GTX-102
- Generic Name
- GTX-102
- Brand Names
- -
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- -
- Unique Ingredient Identifier
- -
- 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.
- Associated Conditions
- -
- Associated Therapies
- -
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS
- First Posted Date
- 2024-09-27
- Last Posted Date
- 2024-12-20
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 120
- Registration Number
- NCT06617429
- Locations
- ๐บ๐ธ
Rare Disease Research, Atlanta, Georgia, United States
๐บ๐ธRush University, Chicago, Illinois, United States
๐ช๐ธClinical Trial Site, Seville, Spain
Long-term Extension of GTX-102 in Angelman Syndrome
- First Posted Date
- 2024-05-16
- Last Posted Date
- 2024-11-15
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 75
- Registration Number
- NCT06415344
- Locations
- ๐บ๐ธ
Rare Disease Research, LLC, Atlanta, Georgia, United States
๐ฆ๐บQueensland Children's Hospital, South Brisbane, Queensland, Australia
๐จ๐ฆChildrens Hospital London Health Sciences Centre, London, 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
- 2024-11-28
- 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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