Clinical trials: Early Manifest Huntington Disease
Huntington's Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease
This study tested the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of ISIS 443139 administered intrathecally to adult participants with early manifest Huntington's Disease.
- Trial name
- NCT02519036
- Testing
- ISIS 443139 10 mg, ISIS 443139 30 mg, ISIS 443139 60 mg
- Where
- 9 sites in Canada, Germany and 1 more
- Volunteers
- 46 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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
Early Manifest Huntington Disease
A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease
This is the first study of branaplam in adults with Huntington's Disease (HD) to determine the correct dose required to lower mutant huntingtin protein (mHTT) levels in the cerebrospinal fluid (CSF) to a degree expected to be…
- Trial name
- VIBRANT-HD (NCT05111249)
- Testing
- Branaplam
- Where
- 12 sites in Canada, France and 3 more
- Volunteers
- 26 people
- Sponsor
- Novartis Pharmaceuticals
Huntington Disease
The Safety and Tolerability Study With ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease
This is an open label, dose escalation clinic trial of ER2001 intravenous injection to evaluate safety, tolerability and pharmacokinetics of ascending single and multiple doses of intravenously administered ER2001 in patients…
- Trial name
- NCT06024265
- Testing
- ER2001 injection
- Where
- China
- Volunteers
- 10 people
- Sponsor
- ExoRNA Bioscience
Huntington's Disease
A Study in Subjects With Late Prodromal & Early Manifest HD to Assess the Safety, Tolerability, pk, and Efficacy of Pepi
The purpose of this study is to evaluate the safety, tolerability, PK, and efficacy of Pepinemab in subjects with late prodromal and early manifest Huntington's disease.
- Trial name
- SIGNAL-HD (NCT02481674)
- Testing
- VX15/2503
- Where
- 32 sites in United States, Canada
- Volunteers
- 301 people
- Sponsor
- Vaccinex Inc.
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.
Huntington Disease
The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease.
This is an open label, dose escalation clinic trial to evaluate safety, tolerability and pharmacokinetics with ER2001 Intravenous Injection repetitive treatment in Huntington's Disease patients who Completed Study…
- Trial name
- NCT07344519
- Testing
- ER2001 injection
- Where
- China
- Volunteers
- 8 people
- Sponsor
- ExoRNA Bioscience
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'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
An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients With SEN0014196
The primary objective of this study is to provide biological samples from patients with Huntington's disease to allow characterisation of the pharmacological mechanism of action of SEN0014196.
- Trial name
- PADDINGTON (NCT01485952)
- Testing
- SEN0014196 (Low Dose), SEN0014196 (High Dose)
- Where
- Germany
- Volunteers
- 55 people
- Sponsor
- Siena Biotech S.p.A.
Huntington Disease
Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease
This is the first clinical study of VY-HTT01, a gene therapy for early-stage Huntington's Disease (HD) patients. The primary goal of this trial is to evaluate the safety and tolerability of VY-HTT01.
- Trial name
- NCT04885114
- Testing
- Intraparenchymal rAAV1 - (mi)RNA HTT
- Sponsor
- Voyager Therapeutics
Huntington's Disease
FuRST 2.0 Cognitive Pre-Testing
The FuRST 2.0 scale is being developed as a Patient Reported Outcome (PRO) with information coming directly from the Huntington's Disease Gene Expansion Carrier (HDGEC) and companion through self-report.
- Trial name
- NCT02881931
- Testing
- FuRST 2.0
- Where
- 4 sites in United States, Canada
- Volunteers
- 75 people
- Sponsor
- CHDI Foundation, Inc.
Huntington's Disease
Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD).
- Trial name
- NCT03664804
- Testing
- No Study Drug was Administered in this Study
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
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 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.