Clinical trials: Gene Therapy
Synovial Sarcomas
Multi-center Study of TBI-1301 (INN: Mipetresgene Autoleucel; Mip-cel) in Patients With NY-ESO-1 Positive Synovial Sarcoma
The purpose of this study is to verify the safety and the efficacy of TBI-1301 for NY-ESO-1 expressing synovial sarcoma when administered following cyclophosphamide/fludarabine pre-treatment.
- Trial name
- NCT07174427
- Testing
- TBI-1301, Cyclophosphamide, Fludarabine
- Where
- 3 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takara Bio Inc.
Non Small Cell Lung Cancer
Quaratusugene Ozeplasmid (Reqorsa) in Combination With Pembrolizumab in Previously Treated Non-Small Lung Cancer
The purpose of this study is to determine the safety and efficacy of quaratusugene ozeplasmid (Reqorsa), in combination with pembrolizumab in patients with previously treated NSCLC.
- Trial name
- Acclaim-2 (NCT05062980)
- Testing
- quaratusugene ozeplasmid, pembrolizumab, docetaxel
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Genprex, Inc.
Neovascular Age-Related Macular Degeneration (nAMD)
Open-laBel Dose-escalation Study for CRISPR/cas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial
Age-related macular degeneration (AMD) leads to severe and irreversible vision loss, while neovascular AMD (nAMD) accounts for 80-90% of AMD blindness.
- Trial name
- BRIGHT (NCT06623279)
- Testing
- HG202
- Volunteers
- 15 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Hemophilia B, Blood Coagulation Disorders, Inherited
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Freeline is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB).
- Conditions
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease
- Trial name
- ECLIPSE (NCT04272554)
- Where
- 56 sites in United States, Australia and 10 more
- Volunteers
- 37 people
- Sponsor
- Spur Therapeutics
Hemophilia A
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any…
- Trial name
- NCT03432520
- Testing
- SPK-8011, SPK-8016
- Where
- 12 sites in United States, Australia and 1 more
- Volunteers
- 29 people
- Sponsor
- Spark Therapeutics, Inc.
Alzheimer's Disease
CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease
This is a Phase I clinical study to assess the safety, tolerability and biologic activity of in vivo AAV-mediated delivery of CERE-110. Up to 12 subjects will receive open label CERE-110 in dose-escalating fashion.
- Trial name
- NCT00087789
- Testing
- CERE-110: Adeno-Associated Virus Delivery of NGF
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sangamo Therapeutics
Spinal Muscular Atrophy (SMA)
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .
- Trial name
- NCT06888661
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 4 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Neovascular Age-related Macular Degeneration
Safety and Efficacy of ADVM-022 in Treatment-Experienced Patients With Neovascular Age-related Macular Degeneration [LUNA]
Neovascular or wet age-related macular degeneration (nAMD) is a degenerative ocular disease associated with the infiltration of abnormal blood vessels in the retina from the underlying choroid layer and is a leading cause of…
- Trial name
- NCT05536973
- Testing
- ADVM-022
- Where
- 39 sites in United States, France and 1 more
- Volunteers
- 69 people
- Sponsor
- Adverum Biotechnologies, Inc.
Advanced NSCLC
Real-World Study on Gene Profile in Patients With Advanced NSCLC Who Progressed on First-Line Osimertinib Therapy(GPS).
Although some small sample studies have reported the possible resistance mechanisms of Osimertinib in the first-line treatment, it is still an urgent need to explore the whole gene profile in EGFRm advanced NSCLC patients post…
- Trial name
- GPS (NCT05219162)
- Testing
- Gene Profile explore
- Where
- 16 sites in China
- Volunteers
- 182 people
- Sponsor
- AstraZeneca
Achromatopsia
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene.
- Trial name
- NCT02935517
- Testing
- AGTC-402
- Where
- 6 sites in United States, Israel
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics