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NCT04120493 · ClinicalTrials.gov registry record · Phase 1
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
A Phase 1 study of Huntington's Disease, sponsored by UniQure Biopharma B.V..
- Active
- Registry status
- Phase 1
- Development phase
- 43
- Enrollment target
- 12
- Study locations
NCT04120493: Active Phase 1 study of Huntington's Disease, sponsored by UniQure Biopharma B.V..
NCT04120493 is a Phase 1 study of Huntington's Disease that is active but no longer recruiting, run by UniQure Biopharma B.V.. The registered enrollment target is 43 participants, below the 4,788-participant average among 8 other Huntington's Disease trials with a reported enrollment target (99% lower). The trial reports 12 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04120493, a Phase 1 study of Huntington's Disease, is active but no longer recruiting, sponsored by UniQure Biopharma B.V..
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 43 participants
- Enrollment target
- 12
- Study locations
Study Summary
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. The first three cohorts of the study have completed enrollment, including the randomized, double-blind, sham-controlled cohorts. Cohort 4 is open-label. Cohort 4 participants will receive high dose AMT-130.
Primary Outcome
Safety will be assessed by adverse events (AEs) related to clinical safety laboratory tests, vital signs, electrocardiograms (ECGs), neurological and physical examinations, rAAV5 vector shedding, immunogenicity response (Cohorts 1, 2 \& 3), suicidality risk \[Columbia-Suicide Severity Rating Scale \[C-SSRS)\], changes in global cognitive functioning \[Montreal Cognitive Assessment Scale (MoCA)\] and MRI measures of edema, inflammation, volume loss and structural changes.
Conditions Studied
Interventions
- GENETIC intra-striatal rAAV5-miHTT
- OTHER Imitation (sham) surgery
Study Locations (12)
Alabama
- University of Alabama at Birmingham - Birmingham
Arizona
- University of Arizona (Surgical Site Only) - Tucson
California
- University of California, San Francisco - San Francisco
Colorado
- CenExel Rocky Mountain Clinical Research - Englewood
Illinois
- Rush University Medical Center - Chicago
Maryland
- Johns Hopkins University - Baltimore
Michigan
- University of Michigan Department of Neurology - Ann Arbor
Ohio
- Ohio State University - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 43 participants |
| Start Date | 2019-09-06 |
| Est. Completion | 2029-12 |
| Phase | Phase 1 |
What the registry record for NCT04120493 still lists
NCT04120493 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 43 participants, a relatively small participant target, below the 4,788-participant average among 8 other Huntington's Disease trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Huntington's Disease appearing as the primary indexed condition, and to 2 interventions - of which intra-striatal rAAV5-miHTT is the first listed.
NCT04120493 lists 12 locations in 12 states (Alabama, Arizona, California).
Frequently Asked Questions
What is clinical trial NCT04120493 about?
NCT04120493 is a clinical study titled "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. The first three cohorts of the study have completed enrollment, including the randomized, doubl...
What is the current status of trial NCT04120493?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 43 participants. The study started on 2019-09-06. Estimated completion is 2029-12.
What conditions does trial NCT04120493 study?
This clinical trial studies the following conditions: Huntington's Disease.
What interventions are being tested in trial NCT04120493?
The interventions under investigation include: intra-striatal rAAV5-miHTT (GENETIC), Imitation (sham) surgery (OTHER).
Who is sponsoring clinical trial NCT04120493?
This trial is sponsored by UniQure Biopharma B.V., which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04120493 being conducted?
This trial has 12 study locations across Alabama, Arizona, California, Colorado, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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