Clinical trials: Phase 1
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
The goal of this clinical trial is to learn if sonrotoclax (BGB-11417) is safe and may help treat children and adolescents with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that has come back after treatment…
- Conditions
- Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, B-cell Acute Lymphoblastic Leukemia, Pediatric Cancer
- Trial name
- NCT07770698
- Testing
- sonrotoclax, Azacitidine, Inotuzumab ozogamicin
- Volunteers
- 30 people
- Sponsor
- BeOne Medicines
Focal Seizures
A Study to Evaluate the Efficacy and Safety of Cannabidiol Oral Solution (CBD-OS [GWP42003-P, JZP926]) for the Treatment of Focal-Onset Seizures
Cannabidiol oral solution (CBD-OS) is approved in the US for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or Tuberous sclerosis complex (TSC) in patients 1 year of age and older.
- Trial name
- NCT07233239
- Testing
- CBD-OS
- Where
- 24 sites in United States
- Volunteers
- 100 people
- Sponsor
- Jazz Pharmaceuticals
Nicotine Vaping, Nicotine Use Disorder
Testing the Feasibility and Acceptability of Social Media and Digital Therapeutics to Decrease Vaping Behaviors
Use of vaping products (e.g., electronic nicotine delivery systems, e-cigarettes) has been increasing rapidly, particularly among teens and young adults.
- Trial name
- NCT05994209
- Testing
- quitSTART Mobile Application, Chatbot Feature in quitSTART Mobile Application
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Washington University School of Medicine
Catecholaminergic Polymorphic Ventricular Tachycardia
A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
This is a Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in participants with catecholaminergic polymorphic ventricular tachycardia (CPVT).
- Trial name
- ARTEMIS (NCT07148089)
- Testing
- SGT-501
- Where
- 5 sites in United States, Canada
- Volunteers
- 18 people
- Sponsor
- Solid Biosciences Inc.
Myelodysplastic Syndromes
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults.
- Trial name
- NCT06303193
- Testing
- pacritinib
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- National Cancer Institute (NCI)
B-NHL, B-Non Hodgkin Lymphoma
CD19/CD22 Bicistronic Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies
Background: Acute lymphoblastic leukemia (ALL) is the most common cancer in children. About 90% of children and young adults who are treated for ALL can now be cured.
- Conditions
- B-NHL, B-Non Hodgkin Lymphoma, Acute Lymphocytic Leukemia, Acute Lymphoblastic Leukemia
- Trial name
- NCT05442515
- Testing
- CD19/CD22-CAR-transduced T cells, cyclophosphamide, fludarabine
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Cancer Institute (NCI)
Primary Extra-cranial Solid Tumor, Recurrent or Refractory Glypican 3 (GPC3)
A Study of Codrituzumab in Children and Young Adults With Solid Tumors and Have Not Responded to Treatment or Have Come Back After Treatment
The purpose of this study to find out whether codrituzumab is a safe treatment that causes few or mild side effects in children and young adults who have solid tumors that express the protein GPC3.
- Trial name
- NCT04928677
- Testing
- Codrituzumab
- Where
- 5 sites in United States
- Volunteers
- 50 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
B-cell Acute Lymphoblastic Leukemia, Large B-cell Lymphoma
Gene Therapy for CD19-Positive Hematologic Malignancies (SENTRY-CD19)
This is a Phase 1/2, first-in-human, open-label, dose-escalating trial designed to assess the safety and efficacy of VNX-101 in patients with relapsed or refractory CD19-positive hematologic malignancies.
- Conditions
- B-cell Acute Lymphoblastic Leukemia, Large B-cell Lymphoma, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
- Trial name
- NCT06533579
- Testing
- Dose Level 1, VNX-101, Dose Level 2, VNX-101, Dose Level 3, VNX-101
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Vironexis Biotherapeutics Inc.
Solid Tumors, Brain Tumors
NK White Blood Cells and Interleukin in Children and Young Adults With Advanced Solid Tumors
BACKGROUND: Despite progress, some children and young adults with solid tumors still experience poor survival. Activated NK cells potently kill autologous pediatric solid tumors, and clinical grade procedures are available to…
- Conditions
- Solid Tumors, Brain Tumors, Sarcoma, Pediatric Cancers
- Trial name
- NCT01875601
- Testing
- Recombinant human interleukin-15 (rhIL-15), NK Cell Infusion
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell…
- Trial name
- NCT03279094
- Testing
- Hematopoietic stem cell transplantation
- Sponsor
- City of Hope Medical Center
Hyper IgE Syndrome From STAT3 Mutation, Job s Syndrome
Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system.
- Conditions
- Hyper IgE Syndrome From STAT3 Mutation, Job s Syndrome, HIES, Lupus
- Trial name
- NCT07262983
- Testing
- baricitinib
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
WHIM, Warts
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
Background: Warts, hypogammaglobulinemia, infections and myelokathexis syndrome (WHIMs) is a rare disorder that affects the immune system. People with WHIMs can have severe infections all over their body.
- Conditions
- WHIM, Warts, Hypogammaglobulinemia, Immunodeficiency
- Trial name
- NCT07775313
- Testing
- Busulfan, Palifermin, Plerixafor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Tetralogy of Fallot, Double Outlet Right Ventricle
Beta-blocker Administration for Cardiomyocyte Division
Heart failure is a common long-term complication in patients with congenital heart disease (CHD). Medical treatments to promote regeneration of new healthy heart muscle cells have the potential to provide new heart failure…
- Trial name
- NCT04713657
- Testing
- N-thymidine, Urine Collection, Echocardiogram
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- Weill Medical College of Cornell University
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Pneumococcal Disease
A Study to Understand the Safety of the 20vPnC Vaccine in Healthy Chinese Adults, Children, and Infant
The purpose of this study is to learn about the safety of the 20-valent pneumococcal conjugate vaccine (20vPnC) among adults, children, and infants, as well as the immunogenicity of 20vPnC among infants and children under 2 years…
- Trial name
- NCT07023081
- Testing
- 20vPnC, 13vPnC
- Where
- 2 sites in China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Pfizer
Giant Axonal Neuropathy, Gene Transfer
Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background: The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly.
- Trial name
- NCT02362438
- Testing
- scAAv9/JeT-GAN
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Acute Myeloid Leukemia Post Cytotoxic Therapy, Recurrent Acute Myeloid Leukemia
A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia
This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that…
- Conditions
- Acute Myeloid Leukemia Post Cytotoxic Therapy, Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
- Trial name
- NCT07012044
- Testing
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy
- Where
- 15 sites in United States
- Volunteers
- 47 people
- Sponsor
- National Cancer Institute (NCI)
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1A), aged ≥48 months to <18 years (Part 1B), and…
- Trial name
- ENDEAVOR (NCT05419492)
- Testing
- ETX101
- Where
- 14 sites in United States, Australia and 1 more
- Volunteers
- 113 people
- Sponsor
- Encoded Therapeutics
Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia
Study of AZD3632 Monotherapy or in Combination With Anticancer Agents in Participants With Advanced Haematologic Malignancies With KMT2Ar, NPM1m, or Other Genotypes Associated With HOX Overexpression
The purpose of this study is to understand the safety, tolerability, efficacy, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of orally administered AZD3632 in participants with advanced haematologic…
- Conditions
- Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia, Higher-risk Myelodysplastic Syndromes
- Trial name
- MOMENTUM (NCT07155226)
- Testing
- AZD3632, Posaconazole
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- AstraZeneca
Healthy Volunteer, Muscular Atrophy, Spinal
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
- Trial name
- NCT05575011
- Testing
- BIIB115
- Where
- 16 sites in Belgium, Canada and 7 more
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Biogen
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