Sapropterin

Generic Name: Sapropterin

Brand Names: Javygtor, Kuvan, Sapropterin Dipharma

Drug Type: Small Molecule

Chemical Formula: C_₉H_₁₅N_₅O_₃

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

Associated Therapies: -

Clinical Trials

A Study for Assessing the Efficacy and Safety of BH4 in Radiation Enteritis

Phase 2

Conditions: Radiation Enteritis

Interventions: Drug: Tetrahydrobiopterin

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

Phase 1

Conditions: Radiation-Induced Dermatitis

Interventions: Drug: Tetrahydrobiopterin

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

O2 Transport and Utilization in Health and Lung Disease

Phase 1

Withdrawn

Conditions: COPD
Tetrahydrobiopterin Deficiency
Oxidative Stress

Interventions: Drug: Placebo oral tablet
Drug: Tetrahydrobiopterin

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

Nutritional Status in Phenylketonuria

Completed

Conditions: Phenylketonurias

Interventions: Other: Diet
Drug: Sapropterin

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

Neurovascular Transduction During Exercise in Chronic Kidney Disease

Phase 2

Recruiting

Conditions: Renal Insufficiency, Chronic

Interventions: Other: 6R-BH4 placebo
Drug: 6R-BH4
Dietary Supplement: Folic acid
Dietary Supplement: Histidine and beta-alanine supplementation
Other: Histidine and beta-alanine placebo
Other: Exercise training
Other: Stretching

First Posted Date: 2016-10-28

Last Posted Date: 2023-10-25

Lead Sponsor: Emory University

Target Recruit Count: 150

Registration Number: NCT02947750

Locations: 🇺🇸
Emory University Hospital Midtown, Atlanta, Georgia, United States
🇺🇸
Emory University Hospital, Atlanta, Georgia, United States
🇺🇸
Atlanta VA Health Care System, Decatur, Georgia, United States

Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

Phase 2

Terminated

Conditions: Phenylketonuria

Interventions: Drug: Placebo
Drug: Sapropterin

First Posted Date: 2013-11-07

Last Posted Date: 2018-02-22

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 2

Registration Number: NCT01977820

Locations: 🇮🇹
Research Site, Bologna, Italy
🇨🇭
Research site, Lausanne, Switzerland

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

Not Applicable

Terminated

Conditions: Hyperphenylalaninemia
Phenylketonuria

Interventions: Drug: Sapropterin

First Posted Date: 2012-03-01

Last Posted Date: 2015-07-07

Lead Sponsor: The University of Texas Health Science Center, Houston

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

Phase 2

Completed

Conditions: Liver Cirrhosis
Portal Hypertension

Interventions: Drug: sapropterin

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

Phase 2

Completed

Conditions: Alzheimer's Disease

Interventions: Drug: Simvastatin
Drug: L-Arginine
Drug: Tetrahydrobiopterin

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

Phase 1

Completed

Conditions: GTP Cyclohydrolase Deficiency

Interventions: Drug: Sapropterin

First Posted Date: 2011-08-30

Last Posted Date: 2023-06-02

Lead Sponsor: University of Utah

Target Recruit Count: 12

Registration Number: NCT01425528

Locations: 🇺🇸
University of Utah, Salt Lake City, Utah, United States

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