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
Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders
- Conditions
- PAH Gene ExpressionPhenylketonuriaBehavior and Behavior Mechanisms
- Interventions
- First Posted Date
- 2011-01-11
- Last Posted Date
- 2023-08-14
- Lead Sponsor
- Tulane University School of Medicine
- Target Recruit Count
- 21
- Registration Number
- NCT01274026
- Locations
- 🇺🇸
Tulane University Health Science Center, New Orleans, Louisiana, United States
Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
- Conditions
- Autism Spectrum Disorder
- Interventions
- First Posted Date
- 2010-06-10
- Last Posted Date
- 2014-04-01
- Target Recruit Count
- 10
- Registration Number
- NCT01141595
- Locations
- 🇺🇸
Arkansas Children's Hospital Research Institute, Little Rock, Arkansas, United States
Impact of KUVAN® on Gastric Relaxation in Women With Diabetic Gastroparesis.
- First Posted Date
- 2010-06-02
- Last Posted Date
- 2019-09-10
- Lead Sponsor
- Stanford University
- Target Recruit Count
- 9
- Registration Number
- NCT01135186
- Locations
- 🇺🇸
Stanford University School of Medicine, Stanford, California, United States
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
- Conditions
- Phenylketonuria
- Interventions
- Drug: Placebo
- First Posted Date
- 2010-05-03
- Last Posted Date
- 2016-02-01
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 206
- Registration Number
- NCT01114737
Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period
- First Posted Date
- 2010-03-08
- Last Posted Date
- 2014-02-27
- Lead Sponsor
- Merck KGaA, Darmstadt, Germany
- Target Recruit Count
- 59
- Registration Number
- NCT01082328
- Locations
- 🇳🇴
Department of Paediatric Research, Division of Paediatrics, Oslo University Hospital, Rikshospitalet, Oslo, Norway
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
Open-Label Extension Study of Kuvan for Autism
- First Posted Date
- 2009-07-22
- Last Posted Date
- 2018-05-02
- Lead Sponsor
- The Children's Health Council
- Target Recruit Count
- 41
- Registration Number
- NCT00943579
- Locations
- 🇺🇸
The Children's Health Council, Palo Alto, California, United States
Trial of Kuvan in Lesch-Nyhan Disease
- Conditions
- Behavioral Manifestations of Lesch-Nyhan Disease
- 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
Drug Development Updates
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