Clinical trials: Early Phase 1
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
Relapsed/Refractory Acute Myeloid Leukemia
Safety and Efficacy of NK520 to Treat Pediatric Relapsed/Refractory Acute Myeloid Leukemia
This study will evaluate the safety and efficacy of NK520 in the treatment of pediatric relapsed/refractory acute myeloid leukemia. NK520 will be administered by intravenous injection.
- Trial name
- NCT06541405
- Testing
- NK520
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Base Therapeutics (Shanghai) Co., Ltd.
Bardet-Biedl Syndrome 1, Retinal Degeneration
First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration
The goal of this first in human study is to evaluate the preliminary safety and tolerability of AXV-101 in participants with BBS1.
- Trial name
- AXIS (NCT07269665)
- Testing
- AXV-101
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Axovia Therapeutics
Duchenne Muscular Dystrophin (DMD)
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys.
- Trial name
- MUSCLE (NCT06594094)
- Testing
- HG302
- Where
- China
- Volunteers
- 4 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Hailey Hailey Disease, Skin Diseases
A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease
- Conditions
- Hailey Hailey Disease, Skin Diseases, Lesion Skin
- Trial name
- HALITE-1 (NCT07717346)
- Testing
- KB111
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Krystal Biotech, Inc.
Beta-Thalassemia
CS-101 in Patients With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06328764
- Testing
- CS-101
- Where
- China
- Volunteers
- 10 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Childhood CNS Tumor
SGT-53 in Children With Recurrent or Progressive CNS Malignancies
An early phase 1 for pediatric patients with recurrent or progressive CNS malignancies
- Trial name
- NCT03554707
- Testing
- SGT-53, Radiation, Irinotecan
- Where
- United States
- Sponsor
- SynerGene Therapeutics, Inc.
B Cell Lymphoma, B Cell Leukemia
Universal CAR-T Cells (REVO-UWD-19) for Refractory and Relapsed B-Cell Tumors
This study is a single-arm, single-center, investigator-initiated clinical trial. The primary objective is to evaluate the safety and preliminary efficacy of administering universal CD19 CAR-T cells to subjects with refractory…
- Conditions
- B Cell Lymphoma, B Cell Leukemia, B Cell Malignancy
- Trial name
- NCT06662227
- Testing
- Single dose injection of certain dose of UWD-19, MMF Immunosuppression
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Wondercel Biotech (ShenZhen)
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
X-linked Retinoschisis
Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients
This trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
- Trial name
- NCT06066008
- Testing
- ZM-01-L, ZM-01-H
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Zhongmou Therapeutics
Sensorineural Hearing Loss, Tinnitus
Regenerative Medicine and Stem Cell-Based Interventions for Inner Ear Trauma, Tinnitus, and Sensorineural Hearing Loss
The HEAR-RESTORE study is an exploratory clinical research program designed to evaluate new diagnostic and regenerative approaches for individuals experiencing inner ear injury, tinnitus, and temporary or permanent hearing loss.
- Conditions
- Sensorineural Hearing Loss, Tinnitus, Acoustic Trauma, Inner Ear Injury
- Trial name
- HEAR-RESTORE (NCT07472023)
- Testing
- Audiologic Monitoring, Investigational Regenerative Pharmacologic Therapy, Stem Cell Regenerative Therapy
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Truway Health, Inc.
Beta-Thalassemia
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06024876
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Sickle Cell Disease
CS-206 in Patients With Sickle Cell Disease
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.
- Trial name
- NCT06565026
- Testing
- CS-206
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Pediatric Bronchiolitis Obliterans
Treatment of Pediatric Bronchiolitis Obliterans by Airway Basal Stem Cells
Bronchiolitis obliterans (BO) is a chronic lung disease which was initiated with injury of the bronchiolar epithelium and resulted in nonuniform luminal obliteration or narrowing.
- Trial name
- NCT07239895
- Testing
- Airway Basal Stem Cells
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Regend Therapeutics
Colorectal Cancer Metastatic, Colorectal Cancer Stage III
COLONYVAQ™, a Quantum-Classical Guided Personalized Neoantigen Vaccine for MSS Stage III Colorectal Cancer
This is an early phase I, single-arm, open-label clinical study designed to evaluate the safety, tolerability, and feasibility of COLONYVAQ-CRC, a physics-aware, quantum-classical AI-guided personalized neoantigen peptide…
- Conditions
- Colorectal Cancer Metastatic, Colorectal Cancer Stage III, Colorectal Cancer Stage IV
- Trial name
- COLONYVAQ™-CRC (NCT07328087)
- Testing
- Experimental: COLONYVAQ-CRC + mFOLFOX6 or CAPOX + Nivolumab
- Where
- Greece
- Volunteers
- 12 people
- Sponsor
- Biogenea Pharmaceuticals Ltd.
Endometrial Carcinoma, Endometrial Carcinoma Stage III
GYNORYLAQ™-VLINIVAL™: Ψ-Guided Personalized Neoantigen Peptide Vaccine for High-Risk Endometrial Cancer
GYNORYLAQ-VLINIVAL is an Early Phase I, non-randomized, single-arm, open-label clinical trial enrolling 40 patients with high-risk or recurrent endometrial carcinoma.
- Trial name
- GYNORYLAQ™ (NCT07316361)
- Testing
- • Biological: GYNORYLAQ-TM Personalized Neoantigenic Peptide Vaccine
- Where
- Greece
- Volunteers
- 40 people
- Sponsor
- Biogenea Pharmaceuticals Ltd.
Cellular Injury and Post-Cryogenic Recovery, Cryogenic Cellular Stress
Evaluation of In-Vitro Cryo Therapeutic Protocols on Human Cell Samples (TWH-CRYO-001)
This laboratory-based study evaluates the effects of controlled cryogenic preservation on human cell samples using Truway Health's in-vitro cryo therapeutic methodology.
- Conditions
- Cellular Injury and Post-Cryogenic Recovery, Cryogenic Cellular Stress, Cold-Induced Cellular Injury, Thermal Injury Response
- Trial name
- CRYO-IVT (NCT07321028)
- Testing
- Standard Laboratory Cryopreservation Procedure, Enhanced Laboratory Cryopreservation Procedure, Normothermic Cell Culture Control
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Truway Health, Inc.
Healthy Volunteer
A Single-volunteer Study of Immune Response Following OTS-412 Vaccinia Virus Systemic Injection(Self-experiment)
Following oncolytic virus (OV) administration, the host immune response is initiated immediately and evolves dynamically. However, in clinical OV therapy, it is virtually impossible to capture these immune events in real time.
- Trial name
- NCT07302945
- Testing
- OTS-412
- Where
- South Korea
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Bionoxx Inc.
NSCLC (Non-small Cell Lung Cancer), Squamous Lung Cancer With FGFR1 Amplification
Clinical Study of the Therapeutic Effectiveness of In-silico-Designed, Machine Learning Inspired, and Quantum-molecularly Coupled Personalized Neoantigenic Vaccines Microlyvaq™ in Patients With Advanced Non-small Cell Lung Cancer
Microlyvaq™ is a first-line, non-randomized, two-arm clinical trial in advanced non-small cell lung cancer (NSCLC). In both arms, patients receive a personalized multi-epitope vaccine (Microlyvaq™) on top of standard-of-care…
- Conditions
- NSCLC (Non-small Cell Lung Cancer), Squamous Lung Cancer With FGFR1 Amplification, Non-Squamous Non Small Cell Lung Cancer
- Trial name
- Microlyvaq™ (NCT07285434)
- Testing
- Microlyvaq™ (Personalized Multi-Epitope Immunotherapeutic)
- Where
- Greece
- Volunteers
- 90 people
- Sponsor
- Biogenea Pharmaceuticals Ltd.
Chronic Disease, Type 2 Diabetes Mellitus (T2DM), Obesity, Overweight
Truway Diagnostic Tools in Primary Care
This study evaluates the effectiveness of Truway Health-sourced portable diagnostic devices, including ultrasound and blood glucose monitors, in early detection of chronic conditions in outpatient primary care settings.
- Conditions
- Chronic Disease, Type 2 Diabetes Mellitus (T2DM), Obesity, Overweight, Hypertension, Cardiovascular Disease Acute
- Trial name
- TruDxPC (NCT07209527)
- Testing
- Truway Portable Ultrasound Device, Truway Blood Glucose Monitor, Standard Oral Hypoglycemic Agent
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Truway Health, Inc.