- Conditions
- Angelman Syndrome
- Interventions
- GTX-102, Sham-LP
- Drug · Procedure
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 4 Years to 17 Years
- Enrollment
- 129 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Diego, California • San Francisco, California + 11 more
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- Conditions
- Angelman Syndrome
- Interventions
- GTX-102
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 4 Years to 17 Years
- Enrollment
- 74 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 6
- States / cities
- Los Angeles, California • San Diego, California • Atlanta, Georgia + 3 more
- Conditions
- Angelman Syndrome
- Interventions
- GTX-102
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 255 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 6
- States / cities
- Los Angeles, California • San Diego, California • Atlanta, Georgia + 3 more
- Conditions
- Angelman Syndrome
- Interventions
- No intervention, GTX-102
- Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 1 Year to 64 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 5 more