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 | ||
|---|---|---|---|
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) | ||
| NCT05579548 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | ||
| NCT04480567 | AAV Gene Therapy Study for Subjects with PKU | ||
| NCT06224907 | Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A | ||
| NCT03505125 | A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults | ||
| NCT02329769 | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT05768386 | A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) | ||
| NCT01435772 | Extension Study for Patients Who Have Participated in a BMN 701 Study | ||
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | ||
| NCT03150069 | Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | ||
| 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 | ||
| NCT03989947 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | ||
| NCT00838435 | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU | ||
| NCT01275066 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT03694353 | Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU | ||
| NCT06138327 | A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria | ||
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | ||
| NCT04800692 | The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication | ||
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | ||
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | ||
| NCT01920828 | Gait Analysis in MPS IVA | ||
| NCT01515956 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT02353312 | Rhode Island Diastolic Dysfunction - Heart Failure | ||
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors | ||
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease | ||
| NCT03520712 | Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 | ||
| NCT00067470 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | ||
| NCT04560933 | A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | ||
| NCT01603095 | A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia | ||
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | ||
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | ||
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | ||
| NCT05314751 | Learning to Live With Non-severe Haemophilia | ||
| NCT00048711 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | ||
| NCT01806051 | A Pilot Study on Diurnal Variation | ||
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | ||
| NCT02537561 | Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors | ||
| NCT03864029 | Retrospective Observational Safety Effectiveness With Kuvan in hpA | ||
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | ||
| NCT06168201 | VIrtual STudy in Achondroplasia for the US (VISTA) | ||
| NCT01826474 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | ||
| NCT01752296 | Psychological Concomitants of Morquio Syndrome (The MAP Study) | ||
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI |
