BioMarin Pharmaceutical: all 172 studies
Page 2 of 2. Open to participants first (21), then studies with results posted, then the rest; newest registration first within each.
- Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaActive, not recruitingNCT06455059Phase 3
- PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 DeficiencyTerminatedNCT06302439
- SEAPORT 1: Study of INZ-701 in Adults With Hemodialysis-Dependent End-Stage Kidney DiseaseCompletedNCT06283589Phase 1
- A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular DystrophyActive, not recruitingNCT06280209Phase 1/2
- Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia AActive, not recruitingNCT06224907Phase 3
- A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And HyperoxaluriaWithdrawnNCT06138327Phase 1
- The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 DeficiencyTerminatedNCT06046820Phase 3
- Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy ParticipantsTerminatedNCT05813314Phase 1
- The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 DeficiencyTerminatedNCT05734196Phase 1
- A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)TerminatedNCT05580692Not applicable
- A Gene Therapy Study of BMN 331 in Subjects With Hereditary AngioedemaActive, not recruitingNCT05121376Phase 1/2
- Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 DeficiencyCompletedNCT05050669
- Understanding Fabry Disease Therapy Choices Through the Eyes of the PatientsCompletedNCT04804566
- Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 DeficiencyCompletedNCT04686175Phase 1/2
- Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior InhibitorsCompletedNCT04684940Phase 1/2
- French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With MigalastatCompletedNCT04602364
- A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia ACompletedNCT04560933
- A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With AchondroplasiaActive, not recruitingNCT04554940Phase 2
- AAV Gene Therapy Study for Subjects with PKUActive, not recruitingNCT04480567Phase 1/2
- Cerliponase Alfa Observational Study in the USActive, not recruitingNCT04476862
- A Prospective Clinical Study of Phenylketonuria (PKU)CompletedNCT04452513
- Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 DeficiencyCompletedNCT04372446
- Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and PreferencesCompletedNCT04043273
- A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal DiseaseActive, not recruitingNCT04020055Phase 3
- An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03989947Phase 2
- ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDActive, not recruitingNCT03911505Phase 3
- Lifetime Impact Study for AchondroplasiaCompletedNCT03872531
- Retrospective Observational Safety Effectiveness With Kuvan in hpACompletedNCT03864029
- A Non-Interventional Interview Study of Phenylketonuria (PKU) AdultsCompletedNCT03505125
- Lifetime Impact of Achondroplasia Study in Europe-LIAISECompletedNCT03449368
- An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03424018Phase 3
- STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERTTerminatedNCT03347253
- Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis BullosaCompletedNCT03158662
- A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 DiseaseApproved for marketingNCT02963350
- Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)TerminatedNCT02958202Phase 2
- A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)Active, not recruitingNCT02724228Phase 2
- Extension Study of Drisapersen in DMD SubjectsNo longer availableNCT02636686
- Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical ConditionsStatus unknownNCT02365376
- Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)TerminatedNCT02329769Phase 2
- A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)CompletedNCT02294877
- A Prospective, Noninterventional, Observational Study of Late-Onset Pompe DiseaseTerminatedNCT02221362
- A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy VolunteersCompletedNCT02082327Phase 1
- BMN 110 Phase 3B in Australian PatientsCompletedNCT01966029Phase 3
- Kuvan®'s Effect on the Cognition of Children With PhenylketonuriaCompletedNCT01965912Phase 4
- A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)TerminatedNCT01957059Phase 1/2
- 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 AdministrationTerminatedNCT01910649Phase 2
- BMN 110 US Expanded Access ProgramApproved for marketingNCT01858103
- A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese SubjectsCompletedNCT01853852Phase 1
- Phase IIb Study of PRO045 in Subjects With Duchenne Muscular DystrophyTerminatedNCT01826474Phase 1/2
- A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian SubjectsTerminatedNCT01803412Phase 3
- A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.TerminatedNCT01753804
- A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)CompletedNCT01730482Phase 1
- Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal FunctionCompletedNCT01730469Phase 1
- A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With AchondroplasiaCompletedNCT01603095
- A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult VolunteersCompletedNCT01590446Phase 1
- Migalastat Food Effect StudyCompletedNCT01489995Phase 1
- Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02CompletedNCT01387854
- Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 DeficiencyCompletedNCT01016392
- A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101TerminatedNCT00875160Phase 1
- A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult SubjectsCompletedNCT00789568Phase 1
- A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)TerminatedNCT00787995
- PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryCompletedNCT00778206
- Safety and Tolerability Study of rAvPAL-PEG to Treat PhenylketonuriaCompletedNCT00634660Phase 1
- A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe DiseaseCompletedNCT00515398
- Sapropterin Expanded Access ProgramApproved for marketingNCT00484991
- Study to Evaluate Blood Cell Lines From Patients With Gaucher DiseaseCompletedNCT00351156
- A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic HypertensionCompletedNCT00325962Phase 2
- A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine LevelsCompletedNCT00225615Phase 3
- Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)CompletedNCT00214773
- Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With PhenylketonuriaCompletedNCT00104260Phase 2
- Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VICompletedNCT00048711Phase 2
- Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VICompletedNCT00048620Phase 1