Search Results
Dissecting the role of acetaldehyde in oral carcinogenesis
The goal of this study is to better understand how drinking alcohol may lead to oral cancers. Acetaldehyde, a chemical formed when the body breaks down alcohol, is believed to play an important role. This study will measure acetaldehyde and DNA damage levels in the mouth of participants after a low dose of alcohol. The levels will be compared between three groups, all having different degrees of risk for developing oral cancer, in order to identify DNA damage that might be crucial to cancer formation.
• 21 to 45 years of age: alcohol drinker who experiences flushing (reddening or warming of face) when you drink
• 21 to 45 years of age: alcohol drinker who have Fanconi Anemia
• 18 to 45 years of age: non-drinkers
• Tobacco or nicotine users
HM2022-48: A Phase 1/2 Dose Escalation Study of the BCL-2 Inhibitor ZN-d5 and the Wee1 Inhibitor ZN-c3 in Subjects with Acute Myeloid Leukemia
This study is being performed to determine the safety and tolerability of ZN-c3 alone and the combination of ZN-c3 and ZN-d5 in Acute Myeloid Leukemia (AML). We want to identify the best doses of the study drugs and learn if either drug effects the blood levels of the other. We will also assess how effective the Study Drugs are in treating AML and explore whether certain aspects of AML can predict whether leukemia responds to the study drug(s).
• adults with Acute Myeloid Leukemia (AML) (including secondary or therapy-related), relapsed from or refractory to one or more prior lines of therapy
• able to walk and do selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• women of childbearing potential must not be pregnant and must use effective birth control during the study and for 6 months after the last dose of study drugs
• men must agree to use a condom when having intercourse during the study and for 3 months after the last dose of study drugs
• active central nervous system (CNS) involvement
• significant cardiovascular disease
• active hepatitis B or hepatitis C infection
• additional exclusion criteria (study staff will review)
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.
• diagnosis of a relapse or refractory large B cell lymphoma, for which treatment with Kymriah is planned
• received two or more lines of systemic therapy
• able to walk and do all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• participants of child bearing age must use birth control for 30 days following completion of treatment
• additional inclusion criteria (study staff will review)
• women who are pregnant or breast feeding
• CNS involvement by malignancy
• eye disease or complaints visual acuity impairment, color or shape distortion, or blurred vision - potential participants are required to have an eye exam as part of screening
• additional exclusion criteria (study staff will review)
An Observational Registry of Abatacept in Patients with Juvenile Idiopathic Arthritis (BMS Protocol IM101240)
The objective of this study is to create an international registry with long-term follow-up to characterize and evaluate the safety of abatacept in juvenile idiopathic arthritis (JIA). The primary objective of the JIA registry is to describe the long-term safety of abatacept treatment for JIA by quantifying the incidence rates of serious infections, autoimmune disorders, and malignancies.
• less than 18 years of age (unless currently or previously enrolled in an abatacept clinical trial and received abatacept)
• diagnosis of Juvenile Idiopathic Arthritis (JIA)
• currently receiving abatacept per treating physician's decision or received abatacept in a clinical trial
• pregnant or breast feeding
• history of cancer unless cancer free for at least 5 years
• any serious acute or chronic medical condition other than JIA
• history of frequently missing clinic appointments
INHALE-1: A 26-week Primary Treatment Phase, with 26-week Extension, Open-label, Randomized Clinical Trial Evaluating the Efficacy and Safety of Afrezza? Versus Rapid-acting Insulin Analog Injections, Both in Combination with a Basal Insulin, in Pediatric Subjects with Type 1 or Type 2 Diabetes Mellitus (INHALE-1)
To assess the safety of Afrezza in a pediatric population when compared to the usual standard of care insulin.
• 4 years to under 18 years old
• diagnosis of type 1 diabetes mellitus (T1DM) or type 2 diabetes mellitus (T2DM)
• using insulin for at least 6 months for T1DM, or at least 3 months for T2DM
• treated with basal-bolus insulin therapy delivered by multiple daily injections for at least 2 weeks
• bolus insulins are restricted to the RAAs insulin lispro, insulin aspart or insulin glulisine, including biosimilar products
• basal insulins are restricted to insulin glargine, insulin degludec or insulin detemir, including biosimilar products
• HbA1c between 7.0% and 11%
• average prandial dose of insulin 2 or more units per meal
• used CGM for at least 70% of the time over a consecutive 14-day period before starting the study
• access to stable WiFi connection
• history of recent blood transfusions (within previous 3 months)
• recent history of asthma (defined as using any medications to treat within the last year) or any other clinically important lung disease
• history of serious complications of diabetes
• any other illness that isn't stable (study staff will review)
• uncontrolled eating disorder (e.g., anorexia or bulimia nervosa)
• current drug or alcohol abuse or a history of drug or alcohol abuse
• smoking (includes cigarettes, cigars, pipes, marijuana, and vaping devices) for the preceding 6 months
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 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.
• adults with classic Congenital Adrenal Hyperplasia (CAH)
• on stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
• no prior gene therapy or AAV-mediated therapy
• positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
• history of adrenalectomy and/or significant liver disease
• women who are pregnant
A Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Efficacy, Safety and Pharmacokinetics of Voclosporin in Adolescents with Lupus Nephritis (VOCAL)
The aim of this study is to investigate whether voclosporin, added to standard treatment, is able to reduce activity of lupus nephritis over a study treatment period of 24 weeks, and to determine its safety as well as the best dose for treatment of lupus nephritis in children or adolescents.
• 12 to 17 years old
• diagnosis of systemic lupus erythematosus (SLE)
• active lupus nephritis confirmed by a kidney biopsy
• currently need dialysis
• clinically significant active medical or mental health conditions (study staff will review)
• certain medications, including: immunosuppression biologic agents, cyclophosphamide, calcineurin inhibitors (CNIs), start or change dose of ACE inhibitors/ARBs within 4 weeks prior to starting study, IV corticosteroids and IV immunoglobulin within 2 weeks of starting study
A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome - RECONNECT (RECONNECT)
The purpose of this study is to investigate how effective and safe ZYN002, a transdermal gel, is in participants with FXS. The drug product ZYN002 is a pharmaceutically manufactured CBD. It is being developed as a clear gel that can be applied to the skin (called transdermal delivery), to provide consistent, controlled levels of CBD in the blood when it is given twice a day. Participants will be assigned by chance to get one of the following study treatments: Active study drug – ZYN002 or placebo. Assigning study drug by chance is called “randomization,” and it is an important part of testing an experimental study drug. Participants will be randomly assigned to study treatment according to a computer program and will have 1 in 2 chance of receiving the active study drug.
• ages 3 to less than 23 years
• resides with caregiver who will continue to provide consistent care throughout the study
• diagnosis of Fragile X Syndrome (FXS) through molecular documentation
• body mass index between 12-30 kg/m2
• in generally good health based upon the results of medical history, physical exam, 12-lead ECG and clinical laboratory test results
• contact study staff for additional requirements
• women who are pregnant, nursing or planning a pregnancy
• has transitioned to independent living or living in a residential facility such as a university setting or congregate care
• use of cannabis or any THC or CBD-containing product within 3 months first study visit or during the study
• positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines (except midazolam or comparable administered for blood draws and ECG collection), and opiates
• additional medical or mental health diagnosis (study staff will review)
MT2023-16: A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of TAK-186 (also known as MVC-101), An EGFR x CD3 COnditional Bispecific Redirected Activation (COBRA) Protein in Patients with Unresectable Locally Advanced or Metastatic Cancer
This study will test TAK-186, an antibody that selectively targets EGRF-expressing tumor cells, to see if TAK-186 is safe in patients with unresectable, locally advanced or metastatic cancer.
• unresectable, locally advanced or metastatic solid tumors are considered to express epidermal growth factor receptor (EGFR)
• willing to have new biopsy(s) for the study if it is possible
• able to care for self and do light work
• women of childbearing potential must be willing to use 2 forms of contraception throughout the study, starting at screening through 90 days after the last dose of TAK-186
• Males with partners of childbearing potential must use barrier contraception during the entire study treatment period through 120 days after the last dose of study drug and must not donate sperm during this period. Must also have partner use 2 forms of contraception (see above requirement)
• contact study staff for additional study requirements
• history of known autoimmune disease with some exceptions
• major surgery or traumatic injury within 8 weeks before first dose of study drug
• unhealed wounds from surgery or injury
• serious underlying medical or psychiatric condition that would impair the ability of the participant to consent, receive or tolerate the planned treatment (study staff will review)
• women who are pregnant or breast feeding
A Phase 3, Open-Label, Multi-Center, Randomized Study Evaluating the Efficacy and Safety of TAR-200 in Combination with Cetrelimab or TAR-200 Alone Versus Intravesical Bacillus Calmette-Guerin (BCG) in Participants with BCG-naive High-Risk Non-Muscle Invasive Bladder Cancer (HR-NMIBC) (SunRISe-3)
The purpose of this study is to compare the effects (both good and bad) of an investigational drug delivery system (TAR-200) in combination with cetrelimab or TAR-200 alone to the effects of study drug comparator intravesical (medicine that is put directly into the bladder instead of being taken like a pill or put into veins) BCG in patients with HR-NMIBC. Cetrelimab is a medicine that may treat certain cancers by working with the immune system (it is also known as immunotherapy). Immunotherapy is the use of medicines to help a person’s own immune system recognize and destroy cancer cells.
• diagnosis of high grade non-muscle invasive bladder cancer (HR-NMIBC) (high-grade Ta, any T1 or carcinoma in-situ [CIS])
• have not received Bacillus Calmette Guerin (BCG)
• cancer must be surgically removed
• able to walk and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• more extensive bladder cancer (muscle invasive, locally advanced, nonresectable, or metastatic urothelial carcinoma (that is, greater than and equal to [>=] T2))
• history of clinically significant polyuria with recorded 24-hour urine volumes greater than 4000 milliliters (mL)
• Indwelling catheters are not permitted; however, intermittent catheterization is acceptable
• additional exclusion criteria (study staff will review)
MT2023-06: A CLINICAL STUDY TO ASSESS THE EFFICACY AND SAFETY OF LERIGLITAZONE IN ADULT MALE SUBJECTS WITH CEREBRAL ADRENOLEUKODYSTROPHY (CALYX)
This study has 2 parts: a double-blind period and an open-label extension. In the double-blind period of this study, the study medicine will be compared to a placebo. A placebo is a treatment that looks and tastes exactly like the study medicine but does not contain any active ingredient. In this study, you will receive leriglitazone or placebo. Whether you receive leriglitazone or placebo will be decided randomly (by chance, like flipping a coin). In this study, 1 out of every 2 subjects (50%) will receive leriglitazone and 1 out of every 2 subjects (50%) will receive placebo. To make this study fair, you and the study doctor will not be told which treatment you will receive, this is called “blinding”. In the open-label extension, all subjects will receive leriglitazone.
• diagnosis of progressive cerebral adrenoleukodystrophy (cALD), defined as GdE with brain lesions
• bone marrow transplantation (HSCT) is not recommended patient is not willing to undergo HSCT
• no major cognitive impairment
• see link to clinicaltrials.gov for additional inclusion criteria
• or treatment with ex-vivo gene therapy (eli-Cel).
• known type 1 or type 2 diabetes
• see link to clinicaltrials.gov for additional exclusion criteria
An Open-label, Multicenter, Multicohort, Phase 2 Study to Evaluate Enfortumab Vedotin in Subjects with Previously Treated Locally Advanced or Metastatic Malignant Solid Tumors (EV-202)
One goal of this study is to find out if enfortumab vedotin is effective and safe as a treatment for people with breast, lung, head and neck, gastric, gastroesophageal junction, or esophageal cancer. Researchers will look at how enfortumab vedotin can act in the body. Enfortumab vedotin is expected to work by attacking cells that have a protein called Nectin-4, commonly found in cancer cells. Another goal of this study is to find out if enfortumab vedotin is effective and safe when combined with another US Food and Drug Administration (FDA) approved medicine, pembrolizumab (brand name KEYTRUDA®), and used as a treatment for people with head and neck cancer who have not received previous chemotherapy treatment other than the chemotherapy that may have been given in combination with radiation therapy or right before or right after surgery in the past.
• locally advanced or metastatic disease of breast, lung, head and neck, gastric, gastroesophageal junction, or esophagus, that is not amenable to curative intent treatment
• evidence of progression on or after the last regimen received
• restricted strenuous activity but able to walk carry out work of a light or sedentary nature, e.g., light house work, office work
• see link to clinicaltrials.gov for specific requirements by type of cancer
• study staff will review
A Randomized Phase III, Two-Arm Trial of Paclitaxel/Carboplatin/Maintenance Letrozole Versus Letrozole Monotherapy in Patients with Stage II-IV, Primary Low-Grade Serous Carcinoma of the Ovary or Peritoneum
The purpose of this study is to compare the treatment of carboplatin/paclitaxel and letrozole hormonal therapy to letrozole alone. Letrozole is a drug called an aromatase inhibitor, which indirectly stops the body from producing estrogen. The use of the hormonal therapy drug, letrozole without chemotherapy may shrink or stabilize your cancer in the same way that chemotherapy also does, but without the added side effects of chemotherapy. Half of women in this study will receive letrozole with paclitaxel/carboplatin and the other half will receive letrozole alone.
• newly diagnosed, stage II-IV low-grade serous ovarian cancer: Ovarian cancer includes ovarian, fallopian tube and primary peritoneal cancers
• must have undergone an attempt at maximal cytoreductive surgery and a bilateral salpingo-oophorectomy
• ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• surgery no more than eight weeks before starting the study
• able to take medications by mouth
• contact study staff for additional inclusion criteria
• have received neoadjuvant or adjuvant chemotherapy or radiotherapy for the treatment of this disease
• received previous hormone therapy for the treatment of this disease
• history of severe cardiac disease
• central nervous system metastases
• active (except for uncomplicated urinary tract infection) or uncontrolled systemic infection
• neuropathy causing more than moderate pain and affecting daily activity
A randomized phase II trial of adjuvant Pembrolizumab versus observation following curative resection for stage I non-small cell lung cancer (NSCLC) with primary tumors between 1-4 cm: Big Ten Cancer Research Consortium BTCRC-LUN18-153
This is a research study to find out if giving a drug called pembrolizumab after lung cancer surgery does a better job at keeping the cancer from coming back than surgery alone.
• at least 18 years old
• diagnosis of non-small cell lung cancer (NSCLC)
• tumor size between 1 and 4 cm in size
• had a complete surgical resection of stage I NSCLC between 4-12 weeks ago
• able to walk and carry out basic activities of living
• women are willing to use highly effective birth control for 120 days after last dose of study drug
• certain laboratory values are required (study staff will review)
• chemotherapy, radiation therapy, or immunotherapy for the treatment of this lung cancer
• active additional cancer that is progressing or has required treatment within the past 3 years
• diagnosis of immunodeficiency or receiving chronic steroid therapy
• women who are pregnant or breast feeding
• other active diseases (study staff will review)
MT2023-33 A Phase II Study of Reduced Dose Post Transplantation; Cyclophosphamide as GvHD Prophylaxis in Adult Patients with Hematologic Malignancies Receiving HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation (OPTIMIZE)
Cyclophosphamide is a chemotherapy (chemo) drug often given after a transplant to prevent graft-versus-host disease (GvHD). We are doing this study to see if a lower dose of cyclophosphamide after transplant is as safe and works just as well. This study does not include any new or untested drugs. The drugs and procedures in this study are standard for transplant.
• between 18 and 66 years old
• receiving an unrelated Donor Peripheral Blood Stem Cell Transplantation
• willing to comply with all study procedures and availability for the duration of the study
• see link to clinicaltrials.gov for complete Inclusion Criteria
• prior allogeneic transplant
• autologous transplant within the past 3 months
• women who are pregnant or breast feeding
• HIV+ with persistently positive viral load
• study staff will review
MT2023-22: PHASE 1/2 STUDY OF IDP-023 AS A SINGLE AGENT AND IN COMBINATION WITH ANTIBODY THERAPIES IN PATIENTS WITH ADVANCED HEMATOLOGIC CANCERS
There are 2 phases to this clinical research study: Phase 1 (dose escalation) and Phase 2 (dose expansion). The goal of Phase 1 is to find the recommended dose of the study drug IDP-023 that can be given alone (referred to as a “monotherapy”), with or without interleukin-2 (IL-2) and in combination with another anti-cancer drug, either daratumumab in subjects with relapsed/refractory MM or rituximab in subjects with relapsed/refractory NHL. The goal of Phase 2 is to learn if the recommended dose of IDP-023 found in Phase 1 with or without IL-2 can help to control advanced MM or NHL when given in combination with daratumumab or rituximab, respectively.
Collaborative Solutions for Breaking Up Sedentary Time in Black Older Adults with Type 2 Diabetes: The Interrupt T2D Study
Black Americans are 60% more likely to be diagnosed with Type 2 Diabetes (T2D) and 2 times more likely to die from T2D than non-Hispanic White Americans. Recent research has shown that sedentary behavior (such as sitting or lying down) have a significant negative impact on health. We are doing a small study with older people who identify as Black or African American and have T2D to look at ways to reduce sedentary behavior. The study will last about 1-2 months for each participant.
• self-identify as Black or African American.
• 55 years and older
• speak and read English.
• self-reported diagnosis of type 2 diabetes (T2D)
• evidence of cognitive impairment that could impact ability to consent and/or participation
• physical impairment or disability that interferes with ability to engage in physical activity such as severe osteoarthritis, lower extremity amputation (other than toe(s) or partial foot), regular use of a walker or wheelchair, etc.
• unstable medical/psychiatric condition (study staff will review)
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants with Type 2 Gaucher Disease
PR001A is designed to deliver a normal GBA1 gene copy into the body to increase the activity of GCase, which is low in Type 2 Gaucher Disease (GD2) patients. The new GBA1 gene will remain a child’s body cells for many years and possibly for the rest of their life. A participant will need one surgery during which the study drug will be given and will stay in the hospital for at least 48 hours following the surgery.
• 0 to 24 months of age
• clinical diagnosis on Gaucher disease, Type 2 (GD2)
• Bi-allelic GBA1 mutation
• child has a reliable caregiver (i.e., parent/legal guardian) who is willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities
• diagnosis of a significant CNS disease other than GD2
• able to walk independently
• any other significant medical diagnosis (study staff will review)
• significant laboratory test result abnormalities
• unable to tolerate diagnostic imaging (MRI, CT scan) or unable to tolerate contrast agent
• unable to have sedation or anesthesia
Research Evaluating Vagal Excitation and Anatomical Links
We are studying the effects of stimulating the vagus nerve. The vagus nerve connects the brain to many organs in the body. Vagus nerve stimulation (VNS) is already approved by the United States Food and Drug Administration (FDA) to treat depression and epilepsy. We want to learn more about how it affects other parts of our bodies, such as the heart, metabolism, the immune system, and the nervous system. We hope that by understanding how VNS affects the body as a whole, we can develop new treatments for other conditions, or help to improve its use for depression and epilepsy.
• previously implanted with a vagal nerve stimulator (VNS) device to treat Major Depressive Disorder and on stable medications for at least 2 months
• OR will receive a VNS implant as standard clinical care, for Major Depressive Disorder and will receive VNS clinical standard of care programming after study completion. standard clinical care, for Major Depressive Disorder and will receive VNS clinical standard of care programming after completing the study
• OR previously been implanted with a VNS for Epilepsy that isn't controlled with medication
• OR will receive a VNS implant as standard clinical care, and will receive VNS clinical standard of care programming after study completion
• Contact study staff for additional requirements for each group
• willing to use effective birth control for the entire time period of the study
• has a prior implantable stimulation device, other than a VNS device
• uses or is expected during the study to use short-wave diathermy, microwave, diathermy, or therapeutic ultrasound diathermy
• unable to speak English
• additional medical or mental health issues (study staff will review)
Long COVID Ultrasound Trial
This research is being performed to study whether an investigational ultrasound treatment of the spleen can reduce the symptoms of Long COVID by reducing inflammation in the body. Ultrasound is widely used in human medicine because it is designed to be safe, non-invasive, and painless. The same kind of ultrasound that is used for imaging (for example, to visualize babies in utero) may be able to treat Long COVID.
• 18 to 65 years old
• diagnosis of COVID-19 within one year of starting the study, documented by a positive test in which there is evidence in the medical records, physical or photographic evidence, or a note in the medical records from a medical professional documenting that they were positive for COVID-19
• symptoms present for 12 or more weeks including fatigue and one or more of: a. Myalgia b. General aches/pains c. Joint pain d. Cognitive dysfunction (brain fog)
• history of intubation or admission to ICU to treat COVID-19
• pre-existing lung conditions such as chronic obstructive pulmonary disease (COPD), interstitial lung disease (ILD), or severe asthma
• women who are pregnant
• history of coronary artery disease (CAD) or stroke
• history of smoking
• any non-marijuana drug abuse history within 30 days
• history of alcohol abuse: greater than 2 drinks a day for men and 1 drink for females
• participant does not speak English
• additional medical and mental health diagnosis (study staff will review)
A Study of Imlunestrant Versus Standard Endocrine Therapy in Participants With Early Breast Cancer (EMBER-4)
Disruption of estrogen signaling by drugs called selective estrogen receptor degraders (SERDs) is one of the treatment options for patients with estrogen receptor positive (ER+) cancers. Imlunestrant is a SERD that disrupts estrogen signaling, and therefore should stop or slow down tumor growth in ER+ cancers. This study will help answer research questions about the safety of imlunestrant and any side effects, and how imlunestrant compares to standard-of-care endocrine therapy.
• diagnosis of ER+, HER2- early-stage invasive breast cancer without evidence of distant metastasis
• completed surgery
• received at least 24 months but not more than 60 months of any endocrine therapy after treatment
• may be limited with strenuous activity but able to walk and carry out work of a light or sedentary nature, e.g., light house work, office work
• any evidence of metastatic disease
• more than a 6 month consecutive gap in therapy during the course of prior adjuvant endocrine therapy
• history of any other cancer
• women who are pregnant, breastfeeding, or expecting to conceive or men expecting to father children
MT2021-29: Evaluation of intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients with mucopolysaccharidosis type IH
In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.
• between 0 to 3 years of age
• meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
• planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course
• patient's parent/ legal guardians are unable to provide informed consent.
MT2015-25: Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
A Randomized Double Blind Phase II Trial of Restorative Microbiota Therapy (RMT) or Placebo in Combination with Durvalumab (MEDI4736) and Tremelimumab With Chemotherapy in Treatment Naive Advanced or Metastatic Adenocarcinoma Non-Small Cell Lung Cancer
The investigational therapy in this study is referred to as Restorative Microbiota Therapy (RMT). It is prepared by extracting healthy bacteria from the stool of healthy human donors and making it into capsules taken by mouth. The donor stool samples are rigorously tested for harmful bacteria and viruses before processing. There is scientific evidence to suggest that RMT might make immunotherapy more effective. The primary goal of the study is to test if RMT makes durvalumab + tremelimumab treatment with chemotherapy more effective to control lung cancer.
• confirmed adenocarcinoma of the lung that is stage IIIB/C or stage IV that can't be surgically removed
• prior chemotherapy or immunotherapy as adjuvant therapy for lung cancer is permitted as long as it has been more than 6 months from last dose
• people who have treated brain metastasis are eligible as long as they have stable symptoms, are more than 2 weeks from completion of therapy, and do not require more than 10mg of daily prednisone or equivalent
• restricted in strenuous physical activity but can walk and carry out work of a light or sedentary nature, e.g., light house work, office work
• weigh at least 30 kg (66 lbs.)
• contact study staff for additional requirements
• women who are pregnant or breast feeding
• unable to swallow medications
• additional medical and mental health diagnosis (study staff will review)
MT2022-54 A MULTINATIONAL, MULTICENTER, DOSE ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY ACTIVITY OF FP-045 IN PATIENTS WITH FANCONI ANEMIA (FuschiA Study)
The purpose of this research study is to determine the best dose of FP-045 for Fanconi anemia pediatric and adolescent participants. The study will look at whether the participants have any side effects and if there are any possible changes in something called “biomarkers,” which are blood proteins that will be checked to see if they change when taking FP-045 and that may indicate if FP-045 can delay or prevent disease symptoms. Every participant will receive FP-045.
• 3 to 25 years old
• documented Fanconi anemia by chromosome breakage analysis
• women of child-bearing potential and males required to use highly effective birth control
• history of any cancer except squamous cell or basal cell carcinoma of the skin or carcinoma in situ of cervix
• myelodysplastic syndrome or acute leukemia
• history of any significant medical conditions
• history of bone marrow or stem cell transplant
• see link to clinicaltrials.gov for complete criteria
A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects with Selected Proteinuric Glomerular Diseases (EPPIK) (EPPIK)
Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The study will look at the safety, efficacy, and pharmacokinetic (PK) trial in children ≥1 to <18 years treated for up to 108 weeks with the drug sparsentan.
• Child 1 to 18 years old
• Diagnosed by biopsy with specific types of glomerular disease & protein in the urine
• Blood pressure is within normal range for age
• Maintained on a stable dose of immunosuppressive medications
• Weight less than 7.3 kg 16 pounds) at screening.
• Disease due to to viral infections, drug toxicities, or cancer.
• Kidney function is below the minimum required
A MULTI-CENTER STUDY OF NON-INVASIVE COLORECTAL CANCER EVALUATION IN CYSTIC FIBROSIS (NICE-CF) (NICE-CF)
We are comparing the results of stool sample testing to colonoscopy for people who have Cystic Fibrosis (CF). We want to find out how effective stool sample testing is in detecting adenomas, including colorectal cancer.
• Ages 18 - 75 years of age without history of transplant, or adults with CF age 18 - 75 who have had a transplant
• Diagnosis of Cystic Fibrosis with a sweat chloride test result of at least 60 mmol/L and/or documented CF-causing CFTR mutations and clinical
• Speak and write English or Spanish
• Having a screening or surveillance colonoscopy for colorectal cancer (CRC)
• Women who are pregnant
• Active inflammatory bowel disease (Crohns Disease or Ulcerative Colitis)
• History of colon cancer diagnosis and treatment within 5 years of enrollment
• Symptoms that indicate colonoscopy is for diagnostic purposes rather than as screening for CRC
MT2020-08 A Phase 1/1b Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate the Safety and Clinical Activity of PBCAR0191(azercabtagene zapreleucel or “azer-cel”), in Subjects with Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma (NHL) and r/r B-cell Acute Lymphoblastic Leukemia (B-ALL)
The purpose of this research study is to obtain information on the safety and effectiveness of PBCAR0191 to treat certain types of cancers, such as Non-Hodgkin Lymphoma and B-cell Acute Lymphoblastic Leukemia. It is made from a type of blood cells known as T cells. The T cells in PBCAR0191 came from people who have donated their blood. The donated T cells have been genetically changed, so that they may be able to kill specific cancer cells commonly present in Non-Hodgkin Lymphoma and B-cell Acute Lymphoblastic Leukemia.
• diagnosis of Non-Hodgkin Lymphoma
• received at least 2, but no more than 7 prior chemotherapy-containing treatment regimens
• 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
• restricted in strenuous activity but able to walk and able to carry out light work e.g., light house work, office work
• adequate bone marrow, renal, hepatic, pulmonary, and cardiac function (study staff will review)
• prior or active CNS disease
• uncontrolled and serious fungal, bacterial, viral, protozoal, or other infection
• active hepatitis B or hepatitis C
• any known uncontrolled cardiovascular disease
• contact study staff for additional exclusion criteria
Surgical Window of Opportunity Study of Megestrol Acetate Compared with Megestrol Acetate and Metformin for Endometrial Intraepithelial Neoplasia
The purpose of this study is to compare the effectiveness of megestrol alone, or combined with metformin, on the growth of Endometrial Intraepithelial Neoplasia (EIN). Participants will receive medication directed against EIN prior to the planned procedure (hysterectomy or progestin IUD placement). Women will receive either megestrol acetate pills by mouth twice a day for 3 to 5 weeks, or megestrol acetate and metformin pills twice a day by mouth for 3 to 5 weeks.
• endometrial intraepithelial neoplasia (EIN) on an endometrial biopsy or dilation and curettage specimen
• willing to have surgery (hysterectomy) or non-surgical treatment with a progestin IUD
• if diabetic, blood glucose must be appropriately controlled as evidenced by a hemoglobin A1c of < 8.0 in the last three months prior to enrollment
• women of child-bearing potential must agree to use adequate contraception (barrier method of birth control; abstinence) prior to study entry and for the duration of study participation
• Current hormonal contraceptives or post-menopausal hormone replacement therapy, and uses of progestins (including progestin containing intrauterine device (there are exceptions, study staff will review)
• current use of metformin therapy. If previously used, it must be discontinued at least a year ago
• women who are pregnant or breast feeding
• history of pulmonary embolism, thrombotic stroke, arterial thrombosis or deep vein thrombosis
• see link to clinicaltrials.gov for additional inclusion and exclusion criteria
FAM (Follower, Action Plan, and Remote Monitoring) Intervention to Reduce Severe Hyperglycemia in Adults with Type 1 Diabetes Mellitus at Risk for Diabetic Ketoacidosis (Aim 3)
The purpose of this study is to implement an intervention using a Follower, Action plan, and remote Monitoring (FAM) of glucose data to reduce severe hyperglycemia in adults with Type 1 Diabetes Mellitus at risk for diabetic ketoacidosis. You and your chosen “follower” (family member, caregiver, or friend) will be asked to attend 6 visits (in- person or remote) as a pair (“dyad”) with the study team that will last up to 30 minutes to 2 hours each across a span of 4 months.
• ages 18 to 65
• people with Type 1 Diabetes (T1DM) for at least 1 year
• HbA1C between 8.0% and 14.0%
• "Follower" (family member, friend, or caregiver who is at least 18 years old), willing to participate in the study and follow glucose data and has no self-reported cognitive impairment
• active treatment with a sodium-glucose cotransporter-2 inhibitor or planning to start a sodium-glucose cotransporter-2 inhibitor in the next 6 months
• active cancer with the exception of non-melanoma skin cancer
• receiving hospice care