A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome (NCT07181837)
A Multi-Center, Open-label, Phase 1/2 Trial of the Safety and Efficacy of MVX-220 Gene Therapy Administered by Intra-Cisterna Magna Injection to Participants With Angelman Syndrome
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.
Who can join: Review the eligibility criteria below.
Location: Cedars-Sinai Medical Center — Los Angeles, California
Age range: 4 Years to 50 Years
🧪 Interventions in This Study
- MVX-220 (GENETIC)
📋 "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 (3 Sites)
- Cedars-Sinai Medical Center — Los Angeles, California
- Rush University Medical Center — Chicago, Illinois
- Boston Children's Hospital — Boston, Massachusetts
📞 Contact the Study Coordinator
Phone: (978) 538-8554
Email: info@mvxbio.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
- 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.