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
Effects of Kuvan on Melatonin Secretion
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Dietary Supplement: Large Neutral Amino Acid Therapy
- First Posted Date
- 2012-06-12
- Last Posted Date
- 2016-11-18
- Lead Sponsor
- University of Southern California
- Target Recruit Count
- 10
- Registration Number
- NCT01617070
- Locations
- 🇺🇸
Keck School of Medicine, Clinical Trials Unit at Keck Medical Center of USC, Los Angeles, California, United States
Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
- Conditions
- HyperphenylalaninemiaPhenylketonuria
- Interventions
- First Posted Date
- 2012-03-01
- Last Posted Date
- 2015-07-07
- Target Recruit Count
- 6
- Registration Number
- NCT01541397
- Locations
- 🇺🇸
The University of Texas Health Science Center at Houston, Houston, Texas, United States
Randomized Controlled Trial to Assess the Effects of Sapropterin on Hepatic and Systemic Hemodynamics in Patients With Liver Cirrhosis and Portal Hypertension
- First Posted Date
- 2011-10-20
- Last Posted Date
- 2013-10-07
- Lead Sponsor
- Fundacion Clinic per a la Recerca Biomédica
- Target Recruit Count
- 42
- Registration Number
- NCT01456286
- Locations
- 🇪🇸
Hospital Clínic de Barcelona, Barcelona, Spain
🇪🇸Hospital Ramón y Cajal, Madrid, Madrid Community, Spain
Endothelial Facilitation in Alzheimer's Disease
- Conditions
- Alzheimer's Disease
- Interventions
- First Posted Date
- 2011-09-23
- Last Posted Date
- 2019-07-30
- Lead Sponsor
- University of Massachusetts, Worcester
- Target Recruit Count
- 11
- Registration Number
- NCT01439555
- Locations
- 🇺🇸
UMass Medical School/ UMass Memorial Medical Center, Worcester, Massachusetts, United States
Study of Kuvan Treatment in Adults With GTPCH Deficiency
- First Posted Date
- 2011-08-30
- Last Posted Date
- 2025-04-01
- Lead Sponsor
- University of Utah
- Target Recruit Count
- 12
- Registration Number
- NCT01425528
- Locations
- 🇺🇸
University of Utah, Salt Lake City, Utah, United States
Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria
- Conditions
- PKU
- Interventions
- Dietary Supplement: diet
- First Posted Date
- 2011-08-09
- Last Posted Date
- 2014-12-17
- Lead Sponsor
- Children's National Research Institute
- Registration Number
- NCT01412437
- Locations
- 🇺🇸
Children's Research Institute, Washington, District of Columbia, United States
Nitric Oxide Bioavailability in Chronic Obstructive Pulmonary Disease (COPD)
- Conditions
- Pulmonary Disease, Chronic Obstructive
- Interventions
- Dietary Supplement: Antioxidant Cocktail
- First Posted Date
- 2011-07-21
- Last Posted Date
- 2017-12-11
- Lead Sponsor
- Augusta University
- Target Recruit Count
- 60
- Registration Number
- NCT01398943
- Locations
- 🇺🇸
Augusta University, Augusta, Georgia, United States
Phenylketonuria, Oxidative Stress, and BH4
- First Posted Date
- 2011-07-15
- Last Posted Date
- 2014-08-28
- Lead Sponsor
- Emory University
- Target Recruit Count
- 12
- Registration Number
- NCT01395394
- Locations
- 🇺🇸
Emory University, Decatur, Georgia, United States
Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
- Conditions
- Phenylketonuria
- Interventions
- Other: Phenylalanine (Phe)-restricted diet
- First Posted Date
- 2011-06-20
- Last Posted Date
- 2017-09-15
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 56
- Registration Number
- NCT01376908
- Locations
- 🇬🇧
Research site, London, United Kingdom
🇬🇧Research Site, Birmingham, United Kingdom
Exercise Intolerance in Renal Failure
- Conditions
- Kidney Failure, Chronic
- 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
Drug Development Updates
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