Clinical trials: Phase 1
Peanut Allergy
Study of ENP-501 in Peanut-Allergic and Non-Allergic Participants
This is a Phase 1/2 study to evaluate the safety and clinical activity of ENP-501 in non-peanut allergic (NPA) healthy participants and participants with an established peanut allergy (PA)
- Trial name
- NCT07117669
- Testing
- ENP-501
- Where
- United States
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- N-Fold, LLC
Pediatric Brain Tumor, Recurrent Pediatric Brain Tumor
Intra-Tumoral Injections of Natural Killer Cells for Recurrent Malignant Pediatric Brain Tumors
This phase I trial tests the safety, side effects, and best dose of ex vivo expanded natural killer cells in treating patients with cancerous (malignant) tumors affecting the upper part of the brain (supratentorial) that have…
- Conditions
- Pediatric Brain Tumor, Recurrent Pediatric Brain Tumor, Pediatric Neoplasm
- Trial name
- PNOC028 (NCT05887882)
- Testing
- Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Implantation, Magnetic Resonance Imaging (MRI)
- Where
- 10 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sabine Mueller, MD, PhD
Neuroblastoma
Multi-modular Chimeric Antigen Receptor Targeting GD2 in Neuroblastoma
MAGNETO is a single-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children and teenagers aged 1-16 years with relapsed or refractory neuroblastoma.
- Trial name
- MAGNETO (NCT05990751)
- Testing
- GD2 CAR T cells
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University College, London
Hemophagocytic Lymphohistiocytosis
Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis
This study is a multi-site Phase Ib/II, 2-arm non-randomized clinical trial to determine the efficacy and tolerability of a response-adapted regimen combining ruxolitinib, dexamethasone, and etoposide as Frontline therapy for…
- Trial name
- NCT04551131
- Testing
- Ruxolitinib, Dexamethasone, Etoposide
- Where
- 13 sites in United States
- Volunteers
- 10 people
- Sponsor
- St. Jude Children's Research Hospital
Malaria (Plasmodium Falciparum), Malaria Falciparum
Safety and PK of MMV371 LAI in Healthy Adults and Adolescents in Rwanda
This Phase 1b study will assess the safety, tolerability and pharmacokinetics (PK, this measures the levels of study drug in the body) of a single injection of MMV371 in healthy adult and adolescent participants in Rwanda.
- Conditions
- Malaria (Plasmodium Falciparum), Malaria Falciparum, Malaria Infection, Malaria Prophylaxis
- Trial name
- NCT07548021
- Testing
- MMV371
- Where
- Rwanda
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Medicines for Malaria Venture
Osteonecrosis of Femoral Head
The Study of Early Stage Osteonecrosis of Femoral Head With Human Umbilical Cord Mesenchymal Stem Cells (19#iSCLife®-ONFH)
The purposes of the study is to evaluate the safety and efficacy of human umbilical cord mesenchymal stem cells (hUC-MSC) in treating osteonecrosis of femoral head patients.
- Trial name
- NCT03180463
- Testing
- Allogeneic umbilical cord mesenchymal stem cells (SCLnow 19#), core decompression
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Sclnow Biotechnology Co., Ltd.
Malignant Glioma, High Grade Glioma
Adoptive Cellular Therapy in Pediatric Patients With High-grade Gliomas
It is believed that the body's immune system protects the body by attacking and killing tumor cells. T-lymphocytes (T-cells) are part of the immune system and can attack when they recognize special proteins on the surface of…
- Trial name
- ACTION (NCT03334305)
- Testing
- TTRNA-DC vaccines with GM-CSF, Dose-intensified TMZ, Autologous Hematopoietic Stem cells (HSCs)
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- University of Florida
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease
Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures.
- Trial name
- NCT03077542
- Testing
- haploidentical stem cell transplant, Sirolimus, Alemtuzumab
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sarcoma,Soft Tissue
MEK Inhibitor and a PDL1 Inhibitor Patients With Locally Advanced and/or Metastatic Soft Tissue Sarcoma
The proposed study conducted in adult and pediatric patients aims to evaluate the safety and clinical activity of atezolizumab + cobimetinib in advanced/metastatic soft tissue sarcomas (up to 80 patients).
- Trial name
- COTESARC (NCT04216953)
- Testing
- Cobimetinib, Atezolizumab
- Where
- 6 sites in France
- Volunteers
- 229 people
- Sponsor
- Centre Leon Berard
CAR T Cell Therapy, CD33 Positive Acute Myelogenous Leukemia
Functionally Optimized CD33 CAR-T Cell Therapy Targeting Recurrent/Refractory Acute Myeloid Leukemia
Relapsed/refractory acute myeloid leukemia (R/R AML) currently lacks effective CAR-T therapeutic agents due to the absence of tumor-specific target antigens.
- Trial name
- NCT07538713
- Testing
- Functionally optimized CD33 CAR-T
- Volunteers
- 18 people
- Sponsor
- Qi deng
Acute Lymphocytic Leukemia, Acute Myeloid Leukemia
Pilot Imaging Study of Leukemia
This is a prospective pilot study, the primary aim of which is to determine whether the presence of 18F FLT imaging signal uptake abnormalities correlate with clinically validated evidence of hematopoietic malignant disease (e.g.
- Conditions
- Acute Lymphocytic Leukemia, Acute Myeloid Leukemia, Ambiguous Lineage Leukemia or Lymphoma, Myeloma
- Trial name
- REALIZE (NCT03633955)
- Testing
- FLT
- Where
- 3 sites in United States
- Volunteers
- 60 people
- Sponsor
- University of Oklahoma
Healthy Volunteers, OTC Deficiency
Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype
The objective of this clinical study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending doses of CMP-CPS-001 administered as a subcutaneous injection in adult healthy…
- Trial name
- NCT06247670
- Testing
- CMP-CPS-001
- Where
- 2 sites in Australia, Netherlands
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- CAMP4 Therapeutics Corporation
Osteosarcoma, Neuroblastoma
iC9-GD2-CAR-VZV-CTLs/Refractory or Metastatic GD2-positive Sarcoma and Neuroblastoma
The purpose of this study is to find the largest safe dose of GD2-T cells (also called iC9-GD2-CAR-VZV-CTLs) in combination with a varicella zoster vaccine and lymohodepleting chemotherapy.
- Trial name
- VEGAS (NCT01953900)
- Testing
- GD2 T cells, VZV vaccine, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 26 people
- Sponsor
- Baylor College of Medicine
Acute Lymphoid Leukemia Relapse, Acute Lymphoid Leukemia Relapse After Bone Marrow Transplant
Safety of Clofarabine With Multiagent Chemotherapy in Childhood Acute Lymphoblastic Leukemia
The purpose of this study is to determine Maximum Tolerated Dosage (MTD), Dosage Limited Toxicities (DLT), and the Rate Phase 2 Dosage of clofarabine when used in combination with etoposide, asparaginase, mitoxantrone and…
- Trial name
- Vandevol (NCT01279096)
- Testing
- Clofarabine
- Where
- 2 sites in France
- Volunteers
- 20 people
- Sponsor
- University Hospital, Lille
Acute Myeloid Leukemia
Study of Talazoparib in Combination With Chemotherapy in Relapsed Pediatric AML to Determine Safety and Efficacy
This is a Phase 1, open label, multicenter, dose finding study with dose expansion intended to evaluate the safety and tolerability of talazoparib in combination with conventional chemotherapy.
- Trial name
- PARPAML (NCT05101551)
- Testing
- Talazoparib, Topotecan, Gemcitabine
- Where
- 9 sites in United States
- Volunteers
- 34 people
- Sponsor
- Norman J. Lacayo
Giant Axonal Neuropathy (GAN)
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous…
- Trial name
- NCT07543991
- Testing
- scAAV9/JeT-GAN
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Metachromatic Leukodystrophy (MLD)
An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy
The purpose of this study is to collect long-term safety data in participants with metachromatic leukodystrophy (MLD) who are receiving HGT-1110 and have participated in Study HGT-MLD-070 (NCT01510028) through Week 40.
- Trial name
- NCT01887938
- Testing
- HGT-1110
- Where
- 15 sites in Australia, Brazil and 5 more
- Volunteers
- 24 people
- Sponsor
- Shire
Brain Tumor
Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric DIPG
The purpose of this study is to evaluate the safety and efficacy of targeted blood brain barrier disruption with Exablate Model 4000 Type 2.0/2.1 in combination with Doxorubicin therapy for the treatment of DIPG in pediatric…
- Trial name
- NCT05615623
- Testing
- Exablate, Doxorubicin
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- InSightec
Severe Combined Immunodeficiency
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat…
- Trial name
- NCT01129544
- Testing
- Gene transfer
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- David Williams