Clinical trials: Phase 1
Neuroblastoma, Neuroblastoma Recurrent
Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma
This research is being done to investigate a treatment regimen of Irinotecan, Temozolomide, and Sargramostin, and an immunotherapy called Naxitamab and whether giving Naxitamab more slowly reduces the side effects for…
- Conditions
- Neuroblastoma, Neuroblastoma Recurrent, Relapsed Neuroblastoma, Refractory Neuroblastoma
- Trial name
- NCT07027748
- Testing
- Naxitamab, Irinotecan, Temozolomide
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Steven DuBois, MD
Amyotrophic Lateral Sclerosis (ALS)
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
- Trial name
- NCT07703462
- Testing
- nL-TARDB-002
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- n-Lorem Foundation
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)
This is a long-term follow-up study of participants in the RB001-101 gene replacement therapy clinical trial for Children With SHANK3-related Phelan-McDermid Syndrome (PMS).
- Trial name
- RB001-101-LTFU (NCT07690527)
- Testing
- RB001
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking University First Hospital
Malignant Neoplasm, Carcinoma, Merkel Cell
A Study of Pembrolizumab in Japanese Pediatric Participants With Solid Tumors or Lymphomas and Japanese Adult Participants With Merkel Cell Carcinoma (MK-3475-G21/KEYNOTE-G21)
Researchers are looking for new ways to treat people with solid tumors, lymphomas (blood cancers), and a certain type of skin cancer.
- Conditions
- Malignant Neoplasm, Carcinoma, Merkel Cell, Lymphoma
- Trial name
- NCT07302347
- Testing
- Pembrolizumab
- Where
- 7 sites in Japan
- Volunteers
- 20 people
- Sponsor
- Merck Sharp & Dohme LLC
The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3
Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.
The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome.
- Trial name
- THERIPEX (NCT07697118)
- Testing
- FOXP3-T4 drug product, ILT-101
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma
CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR)
This study is for patients who have lymphoma or leukemia that has come back or has not gone away after treatment. Because there is no standard treatment for this cancer, patients are being asked to volunteer for a gene transfer…
- Conditions
- Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma, Relapsed Adult ALL, Relapsed CLL
- Trial name
- NCT03774654
- Testing
- CD19.CAR-aNKT cells
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Baylor College of Medicine
Allogeneic Hematopoietic Stem Cell Transplant Recipient, Hematopoietic and Lymphoid Cell Neoplasm
Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant
This phase I trial studies the side effects of human lysozyme goat milk in preventing graft versus host disease in patients with blood cancer undergoing a donor stem cell transplant.
- Trial name
- NCT04177004
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Etoposide
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- City of Hope Medical Center
Diffuse Midline Glioma, H3 K27M-Mutant
Genetically Modified Cells (KIND T Cells) for the Treatment of HLA-A*0201-Positive Patients With H3.3K27M-Mutated Glioma
This phase I, first-in-human trial tests the safety, side effects, and best dose of genetically modified cells called KIND T cells after lymphodepletion (a short dose of chemotherapy) in treating patients who are…
- Trial name
- PNOC018 (NCT05478837)
- Testing
- Cyclophosphamide, Fludarabine, Autologous Anti-H3.3K27M TCR-expressing T-cells
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of California, San Francisco
Friedreich Ataxia
A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
- Trial name
- NCT06681766
- Testing
- Nomlabofusp
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Larimar Therapeutics, Inc.
Tuberculosis, HIV
Evaluating the Pharmacokinetics, Safety, and Tolerability of Bedaquiline in Infants, Children, and Adolescents With Multidrug-Resistant Tuberculosis, Living With or Without HIV
P1108 was a Phase I/II, open-label, single-arm, exposure-controlled dose finding study of BDQ in infants, children, and adolescents living with and without HIV, with clinically diagnosed or bacteriologically confirmed…
- Trial name
- NCT02906007
- Testing
- Bedaquiline
- Where
- 4 sites in Haiti, South Africa
- Volunteers
- 54 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Pediatric Cancer
European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors
This proof-of-concept platform trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe (Geoerger 2017; Geoerger 2019).
- Trial name
- ESMART (NCT02813135)
- Testing
- Ribociclib, Topotecan, Temozolomide
- Where
- 23 sites in Denmark, France and 4 more
- Volunteers
- 472 people
- Sponsor
- Gustave Roussy, Cancer Campus, Grand Paris
Nerve Sheath Tumors
Neoadjuvant Nivolumab Plus Ipilimumab for Newly Diagnosed Malignant Peripheral Nerve Sheath Tumor
The purpose of the study is to evaluate safety and feasibility of neoadjuvant nivolumab plus ipilimumab prior to standard therapy (surgery, chemotherapy or radiation therapy) in patients with Neurofibromatosis Type 1 (NF1) and…
- Trial name
- NCT04465643
- Testing
- Nivolumab, Ipilimumab
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Low Grade Glioma, High Grade Glioma
MAPK Inhibition Combined With Anti-PD1 Therapy for BRAF-altered Pediatric Gliomas
Pediatric gliomas harboring BRAF-alterations, commonly BRAFV600 mutation or KIAA1549-BRAF fusion, are currently treated with either chemotherapy or mitogen activated protein kinase (MAPK) inhibitors, such as, dabrafenib and/or…
- Trial name
- NCT06712875
- Testing
- Trametinib and Nivolumab, Dabrafenib, trametinib, nivolumab
- Where
- 3 sites in United States
- Volunteers
- 27 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Refractory Systemic Lupus Erythematosus
CD19 CAR T-Cell Therapy for Refractory Systemic Lupus Erythematosus
The goal of this Phase I clinical trial is to evaluate the safety and tolerability of autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy in adults with refractory systemic lupus erythematosus who have…
- Trial name
- NCT07432334
- Testing
- Autologous CD19-Targeted CAR-T Cells
- Where
- Vietnam
- Volunteers
- 8 people
- Sponsor
- Vinmec Research Institute of Stem Cell and Gene Technology
Sensorineural Hearing Loss
Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
To determine if autologous human umbilical cord blood infusion in children with acquired hearing loss is safe, feasible, improves inner ear function, audition and language development.
- Trial name
- NCT02038972
- Testing
- Autologous Stem Cells
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- James Baumgartner, MD
Non-Hodgkin Lymphoma, B-cell Non-Hodgkin Lymphoma (B-NHL)
Preliminary Safety and Tolerability of CD19x22 CAR T Cells in Adolescent and Adult R/R B-NHL Patients
This open-label, single arm phase 1 trial aims to determine the safety and tolerability of anti-CD19 and anti-CD22 chimeric antigen receptor-expressing (CAR) T cells (CD19x22 CAR T) in adolescents and adults with…
- Conditions
- Non-Hodgkin Lymphoma, B-cell Non-Hodgkin Lymphoma (B-NHL), Mantle Cell Lymphoma (MCL), CNS Lymphoma
- Trial name
- NCT05098613
- Testing
- CD19x22 CAR T Cells
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- University of Colorado, Denver
Sickle Cell Disease
Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)
The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose…
- Trial name
- NCT01499888
- Testing
- Allogeneic Non-Myeloablative Stem Cell Transplantation, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- University of Illinois Chicago
Spasticity, Muscle, Microcephaly
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss.
- Conditions
- Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation
- Trial name
- NCT05518188
- Testing
- MELPIDA
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Elpida Therapeutics SPC
IDH1-mutant Glioma, IDH2-mutant Glioma
Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma
The objective of this study is to determine the safety and tolerability of vorasidenib in combination with temozolomide (TMZ) and to establish the recommended combination dose (RCD) of vorasidenib.
- Trial name
- NCT06478212
- Testing
- Vorasidenib, Temozolomide (TMZ)
- Where
- 28 sites in United States, Austria and 9 more
- Volunteers
- 51 people
- Sponsor
- Institut de Recherches Internationales Servier
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
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.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
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