Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

ClinicalTrials.gov ID: NCT06270316

Public ClinicalTrials.gov record NCT06270316. 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.

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ClinicalTrials.gov public records Last synced Oct 25, 2026, 4:46 AM EDT

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, Single Dose, Dose Ranging Study of Intravenous AAV5-GLA (AMT-191) in Adult Males With Classic Fabry Disease

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.

Study identification

NCT ID: NCT06270316
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 1, Phase 2
Lead sponsor: UniQure Biopharma B.V.; Industry
Enrollment: 20 participants

Conditions and interventions

Conditions

Fabry Disease

Interventions

AMT-191 Drug

Drug

Eligibility (public fields only)

Age range: 18 Years to 65 Years
Sex: Male
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Jun 5, 2024 (Actual)
Primary completion: Dec 1, 2027 (Estimated)
Completion: Nov 30, 2031 (Estimated)
Last update posted: Sep 24, 2026

2024 – 2031

United States locations

U.S. sites: 10
U.S. states: 10
U.S. cities: 10

Facility	City	State	ZIP	Site status
The Kirklin Clinic Of University of Alabama Birmingham Hospital	Birmingham	Alabama	35233	Recruiting
University of California Los Angeles (UCLA)	Los Angeles	California	90095	Recruiting
Emory University School of Medicine	Atlanta	Georgia	30322	Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	60611	Recruiting
MHealth Fairview University of Minnesota Medical Center East Bank	Minneapolis	Minnesota	55455	Recruiting
NYC Health + Hospitals/Metropolitan	New York	New York	10029	Recruiting
Children's Hospital Medical Center	Cincinnati	Ohio	45229	Recruiting
UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania	15224	Recruiting
University of Utah, Clinical and Translational Sciences Institute	Salt Lake City	Utah	84108	Recruiting
Lysosomal & Rare Disorders Research and Treatment Center, Inc	Fairfax	Virginia	22030	Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT06270316, 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 Sep 24, 2026 · Synced Oct 25, 2026

Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT06270316 live on ClinicalTrials.gov.

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