Testing the Combination of the Anti-cancer Drugs ZEN003694 (ZEN-3694) and Talazoparib in Patients With Advanced Solid Tumors, The ComBET Trial
NCT05327010
Recruiting
This phase II trial tests whether ZEN003694 (ZEN-3694) in combination with talazoparib works to shrink tumors in patients with solid tumors that are unlikely to be cured or controlled with treatment and that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced). Another aim of this study is to find out if, and how, patients' genes influence their response to this specific drug combination. For this part of the study, investigators will... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: City of Hope Comprehensive Cancer Center, Duarte, California +34 locations
Conditions: Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, Unresectable Malignant Solid Neoplasm
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A Study of the Drug Selinexor With Radiation Therapy in Patients With Newly-Diagnosed Diffuse Intrinsic Pontine (DIPG) Glioma and High-Grade Glioma (HGG)
NCT05099003
Recruiting
This phase I/II trial tests the safety, side effects, and best dose of selinexor given in combination with standard radiation therapy in treating children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG) or high-grade glioma (HGG) with a genetic change called H3 K27M mutation. It also tests whether combination of selinexor and standard radiation therapy works to shrink tumors in this patient population. Glioma is a type of cancer that occurs in the brain or spine. Gl... Read More
Gender:
ALL
Ages:
Between 12 months and 21 years
Trial Updated:
08/22/2025
Locations: Children's Hospital of Alabama, Birmingham, Alabama +123 locations
Conditions: Malignant Glioma
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A Trial to Learn if the Combination of Fianlimab, Cemiplimab, and Chemotherapy is Safe and Works Better Than the Combination of Cemiplimab and Chemotherapy in Adult Patients With Non-Small Cell Lung Cancer That Can be Treated With Surgery
NCT06161441
Recruiting
This study is researching an experimental drug called fianlimab (also called REGN3767) with two other medications called cemiplimab and platinum-doublet chemotherapy, individually called a "study drug" or collectively called "study drugs", when combined in this study. The study is being conducted in patients who have resectable stage II to IIIB (N2) non-small cell lung cancer (NSCLC) that can be treated with surgery. The aim of the study is to see how effective the combination of fianlimab, cem... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: University of Alabama at Birmingham, Birmingham, Alabama +117 locations
Conditions: Resectable Non-small Cell Lung Cancer
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
NCT05861986
Recruiting
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
Gender:
ALL
Ages:
Between 3 months and 24 months
Trial Updated:
08/22/2025
Locations: University of Arkansas for Medical Sciences, Little Rock, Arkansas +15 locations
Conditions: Muscular Atrophy, Spinal
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4D-710 in Adult Patients With Cystic Fibrosis
NCT05248230
Recruiting
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: University of Alabama Child Health Research Unit, Birmingham, Alabama +18 locations
Conditions: Cystic Fibrosis Lung
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A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
NCT06138639
Recruiting
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will... Read More
Gender:
MALE
Ages:
Between 0 years and 17 years
Trial Updated:
08/22/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +9 locations
Conditions: Duchenne Muscular Dystrophy
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A Nature-Based Virtual Reality (VR) Intervention in Family Caregivers of Allogeneic Hematopoietic Stem Cell Transplant (HSCT) Recipients
NCT05909202
Recruiting
Background: People who receive an allogeneic hematopoietic stem cell transplant (HSCT) require long-term care at home afterwards. Their caregivers often experience high levels of stress, which can lead to symptoms such as depression, anxiety, poor sleep, fatigue, and difficulties with concentration and memory. Objective: To explore whether a nature-based immersive virtual reality (VR) program helps reduce stress in people who care for HSCT patients. Eligibility: People aged 18 and older who... Read More
Gender:
ALL
Ages:
Between 18 years and 100 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Neoplasm, Caregivers
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Magnetic Resonance Imaging (MRI) to Evaluate Activity of Multiple Sclerosis (MS)
NCT00001248
Recruiting
Studies performed under 89-N-0045 are designed to examine the natural history of multiple sclerosis (MS) using MRI and immunological measures. In addition to studying the natural history of untreated patients, the natural history of patients receiving approved disease-modifying therapies of MS will be examined. In both cohorts of patients levels of disease activity on MRI will be compared with immunological characteristics in order to help identify disease mechanism. Patients with either definit... Read More
Gender:
ALL
Ages:
Between 18 years and 120 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Multiple Sclerosis
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Rapid Autopsy and Procurement of Cancer Tissue
NCT01851395
Recruiting
Background: - Individuals with cancer of the lung, chest cavity, ovary, or bladder, as well as patients who have been treated with adoptive cell therapy unfortunately commonly succumb to their disease. Some agree to donate their bodies to cancer research that may help the medical community better understand these diseases. Studies of cancer tumor tissue obtained soon after death may be used to answer questions about the origins, progression, and treatment of cancer. Researchers want to conduct... Read More
Gender:
ALL
Ages:
Between 18 years and 120 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Thymus Tumors, Adoptive Cellular Therapy, Mesothelioma, Genitourinary Cancers, Lung Cancers
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Diagnosing and Treating Low Blood Sugar Levels
NCT00001276
Recruiting
Hypoglycemia is the term used to refer to lower than normal levels of blood sugar. This study will continue to research the causes of hypoglycemia. Patients involved in the study will be admitted to the Clinical Center of the National Institutes of Health and undergo tests for evaluating blood sugar. Patients will be required to refrain from eating for a set period of time and will undergo blood tests for insulin levels and several other specific diagnostic tests related to insulin secretion. T... Read More
Gender:
ALL
Ages:
Between 18 years and 120 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Hypoglycemia, Insulinoma
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Surgery as a Treatment for Medically Intractable Epilepsy
NCT01273129
Recruiting
Background: - Drug resistant epilepsy is the term used to describe epilepsy that cannot be controlled by medication. Many people whose seizures do not respond to medication will respond to surgical treatment, relieving seizures completely or almost completely in one-half to two-thirds of patients who qualify for surgery. The tests and surgery performed as part of this treatment are not experimental, but researchers are interested in using the data collected as part of routine standard epilepsy... Read More
Gender:
ALL
Ages:
Between 8 years and 99 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Epilepsy, Epilepsy, Temporal Lobe, Partial Epilepsy
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Genetic Analysis of Immune Disorders
NCT00001467
Recruiting
The purposes of this study are to 1) identify the genes responsible for certain immune disorders, 2) learn about the medical problems they cause, and 3) learn how to predict who is likely to develop these disorders and what the risk is of passing them on to children. The immune system is the body s defense system. Some immune deficiencies impair a person s ability to fight infections; others render a person susceptible to allergies, or to autoimmune diseases such as lupus or arthritis, in which... Read More
Gender:
ALL
Ages:
Between 1 day and 101 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: DOK 8, STAT1, GATA2, Immunodeficiency, STAT3
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