Clinical trials: Phase 1
Leukemia, Lymphoma
A Pediatric and Young Adult Trial of Genetically Modified T Cells Directed Against CD22 for Relapsed/Refractory Leukemia or Lymphoma
Patients with relapsed or refractory leukemia or lymphoma are often refractory to further chemotherapy. In this study, the investigators will attempt to use T cells obtained directly from the patient, which can be genetically…
- Trial name
- NCT04571138
- Testing
- SCRI-CAR22v2
- Where
- 4 sites in United States
- Volunteers
- 35 people
- Sponsor
- Seattle Children's Hospital
Ependymoma, High Grade Gliomas
Determine Maximum Tolerated Dose, Safety, and Tolerability of Rhenium (186Re) in Pediatric Recurrent, Refractory or Progressive Ependymoma and High-Grade Glioma
Pediatric patients 6-21 years of age with supratentorial recurrent, refractory, or progressive pediatric ependymoma and high-grade glioma (HGG) will be included in this study of treatment with Rhenium-186 Nanoliposome (186RNL).
- Trial name
- NCT07061626
- Testing
- Rhenium-186 Nanoliposome
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Cerenome
T-cell Acute Lymphoblastic Leukemia, T-Cell Acute Lymphoblastic Lymphoma
Targeted Immunotherapy After Myeloablative TBI-Based Conditioning & AlloHCT in CAYA With High Risk T-Cell ALL & Lymphoma
A Phase I trial to determine the safety of targeted immunotherapy with daratumumab (DARA) IV after total body irradiation (TBI)-based myeloablative conditioning and allogeneic hematopoietic cell transplantation (HCT) for…
- Trial name
- ALLO-T-DART (NCT04972942)
- Testing
- Daratumumab
- Where
- 16 sites in United States
- Volunteers
- 30 people
- Sponsor
- New York Medical College
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
- Trial name
- ENDEAVOR (NCT04626674)
- Testing
- delandistrogene moxeparvovec
- Where
- 12 sites in United States
- Volunteers
- 83 people
- Sponsor
- Sarepta Therapeutics, Inc.
Low-Grade Glioma, Recurrent Low-Grade Glioma
SJ901: Evaluation of Mirdametinib in Children, Adolescents, and Young Adults With Low-Grade Glioma
This is an open-label, multi-center, Phase 1/2 study of the brain-penetrant MEK inhibitor, mirdametinib (PD-0325901), in patients with pediatric low-grade glioma (pLGG).
- Conditions
- Low-Grade Glioma, Recurrent Low-Grade Glioma, Progressive Low-Grade Glioma
- Trial name
- NCT04923126
- Testing
- Mirdametinib
- Where
- United States
- Volunteers
- 132 people
- Sponsor
- St. Jude Children's Research Hospital
Malaria,Falciparum
Safety and Immunogenicity of SUM-101 Malaria Vaccine in Children and Infants Living in Burkina Faso
This clinical trial aims to learn about the safety and immunogenicity of the blood-stage malaria vaccine candidate SUM-101 in infants and children, paving the way for its incorporation into a multi-stage malaria vaccine.
- Trial name
- SUM-101 (NCT06618885)
- Testing
- SUM-101
- Where
- Burkina Faso
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- European Vaccine Initiative
Low Grade Glioma (LGG) of Brain With BRAF Aberration, High Grade Glioma (HGG) of the Brain With BRAF Aberration
A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration
This phase I/II trial is designed to study the side effects, best dose and efficacy of adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or high grade brain tumors previously treated with…
- Conditions
- Low Grade Glioma (LGG) of Brain With BRAF Aberration, High Grade Glioma (HGG) of the Brain With BRAF Aberration, Low Grade Glioma of Brain With Neurofibromatosis Type 1
- Trial name
- NCT04201457
- Testing
- Dabrafenib, Trametinib, Hydroxychloroquine
- Where
- 15 sites in United States
- Volunteers
- 57 people
- Sponsor
- Pediatric Brain Tumor Consortium
Type 1 Diabetes Mellitus, T1D
CNP-103 in Adolescent and Adult Subjects Ages 12-35 With Recently Diagnosed (Within 6 Months) Stage 3 Type 1 Diabetes (T1D)
This study is a Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-103.
- Conditions
- Type 1 Diabetes Mellitus, T1D, T1DM, T1DM - Type 1 Diabetes Mellitus
- Trial name
- NCT06783309
- Testing
- CNP-103
- Where
- 45 sites in United States
- Volunteers
- 72 people
- Sponsor
- COUR Pharmaceutical Development Company, Inc.
Diffuse Midline Glioma, H3 K27-Altered, Metastatic Malignant Neoplasm in the Central Nervous System
CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma
This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with solid tumors, including central nervous system (CNS) tumors or lymphoma that has come back (relapsed) or does…
- Conditions
- Diffuse Midline Glioma, H3 K27-Altered, Metastatic Malignant Neoplasm in the Central Nervous System, Recurrent Diffuse Intrinsic Pontine Glioma, Recurrent Diffuse Midline Glioma, H3 K27-Altered
- Trial name
- NCT04870944
- Testing
- Biospecimen Collection, Bone Marrow Aspirate, Bone Marrow Biopsy
- Where
- 35 sites in United States, Australia
- Volunteers
- 63 people
- Sponsor
- Children's Oncology Group
Neuroblastoma
Apatinib Mesylate Combined With IT Regimen for the Treatment of Recurrent or Refractory Neuroblastoma: A Single-arm, Phase I/II，Multi-center, Clinical Study.
The survival rate of recurrent and refractory neuroblastoma is low and the prognosis is poor. Apatinib mesylate is a highly selective small-molecule vasoendothelial growth factor receptor-2 (VEGFR-2) tyrosine kinase inhibitor.
- Trial name
- NCT05027386
- Testing
- Apatinib, Irinotecan, Temozolomide
- Where
- China
- Volunteers
- 125 people
- Sponsor
- Sun Yat-sen University
Safety Issues
Safety, Tolerability and Pharmacokinetics of ETD-001 Gel
This is a single-center, randomized, double-blind, placebo-controlled Phase I clinical study, which comprises two parts: Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) in healthy subjects.Three dose groups are…
- Trial name
- ETD-001 gel (NCT07674862)
- Testing
- Dose 1, dose 2, dose 3
- Where
- China
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Etern Therapeutics Wuxi Limited
Acute Lymphocytic Leukemia
Inotuzumab Ozogamicin Post-Transplant For Acute Lymphocytic Leukemia
This study has two phases, Phase I and Phase II. The main goal of the Phase I portion of this research study is to see what doses post-transplant inotuzumab ozogamicin can safely be given to subjects without having too many side…
- Trial name
- NCT03104491
- Testing
- Inotuzumab Ozogamicin
- Where
- 7 sites in United States
- Volunteers
- 44 people
- Sponsor
- Leland Metheny
Attention, Visual Perceptual Weakness
Result of tDCS in ASD Children With Comorbidities Like PANDAS, Rare Genetic Diseases or Autoimmune Disorders
Results of the application of 100 sessions of tDCS for 12 months in children between 6 and 11 years old with autism spectrum disorder with rare diseases, genetic problems or PANDAS
- Conditions
- Attention, Visual Perceptual Weakness, Social Behavior, Fluency Disorder
- Trial name
- tDCS&ASD (NCT06368726)
- Testing
- tDCS or Transcranial Direct Current Stimulation, Risperidone
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- Spanish Foundation for Neurometrics Development
Duchenne Muscular Dystrophy
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)
This study is a single-center, single-arm, non-randomized, open-label, non-controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK007 injection in…
- Trial name
- NCT06114056
- Testing
- JWK007 Single intravenous infusion administration
- Where
- China
- Volunteers
- 3 people
- Sponsor
- West China Hospital
HIV, HIV Infections
The Tatelo Plus Study
The purpose of this study is to advance pediatric HIV treatment and cure research by evaluating the impact of a combination of three anti-HIV-1 broadly neutralizing antibodies (bNAbs) or analytic treatment interruption (ATI) on…
- Trial name
- NCT06508749
- Testing
- PGDM1400LS, VRC07-523LS, PGT121.414.LS
- Where
- 2 sites in Botswana
- Volunteers
- 41 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Dyskeratosis Congenita, Telomere Disease
Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD)
The purpose of this study is to see if a vitamin-like substance called quercetin is safe for people who have a rare condition called Dyskeratosis congenita (DC) or telomere biology disorders (TBD).
- Trial name
- NCT07628972
- Testing
- Quercetin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Charcot-Marie-Tooth Disease Type 4J
Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)
First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy of a single lumbar intrathecal dose of ELP-02 to individuals with CMT4J.
- Trial name
- NCT07447557
- Testing
- ELP-02
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Elpida Therapeutics SPC
Rett Syndrome
Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)
This 18-week open-label study examines the effectiveness of Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164) in treating Rett syndrome (RTT) in children and young people.
- Trial name
- NCT06621043
- Testing
- NTI164
- Where
- Australia
- Volunteers
- 14 people
- Sponsor
- Fenix Innovation Group
Biliary Atresia, Kasai Operation
Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia
Biliary atresia is a progressive liver disease in infants where liver transplantation is often the only long-term option once cirrhosis develops.
- Conditions
- Biliary Atresia, Kasai Operation, Liver Fibrosis, Stem Cell
- Trial name
- NCT07797673
- Testing
- Umbilical Cord-Mesenchymal Stem Cells (UC-MSC), Kasai Portoenterostomy
- Where
- Indonesia
- Volunteers
- 40 people
- Sponsor
- Dr Cipto Mangunkusumo General Hospital
Blood Sampling, Child, Only
Two Different Therapeutic Interventions Applied During Blood Sampling in Children
This research is a three-group, randomized controlled experimental study designed to determine the effect of two different therapeutic interventions applied during blood sampling on pain, anxiety, and fear levels in children aged…
- Conditions
- Blood Sampling, Child, Only, Pain, Fear
- Trial name
- NCT07731347
- Testing
- experimental group, experiment group, Control Group
- Where
- Turkey (Türkiye)
- Volunteers
- 105 people, healthy volunteers welcome
- Sponsor
- Igdir University
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