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
Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
- First Posted Date
- 2009-02-11
- Last Posted Date
- 2019-12-03
- Lead Sponsor
- University of Miami
- Target Recruit Count
- 21
- Registration Number
- NCT00841100
- Locations
- 🇺🇸
Department of Biochemistry & Molecular Biology, Miami, Florida, United States
Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
- First Posted Date
- 2009-02-06
- Last Posted Date
- 2020-12-22
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 95
- Registration Number
- NCT00838435
Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
- First Posted Date
- 2009-01-23
- Last Posted Date
- 2013-12-05
- Lead Sponsor
- Washington University School of Medicine
- Target Recruit Count
- 2
- Registration Number
- NCT00827762
- Locations
- 🇺🇸
University of Missouri, Columbia, Missouri, United States
🇺🇸Washington University, St. Louis, Missouri, United States
🇺🇸Oregon Health & Science University, Portland, Oregon, United States
Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
- Conditions
- Isolated Systolic HypertensionEndothelial Dysfunction
- Interventions
- Other: Placebo
- 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
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
- Conditions
- Phenylketonuria
- Interventions
- First Posted Date
- 2008-11-13
- Last Posted Date
- 2021-07-23
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 56
- Registration Number
- NCT00789568
- Locations
- 🇺🇸
PRACS Institute, Ltd., Fargo, North Dakota, United States
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
- First Posted Date
- 2008-10-23
- Last Posted Date
- 2022-10-12
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 1887
- Registration Number
- NCT00778206
- Locations
- 🇺🇸
LAC and USC Medical Center, Los Angeles, California, United States
🇺🇸UCLA, Los Angeles, California, United States
🇺🇸Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Sapropterin in Individuals With Phenylketonuria
- First Posted Date
- 2008-08-08
- Last Posted Date
- 2018-06-18
- Lead Sponsor
- Washington University School of Medicine
- Target Recruit Count
- 45
- Registration Number
- NCT00730080
- Locations
- 🇺🇸
Washington University, Saint Louis, Missouri, United States
A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
- Conditions
- Endothelial Dysfunction
- Interventions
- First Posted Date
- 2007-09-20
- Last Posted Date
- 2021-05-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 52
- Registration Number
- NCT00532844
Sapropterin Expanded Access Program
- Conditions
- Phenylketonuria
- First Posted Date
- 2007-06-12
- Last Posted Date
- 2008-04-15
- Lead Sponsor
- BioMarin Pharmaceutical
- Registration Number
- NCT00484991
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
- First Posted Date
- 2007-03-09
- Last Posted Date
- 2021-02-25
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 32
- Registration Number
- NCT00445978
Drug Development Updates
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