Asitri Dashboard Search Clinical Trials
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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Count: 171
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| NCT ID | Title | ||
|---|---|---|---|
| NCT04560933 | A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A | ||
| NCT01560286 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks | ||
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | ||
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | ||
| NCT04404530 | Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) | ||
| NCT03370913 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) | ||
| NCT01016392 | Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency | ||
| NCT06046820 | The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency | ||
| NCT06280209 | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy | ||
| NCT02055157 | A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia | ||
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | ||
| NCT06168201 | VIrtual STudy in Achondroplasia for the US (VISTA) | ||
| NCT05580692 | A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV) | ||
| NCT01274026 | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders | ||
| NCT02678689 | A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | ||
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | ||
| NCT00332189 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT01230801 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease | ||
| NCT00802893 | Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction | ||
| NCT03989947 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | ||
| NCT00924703 | Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) | ||
| NCT00355264 | Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency | ||
| NCT02049593 | PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors | ||
| NCT01752296 | Psychological Concomitants of Morquio Syndrome (The MAP Study) | ||
| NCT05734196 | The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency | ||
| NCT00925054 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU | ||
| NCT06309979 | A Study to Assess Growth in Children With Idiopathic Short Stature | ||
| NCT01650909 | The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU | ||
| NCT01541397 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | ||
| NCT02636686 | Extension Study of Drisapersen in DMD Subjects | ||
| NCT00214773 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | ||
| NCT01114737 | Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients | ||
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | ||
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | ||
| NCT04323098 | Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A | ||
| NCT00325962 | A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension | ||
| NCT01753804 | A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. | ||
| NCT01920828 | Gait Analysis in MPS IVA | ||
| NCT02576795 | Gene Therapy Study in Severe Haemophilia A Patients (270-201) | ||
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT06212947 | A Multicenter Multinational Observational Study of Children With Hypochondroplasia | ||
| NCT01907087 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT03856203 | Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase | ||
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 | ||
| NCT07573631 | An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy | ||
| NCT06138327 | A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria | ||
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency |
