Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease (NCT06270316)
A Phase 1/2, Single Dose, Dose Ranging Study of Intravenous AAV5-GLA (AMT-191) in Adult Males With Classic Fabry Disease
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: The Kirklin Clinic Of university of Alabama Birmingham Hospital — Birmingham, Alabama
Age range: 18 Years to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (8 Sites)
- The Kirklin Clinic Of university of Alabama Birmingham Hospital — Birmingham, Alabama
- Emory University School of Medicine — Atlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- MHealth Fairview University of Minnesota Medical Center East Bank — Minneapolis, Minnesota
- NYC Health + Hospitals/Metropolitan — New York, New York
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania
- University of Utah, Clinical and Translational Sciences Institute — Salt Lake City, Utah
- Lysosomal & Rare Disorders Research and Treatment Center, Inc — Fairfax, Virginia
📞 Contact the Study Coordinator
Phone: (866) 520-1257
Email: medinfo@uniqure.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Fabry Disease
- A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease — Birmingham, Alabama
- A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Birmingham, Alabama
- Long-Term Follow-up of Subjects Who Were Treated With ST-920 — Irvine, California
- An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease — La Jolla, California
- A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease — Birmingham, Alabama
- A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease — Atlanta, Georgia
Explore by condition: All Fabry Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.