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
6R-BH4 Pulmonary Arterial Hypertension Study
- Conditions
- Pulmonary Arterial Hypertension
- Interventions
- First Posted Date
- 2007-02-14
- Last Posted Date
- 2013-06-19
- Lead Sponsor
- Vanderbilt University
- Target Recruit Count
- 14
- Registration Number
- NCT00435331
- Locations
- 🇺🇸
Vanderbilt Medical Center, Nashville, Tennessee, United States
Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety
- Conditions
- Phenylalanine Hydroxylase Deficiencies
- First Posted Date
- 2007-02-08
- Last Posted Date
- 2007-09-20
- Lead Sponsor
- Orphanetics Pharma Entwicklungs GmbH
- Target Recruit Count
- 50
- Registration Number
- NCT00432822
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
A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
- Conditions
- Intermittent Claudication
- Interventions
- Other: Placebo
- First Posted Date
- 2006-11-23
- Last Posted Date
- 2021-01-07
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 190
- Registration Number
- NCT00403494
Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
- Conditions
- Tetrahydrobiopterin DeficienciesHyperphenylalaninemia, Non-Phenylketonuric
- Interventions
- First Posted Date
- 2006-07-21
- Last Posted Date
- 2020-09-25
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 12
- Registration Number
- NCT00355264
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
- Conditions
- Phenylketonuria
- First Posted Date
- 2006-06-01
- Last Posted Date
- 2012-11-16
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 111
- Registration Number
- NCT00332189
A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
- Conditions
- Hypertension
- First Posted Date
- 2006-05-15
- Last Posted Date
- 2009-07-21
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 84
- Registration Number
- NCT00325962
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
- Conditions
- Phenylketonurias
- Interventions
- Drug: Placebo
- First Posted Date
- 2006-01-09
- Last Posted Date
- 2015-08-13
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 45
- Registration Number
- NCT00272792
Study of BH4, a New and Simple Treatment of Mild PKU
- Conditions
- Phenylketonuria
- 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
Response to Phenylketonuria to Tetrahydrobiopterin (BH4)
- First Posted Date
- 2005-10-26
- Last Posted Date
- 2021-09-21
- Target Recruit Count
- 57
- Registration Number
- NCT00244218
- Locations
- 🇺🇸
University of Texas Medical Branch, Galveston, Texas, United States
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.