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
Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects
- First Posted Date
- 2024-07-01
- Last Posted Date
- 2024-12-12
- Lead Sponsor
- APR Applied Pharma Research s.a.
- Target Recruit Count
- 16
- Registration Number
- NCT06481709
- Locations
- 🇨🇦
Pharma Medica Research Inc. 4770 Sheppard Ave E,, Toronto, Ontario, Canada
PD-1 Antibody and Sapropterin Dihydrochloride in Patients With PDAC
- Conditions
- Pancreatic Adenocarcinoma Metastatic
- Interventions
- First Posted Date
- 2024-05-02
- Last Posted Date
- 2024-05-02
- Lead Sponsor
- Sun Yat-sen University
- Target Recruit Count
- 20
- Registration Number
- NCT06396637
- Locations
- 🇨🇳
Cancer center of Sun Yat-sen University, Guangzhou, Guangdong, China
BH4 in the Prevention and Treatment of Radiation-induced Skin Reactions for Head and Neck Squamous Cell Carcinoma (Including Nasopharyngeal Carcinoma)
- Conditions
- Head and Neck Squamous Cell Carcinoma
- Interventions
- First Posted Date
- 2022-03-28
- Last Posted Date
- 2022-03-28
- Lead Sponsor
- West China Hospital
- Target Recruit Count
- 18
- Registration Number
- NCT05299203
A Study for Assessing the Efficacy and Safety of BH4 in Radiation Enteritis
- First Posted Date
- 2021-12-01
- Last Posted Date
- 2022-03-02
- Lead Sponsor
- West China Second University Hospital
- Target Recruit Count
- 366
- Registration Number
- NCT05138887
- Locations
- 🇨🇳
est China Second Hospital of Sichuan University, Chengdu, Sichuan, China
A Study for Assessing the Efficacy and Safety of Tetrahydrobiopterin in Radiation-Induced Skin Injury
- First Posted Date
- 2021-11-09
- Last Posted Date
- 2022-02-28
- Lead Sponsor
- West China Second University Hospital
- Target Recruit Count
- 45
- Registration Number
- NCT05114226
- Locations
- 🇨🇳
est China Second Hospital of Sichuan University, Chengdu, Sichuan, China
The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
- Conditions
- Peripheral Artery DiseaseClaudication, IntermittentPeripheral Vascular Diseases
- Interventions
- First Posted Date
- 2021-03-16
- Last Posted Date
- 2025-04-06
- Lead Sponsor
- Louis Messina
- Target Recruit Count
- 10
- Registration Number
- NCT04800692
- Locations
- 🇺🇸
UMASS Memorial Healthcare - University Campus, Worcester, Massachusetts, United States
L-arginine Study for Persistent Symptoms of Schizophrenia
- Conditions
- Schizo Affective DisorderSchizophrenia
- Interventions
- First Posted Date
- 2019-08-13
- Last Posted Date
- 2022-01-20
- Lead Sponsor
- University of Massachusetts, Worcester
- Registration Number
- NCT04054973
- Locations
- 🇺🇸
UMass Medical School, Worcester, Massachusetts, United States
O2 Transport and Utilization in Health and Lung Disease
- Conditions
- COPDTetrahydrobiopterin DeficiencyOxidative Stress
- Interventions
- Drug: Placebo oral tablet
- First Posted Date
- 2019-07-10
- Last Posted Date
- 2023-12-19
- Lead Sponsor
- University of Massachusetts, Amherst
- Registration Number
- NCT04014712
- Locations
- 🇺🇸
Institute of Applied Life Sciences, Amherst, Massachusetts, United States
Retrospective Observational Safety Effectiveness With Kuvan in hpA
- Conditions
- Tetrahydrobiopterin Deficiency
- First Posted Date
- 2019-03-06
- Last Posted Date
- 2019-03-07
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 26
- Registration Number
- NCT03864029
- Locations
- 🇨🇳
West China Hospital of Sichuan University, Chengdu, Sichuan, China
Nutritional Status in Phenylketonuria
- First Posted Date
- 2019-01-29
- Last Posted Date
- 2019-09-10
- Lead Sponsor
- Universidade do Porto
- Target Recruit Count
- 94
- Registration Number
- NCT03820804
- Locations
- 🇵🇹
Centro Hospitalar Universitário do Porto, Porto, Portugal
Drug Development Updates
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