Sapropterin
- Generic Name
- Sapropterin
- Brand Names
- Javygtor, Kuvan, Sapropterin Dipharma
- Drug Type
- Small Molecule
- Chemical Formula
- C9H15N5O3
- CAS Number
- 62989-33-7
- Unique Ingredient Identifier
- EGX657432I
- Background
Sapropterin (tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide. It is also essential in the conversion of phenylalanine to tyrosine by the enzyme phenylalanine-4-hydroxylase; the conversion of tyrosine to L-dopa by the enzyme tyrosine hydroxylase; and conversion of tryptophan to 5-hydroxytryptophan via tryptophan hydroxylase.
- Indication
For the treatment of tetrahydrobiopterin (BH4) deficiency.
- Associated Conditions
- Hyperphenylalaninemia
- Associated Therapies
- -
Exercise Intolerance in Renal Failure
- Conditions
- Interventions
- First Posted Date
- 2011-05-20
- Last Posted Date
- 2015-06-01
- Lead Sponsor
- Emory University
- Target Recruit Count
- 74
- Registration Number
- NCT01356966
- Locations
- 🇺🇸
Atlanta VA Medical Center, Decatur, Georgia, United States
🇺🇸Emory University Hospital, Atlanta, Georgia, United States
Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy
- First Posted Date
- 2009-09-30
- Last Posted Date
- 2015-06-08
- Lead Sponsor
- Children's Hospital of Philadelphia
- Target Recruit Count
- 6
- Registration Number
- NCT00986973
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Section of Metabolism,PKU program, Philadelphia, Pennsylvania, United States
The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
- First Posted Date
- 2009-08-24
- Last Posted Date
- 2018-01-04
- Lead Sponsor
- University of Missouri-Columbia
- Target Recruit Count
- 20
- Registration Number
- NCT00964236
- Locations
- 🇺🇸
University of Missouri, Columbia, Missouri, United States
Trial of Kuvan in Lesch-Nyhan Disease
- Conditions
- Interventions
- First Posted Date
- 2009-07-09
- Last Posted Date
- 2019-08-02
- Lead Sponsor
- University of California, San Diego
- Registration Number
- NCT00935753
- Locations
- 🇺🇸
UCSD Mitochondrial and Metabolic Disease Center, San Diego, California, United States
Sapropterin as a Treatment for Autistic Disorder
- First Posted Date
- 2009-02-24
- Last Posted Date
- 2018-05-01
- Lead Sponsor
- The Children's Health Council
- Target Recruit Count
- 46
- Registration Number
- NCT00850070
- Locations
- 🇺🇸
The Children's Health Council, Palo Alto, California, United States
Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
- Conditions
- Interventions
- First Posted Date
- 2008-12-05
- Last Posted Date
- 2017-08-21
- Lead Sponsor
- Johns Hopkins University
- Target Recruit Count
- 3
- Registration Number
- NCT00802893
- Locations
- 🇺🇸
Johns Hopkins University, Baltimore, Maryland, United States
Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
- First Posted Date
- 2007-01-18
- Last Posted Date
- 2008-08-07
- Lead Sponsor
- University of Oxford
- Target Recruit Count
- 66
- Registration Number
- NCT00423280
- Locations
- 🇬🇧
Department of Cardiovascular Medicine, University of Oxford, Oxford, United Kingdom
Study of BH4, a New and Simple Treatment of Mild PKU
- Conditions
- First Posted Date
- 2005-12-01
- Last Posted Date
- 2006-04-13
- Lead Sponsor
- The Kennedy Institute-National Eye Clinic
- Target Recruit Count
- 15
- Registration Number
- NCT00260000
- Locations
- 🇩🇰
The Kennedy Institute-National Eye Clinic, Glostrup, Copenhagen, Denmark
Effects of Tetrahydrobiopterin on Blood Pressure
- First Posted Date
- 2005-09-21
- Last Posted Date
- 2013-11-18
- Lead Sponsor
- Emory University
- Target Recruit Count
- 24
- Registration Number
- NCT00208780
- Locations
- 🇺🇸
Emory University, Atlanta, Georgia, United States
Drug Development Updates
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