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: 164
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| NCT ID | Title | ||
|---|---|---|---|
| NCT03392974 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg | ||
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | ||
| NCT04055051 | ATHN 11: Liver Transplantation Outcomes Study | ||
| NCT03792451 | Nutrition Status of Adults Treated With Pegvaliase | ||
| NCT01966029 | BMN 110 Phase 3B in Australian Patients | ||
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | ||
| NCT00484991 | Sapropterin Expanded Access Program | ||
| NCT04323098 | Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A | ||
| NCT01819727 | An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 | ||
| NCT01924845 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) | ||
| NCT03520712 | Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 | ||
| NCT01806051 | A Pilot Study on Diurnal Variation | ||
| NCT03424018 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT00445978 | A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease | ||
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | ||
| NCT00532844 | A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction | ||
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors | ||
| NCT06168201 | VIrtual STudy in Achondroplasia for the US (VISTA) | ||
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | ||
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | ||
| NCT01415427 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | ||
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria | ||
| NCT06280209 | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy | ||
| NCT00802893 | Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction | ||
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 | ||
| NCT00925054 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU | ||
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | ||
| NCT06455059 | Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia | ||
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | ||
| NCT06309979 | A Study to Assess Growth in Children With Idiopathic Short Stature | ||
| NCT02353312 | Rhode Island Diastolic Dysfunction - Heart Failure | ||
| NCT01803412 | A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects | ||
| NCT04560933 | A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A | ||
| NCT00935753 | Trial of Kuvan in Lesch-Nyhan Disease | ||
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT01541397 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | ||
| NCT01889862 | Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU | ||
| NCT00789568 | A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects | ||
| NCT00332189 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | ||
| NCT02401347 | Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors | ||
| NCT02537561 | Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors | ||
| NCT03370913 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) | ||
| NCT00730080 | Sapropterin in Individuals With Phenylketonuria | ||
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | ||
| NCT03449368 | Lifetime Impact of Achondroplasia Study in Europe-LIAISE | ||
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) | ||
| NCT01560286 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks | ||
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder |
