Clinical trials: First in Man Study to Evaluate Initial Safety
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
Cancer
Caspofungin Acetate Compared With Amphotericin B Liposomal in Treating Patients With Persistent Fever and Neutropenia Following Cancer Treatment
RATIONALE: Caspofungin acetate or amphotericin B liposomal may be effective in preventing or controlling fever and neutropenia caused by chemotherapy, bone marrow transplantation, or peripheral stem cell transplantation.
- Trial name
- NCT00008359
- Testing
- caspofungin acetate, liposomal amphotericin B
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
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.
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
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
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
Vaccine Delivery, Tetanus
A First in Human Study Investigating the Safety of an Unloaded and Loaded (With a Tetanus-containing Vaccine Booster) Microneedle Patch in Healthy Adults
The purpose of this research study is to check whether the MNS Miracus Patch (the study's investigational device, a small patch made up of microneedles that dissolve in the skin and can potentially deliver vaccines in a pain-free…
- Trial name
- Miracus™ FIH (NCT07624669)
- Testing
- Microneedle Patch, Tetanus-containing vaccine
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Microneedle Solutions Ltd
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
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
MECP2 Duplication Syndrome
A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)
Methyl-CpG binding protein 2 (MECP2) is a dosage-sensitive, X-linked gene critical for central nervous system development and functional maintenance, which gain-of-function causes MECP2 duplication syndrome (MDS).
- Trial name
- HERO (NCT06615206)
- Testing
- HG204
- Where
- China
- Volunteers
- 6 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
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
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
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
Neuronal Ceroid Lipofuscinosis Type 2
A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease
This is a registrational, first-in-human, open-label, dose escalation and expansion study evaluating the safety, tolerability and effectiveness of TTX-381, administered by injection under the retina, for the treatment of eye…
- Trial name
- NCT05791864
- Testing
- TTX-381
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Tern Therapeutics, LLC
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
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
Netherton Syndrome
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
This is a first-in-human, Phase 1/1b, 4-part study that includes the evaluation of safety, tolerability, pharmacokinetics (PK), and immunogenicity of BCX17725 when administered via single and multiple doses in healthy adult…
- Trial name
- NCT06539507
- Testing
- BCX17725
- Where
- 12 sites in United States, Australia and 3 more
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
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
Menopause, Urinary Incontinence
Evaluation of the Safety and Performance of the MILTA GYNECO Vaginal Probe in Postmenopausal Women With Vaginal Atrophy
Vulvovaginal atrophy is common after menopause, with a significant negative effect on quality of life. Large cohort studies have reported the prevalence of vaginal dryness to be between 27% and 55% and dyspareunia between 32% and…
- Conditions
- Menopause, Urinary Incontinence, Atrophic Vaginitis
- Trial name
- SIVAM (NCT05058313)
- Testing
- Photobiomodulation
- Where
- 3 sites in France
- Volunteers
- 25 people
- Sponsor
- Centre Hospitalier Universitaire de Nīmes
Osteosarcoma Metastatic, Soft Tissue Sarcoma (STS)
A Study to Assess Safety and Efficacy of SOT106 in Patients With LRRC15-positive Advanced Unresectable or Metastatic Osteosarcoma or Soft Tissue Sarcoma
SOT106 is a special cancer medicine designed to find and kill certain cancer cells that carry a marker called LRRC15, while causing less harm to healthy cells. The study consists of two parts, Part A and Part B.
- Conditions
- Osteosarcoma Metastatic, Soft Tissue Sarcoma (STS), Osteosarcoma, Advanced
- Trial name
- NCT07801222
- Testing
- SOT106
- Where
- Moldova
- Volunteers
- 70 people
- Sponsor
- SOTIO Biotech a.s.