Clinical Trials
61
Trial Phases
4 Phases
Drug Approvals
4
Clinical Trials
Distribution across different clinical trial phases (40 trials with phase data)β’ Click on a phase to view related trials
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
- First Posted Date
- 2025-09-05
- Last Posted Date
- 2025-11-03
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 60
- Registration Number
- NCT07157254
- Locations
- π¬π§
Clinical Trial Site, Oxford, United Kingdom
πΊπΈRare Disease Research, Hillsborough, North Carolina, United States
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
- First Posted Date
- 2024-10-10
- Last Posted Date
- 2025-09-17
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 6
- Registration Number
- NCT06636071
- Locations
- π―π΅
Osaka Metropolitan University Hospital, Osaka, Japan
π―π΅Osaka University Hospital, Osaka, Japan
π―π΅Keio University Hospital, Tokyo, Japan
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
- Conditions
- Glycogen Storage Disease Type Ia
- First Posted Date
- 2024-10-10
- Last Posted Date
- 2025-11-05
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 140
- Registration Number
- NCT06636383
- Locations
- πΊπΈ
Children's Hospital of Orange County, Orange, California, United States
πΊπΈChildren's Hospital Colorado, Denver, Colorado, United States
πΊπΈUniversity of Connecticut Health Center, Hartford, Connecticut, United States
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
- Conditions
- Angelman Syndrome
- Interventions
- Procedure: Sham-LP
- First Posted Date
- 2024-09-27
- Last Posted Date
- 2025-11-06
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 129
- Registration Number
- NCT06617429
- Locations
- πΊπΈ
Cedars Sinai, Los Angeles, California, United States
πΊπΈUCSD, Rady Children's Hospital, San Diego, California, United States
πΊπΈUCSF, San Francisco, California, United States
Long-term Extension of GTX-102 in Angelman Syndrome
- First Posted Date
- 2024-05-16
- Last Posted Date
- 2025-10-08
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 255
- Registration Number
- NCT06415344
- Locations
- πΊπΈ
University of California, Los Angeles (UCLA), Los Angeles, California, United States
πΊπΈUniversity of California, San Diego - Rady Children's Hospital, San Diego, California, United States
πΊπΈRare Disease Research, LLC, Atlanta, Georgia, United States
- Prev
- 1
- 2
- 3
- 4
- 5
- 13
- Next
News
Gene Therapy Clinical Trial Pipeline Surges with Over 180 Companies Leading Innovation
β’ The gene therapy market is experiencing substantial growth, fueled by technological advancements, increased investments, and a deeper understanding of genetic diseases. β’ Key players are actively engaging in collaborations, mergers, and acquisitions to enhance their research and development capabilities and broaden their product portfolios. β’ Regulatory bodies like the FDA and EMA are expediting the approval process for gene therapies, acknowledging their potential in addressing unmet medical needs. β’ Ethical considerations and public perception surrounding gene editing, particularly germline modifications, remain critical factors influencing market dynamics and regulatory policies.