NCT06270316

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

A Phase 1/2, Single Dose, Dose Ranging Study of Intravenous AAV5-GLA (AMT-191) in Adult Males With Classic Fabry Disease

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: UniQure Biopharma B.V. · Industry
• Sex: Male
• Age: 18 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

In Fabry disease, the enzyme α-galactosidase A is deficient. AMT-191 is an investigational gene therapy that encodes a recombinant serotype 5 based adeno-associated viral vector (rAAV5). AMT-191 is designed to target the liver for production of the enzyme α-galactosidase A (αGAL). AMT-191 is delivered via a single (one-time) intravenous (IV) infusion. In this first-in-human study of AMT-191, two or more dose levels will be tested. All eligible participants will receive AMT-191 at one of the dose levels; there is no placebo in this study. The starting dose level is decided based on accepted rules for dose translation from preclinical (animal) studies to humans. Subsequent dose cohort levels are decided based on the review of safety, tolerability, and PK/PD results by an Independent Data Monitoring Committee and in agreement with the Sponsor. Participants will be monitored through study site visits, blood tests, imaging questionnaires, and other assessments as per the study protocols.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	AMT-191	A recombinant serotype 5 based adeno-associated viral vector (AMT-191) for one-time intravenous (IV) administration will be investigated in this study. This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A. Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma levels in patients with Fabry disease.

Timeline

• Start date: 2024-06-05
• Primary completion: 2027-12-01
• Completion: 2031-04-30
• First posted: 2024-02-21
• Last updated: 2025-10-23

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study