Condition: Angelman Syndrome · Sponsor: Ultragenyx Pharmaceutical Inc
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
This description comes directly from the study's public registry record.
Patients Contact Trial Recruitment · 1-888-756-8657 · Trialrecruitment@ultragenyx.com
HCPs Contact: Medical Information · 1-888-756-8657 · medinfo@ultragenyx.com
Always discuss trial participation with your own doctor first.
| Cedars Sinai Medical Center | Los Angeles, California, United States | Recruiting |
| Rush University Medical Center | Chicago, Illinois, United States | Recruiting |
| Clinical Trial Site | Baltimore, Maryland, United States | Not Yet Recruiting |
| Clinical Trial Site | Kansas City, Missouri, United States | Not Yet Recruiting |
| Rare Disease Research | Hillsborough, North Carolina, United States | Recruiting |
| Akron Children's Hospital | Akron, Ohio, United States | Recruiting |
| UT Health Austin | Austin, Texas, United States | Recruiting |
| Carum Research Inc. | Dallas, Texas, United States | Recruiting |
| Hospital Universitario Austral | Pilar, Buenos Aires, Argentina | Recruiting |
| Hospital de Crianças César Pernetta e Hospital Pequeno Príncipe | Curitiba, Paraná, Brazil | Recruiting |
| Casa dos Raros | Santa Cecília, Porto Alegre, Brazil | Recruiting |
| Hopital de la Timone | Marseille, France | Recruiting |
+ 9 more locations — full list on the registry record.
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.
Source record: clinicaltrials.gov/study/NCT07157254