DeepMed Research

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

Clinical Trials

Effects of Tetrahydrobiopterin (BH4) on Leg Blood Flow and Exercise Capacity in Patients With Peripheral Artery Disease

Phase 4

Withdrawn

Conditions: Peripheral Arterial Disease
Peripheral Artery Disease

Interventions: Drug: Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)
Drug: Placebo

First Posted Date: 2018-04-10

Last Posted Date: 2023-08-15

Lead Sponsor: University of Nebraska

Registration Number: NCT03493412

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: 2025-03-05

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

Long Term Nitric Oxide Bioavailability on Vascular Health in Chronic Obstructive Pulmonary Disease

Phase 2

Completed

Conditions: Chronic Obstructive Pulmonary Disease (COPD)

Interventions: Drug: Tetrahydrobiopterin (BH4)
Dietary Supplement: Antioxidant Cocktail

First Posted Date: 2016-05-17

Last Posted Date: 2021-02-23

Lead Sponsor: Augusta University

Target Recruit Count: 15

Registration Number: NCT02774226

Locations: 🇺🇸
Georgia Prevention Institute, Augusta, Georgia, United States

Rhode Island Diastolic Dysfunction - Heart Failure

Not Applicable

Completed

Conditions: Heart Failure
Cardiovascular Disease

Interventions: Drug: Kuvan

First Posted Date: 2015-02-02

Last Posted Date: 2019-01-30

Lead Sponsor: Providence VA Medical Center

Target Recruit Count: 28

Registration Number: NCT02353312

Locations: 🇺🇸
Providence VAMC, Providence, Rhode Island, United States

Sex Hormones and Atherosclerosis Prevention in Perimenopausal Women

Early Phase 1

Completed

Conditions: Aging
Menopause

Interventions: Drug: KUVAN
Drug: Placebo
Drug: Cetrotide
Drug: Placebo transdermal patch
Drug: Climara

First Posted Date: 2014-01-22

Last Posted Date: 2019-07-11

Lead Sponsor: University of Colorado, Denver

Target Recruit Count: 100

Registration Number: NCT02042196

Locations: 🇺🇸
University of Colorado Denver, Aurora, Colorado, 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

Kuvan®'s Effect on the Cognition of Children With Phenylketonuria

Phase 4

Completed

Conditions: Phenylketonuria

Interventions: Drug: Kuvan®

First Posted Date: 2013-10-18

Last Posted Date: 2023-04-24

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 34

Registration Number: NCT01965912

Locations: 🇮🇹
Research site - Bambino Gesu, Roma, Italy
🇬🇧
Research site, Bristol, United Kingdom
🇮🇹
Research site - La Sapienza, Roma, Italy

and more 3 locations

A Pilot Study on Diurnal Variation

Not Applicable

Terminated

Conditions: Phenylketonuria (PKU)

Interventions: Drug: Kuvan

First Posted Date: 2013-03-07

Last Posted Date: 2016-04-20

Lead Sponsor: Dr. Linda Randolph

Target Recruit Count: 6

Registration Number: NCT01806051

Locations: 🇺🇸
Children's Hospital Los Angeles, Los Angeles, California, United States

Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria

Phase 3

Completed

Conditions: Phenylketonuria

Interventions: Drug: Kuvan®

First Posted Date: 2012-11-22

Last Posted Date: 2014-07-28

Lead Sponsor: Merck KGaA, Darmstadt, Germany

Target Recruit Count: 90

Registration Number: NCT01732471

Locations: 🇩🇪
Please contact Merck KGaA Communication Center, Darmstadt, Germany

Kuvan in People With Schizophrenia and Schizoaffective Disorder

Phase 1

Completed

Conditions: Schizophrenia
Schizoaffective Disorder

Interventions: Drug: Kuvan
Drug: Multivitamin

First Posted Date: 2012-10-15

Last Posted Date: 2017-09-13

Lead Sponsor: New York State Psychiatric Institute

Target Recruit Count: 27

Registration Number: NCT01706965

Locations: 🇺🇸
New York State Psychiatric Institute, New York, New York, United States

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