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NCT06100276 · ClinicalTrials.gov registry record · Phase 1
Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)
A Phase 1 study of Amyotrophic Lateral Sclerosis, sponsored by UniQure Biopharma B.V..
- Active
- Registry status
- Phase 1
- Development phase
- 20
- Enrollment target
- 12
- Study locations
NCT06100276: Active Phase 1 study of Amyotrophic Lateral Sclerosis, sponsored by UniQure Biopharma B.V..
NCT06100276 is a Phase 1 study of Amyotrophic Lateral Sclerosis that is active but no longer recruiting, run by UniQure Biopharma B.V.. The registered enrollment target is 20 participants, below the 898-participant average among 102 other Amyotrophic Lateral Sclerosis trials with a reported enrollment target (98% lower). The trial reports 12 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06100276, a Phase 1 study of Amyotrophic Lateral Sclerosis, is active but no longer recruiting, sponsored by UniQure Biopharma B.V..
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 20 participants
- Enrollment target
- 12
- Study locations
Study Summary
This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.
Primary Outcome
Occurrence of TEAEs upon administration of ascending doses of AMT-162
Conditions Studied
Interventions
- DRUG AMT-162
Study Locations (12)
California
- University of California Irvine - Irvine
- California Pacific Medical Center - San Francisco
Arizona
- Barrow Neurological Institute - Phoenix
Florida
- Mayo Clinic Florida - Jacksonville
Georgia
- Winship Cancer Institute of Emory University - Atlanta
Illinois
- Northwestern University Feinberg School of Medicine - Chicago
Kansas
- University of Kansas Medical Center - Fairway
Massachusetts
- Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research - Boston
Minnesota
- Mayo Clinic Rochester - Rochester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2024-08-01 |
| Est. Completion | 2031-06-30 |
| Phase | Phase 1 |
What the registry record for NCT06100276 still lists
NCT06100276 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 898-participant average among 102 other Amyotrophic Lateral Sclerosis trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Amyotrophic Lateral Sclerosis appearing as the primary indexed condition, and to 1 intervention - of which AMT-162 is the first listed.
NCT06100276 lists 12 locations in 11 states (California, Arizona, Florida).
Frequently Asked Questions
What is clinical trial NCT06100276 about?
NCT06100276 is a clinical study titled "Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)". This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.
What is the current status of trial NCT06100276?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 20 participants. The study started on 2024-08-01. Estimated completion is 2031-06-30.
What conditions does trial NCT06100276 study?
This clinical trial studies the following conditions: Amyotrophic Lateral Sclerosis.
What interventions are being tested in trial NCT06100276?
The interventions under investigation include: AMT-162 (DRUG).
Who is sponsoring clinical trial NCT06100276?
This trial is sponsored by UniQure Biopharma B.V., which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06100276 being conducted?
This trial has 12 study locations across Arizona, California, Florida, Georgia, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Amyotrophic Lateral Sclerosis
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06100276's enrollment target sits among peer trials
20 78th of 102 higher than 23 of 102 other Amyotrophic Lateral Sclerosis trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Amyotrophic Lateral Sclerosis trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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