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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: 165
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NCT IDTitle
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT02963350A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
NCT05368038ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT03150069Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
NCT04560933A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
NCT00484991Sapropterin Expanded Access Program
NCT03520712Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
NCT00104234Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT01924026Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
NCT02208661Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
NCT03204370Natural History of Atypical Morquio A Disease
NCT06168201VIrtual STudy in Achondroplasia for the US (VISTA)
NCT01924845BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
NCT02156674Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
NCT01920828Gait Analysis in MPS IVA
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT03583697A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT01819727An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT02191917A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT01016392Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
NCT00299000A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
NCT03017677A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT01650909The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT00730080Sapropterin in Individuals With Phenylketonuria
NCT02468570A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
NCT00787995A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT00943579Open-Label Extension Study of Kuvan for Autism
NCT02576795Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT01515956Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01609062Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
NCT06305234A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT02678689A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
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
NCT01230801Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
NCT06224907Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT06382155A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature