Sepiapterin
- Generic Name
- Sepiapterin
- Drug Type
- Small Molecule
- Chemical Formula
- C9H11N5O3
- CAS Number
- 17094-01-8
- Unique Ingredient Identifier
- CJQ26KO7HP
- Background
Sepiapterin is under investigation in clinical trial NCT03519711 (A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia).
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
- First Posted Date
- 2024-03-08
- Last Posted Date
- 2025-04-29
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 56
- Registration Number
- NCT06302348
- Locations
- ๐บ๐ธ
Indiana University, Indianapolis, Indiana, United States
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
- First Posted Date
- 2021-12-21
- Last Posted Date
- 2025-05-08
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 200
- Registration Number
- NCT05166161
- Locations
- ๐บ๐ธ
Children's Hospital Colorado, Aurora, Colorado, United States
๐บ๐ธIndiana University School of Medicine Department of Medical and Molecular Genetics, Indianapolis, Indiana, United States
๐บ๐ธUniversity of Utah, Salt Lake City, Utah, United States
A Study of PTC923 in Participants With Phenylketonuria
- First Posted Date
- 2021-10-29
- Last Posted Date
- 2024-01-10
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 157
- Registration Number
- NCT05099640
- Locations
- ๐ซ๐ท
Bretonneau Hospital - CHRU de Tours, Tours, Centre-Val De Loire, France
๐บ๐ธBoston Children's Hospital, Boston, Massachusetts, United States
๐บ๐ธIcahn School of Medicine at Mount Sinai (ISMMS), New York, New York, United States
A Study of CNSA-001 in Women With Diabetic Gastroparesis
- First Posted Date
- 2018-10-19
- Last Posted Date
- 2022-01-05
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 21
- Registration Number
- NCT03712124
- Locations
- ๐บ๐ธ
GW Research, Inc., Chula Vista, California, United States
๐บ๐ธLMG Research, Miami, Florida, United States
๐บ๐ธJohns Hopkins University, Baltimore, Maryland, United States
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
- First Posted Date
- 2018-05-09
- Last Posted Date
- 2023-11-14
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 8
- Registration Number
- NCT03519711
- Locations
- ๐บ๐ธ
UT Southwestern, Dallas, Texas, United States
๐บ๐ธUniversity of Minnesota, Minneapolis, Minnesota, United States
๐บ๐ธUniversity of Utah Hospital, Salt Lake City, Utah, United States
Drug Development Updates
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