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The U.S. National Library of Medicine provides a database of clinical trials worldwide. Use the fields below to search the database. Expand your results and select terms or phrases and right-click to search using Google, MalaCards, MedlinePlus, PubMed, or Wikipedia.

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Clinical Trials Search Results

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Count: 164
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NCT IDTitle
NCT00935753Trial of Kuvan in Lesch-Nyhan Disease
NCT01387854Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
NCT00789568A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
NCT00634660Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT01016392Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
NCT03583697A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT04368624PKU Skin Stripping
NCT06224907Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT04227080BH4 Responsiveness in PAH Deficiency PKU Patients
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT00445978A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
NCT02329769Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT01617070Effects of Kuvan on Melatonin Secretion
NCT00272792Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
NCT01376908Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
NCT01733615Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
NCT01910649A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT04480567AAV Gene Therapy Study for Subjects with PKU
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
NCT00838435Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT03872531Lifetime Impact Study for Achondroplasia
NCT00325962A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT04323098Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT04055051ATHN 11: Liver Transplantation Outcomes Study
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT06668805A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT01650909The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT01435772Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT00730080Sapropterin in Individuals With Phenylketonuria
NCT01961518Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
NCT00728676Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors
NCT01425528Study of Kuvan Treatment in Adults With GTPCH Deficiency