Clinical trials: Early Manifest Huntington Disease, active
Huntington Disease
GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).
- Trial name
- NCT05686551
- Testing
- Tominersen
- Where
- 70 sites in United States, Argentina and 13 more
- Volunteers
- 301 people
- Sponsor
- Hoffmann-La Roche
Huntington Disease
Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease
This is the second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon…
- Trial name
- NCT05243017
- Testing
- intra-striatal rAAV5-miHTT
- Where
- 4 sites in Poland, United Kingdom
- Volunteers
- 14 people
- Sponsor
- UniQure Biopharma B.V.
ALS, Amyotrophic Lateral Sclerosis
NYSCF Scientific Discovery Biobank
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate diverse disease research using cells from the body (such as skin or blood cells) to make stem cells and other types of cells…
- Conditions
- ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset
- Trial name
- NCT06203106
- Testing
- Biological Sample Collection
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- New York Stem Cell Foundation Research Institute
Huntington Disease
A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.
This is a dose escalation and expansion clinical study to evaluate the safety, tolerability, PK profile and preliminary efficacy of ER2001 Injection vs. placebo in subjects with definitive diagnosis of early manifest HD.
- Trial name
- NCT07339514
- Testing
- ER2001 intravenous injection
- Where
- 3 sites in China
- Volunteers
- 27 people
- Sponsor
- ExoRNA Bioscience
Huntington's Disease
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study.
- Trial name
- NCT04120493
- Testing
- intra-striatal rAAV5-miHTT
- Where
- 12 sites in United States
- Volunteers
- 43 people
- Sponsor
- UniQure Biopharma B.V.
Huntington Disease
Evaluation of Three Tests to Assess Social Cognition in Huntington Disease
The goal of this observational study is to learn about the usefulness of a test of social functioning in persons with Huntington disease.
- Trial name
- NCT07253038
- Where
- Sweden
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Region Skane
Huntington Disease
A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease
A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease
- Trial name
- NCT05541627
- Testing
- AB-1001 Gene Therapy
- Where
- France
- Volunteers
- 5 people
- Sponsor
- AskBio France, SAS, a subsidiary of AskBio Inc.