| sapropterin / Generic mfg. |
2010-021343-41: Variations of blood phenylalanine and tyrosine in children with phenylketonuria under sapropterin |
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| Ongoing | 4 | 20 | Europe | Soluble tablet, Kuvan | University Medical Center Groningen, Merck KGaA | Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene for the enzyme phenylalanine hydroxylase (PAH), rendering it nonfunctional. Left untreated, the disease will result in high concentrations of phenylalanine (Phe) in blood and tissues, likely resulting in severe mental retardation and behavioural problems. Treatment focusus on the restriction of dietary phenylalanine intake with supplementation of a synthetic phenylalaninefree amino acid mixture., Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder. Untreated, PKU can lead to mental retardation and behavioural problems., Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
| Completed | 4 | 34 | Europe | Kuvan®, Sapropterin dihydrochloride | BioMarin Pharmaceutical | Phenylketonuria | 01/23 | 01/23 | | |
NCT06396637: PD-1 Antibody and Sapropterin Dihydrochloride in Patients With PDAC |
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| Recruiting | 2 | 20 | RoW | Sapropterin Dihydrochloride, PD-1 Antibody | Sun Yat-sen University, Innovent Biologics (Suzhou) Co. Ltd. | Pancreatic Adenocarcinoma Metastatic | 04/25 | 04/25 | | |
| Recruiting | 2 | 150 | US | 6R-BH4, Tetrahydrobiopterin, Kuvan, Sapropterin dihydrochloride, 6R-BH4 placebo, Folic acid, Exercise training, Stretching, Control for exercise training, Histidine and beta-alanine supplementation, Histidine and beta-alanine placebo | Emory University, National Heart, Lung, and Blood Institute (NHLBI) | Renal Insufficiency, Chronic | 10/24 | 10/24 | | |
NCT05299203: BH4 in the Prevention and Treatment of Radiation-induced Skin Reactions for Head and Neck Squamous Cell Carcinoma (Including Nasopharyngeal Carcinoma) |
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| Not yet recruiting | 1a/1b | 18 | NA | Sapropterin dihydrochloride tablets | West China Hospital | Head and Neck Squamous Cell Carcinoma | 12/22 | 05/23 | | |
NCT06312748: Novel Approaches for Improving Vascular Function in Veterans With HFpEF |
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| Recruiting | 1 | 90 | US | L-Citrulline, Placebo for L-Citrulline, BH4, Kuvan®, sapropterin dihydrochloride, Placebo for BH4, Atorvastatin, Lipitor®, Placebo for Atorvastatin | D. Walter Wray, US Department of Veterans Affairs | HFpEF - Heart Failure With Preserved Ejection Fraction, Inflammation | 09/28 | 09/30 | | |
ChiCTR2100054693: Phase Ia/Ib clinical trial of Sapropterin dihydrochloride tablets in the prevention and treatment of radiation-induced skin reactions in intensity modulated radiotherapy for head and neck squamous cell carcinoma (including nasopharyngeal carcinoma) |
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| Not yet recruiting | 1 | 18 | | Dose Escalation: Sapropterin dihydrochloride tablets ;Dose Expansion: Sapropterin dihydrochloride tablets | West China Hospital, Sichuan University; West China Hospital, Sichuan University, Project funding | Head and neck squamous cell carcinoma (including nasopharyngeal carcinoma) | | | | |
ATLAS, NCT04800692: The Effects of Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication |
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| Recruiting | 1 | 10 | US | Tetrahydrobiopterin 10 mg/kg, sapropterin, kuvan, Tetrahydrobiopterin 20 mg/kg, L-Ascorbate, L-Arginine | Louis Messina, BioMarin Pharmaceutical | Claudication, Intermittent, Peripheral Artery Disease, Peripheral Vascular Diseases | 07/25 | 12/25 | | |
| Recruiting | 1 | 140 | US | Antioxidant, Tetrahydrobiopterin (BH4), Kuvan, Exercise training | VA Office of Research and Development, University of Utah | Heart Failure With Reduced Ejection Fraction | 05/25 | 05/26 | | |
NCT06481709: Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects |
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| Completed | 1 | 16 | Canada | RLF-OD032, Sapropterin dihydrochloride, Kuvan | APR Applied Pharma Research s.a. | Bioavailability | 08/24 | 08/24 | | |
| domofenogene zalfaparvovec (BMN 307) / BioMarin |
2019-001878-28: A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuria |
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| Not yet recruiting | 1/2 | 100 | Europe | BMN 307, Solution for infusion | BioMarin Pharmaceutical Inc., BioMarin Pharmaceutical Inc. | Phenylketonuria, metabolic disorder, Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
| Active, not recruiting | 1/2 | 100 | Europe, US | BMN 307 | BioMarin Pharmaceutical, BioMarin Pharmaceutical Inc. | Phenylketonuria (PKU) | 12/27 | 12/27 | | |
