- 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
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–6
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matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Spinal Muscular Atrophy
- Interventions
- Nusinersen, Sham procedure
- Drug · Procedure
- Lead sponsor
- Biogen
- Industry
- Eligibility
- 2 Years to 12 Years
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Palo Alto, California • Aurora, Colorado + 8 more
- Conditions
- Type 2 Spinal Muscular Atrophy
- Interventions
- OAV101, Sham control
- Genetic · Procedure
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 6
- States / cities
- Farmington, Connecticut • Chicago, Illinois • Strasburg, Pennsylvania + 2 more
- Conditions
- Leukemia-Lymphoma
- Interventions
- EMLA, Lidocaine
- Drug
- Lead sponsor
- CAMC Health System
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Charleston, West Virginia
- Conditions
- Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
- Interventions
- ION440, Sham procedure
- Drug · Procedure
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 65 Years · Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2030
- U.S. locations
- 8
- States / cities
- San Diego, California • Aurora, Colorado • Baltimore, Maryland + 5 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Adrabetadex
- Drug
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 15
- States / cities
- Birmingham, Alabama • Orange, California • San Francisco, California + 12 more