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: 169
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| NCT ID | Title | ||
|---|---|---|---|
| NCT01275066 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT00484991 | Sapropterin Expanded Access Program | ||
| NCT01650909 | The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU | ||
| NCT02724228 | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | ||
| 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 | ||
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | ||
| NCT02221362 | A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT01515956 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT07477691 | Immune Modulation During Palynziq® Treatment in Adults (IMPALA) | ||
| NCT00884949 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA | ||
| NCT00778206 | PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry | ||
| 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 | ||
| NCT00852358 | A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I | ||
| NCT02191917 | A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD) | ||
| NCT04684940 | Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors | ||
| NCT01376908 | Kuvan® in Phenylketonuria Patients Less Than 4 Years Old | ||
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency | ||
| NCT01707433 | Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease | ||
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | ||
| NCT00730080 | Sapropterin in Individuals With Phenylketonuria | ||
| NCT02583152 | New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis | ||
| NCT05813314 | Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants | ||
| NCT02365376 | Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions | ||
| NCT02677870 | The Effectiveness of Kuvan in Amish PKU Patients | ||
| NCT02208661 | Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) | ||
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | ||
| NCT03197766 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT03392974 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg | ||
| NCT01141595 | Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders | ||
| NCT00964236 | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | ||
| NCT02401347 | Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors | ||
| 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 | ||
| NCT03424018 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 | ||
| NCT00935753 | Trial of Kuvan in Lesch-Nyhan Disease | ||
| NCT00355264 | Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency | ||
| NCT04055051 | ATHN 11: Liver Transplantation Outcomes Study | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT03872531 | Lifetime Impact Study for Achondroplasia | ||
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | ||
| NCT02537561 | Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors | ||
| NCT01274026 | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders | ||
| NCT03862274 | Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease | ||
| NCT04368624 | PKU Skin Stripping | ||
| NCT00325962 | A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension | ||
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria | ||
| NCT00802893 | Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction | ||
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease |
