🏛️ Verified NIH Record • Updated 2026-08-05

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) (NCT07157254)

🟢 Recruiting Phase2 💊 Investigational Drug / Biologic Sponsor: Ultragenyx Pharmaceutical Inc

🩺 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: To join this study, participants generally need to meet the listed inclusion criteria (10 items) and avoid the listed exclusions (9 items).

Location: Cedars Sinai Medical Center — Los Angeles, California

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Usually NOT eligible if any of the following apply:

  • Exclusion Criteria:
  • Any change in medications or diet/supplements intended to treat symptoms of Angelman Syndrome (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments)
  • Any condition that creates an increased risk of unsuccessful lumbar puncture
  • Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)
  • Known hypersensitivity to GTX-102 or its excipients or required premedication that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
  • Presence or history of any condition, lab abnormality, or infection that, in the judgment of the Investigator, would interfere with study participation, pose undue safety risk, or would confound interpretation of results
  • Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
  • Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit, or any prior use of gene therapy or an ASO regardless of length of time since last use
  • Concurrent participation in any interventional study

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 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,

💰 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.
🏛️ View Official NIH Record ↗