48 completed, 8 terminated, 5 unknown, 5 withdrawn. 28 Phase 2, 14 Phase 1, 12 Phase 3, 9 Not applicable, 4 Phase 4.
Follow sapropterin: new trials by email Literature Review of sapropterin trials Research Report on sapropterin
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Single Patient Study of Sapropterin for Multisystem Smooth Muscle Dysfunction Syndrome Kuvan (Sapropterine) - Multisystemic Smooth Muscle Dysfunction Syndrome | Not applicable | Not yet recruiting | Gregor Andelfinger | 1 | 2026-06-01 | 2026-05-07 |
| Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU Phenylalanine-restricted diet alone, Sapropterin Dihydrochloride, Sepiapterin - Phenylketonuria | Recruiting | Birmingham Women's and Children's NHS Foundation Trust | 148 | 2025-10-01 | 2026-10-06 | |
| Evaluation of BH4 Responsiveness in Our PKU Patients Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin) - Phenylketonuria (PKU) | Phase 1 | Active not recruiting | Sohag University | 20 | 2025-09-01 | 2025-12-01 |
| Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects Under Fed Conditions RLF-OD032 100 mg/mL oral suspension, Kuvan 100 MG Powder for Oral Solution - Bioequivalence | Phase 1 | Completed | APR Applied Pharma Research s.a. | 42 | 2025-08-20 | 2026-04-28 |
| Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects RLF-OD032, Kuvan - Bioavailability | Phase 1 | Completed | APR Applied Pharma Research s.a. | 16 | 2024-07-30 | 2025-06-12 |
| PD-1 Antibody and Sapropterin Dihydrochloride in Patients With PDAC Sapropterin Dihydrochloride - Pancreatic Adenocarcinoma Metastatic | Phase 2 | Unknown | Sun Yat-sen University | 20 | 2024-04-30 | 2024-05-02 |
| BH4 in the Prevention and Treatment of Radiation-induced Skin Reactions for Head and Neck Squamous Cell Carcinoma (Including Nasopharyngeal Carcinoma) Sapropterin dihydrochloride tablets - Head and Neck Squamous Cell Carcinoma | Phase 1 | Unknown | West China Hospital | 18 | 2022-05-01 | 2022-03-28 |
| A Study for Assessing the Efficacy and Safety of BH4 in Radiation Enteritis Tetrahydrobiopterin - Radiation Enteritis | Phase 2 | Unknown | West China Second University Hospital | 366 | 2022-03-01 | 2022-03-02 |
| A Study for Assessing the Efficacy and Safety of Tetrahydrobiopterin in Radiation-Induced Skin Injury Tetrahydrobiopterin - Radiation-Induced Dermatitis | Phase 1 | Unknown | West China Second University Hospital | 45 | 2022-03-01 | 2022-02-28 |
| The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication Tetrahydrobiopterin 10 mg/kg, Tetrahydrobiopterin 20 mg/kg, L-Ascorbate - Claudication, Intermittent, Peripheral Artery Disease | Phase 1 | Active not recruiting | Louis Messina | 10 | 2021-06-15 | 2026-04-15 |
| Novel Approaches for Improving Vascular Function in Veterans With HFpEF L-Citrulline, Placebo for L-Citrulline, BH4 - Heart Failure With Preserved Ejection Fraction, Inflammation | Phase 1 | Recruiting | VA Office of Research and Development | 90 | 2021-05-19 | 2026-04-23 |
| O2 Transport and Utilization in Health and Lung Disease Tetrahydrobiopterin, Placebo oral tablet - COPD, Tetrahydrobiopterin Deficiency | Phase 1 | Withdrawn | University of Massachusetts, Amherst | 2021-04-01 | 2023-12-19 | |
| L-arginine Study for Persistent Symptoms of Schizophrenia L-arginine, Sapropterin Dihydrochloride - Schizo Affective Disorder, Schizophrenia | Phase 2 | Withdrawn | University of Massachusetts, Worcester | 2019-09-11 | 2022-01-20 | |
| Effects of Tetrahydrobiopterin (BH4) on Leg Blood Flow and Exercise Capacity in Patients With Peripheral Artery Disease Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin), Placebo - Peripheral Artery Disease, Peripheral Arterial Disease | Phase 4 | Withdrawn | University of Nebraska | 2019-07-01 | 2024-09-03 | |
| Nutritional Status in Phenylketonuria Diet, Sapropterin - Phenylketonurias | Completed | Universidade do Porto | 94 | 2019-01-07 | 2019-09-10 | |
| Exercise and NO in HFrEF Antioxidant, Tetrahydrobiopterin (BH4), Exercise training - Heart Failure With Reduced Ejection Fraction | Early Phase 1 | Completed | VA Office of Research and Development | 68 | 2018-06-01 | 2025-08-06 |
| Retrospective Observational Safety Effectiveness With Kuvan in hpA KUVAN - Tetrahydrobiopterin Deficiency | Completed | BioMarin Pharmaceutical | 26 | 2017-10-10 | 2019-03-07 | |
| Neurovascular Transduction During Exercise in Chronic Kidney Disease 6R-BH4, 6R-BH4 placebo, Folic acid - Renal Insufficiency, Chronic | Phase 2 | Recruiting | Emory University | 150 | 2016-10-01 | 2026-05-18 |
| Systemic Sclerosis (SSc) Vasculopathy: Improved Clinical Monitoring and Treatment BH4, Vasculopathy assessment, Placebo - Rheumatologic Disease | Phase 1 / Phase 2 | Completed | VA Office of Research and Development | 12 | 2016-01-01 | 2021-04-05 |
| Long Term Nitric Oxide Bioavailability on Vascular Health in Chronic Obstructive Pulmonary Disease Tetrahydrobiopterin (BH4), Antioxidant Cocktail - Chronic Obstructive Pulmonary Disease (COPD) | Phase 2 | Completed | Augusta University | 15 | 2015-09-01 | 2021-02-23 |
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Phenylketonurias (36) · Hypertension (4) · Peripheral Arterial Disease (3) · Pulmonary Disease, Chronic Obstructive (3) · Autistic Disorder (2) · Behavior (2) · Intermittent Claudication (2) · Kidney Diseases (2) · Motor Activity (2) · Psychotic Disorders (2) · Radiodermatitis (2) · Schizophrenia (2) · Albuminuria (1) · Alzheimer Disease (1) · Anemia, Sickle Cell (1)
BioMarin Pharmaceutical (18) · Emory University (5) · Augusta University (3) · VA Office of Research and Development (3) · APR Applied Pharma Research s.a. (2) · Merck KGaA, Darmstadt, Germany (2) · The Children's Health Council (2) · The University of Texas Health Science Center, Houston (2) · University of Massachusetts, Worcester (2) · Washington University School of Medicine (2) · West China Second University Hospital (2) · Birmingham Women's and Children's NHS Foundation Trust (1) · Children's Hospital of Philadelphia (1) · Children's National Research Institute (1) · Dr. Linda Randolph (1)
United States (52) · Canada (5) · China (4) · United Kingdom (4) · Germany (3) · Italy (3) · Spain (2) · Argentina (1) · Austria (1) · Belgium (1) · Czechia (1) · Denmark (1) · Egypt (1) · Netherlands (1) · Norway (1)
Don Nwose, MD (3) · David W. Wray, PhD (2) · Desiree White, Ph.D. (2) · Dorothy K. Grange, M.D. (2) · Alex Dorenbaum, MD (1) · Andrea L Gropman, M.D. (1) · Andrea Superti-Furga, Prof. (1) · Anita MacDonald, PhD (1) · Arshed A Quyyumi, MD (1) · Can Ficicioglu, MD, PhD (1) · Ece Kucuksayrac, MD (1) · Elizabeth R DeGrush, DO (1) · Glen R Elliott, Ph.D., M.D. (1) · Glen R Elliott, PhD, MD (1)
PMID 28596000, PMID 17693179, PMID 18322548, PMID 19261295, PMID 20837663, PMID 21646032, PMID 22315282, PMID 23006929, PMID 23462988, PMID 23608077, PMID 23782126, PMID 23928118, PMID 24043380, PMID 26962106, PMID 27513937, PMID 28274234, PMID 28465125, PMID 29705218, PMID 33755856, PMID 33967762, PMID 34344399, PMID 34513700, PMID 36115980, PMID 36810250, PMID 37263523
2009 (2), K23HL098744 (1), R01HL135183 (1), R56HL114073 (1)