Clinical trials: Gene Therapy
Multiple Myeloma in Relapse, Myeloma Multiple
A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma
The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.
- Conditions
- Multiple Myeloma in Relapse, Myeloma Multiple, Multiple Myeloma Progression, Neoplasms by Histologic Type
- Trial name
- inMMyCAR (NCT07075185)
- Testing
- KLN-1010
- Where
- 11 sites in United States, Australia
- Volunteers
- 70 people
- Sponsor
- Kelonia Therapeutics, Inc.
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies
Hemophilia A, Gene Therapy
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe…
- Conditions
- Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder
- Trial name
- GENEr8-AAV5+ (NCT03520712)
- Testing
- Valoctocogene Roxaparvovec
- Where
- 9 sites in South Africa, South Korea and 2 more
- Volunteers
- 3 people
- Sponsor
- BioMarin Pharmaceutical
Metastatic (Stage IV) Non-squamous Non-small Cell Lung Cancer (Nsq-NSCLC) With Driver Gene Negativity, Untreated With Systemic Therapy
Clinical Study on Nanocrystalline Megestrol Acetate for Appetite Improvement and Weight Gain in Pre-Cachexia and Cachexia Stages Key Terminology Analysis
The purpose of this study is to evaluate the efficacy and safety of nanocrystalline medroxyprogesterone acetate for improving appetite and weight gain patients with non-small cell lung cancer (NSCLC) and cachexia and…
- Trial name
- NCT06961188
- Testing
- Nano-crystalline Megestrol Acetate Oral Suspension, Standard Therapy
- Where
- China
- Volunteers
- 174 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Dry Age-related Macular Degeneration, Geographic Atrophy
Intravitreal AAVCAGsCD59 for Advanced Dry Age-related Macular Degeneration (AMD) With Geographic Atrophy (GA)
Patients with advanced dry AMD with GA meeting inclusion criteria will be randomized in one eye in a 1:1:1 ratio comparing intravitreal high or low dose AAVCAGsCD59 with a sham injection.
- Conditions
- Dry Age-related Macular Degeneration, Geographic Atrophy, Gene Therapy, Intravitreal Injection
- Trial name
- NCT04358471
- Testing
- Intravitreal AAVCAGsCD59
- Sponsor
- Hemera Biosciences
Macular Degeneration, Age-Related Maculopathies
Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)
This Phase 1 clinical research study will examine the safety and tolerability of an experimental gene transfer agent, AAV2-sFLT01, in patients with Neovascular Age-Related Macular Degeneration (AMD).
- Conditions
- Macular Degeneration, Age-Related Maculopathies, Age-Related Maculopathy, Maculopathies, Age-Related
- Trial name
- NCT01024998
- Testing
- AAV2-sFLT01
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- Genzyme, a Sanofi Company