GTX-102
- Generic Name
- GTX-102
- Drug Type
- Biotech
- Background
GTX-102 is an antisense oligonucleotide designed to target and inhibit the expression of UBE3A-AS. It is being investigated for the treatment of Angelman Syndrome.
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS
- First Posted Date
- 2024-09-27
- Last Posted Date
- 2025-05-14
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 120
- Registration Number
- NCT06617429
- Locations
- ๐ช๐ธ
Clinical Trial Site, Seville, Spain
๐ฉ๐ชUniversity of Leipzig, Leipzig, Germany
๐ฏ๐ตNagoya City University Graduate School of Medical Sciences, Nagoya, Aichi, Japan
Long-term Extension of GTX-102 in Angelman Syndrome
- First Posted Date
- 2024-05-16
- Last Posted Date
- 2025-04-16
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 75
- Registration Number
- NCT06415344
- Locations
- ๐ช๐ธ
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell, Sabadell, Spain
๐บ๐ธUniversity of California, Los Angeles (UCLA), Los Angeles, California, United States
๐บ๐ธUniversity of California, San Diego - Rady Children's Hospital, San Diego, California, United States
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
- Conditions
- Ataxia Telangiectasia
- Interventions
- First Posted Date
- 2022-09-08
- Last Posted Date
- 2023-01-23
- Lead Sponsor
- Acasti Pharma Inc.
- Target Recruit Count
- 48
- Registration Number
- NCT05531890
- Locations
- ๐จ๐ฆ
Clinical Research Unit, Toronto, Ontario, Canada
A Study of the Safety and Tolerability of GTX-102 in Children with Angelman Syndrome
- First Posted Date
- 2020-02-06
- Last Posted Date
- 2025-01-31
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 74
- Registration Number
- NCT04259281
- Locations
- ๐บ๐ธ
Rare Disease Research, Atlanta, Georgia, United States
๐บ๐ธRush University Medical Center, Chicago, Illinois, United States
๐บ๐ธBoston Children's Hospital, Boston, Massachusetts, United States
Drug Development Updates
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