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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Clinical Trials Search Results
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Count: 165
Selected: 0
| NCT ID | Title | ||
|---|---|---|---|
| NCT00272792 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet | ||
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | ||
| NCT03872531 | Lifetime Impact Study for Achondroplasia | ||
| NCT01806051 | A Pilot Study on Diurnal Variation | ||
| NCT01920828 | Gait Analysis in MPS IVA | ||
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria | ||
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | ||
| NCT00214773 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | ||
| NCT03856203 | Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase | ||
| NCT04480567 | AAV Gene Therapy Study for Subjects with PKU | ||
| NCT02329769 | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | ||
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors | ||
| NCT01910649 | A 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 | ||
| NCT01230801 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease | ||
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | ||
| NCT00325962 | A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension | ||
| NCT02208661 | Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) | ||
| NCT02576795 | Gene Therapy Study in Severe Haemophilia A Patients (270-201) | ||
| NCT02294877 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | ||
| NCT00925054 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU | ||
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 | ||
| NCT00355264 | Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency | ||
| NCT05356377 | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU | ||
| NCT02597881 | Achondroplasia Natural History Multicenter Clinical Study | ||
| NCT03694353 | Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU | ||
| NCT03392974 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg | ||
| NCT00778206 | PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry | ||
| NCT00688844 | Nutritional and Neurotransmitter Changes in PKU Subjects on BH4 | ||
| NCT04684940 | Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors | ||
| NCT03449368 | Lifetime Impact of Achondroplasia Study in Europe-LIAISE | ||
| NCT04368624 | PKU Skin Stripping | ||
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | ||
| NCT00943579 | Open-Label Extension Study of Kuvan for Autism | ||
| NCT04323098 | Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A | ||
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | ||
| NCT01869972 | Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia | ||
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | ||
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | ||
| NCT03792451 | Nutrition Status of Adults Treated With Pegvaliase | ||
| NCT02677870 | The Effectiveness of Kuvan in Amish PKU Patients | ||
| NCT00789568 | A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects | ||
| NCT06738017 | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | ||
| NCT01966029 | BMN 110 Phase 3B in Australian Patients | ||
| NCT00964236 | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | ||
| NCT01603095 | A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia | ||
| NCT03197766 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) |
