Clinical trials: First in Man Study to Evaluate Initial Safety
Stargardt Disease, Stargardt Macular Degeneration
A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
- Trial name
- ASTRA (NCT06942572)
- Testing
- SB-007
- Where
- 5 sites in United States
- Volunteers
- 57 people
- Sponsor
- Splice Bio
Acute Myeloid Leukemia
Safety and Efficacy Study of CI-135 CAR-T Cells in Subjects with Relapsed or Refractory Acute Myeloid Leukemia
This is a FIH, single center, open label, non-randomized, single-arm, Phase I clinical trial to evaluate the safety and efficacy of CI-135 CAR-T cells in subjects with relapsed or refractory Acute Lymphoblastic Leukemia.
- Trial name
- NCT05266950
- Testing
- CI-135 CAR-T cells (0.5 x 10^6 CAR-T+ cells/kg，1.0 x 10^6 CAR-T+ cells/kg)
- Where
- China
- Sponsor
- Beijing Boren Hospital
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Infantile GM2 Gangliosidosis (Disorder)
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of…
- Trial name
- NCT04798235
- Testing
- TSHA-101
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Dr. Anupam Sehgal
T-Cell Acute Lymphoblastic Leukemia/Lymphoma
Donor-Derived CD5 CAR T (CT125B) Cells for Relapsed or Refractory T- Cell Acute Lymphoblastic Leukemia/Lymphoma
This is a FIH, single center, open label, non-randomized, single-arm, Phase I clinical trial to evaluate the safety and tolerability of CD5 CAR T (CT125B) cells in subjects with relapsed or refractory T-cell acute lymphoblastic…
- Trial name
- NCT05487495
- Testing
- CD5 CAR T (CT125B)
- Where
- China
- Sponsor
- Beijing Boren Hospital
Stargardt Disease 1, STGD1
A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants
This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following…
- Conditions
- Stargardt Disease 1, STGD1, Healthy Volunteers
- Trial name
- NCT07836452
- Testing
- CITY-RBP4
- Where
- Australia
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- City Therapeutics
FOXG1 Syndrome
Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)
The goal of this clinical trial is to learn if FRF-001 is a safe, tolerable, and efficacious treatment for children and adults with FOXG1 syndrome.
- Trial name
- NCT07293546
- Testing
- FRF-001
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- FOXG1 Research Foundation
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Trial name
- NCT06255782
- Testing
- ECUR-506
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 20 people
- Sponsor
- iECURE, Inc.
Ovarian Cancer, Endometrial Cancer
Phase 1, Safety and Tolerability Study of XmAb541 in Advanced Solid Tumors
The primary purpose of this study is to determine whether the investigational drug XmAb541 is safe and well tolerated, and to determine an optimal and safe dose(s) for further study.
- Conditions
- Ovarian Cancer, Endometrial Cancer, Germ Cell Tumor, Testicular Germ Cell Tumor
- Trial name
- NCT06276491
- Testing
- XmAb541
- Where
- 27 sites in United States, Belgium and 4 more
- Volunteers
- 282 people
- Sponsor
- Xencor, Inc.
Kawasaki Disease Refractory to Initial Therapy With Intravenous Immunoglobulin
Clinical Study of TA-650 in Patients With Refractory Kawasaki Disease
The purpose of this study is to evaluate the efficacy and safety of TA-650 in comparison with a control drug Polyethylene Glycol-treated Human Immunoglobulin (VGIH) in patients with Kawasaki disease refractory to initial therapy…
- Trial name
- NCT01596335
- Testing
- TA-650, Polyethylene Glycol-treated Human Immunoglobulin (VGIH)
- Where
- 8 sites in Japan
- Volunteers
- 31 people
- Sponsor
- Tanabe Pharma Corporation
Netherton Syndrome
A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton Syndrome
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndroms, a rare inflammatory skin disease.
- Trial name
- NCT05211830
- Testing
- SXR1096 cream
- Where
- 5 sites in Austria, France and 2 more
- Volunteers
- 9 people
- Sponsor
- Sixera Pharma
Acute Ischemic Stroke
First-in-man Study of the Cerebrovascular Interventional Procedural Control System
The objective of this study the first human use of a new device in treatment of acute ischemic stroke, which to evaluate the feasibility, safety, and efficacy of the Cerebrovascular Interventional Procedural Control System.
- Trial name
- NCT05972252
- Testing
- Cerebrovascular Interventional Procedural Control System
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Hangzhou Dinova Neuroscience Technology Co., Ltd
Papillomavirus Infections, Coronavirus Disease (COVID-19)
Safety and Immunogenicity of 9-valent Human Papillomavirus (9vHPV) Vaccine Coadministered With Messenger Ribonucleic Acid (mRNA)-1273 Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) (COVID-19) Vaccine (V503-076)
The purpose of this study to evaluate the safety and immunogenicity of a 2-dose regimen of 9vHPV vaccine, where the first dose is administered concomitantly with a first dose of a 2-dose regimen of mRNA-1273 vaccine versus…
- Trial name
- NCT05119855
- Testing
- 9vHPV Vaccine, mRNA-1273 Vaccine
- Where
- 38 sites in United States
- Volunteers
- 165 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Healthy Volunteers, Propionic Acidemia
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.
- Conditions
- Healthy Volunteers, Propionic Acidemia, Methylmalonic Acidemia, Organic Acidemia
- Trial name
- NCT04836494
- Testing
- BBP-671
- Where
- 3 sites in United States
- Volunteers
- 79 people, healthy volunteers welcome
- Sponsor
- CoA Therapeutics, Inc., a BridgeBio company
NSCLC (Advanced Non-small Cell Lung Cancer)
A Study to Learn the Safety and Efficacy of HLX37 in Combination With Chemotherapy for First-line Treatment in Adult Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer
This is a Randomized, Double-Blind, Multicenter, Phase II/III Study to Evaluate HLX37 (Recombinant Human Anti-PD-L1 and Anti-VEGF Bispecific Antibody) in Combination with Chemotherapy Versus Pembrolizumab in Combination with…
- Trial name
- NCT07865910
- Testing
- Investigational drug: HLX37 dose1, IV, Q3W, PEMBROLIZUMAB (alone or when added to a regimen above), Carboplatin (Investigator discretion)
- Where
- 16 sites in China
- Volunteers
- 1,438 people
- Sponsor
- Shanghai Henlius Biotech
Primary Mitochondrial Diseases
A First in Human Study to Evaluate the Safety of Infusion of MNV-BM-PLC (Autologous CD34+ Cells Enriched With Placenta Derived Allogeneic Mitochondria) in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion
The study objectives are to evaluate the safety of a single intravenous (IV) infusion of autologous CD34+ cells enriched with placenta-derived allogeneic mitochondria in participant with primary mitochondrial disease associated…
- Trial name
- NCT04548843
- Testing
- Bone Marrow mobilization, Apheresis, MNV-BM-PLC infusion
- Where
- Israel
- Sponsor
- Minovia Therapeutics Ltd.
Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia
Safety and Tolerability of HSC835 in Patients With Hematological Malignancies
This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies.
- Conditions
- Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT01474681
- Testing
- HSC835
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Pitt Hopkins Syndrome
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
The goal of this clinical trial is to learn if MZ-1866 is a safe and tolerable treatment for children and adults with Pitt Hopkins Syndrome.
- Trial name
- NCT07135050
- Testing
- MZ-1866
- Where
- 4 sites in United States, Israel
- Volunteers
- 12 people
- Sponsor
- Mahzi Therapeutics
T-Cell Acute Lymphoblastic Leukemia
Donor-Derived CD5 CAR T Cells in Subjects with Relapsed or Refractory T-Cell Acute Lymphoblastic Leukemia
This is a FIH, single center, open label, non-randomized, single-arm, Phase I clinical trial to evaluate the safety and tolerability of CD5 CAR T cells in subjects with relapsed or refractory T-cell acute lymphoblastic leukemia.
- Trial name
- NCT05032599
- Testing
- CD5 CART
- Where
- China
- Volunteers
- 19 people
- Sponsor
- Beijing Boren Hospital
Infections, Rotavirus, Rotavirus Vaccines
To Evaluate Safety & Reactogenicity of GSK Bio's Human Rotavirus (HRV) Vaccine in Filipino Infants at Least 6 Weeks of Age at First Vaccination
GSK has submitted a registration file for its live attenuated oral human rotavirus (HRV) vaccine in several countries. In view of its registration in the Philippines, the present study will collect data on the safety of the…
- Trial name
- NCT00353366
- Testing
- Rotarix, Data collection
- Where
- 18 sites in Philippines
- Volunteers
- 1,439 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline