Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)
Public ClinicalTrials.gov record NCT06100276. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).
Study identification
- NCT ID
- NCT06100276
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Enrollment
- 20 participants
Conditions and interventions
Conditions
Interventions
- AMT-162 Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 31, 2024
- Primary completion
- Sep 29, 2026
- Completion
- Jun 29, 2031
- Last update posted
- Oct 21, 2025
2024 – 2031
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | — |
| University of California Irvine | Irvine | California | 92697 | — |
| California Pacific Medical Center | San Francisco | California | 94109 | — |
| Mayo Clinic Florida | Jacksonville | Florida | 32224 | — |
| Winship Cancer Institute of Emory University | Atlanta | Georgia | 30322 | — |
| Northwestern University Feinberg School of Medicine | Chicago | Illinois | 60611 | — |
| University of Kansas Medical Center | Fairway | Kansas | 66205 | — |
| Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research | Boston | Massachusetts | 02114 | — |
| Mayo Clinic Rochester | Rochester | Minnesota | 55905 | — |
| Columbia University Irving Medical Center | New York | New York | 10032 | — |
| University of Pennsylvania School of Medicine | Philadelphia | Pennsylvania | 19104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06100276, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 21, 2025 · Synced Apr 20, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06100276 live on ClinicalTrials.gov.