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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: 169
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NCT IDTitle
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
NCT02724228A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
NCT00838435Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
NCT06668805A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
NCT03520712Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT00532844A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
NCT00332189Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
NCT01819727An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT03792451Nutrition Status of Adults Treated With Pegvaliase
NCT03017677A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT06212947A Multicenter Multinational Observational Study of Children With Hypochondroplasia
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT02049593PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors
NCT07573631An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT00355264Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
NCT04684940Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT02392793Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT01425528Study of Kuvan Treatment in Adults With GTPCH Deficiency
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT00925054Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT00935753Trial of Kuvan in Lesch-Nyhan Disease
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT04368624PKU Skin Stripping
NCT01617070Effects of Kuvan on Melatonin Secretion
NCT06224907Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
NCT01924026Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT01376908Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT01541397Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT00852358A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
NCT01037309Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT02597881Achondroplasia Natural History Multicenter Clinical Study
NCT06738017Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT00104247Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels