IBD) such as abscess, need for ostomy (study staff review)
• current infection in last 6 months
• other significant medical conditions (heart, lungs, liver, endocrine etc.)
Digestive & Liver Health
Clinics and Surgery Center (CSC), Crohn's disease
The Effect of Tirzepatide Versus Dulaglutide on Major Adverse Cardiovascular Events in Patients With Type 2 Diabetes (SURPASS-CVOT) (SURPASS-CVOT)
Les Forgosh
STUDY00009070
Inclusion Criteria:
• diagnosis of type 2 diabetes
• confirmed atherosclerotic cardiovascular disease
• HbA1c between 7.0% and 10.5%
• body mass index (BMI) at least 25 kilograms per meter squared (kg/m?)
Exclusion Criteria:
• major cardiovascular event within the last 60 days
• type 1 diabetes
• history of severe hypoglycemia and/or hypoglycemia unawareness within the last 6 months
• planning treatment for diabetic retinopathy and/or macular edema
• planning a coronary, carotid, or peripheral artery revascularization procedure
• history of pancreatitis
• history of ketoacidosis or hyperosmolar state/coma
• undergone or currently planning gastric bypass (bariatric) surgery or restrictive bariatric surgery
• history of an active or untreated cancer or are in remission from cancer for less than 5 years
• blood transfusion or severe blood loss within last 90 days or have known hematological conditions that may interfere with HbA1c measurement
Diabetes & Endocrine, Heart & Vascular
Heart Disease, Type 2 Diabetes
Neural and Behavioral Markers of the Temporal Dynamics of Language Planning and Phonological Working Memory Processes in Persons Who Stutter
This is a study of persons who do and do not stutter between ages 7 and 65 years. This study is trying to figure out how attention skills influence memory, language, and speech fluency skills.
Jayanthi Sasisekaran
STUDY00013042
Inclusion Criteria:
• age 7 through 65 years
• remaining eligibility will be determined based on response to an intake survey
Children's Health, Community Health
adults, attention, fluency, language, memory, speech production, Stuttering, children
MT2022-40: A Phase 3 Double-Blind, Randomized, Placebo Controlled Study to Evaluate the Efficacy and Safety of AB-205 plus Standard of Care versus Placebo plus Standard of Care in Adults with Lymphoma Undergoing High-Dose Therapy and Autologous Hematopoietic Cell Transplantation (HDT-AHCT) (E-CELERATE) (E-CELERATE)
To demonstrate a reduction in the incidence and severity of regimen related toxicities with AB-205 plus standard of care (SoC) versus placebo plus SoC in adults with lymphoma undergoing HDT-AHCT.
Daniel O'Leary
STUDY00016649
Inclusion Criteria:
Age ≥ 40 years old
Diagnosis of Hodgkin lymphoma (HL) or non-Hodgkin lymphoma (NHL)
Candidates for HDT-AHCT with one of the following conditioning regimens:
BEAM (carmustine, etoposide, cytarabine, melphalan)
BeEAM (bendamustine, etoposide, cytarabine, melphalan)
Achieved CR or PR prior to planned HDT
ECOG ≤ 2
Weight ≤ 1.6 × ideal body weight (IBW) per Devine formula
Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia
AST, ALT, and alkaline phosphatase < 3 × ULN
Creatinine clearance ≥ 30 ml/min (calculated by Cockcroft Gault)
LVEF ≥ 45% by MUGA or resting echocardiogram
Pulmonary function (FEV1 and corrected DLCO) ≥ 45% predicted
Willingness and ability to comply with scheduled visits, drug administration plan, protocol-specified laboratory tests, other study procedures, and study restrictions
Sexually active females of childbearing potential must have a negative urine pregnancy test and agree to use two accepted methods of contraception during the study and for 3 months after their last dose of study drug.
Male subjects who are sexually active and who are partners of females of childbearing potential: agreement to use two forms of contraception as in criterion 12 above and to not donate sperm during the treatment period and for at least 3 months after the last dose of study drug
Ability to provide written informed consent.
Exclusion Criteria:
History of prior HCT
Primary CNS lymphoma
Lymphoma with CNS involvement at time of relapse prior to planned HDT-AHCT
Active malignancy other than the one for which the subject is undergoing HDT AHCT. Subjects with cervical carcinoma in situ or localized basal or squamous cell carcinoma treated with definitive surgery are eligible
Subjects with a serious concomitant medical condition that could interfere with the conduct of the clinical trial, such as unstable angina, renal failure requiring hemodialysis, or active infection requiring IV antibiotics
Subjects with a known history of HIV
Subjects who have known hypersensitivity reactions to bovine (cow) proteins or documented allergy to DMSO
Subject has other conditions that in the opinion of the investigator would require reduced dose (intensity) of BEAM or BeEAM regimensMT2019-09: 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
STUDY00006513
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.ZEUS - Effects of ziltivekimab versus placebo on cardiovascular outcomes in patients with established atherosclerotic cardiovascular disease, chronic kidney disease and systemic inflammation (ZEUS)
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
STUDY00013843
Inclusion Criteria:
Chronic kidney disease defined by one of the below:
Estimated glomerular filtration rate (eGFR) greater than or equal to (>=) 15 and below 60 mL/min/1.73 m^2 (using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation)
Urinary albumin-to-creatinine ratio (UACR) >= 200 milligrams per gram (mg/g) and eGFR >= 60 mL/min/1.73 m2 (using the CKD-EPI creatinine equation)
Serum high-sensitivity C-reactive protein (hs-CRP) greater than or equal to 2 milligram per liter (mg/L)
Evidence of atherosclerotic cardiovascular disease (ASCVD) by one or more of the following:
a) Coronary heart disease defined as at least one of the following: i. Documented history of MI ii. Prior coronary revascularisation procedure iii. greater than or equal to 50% stenosis in major epicardial coronary artery documented by cardiac catheterisation or CT coronary angiography b) Cerebrovascular disease defined as at least one of the following: i. Prior stroke of atherosclerotic origin ii. Prior carotid artery revascularisation procedure iii. greater than or equal to 50% stenosis in carotid artery documented by X-ray angiography, MR angiography, CT angiography or Doppler ultrasound.
c) Symptomatic peripheral artery disease (PAD) defined as at least one of the following: i. Intermittent claudication with an ankle-brachial index (ABI) below or equal to 0.90 at rest ii. Intermittent claudication with a greater than or equal to 50% stenosis in peripheral artery (excluding carotid) documented by X-ray angiography, MR angiography, CT angiography or Doppler ultrasound iii. Prior peripheral artery (excluding carotid) revascularisation procedure iv. Lower extremity amputation at or above ankle due to atherosclerotic disease (excluding e.g. trauma or osteomyelitis).
Exclusion Criteria:
Clinical evidence of, or suspicion of, active infection at the discretion of the investigator.
Myocardial infarction, stroke, hospitalisation for unstable angina pectoris, or transient ischaemic attack within 60 days prior to randomisation (visit 2).
Planned coronary, carotid or peripheral artery revascularisation known on the day of randomisation (visit 2).
Major cardiac surgical, non-cardiac surgical, or major endoscopic procedure (thoracoscopic or laparoscopic) within the past 60 days prior to randomisation (visit 2) or any major surgical procedure planned at the time of randomisation (visit 2). Clinics and Surgery Center (CSC)
Hepatic Energy Fluxes, NASH, and Vertical Sleeve Gastrectomy
This study is looking at the liver function of people who have bariatric surgery. People who want to be in this trial must be obese, have abnormal liver function tests & prediabetes or type 2 diabetes. The study includes assistance with diet and exercise for a year after surgery.
Sayeed Ikramuddin
STUDY00006269
Inclusion Criteria:
• age 18 to 67 years
• diagnosis of non-alcoholic steatohepatitis (NASH)
• Body Mass Index (BMI) 30.0-55.0 kg/m2
• willing to have surgical treatment and have insurance with no exclusion for obesity related treatments or management of obesity surgery complications
• live or work within approximately three-hour traveling time from the study clinic for the duration of the one-year trial
Exclusion Criteria:
• cardiovascular event (myocardial infarction, acute coronary syndrome, coronary artery angioplasty or bypass, stroke) in the past six months
• pulmonary embolus or thrombophlebitis in the past six months
• cancer diagnosis unless disease free for five years
• alcohol intake more than one drink per day
• other physical or mental health disease (study staff will review)
Digestive & Liver Health
Clinics and Surgery Center (CSC), Bariatric surgery, NASH
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
STUDY00006945
Inclusion Criteria:
• ages 22-55
• diagnosis of chronic (greater than or equal to 2 years) depression
• poor response to three or more antidepressant medications (staff will review)
• had or refused ECT therapy
• under the regular care of a psychiatrist
• enrolled in a Medicare program
• have at least two people over 22 years of age and live within 30 minutes of participants residence who could respond to study staff if needed
• able to have a MRI scan
Exclusion Criteria:
• actively suicidal or have a history of an attempt within the last year
• have a history of another major mental health diagnosis
• have a positive drug test
• have an implanted brain device
• pregnant
• history of seizures
Mental Health & Addiction
Clinics and Surgery Center (CSC), Chronic Depression, Depression
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
STUDY00002192
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 lbsAccelerating Therapies for Refractory SJIA Patients
This research study aims to learn more about the causes of Systemic Juvenile Idiopathic Arthritis (sJIA) including its complications such as Macrophage Activation Syndrome (MAS) and sJIA associated lung disease and identify new therapeutic targets. The study is asking for biological samples from those diagnosed with sJIA as well as their family members.
Mona Riskalla
STUDY00015935
Inclusion Criteria:
• at least 6 months old
• new onset SJIA or established SJIA with or without lung and/or liver complications
• parent or sibling of the enrolled child will also be asked to participate
Exclusion Criteria:
• illness sufficient to prohibit study participation
• inability to cooperate with the study
Arthritis & Rheumatic Diseases, Rare Diseases
SJIA, Systemic Juvenile Idiopathic Arthritis