BioMarin Pharmaceutical: all 172 studies

Page 1 of 2. Open to participants first (21), then studies with results posted, then the rest; newest registration first within each.

• An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular DystrophyEnrolling by invitationNCT07573631Phase 2
• Immune Modulation During Palynziq® Treatment in Adults (IMPALA)RecruitingNCT07477691Phase 4
• Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaRecruitingNCT07441876Phase 2/3
• A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 MonthsRecruitingNCT07126262Phase 2
• Long-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiaEnrolling by invitationNCT07073014Phase 3
• A Study of Patients With Fabry Disease (US Specific)RecruitingNCT06906367
• A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsRecruitingNCT06904261Phase 3
• A Phase 2 Study of Vosoritide in Children With Idiopathic Short StatureRecruitingNCT06382155Phase 2
• A Study to Assess Growth in Children With Idiopathic Short StatureRecruitingNCT06309979
• A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)RecruitingNCT06305234
• A Multicenter Multinational Observational Study of Children With HypochondroplasiaRecruitingNCT06212947
• VIrtual STudy in Achondroplasia for the US (VISTA)RecruitingNCT06168201
• A Global Prospective Observational Registry of Patients With Pompe DiseaseRecruitingNCT06121011
• A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)RecruitingNCT05813678
• A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)Enrolling by invitationNCT05768386
• A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and BreastfeedingRecruitingNCT05579548
• A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18RecruitingNCT04808505Phase 3
• Expanded Access for ATB200/AT2221 for the Treatment of IOPDAvailableNCT04327973
• A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingRecruitingNCT04252066
• Expanded Access for ATB200/AT2221 for the Treatment of Pompe DiseaseAvailableNCT03865836
• Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry DiseaseAvailableNCT01476163
• Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With PhenylketonuriaActive, not recruitingNCT05270837Phase 3Results posted
• Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXECompletedNCT05030831Phase 1/2Results posted
• Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia ACompletedNCT04323098Phase 3Results posted
• A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.CompletedNCT04281537Results posted
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)CompletedNCT04138277Phase 3Results posted
• Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry DiseaseCompletedNCT04049760Phase 3Results posted
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe DiseaseCompletedNCT03729362Phase 3Results posted
• Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKUCompletedNCT03694353Phase 3Results posted
• A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With AchondroplasiaCompletedNCT03583697Phase 2Results posted
• Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5TerminatedNCT03520712Phase 1/2Results posted
• Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)CompletedNCT03500094Phase 3Results posted
• Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kgCompletedNCT03392974Phase 3Results posted
• Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)CompletedNCT03370913Phase 3Results posted
• A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaCompletedNCT03197766Phase 3Results posted
• A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 DiseaseCompletedNCT02678689Phase 2Results posted
• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221CompletedNCT02675465Phase 1/2Results posted
• Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis BullosaTerminatedNCT02670330Phase 3Results posted
• Gene Therapy Study in Severe Haemophilia A Patients (270-201)CompletedNCT02576795Phase 1/2Results posted
• An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 DiseaseCompletedNCT02485899Phase 1/2Results posted
• A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302CompletedNCT02468570Results posted
• ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis BullosaCompletedNCT02384460Phase 3Results posted
• Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseCompletedNCT02194985Phase 3Results posted
• A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)TerminatedNCT02191917Results posted
• Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis BullosaTerminatedNCT02090283Phase 2Results posted
• A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With AchondroplasiaCompletedNCT02055157Phase 2Results posted
• Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis BullosaCompletedNCT02014376Phase 2Results posted
• Sapropterin on Cognitive Abilities in Young Adults With PhenylketonuriaTerminatedNCT01977820Phase 2Results posted
• BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)TerminatedNCT01924845Phase 3Results posted
• 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) DiseaseCompletedNCT01907087Phase 1/2Results posted
• Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKUCompletedNCT01889862Phase 3Results posted
• An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165CompletedNCT01819727Phase 3Results posted
• Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited AmbulationTerminatedNCT01697319Phase 2Results posted
• Safety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeTerminatedNCT01609062Phase 2Results posted
• A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 WeeksCompletedNCT01560286Phase 2Results posted
• Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01515956Phase 2Results posted
• Open-Label Phase 3 Long-Term Safety Study of MigalastatTerminatedNCT01458119Phase 3Results posted
• Extension Study for Patients Who Have Participated in a BMN 701 StudyTerminatedNCT01435772Phase 2Results posted
• Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01415427Phase 3Results posted
• Drug-drug Interaction StudyCompletedNCT01380743Phase 2Results posted
• Kuvan® in Phenylketonuria Patients Less Than 4 Years OldCompletedNCT01376908Phase 3Results posted
• A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01275066Phase 3Results posted
• A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)TerminatedNCT01242111Phase 1/2Results posted
• Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe DiseaseCompletedNCT01230801Phase 1/2Results posted
• Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry DiseaseCompletedNCT01218659Phase 3Results posted
• Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)CompletedNCT01212744Phase 2Results posted
• Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry DiseaseCompletedNCT01196871Phase 2Results posted
• Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) PatientsCompletedNCT01114737Phase 3Results posted
• Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)CompletedNCT01037309Phase 1/2Results posted
• Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry DiseaseCompletedNCT00925301Phase 3Results posted
• Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKUCompletedNCT00925054Phase 2Results posted
• Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)CompletedNCT00924703Phase 2Results posted
• A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVACompletedNCT00884949Phase 1/2Results posted
• Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKUCompletedNCT00838435Phase 3Results posted
• A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher PatientsCompletedNCT00813865Phase 2Results posted
• Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe DiseaseTerminatedNCT00688597Phase 2Results posted
• A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial DysfunctionCompletedNCT00532844Phase 2Results posted
• Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 StudyTerminatedNCT00526071Phase 2Results posted
• A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher DiseaseCompletedNCT00446550Phase 2Results posted
• A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell DiseaseCompletedNCT00445978Phase 2Results posted
• A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement TherapyCompletedNCT00433147Phase 2Results posted
• A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial DiseaseCompletedNCT00403494Phase 2Results posted
• Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 DeficiencyCompletedNCT00355264Phase 2Results posted
• Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006CompletedNCT00332189Phase 3Results posted
• A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry DiseaseCompletedNCT00304512Phase 2Results posted
• A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VICompletedNCT00299000Phase 4Results posted
• A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00283959Phase 2Results posted
• A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00283933Phase 2Results posted
• Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted DietCompletedNCT00272792Phase 3Results posted
• A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00214500Phase 2Results posted
• Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsCompletedNCT00104247Phase 3Results posted
• Study of rhASB in Patients With Mucopolysaccharidosis VICompletedNCT00104234Phase 3Results posted
• Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VICompletedNCT00067470Phase 3Results posted
• Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 DeficiencyCompletedNCT07745179
• ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 DeficiencyTerminatedNCT07473973Phase 3
• Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to PalynziqCompletedNCT06780332Phase 4
• The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 DeficiencyWithdrawnNCT06739980Phase 2
• Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult ParticipantsActive, not recruitingNCT06738017Phase 1
• A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentActive, not recruitingNCT06668805Phase 2
• ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 DeficiencyTerminatedNCT06462547Phase 2

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