A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) (NCT07157254)
A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome
🩺 Plain-English Summary (8th-Grade Level)
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
Who can join: Review the eligibility criteria below.
Location: Cedars Sinai Medical Center — Los Angeles, California
Age range: 1 Year to 64 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 (21 Sites)
- Cedars Sinai Medical Center — Los Angeles, California
- Rush University Medical Center — Chicago, Illinois
- Kennedy Kreiger Institute — Baltimore, Maryland
- Clinical Trial Site — Kansas City, Missouri
- Rare Disease Research — Hillsborough, North Carolina
- Akron Children's Hospital — Akron, Ohio
- UT Health Austin — Austin, Texas
- Carum Research Inc. — Dallas, Texas
- Hospital Universitario Austral — Pilar, Buenos Aires
- Hospital de Crianças César Pernetta e Hospital Pequeno Príncipe — Curitiba, Paraná
- Casa dos Raros — Santa Cecília, Porto Alegre
- Hopital de la Timone — Marseille,
- Necker-Enfants Malades Hospital — Paris,
- Sheba Medical Center — Ramat Gan,
- Azienda Ospedaliera Universitaria Meyer IRCCS — Florence,
- Fondazione IRCCS Istituto Neurologico C. Besta — Milan,
- Clinical Trial Site — Rome,
- Hospital de Santa Maria — Lisbon,
- Hospital Santa Joao — Porto,
- Clinical Trial Site — London,
- University of Oxford — Oxford,
📞 Contact the Study Coordinator
Phone: (888) 756-8657
Email: Trialrecruitment@ultragenyx.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 Angelman Syndrome
- HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome — San Diego, California
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) — Los Angeles, California
- Angelman Syndrome Natural History Study — Los Angeles, California
- A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome — Los Angeles, California
- BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. — San Diego, California
- A Natural History Study of Angelman Syndrome — Lexington, Massachusetts
Explore by condition: All Angelman Syndrome 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.