Clinical trials: Phase 1
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
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)
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)
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
Breast-milk
Breast-milk Enema Administration to Stimulate Passage of Meconium
Lack of passage of meconium in preterm infants delays feeding advancement and may represent a risk factor for necrotizing enterocolitis (NEC) and spontaneous intestinal perforation (SIP).
- Trial name
- BEAM (NCT07661992)
- Testing
- breast milk
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Wake Forest University Health Sciences
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
Malaria, Cerebral
DON in Pediatric Cerebral Malaria
The goal of this clinical trial is to evaluate the safety of a single intravenous dose of DON in healthy adults, adults with uncomplicated malaria, and children 12 months-14 years old with clinically defined Cerebral Malaria.
- Trial name
- NCT05478720
- Testing
- 6-diazo-5-oxo-L-norleucine (DON)
- Where
- 2 sites in Malawi
- Volunteers
- 152 people
- Sponsor
- Douglas Postels, MD, MS
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
Diphtheria, Pertussis
A Phase I/II Clinical Trial of the Diphtheria, Tetanus, Acellular Component Pertussis and Sabin Inactivated Poliomyelitis Vaccine Adsorbed
This is a randomized, positive-controlled phase I/II clinical trial to evaluate the safety and preliminary immunogenicity of the Diphtheria, Tetanus, Acellular Component Pertussis and Sabin Inactivated Poliomyelitis Vaccine…
- Conditions
- Diphtheria, Pertussis, Tetanus, Poliomyelitis
- Trial name
- NCT07891104
- Testing
- Diphtheria, Tetanus, Acellular Component Pertussis and Sabin Inactivated Poliomyelitis Vaccine Adsorbed, Diphtheria, Tetanus and Acellular Component Pertussis Combined Vaccine, Diphtheria, Tetanus and Acellular Component Pertussis Combined Vaccine+Inactivated Poliomyelitis Vaccine Made From Sabin Strains
- Where
- 3 sites in China
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- Institute of Medical Biology, Chinese Academy of Medical Sciences
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
Lymphoma, Hodgkin's Disease
Autologous/Allogeneic TGFbeta-resistant LMP-specific CTL, Lymphoma (TGF-beta)
Patients have a type of lymph gland cancer called HD, NHL or lymphoepithelioma (these 3 diseases will be referred to as "Lymphoma"). The lymphoma has come back or has not gone away after treatment.
- Conditions
- Lymphoma, Hodgkin's Disease, Relapse, Lymphoma, Non-Hodgkin
- Trial name
- TGF-beta (NCT00368082)
- Testing
- TGFbeta resistant LMP-specific CTLs
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Baylor College of Medicine
Refractory Acute Myeloid Leukemia, Relapsed Acute Myeloid Leukemia
Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia
This is a research study to find out if adding a new study drug called revumenib to commonly used chemotherapy drugs is safe and if they have beneficial effects in treating patients with acute myeloid leukemia (AML) or acute…
- Conditions
- Refractory Acute Myeloid Leukemia, Relapsed Acute Myeloid Leukemia, Acute Leukemia of Ambiguous Lineage
- Trial name
- NCT06177067
- Testing
- Revumenib, Venetoclax, Azacitidine
- Where
- 10 sites in United States
- Volunteers
- 24 people
- Sponsor
- St. Jude Children's Research Hospital
Fibrolamellar Hepatocellular Carcinoma
DRP-104 (Glutamine Antagonist) in Combination With Durvalumab in Patients With Advanced Stage Fibrolamellar Carcinoma (FLC)
The purpose of this study is to determine whether the combination of subcutaneous DRP-104 in combination with intravenous Durvalumab is safe and yields a clinically compelling antitumor activity measured as based on objective…
- Trial name
- NCT06027086
- Testing
- Durvalumab, DRP-104
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Histiocytosis,Langerhans-Cell
A Single-Arm,Multicenter Phase, Phase II Clinical Trial of Adebrelimab Combined With Trametinib in the Treatment of Refractory Recurrent Langerhans Cell Histiocytosis in Children and Adolescents
Based on clinical observation data from two previously enrolled cases, the adezbelimab combined with trametinib regimen showed good safety in pediatric refractory relapsed LCH, with good drug tolerance.
- Trial name
- NCT07890701
- Testing
- treated group
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Second Affiliated Hospital of Wenzhou Medical University
ABCA4 Retinopathy, Stargardt Disease
Oral Metformin for Treatment of ABCA4 Retinopathy
Background: ABCA4 retinopathy is a genetic disease in which the ABCA4 protein is absent or faulty. It can cause waste material to collect in the eye and may cause cells to die. The cell death can lead to vision loss.
- Conditions
- ABCA4 Retinopathy, Stargardt Disease, Retinal Dystrophy, Retinal Degeneration
- Trial name
- NCT04545736
- Testing
- Metformin hydrochloride
- Where
- 2 sites in United States
- Volunteers
- 55 people
- Sponsor
- National Eye Institute (NEI)
Seasonal Influenza
Phase I Clinical Trial of Trivalent Inactivated Split Influenza Virus Vaccine (MDCK Cell)
This study is a phase I, randomized, double-blind, placebo and active comparator-controlled clinical trial to evaluate the safety and immunogenicity of a trivalent split influenza virus vaccine (MDCK cell) developed by Sinovac…
- Trial name
- NCT07889193
- Testing
- Cell-based Trivalent Split Influenza Virus Vaccine (cTIV), Egg-based Trivalent Split Influenza Virus Vaccine (eTIV)
- Where
- China
- Volunteers
- 175 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Soft Tissue Sarcoma
Preliminary Clinical Study of Entinostat in Children With Malignant Soft Tissue Sarcoma Harboring PAX-family Gene Abnormalities
Pediatric soft tissue sarcomas (STS) are a group of malignant tumors originating from primitive mesenchymal tissues and account for approximately 7% of childhood cancers.
- Trial name
- NCT07888114
- Testing
- Entinostat, VAC/VTC chemotherapy
- Where
- China
- Volunteers
- 16 people
- Sponsor
- Beijing Tongren Hospital
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