Clinical trials: Phase 1
Developmental and Epileptic Encephalopathy
A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy
The goal of this clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy.
- Trial name
- NCT06983158
- Testing
- gene therapy
- Where
- 4 sites in United States
- Volunteers
- 1 people
- Sponsor
- Capsida Biotherapeutics, Inc.
Pediatric Brain Tumor, Pediatric Solid Tumor
Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)
This is a pilot phase Ib study of the safety of dapagliflozin (in addition to standard of care treatment) for the treatment of pediatric patients with recurrent brain tumors and relapsed/refractory solid tumors.
- Trial name
- NCT05521984
- Testing
- Dapagliflozin, Carmustine, Topotecan
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Washington University School of Medicine
Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm
Testing the Combination of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) in Children, Adolescent, and Young Adult Patients With Relapsed/Refractory Cancers That Have an Increased Number of Genetic Changes, The 3CI Study
This phase Ib trial investigates the side effects of the combination of nivolumab and ipilimumab, and to see how well they work in treating patients with cancers that have come back (relapsed) or does not respond to treatment…
- Conditions
- Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Lynch Syndrome, Recurrent Lymphoma
- Trial name
- NCT04500548
- Testing
- Biospecimen Collection, Ipilimumab, Nivolumab
- Where
- 9 sites in United States, Canada
- Sponsor
- National Cancer Institute (NCI)
Glioma, Glioblastomas (GBM)
SM-1 Combined With Temozolomide in Subjects With Refractory or Recurrent High-Grade Gliomas
The aim of this clinical trial is to evaluate the safety, preliminary effectiveness, and pharmacokinetic profiles of SM-1 (Obitrexate Fumarate Enteric-coated Pellet Capsules) combined with temozolomide, and conduct systematic…
- Conditions
- Glioma, Glioblastomas (GBM), High Grade Gliomas
- Trial name
- NCT07635173
- Testing
- SM-1,TMZ
- Where
- China
- Volunteers
- 35 people
- Sponsor
- Shenzhen Zhenxing Medical Technology Co., Ltd
Leukemia
A Phase 1 Study of CD22-CAR TCell Immunotherapy for CD22+ Leukemia and Lymphoma
Patients with relapsed or refractory leukemia often develop resistance to chemotherapy and some patients who relapse following CD19 directed therapy relapse with CD19 negative leukemia.
- Trial name
- PLAT-04 (NCT03244306)
- Testing
- Patient-derived CD22-specific CAR T-cells also expressing an EGFRt
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Seattle Children's Hospital
Combined D,L-2-hydroxyglutaric Aciduria
An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)
This is an individual patient research protocol to treat a patient diagnosed with Combined D,L-2-hydroxyglutaric aciduria (C-2HGA) with ACER-001.
- Trial name
- NCT07125066
- Testing
- Sodium phenylbutyrate
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Jerry Vockley, MD, PhD
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to < 48 months. The study follows and open-label, dose-escalation design.
- Trial name
- UK Only (NCT06283212)
- Testing
- ETX101
- Where
- 3 sites in United Kingdom
- Volunteers
- 5 people
- Sponsor
- Encoded Therapeutics
Refractory Solid Tumors in Children
Clinical Trial of Rapamycin and Irinotecan in Pediatric Patients With Refractory Solid Tumors
Therapeutic solutions to treat solid tumors that are resistant to conventional treatments are now limited. Laboratory data in animals (on pediatric tumors such as brain tumors, sarcomas and neuroblastomas) have shown that the…
- Trial name
- RAPIRI (NCT01282697)
- Testing
- Combined administration of irinotecan and rapamycin
- Where
- 9 sites in France
- Volunteers
- 42 people
- Sponsor
- University Hospital, Strasbourg, France
Relapsed B-Cell Acute Lymphoblastic Leukemia
T-Lymphocytes Genetically Targeted to the B-Cell Specific Antigen CD19 in Pediatric and Young Adult Patients With Relapsed B-Cell Acute Lymphoblastic Leukemia
The purpose of this study is to test the safety of giving the patient special cells made from their own blood called "Modified T-cells".
- Trial name
- NCT01860937
- Testing
- leukapheresis or collection of PBMCs, cyclophosphamide based chemotherapy regimens, modified T cells
- Where
- 2 sites in United States
- Volunteers
- 23 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Clinical High Risk for Psychosis (CHR)
A Study Exploring Changes in a Variety of Biomarkers Following Dosing With MT1988 in Participants at Clinical High Risk for Psychosis
The goal of this clinical trial is to learn how tests undertaken by people at high risk of developing psychosis (aged 17 to 30 years old) change when those people are given the study drug MT1988 daily for 8 weeks.
- Trial name
- NCT07226895
- Testing
- MT1988 Low Dose, MT1988 High Dose
- Where
- 15 sites in United States
- Volunteers
- 150 people
- Sponsor
- Monument Therapeutics Limited
Atypical Choroid Plexus Papilloma, Choroid Plexus Carcinoma
Intra-Arterial Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma Prior to Second-Look Surgery
This study will test the safety and efficacy of intra-arterial chemotherapy in subjects with newly diagnosed, residual, or recurrent atypical choroid plexus papilloma and choroid plexus carcinoma prior to a second surgery.
- Trial name
- NCT04994977
- Testing
- Melphalan, Carboplatin, Topotecan
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Weill Medical College of Cornell University
Osteosarcoma, Pulmonary Metastasis
Safety and Feasibility of Intraoperative Visualization With Cytalux in Children
Pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to undergo pulmonary metastasectomy via and open or minimally invasive approach.
- Conditions
- Osteosarcoma, Pulmonary Metastasis, Fluorescence, Metastatic Sarcoma
- Trial name
- Cytalux (NCT06235125)
- Testing
- Cytalux
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
High Grade Glioma, Diffuse Intrinsic Pontine Glioma
Adjuvant Dendritic Cell Immunotherapy for Pediatric Patients With High-grade Glioma or Diffuse Intrinsic Pontine Glioma
Childhood aggressive gliomas are rare brain tumors with very poor prognosis. Due to the tumor's location and infiltrative nature, surgical removal is not always possible, and even when resection is performed and combined with…
- Trial name
- ADDICT-pedGLIO (NCT04911621)
- Testing
- Dendritic cell vaccination + temozolomide-based chemoradiation, Dendritic cell vaccination +- conventional next-line treatment
- Where
- Belgium
- Volunteers
- 10 people
- Sponsor
- University Hospital, Antwerp
B Cell Leukemia
A Pediatric Trial of Genetically Modified Autologous T Cells Directed Against CD19 for Relapsed CD19+ Acute Lymphoblastic Leukemia
Patients with relapsed leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use a patient's own T cells, which can be genetically modified to expresses a chimeric antigen receptor(CAR).
- Trial name
- NCT01683279
- Testing
- Autologous CD19 CAR+ EGFTt + T cells
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Seattle Children's Hospital
Menstrual Irregularities, Type1 Diabetes Mellitus
Role of Glucagon-like Peptide-1 Receptor Agonists in Menstrual Irregularities in Adolescent Females With Type 1 Diabetes Mellitus
The aim of this study is to measure the frequency of menstrual irregularities among adolescent females with Type 1 diabetes mellitus and to assess the therapeutic effect of glucagon-like peptide-1 receptor agonist on menstrual…
- Conditions
- Menstrual Irregularities, Type1 Diabetes Mellitus, Glucagon-Like Peptide-1 Receptor Agonists, Female Reproductive Health
- Trial name
- NCT07319286
- Testing
- Dulaglutide
- Where
- 2 sites in Egypt
- Volunteers
- 50 people
- Sponsor
- Ain Shams University
CD 19+ Acute Leukemia
Pilot Study of T-APCs Following CAR T Cell Immunotherapy for CD19+ Leukemia
Patients with relapsed or refractory CD 19+ leukemia who have achieved remission after CD19 CAR-T cell treatment sometimes relapse because the CD 19 CAR-T cells decrease in number over time.
- Trial name
- NCT03186118
- Testing
- T-cell Antigen Presenting Cells expressing truncated CD19 (T-APC)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Seattle Children's Hospital
Sickle Cell Disease, Hematopoetic Stem Cell Transplant
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young…
- Conditions
- Sickle Cell Disease, Hematopoetic Stem Cell Transplant, Haploidentical Hematopoietic Stem Cell Transplant, Haploidentical Stem Cell Transplantation
- Trial name
- RUX-HAPLO (NCT07252050)
- Testing
- Ruxolitinib
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- Arkansas Children's Hospital Research Institute
Acute Lymphoblastic Leukemia With Failed Remission
CAR-T Cell Therapy Targeting to CD19 for R/R ALL
Refractory and relapsed (R/R) acute lymphoblastic leukemia (ALL) patients with active disease always have a dismal outcome. Chimeric antigen receptor (CAR) T-cell therapy targeting to Cluster of Differentiation Antigen 19 (CD19)…
- Trial name
- NCT03919240
- Testing
- CAR T-cell therapy
- Where
- China
- Volunteers
- 196 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Advanced Cancer, Metastatic Solid Tumor
A Study of Repotrectinib in Combination With Chemotherapy in Children and Young Adults With Solid Tumor Cancer
This study will test the safety of the study drug, repotrectinib, in combination with chemotherapy (irinotecan and temozolomide) in children and young adults who have advanced or metastatic solid tumors.
- Trial name
- NCT05004116
- Testing
- Repotrectinib, Irinotecan and temozolomide
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Cytomegalovirus
A Study of mRNA-1647 Cytomegalovirus Vaccine in Healthy Participants 9 to 15 Years of Age and Participants 16 to 25 Years of Age
The main purpose of study is to evaluate the safety and immunogenicity of different dose levels of mRNA-1647 versus control in healthy cytomegalovirus (CMV)-seronegative and CMV-seropositive female and male participants 9 to 15…
- Trial name
- NCT05575492
- Testing
- mRNA-1647
- Where
- 57 sites in United States, Canada and 1 more
- Volunteers
- 873 people, healthy volunteers welcome
- Sponsor
- ModernaTX, Inc.
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