RecruitingPhase 1Phase 2NCT06270316

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

Sponsor

UniQure Biopharma B.V.

Enrollment

12 participants

Start Date

Jun 5, 2024

Study Type

INTERVENTIONAL

Conditions

Fabry Disease

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.

Eligibility

Sex: MALEMin Age: 18 YearsMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called AMT-191 for people with fabry disease. The study is currently recruiting participants at 8 locations. People eligible for this study include men aged 18 Years to 50 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGAMT-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.