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Clinical trials
catalyst calendar across sponsors →Studies where BMRN is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
82 interventional · 26 observational · 4 expanded access · 46 with posted results
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| A Multicenter Multinational Observational Study of Children With HypochondroplasiaNCT06212947observationalChange in annualized growth velocity (AGV) | — | Recruiting | — | Hypochondroplasia | 400 | 2043-12-31 | in 6,343 d | 2025-04-03 |
| A Study to Assess Growth in Children With Idiopathic Short StatureNCT06309979observationalChange in annualized growth velocity (AGV) | — | Recruiting | — | Idiopathic Short Stature | 300 | 2040-12-31 | in 5,248 d | 2026-06-23 |
| Long-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiaNCT07073014Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)Open-label · Treatment | Phase 3 | Enrolling by invitation | Drug: Vosoritide | Hypochondroplasia | 140 | Dec 2040 | ≤ 5,248 d | 2025-07-18 |
| A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)NCT05768386observationalEvaluate the long-term safety of BMN 270 | — | Enrolling by invitation | — | Hemophilia A | 172 | Jan 2040 | ≤ 4,913 d | 2026-01-12 |
| An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaNCT03989947Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]Open-label · Treatment | Phase 2 | Active, not recruiting | Drug: Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. | Achondroplasia | 73 | May 2038 | ≤ 4,303 d | 2026-03-13 |
| A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)NCT06305234observationalTo evaluate immunologic and inflammatory responses associated with occurrences of | — | Recruiting | Drug: Pegvaliase | Phenylketonuria (PKU) | 200 | 2033-11-18 | in 2,648 d | 2026-03-24 |
| A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)NCT05813678observationalTo quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real¬ world setting. | — | Recruiting | Drug: Pegvaliase | Phenylketonuria (PKU) | 450 | 2033-11-01 | in 2,631 d | 2026-03-25 |
| A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and BreastfeedingNCT05579548observationalPregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period | — | Recruiting | Drug: Pegvaliase | Phenylketonuria, Maternal | 50 | 2032-10-04 | in 2,238 d | 2026-03-24 |
| An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular DystrophyNCT07573631To assess the long-term safety and tolerability of BMN 351 in participants with DMDOpen-label · Treatment | Phase 2 | Enrolling by invitation | Drug: BMN 351 | Duchenne Muscular Dystrophy (DMD) | 18 | Dec 2031 | ≤ 1,960 d | 2026-07-24 |
| An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaNCT03424018Change from baselines in mean annualized growth velocityOpen-label · Treatment | Phase 3 | Active, not recruiting | Drug: BMN 111 | Achondroplasia | 119 | Jun 2031 | ≤ 1,776 d | 2026-03-13 |
| Cerliponase Alfa Observational Study in the USNCT04476862observationalSafety surveillance of cerliponase alfa | — | Active, not recruiting | Drug: Cerliponase Alfa · Device: Administration Kit | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | 35 | 2030-08-31 | in 1,473 d | 2026-06-30 |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaNCT07441876Phase 2: Predicted Annualized Growth Velocity (AGV) at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data]Randomized · Single-masked · Treatment | Phase 2/3 | Recruiting | Drug: BMN 333, Vosoritide Injection [Voxzogo] | Achondroplasia | 160 | Jun 2029 | ≤ 1,046 d | 2026-07-24 |
| A Gene Therapy Study of BMN 331 in Subjects With Hereditary AngioedemaNCT05121376Number of participants with treatment-emergent adverse events following a single IV administration of BMN 331Open-label · Treatment | Phase 1/2 | Active, not recruiting | Genetic: Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331, Dose 4 of BMN 331, Dose 5 of BMN 331, Dose 6 of BMN 331, Dose 7 of BMN 331 | Hereditary Angioedema, HAE | 44 | Nov 2028 | ≤ 834 d | 2024-05-16 |
| A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 MonthsNCT07126262Incidence of treatment-emergent adverse eventsRandomized · Quadruple-masked · Treatment | Phase 2 | Recruiting | Drug: Vosoritide, Placebo | Hypochondroplasia | 60 | 2028-06-30 | in 681 d | 2026-03-31 |
| VIrtual STudy in Achondroplasia for the US (VISTA)NCT06168201observationalHeight | — | Recruiting | — | Achondroplasia | 170 | Feb 2028 | ≤ 559 d | 2026-06-30 |
| A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)NCT02724228Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]Open-label · Treatment | Phase 2 | Active, not recruiting | Drug: BMN 111 | Achondroplasia | 30 | Dec 2027 | ≤ 499 d | 2026-03-13 |
| AAV Gene Therapy Study for Subjects with PKUNCT04480567Change from baseline in mean Plasma Phe levelsNon-randomized · Open-label · Treatment | Phase 1/2 | Active, not recruiting | Drug: BMN 307 | Phenylketonuria (PKU) | 100 | Dec 2027 | ≤ 499 d | 2024-12-12 |
| A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With AchondroplasiaNCT04554940Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]Randomized · Open-label · Treatment | Phase 2 | Active, not recruiting | Biological: vosoritide | Achondroplasia | 20 | Dec 2027 | ≤ 499 d | 2026-03-13 |
| Immune Modulation During Palynziq® Treatment in Adults (IMPALA)NCT07477691Change in blood Phe levels from baseline to Week 25 and Week 49Non-randomized · Open-label · Treatment | Phase 4 | Recruiting | Biological: Pegvaliase · Drug: Methotrexate | Phenylketonuria | 12 | 2027-09-30 | in 407 d | 2026-06-17 |
| A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular DystrophyNCT06280209To evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters).Non-randomized · Open-label · Treatment | Phase 1/2 | Active, not recruiting | Drug: BMN 351 | Duchenne Muscular Dystrophy | 18 | 2027-04-14 | in 238 d | 2026-06-25 |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentNCT06668805Change from baseline in Annualized Growth Velocity (AGV)Randomized · Triple-masked · Treatment | Phase 2 | Recruiting | Drug: Vosoritide Injection | Noonan Syndrome | 30 | Mar 2027 | ≤ 224 d | 2026-05-11 |
| Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to PalynziqNCT06780332To determine if RDD to Palynziq improves drug tolerability and treatment persistence in adults with PKU experiencing HSRs leading to treatment interruption or reduction of dose or dosing frequency (reactive HSR).Open-label · Treatment | Phase 4 | Active, not recruiting | Drug: RDD to Palynziq | Phenylketonuria, PKU | 9 | Aug 2026 | ≤ 12 d | 2026-04-03 |
| Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult ParticipantsNCT06738017Participant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parametersRandomized · Triple-masked · Treatment | Phase 1 | Active, not recruiting | Drug: BMN 349, Placebo | Alpha 1-Antitrypsin Deficiency | 6 | 2026-08-19 | today | 2026-05-06 |
| Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaNCT06455059Change from baseline in annualized growth velocity (AGV) at Week 52 versus placeboRandomized · Quadruple-masked · Treatment | Phase 3 | Active, not recruiting | Drug: Vosoritide, Placebo | Hypochondroplasia | 80 | 2026-08-01 | — | 2026-01-20 |
| A Phase 2 Study of Vosoritide in Children With Idiopathic Short StatureNCT06382155Change from baseline in Annualized Growth Velocity (AGV)Randomized · Quadruple-masked · Treatment | Phase 2 | Recruiting | Drug: Vosoritide Injection, Human Growth Hormone, Placebo | Idiopathic Short Stature | 100 | Jun 2026 | — | 2026-05-22 |
| Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia ANCT06224907Change in the human coagulation factor VIII (hFVIII) activity, as measured by chromogenic substrate assay, during Weeks 49 to 52 post BMN 270 infusion from Baseline.Open-label · Treatment | Phase 3 | Active, not recruiting | Biological: Valoctocogene roxaparvovec | Hemophilia A | 6 | 2025-04-16actual | — | 2026-03-19 |
| Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With PhenylketonuriaNCT05270837results2026-07-21Change From Baseline in Blood Phe Concentration Following 72 Weeks (Average of the Week 69 and Week 73) on Study.Randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Active, not recruiting | Drug: Pegvaliase · Other: Diet Only | Phenylketonuria (PKU) | 55 | 2025-01-14actual | — | 2026-07-21 |
| Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior InhibitorsNCT04684940Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.Open-label · Treatment | Phase 1/2 | Completed | Biological: Valoctocogene roxaparvovec | Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII | 10 | 2026-04-20actual | — | 2026-07-10 |
| Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5NCT03520712results2025-08-22Number of Participants With Treatment Emergent Adverse EventsOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Terminatedsponsor's stated reason: Early rollover into long-term extension study | Biological: Valoctocogene Roxaparvovec | Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder | 3 | 2024-08-07actual | — | 2025-08-22 |
| A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)NCT05580692To qunatify Seroprevalence of antibodies to selected AAV serotypesOpen-label · Other | Not applicable | Terminatedsponsor's stated reason: Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved. | Procedure: Biospecimen Collection | Hemophilia A | 321 | 2024-07-31actual | — | 2024-08-19 |
| A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And HyperoxaluriaNCT06138327The primary safety endpoint is the incidence of adverse events in adult participants with NAFLD and hyperoxaluriaRandomized · Double-masked · Treatment | Phase 1 | Withdrawnsponsor's stated reason: The study was withdrawn because BioMarin decided to end the overall development program. The study withdrawal was not due to any patient safety concerns. | Drug: BMN 255, Placebo | Hyperoxaluria, Nonalcoholic Fatty Liver Disease, Kidney Stone | 0 | 2024-03-25actual | — | 2024-06-11 |
| A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)NCT02294877observationalSafety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests | — | Completed | Drug: Vimizim® (elosulfase alfa) | Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA | 418 | 2024-02-21actual | — | 2024-03-05 |
| Gene Therapy Study in Severe Haemophilia A Patients (270-201)NCT02576795results2025-04-10Number of Participants With Treatment Emergent Adverse EventsOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Biological: valoctocogene roxaparvovec | Severe Haemophilia A | 15 | 2024-02-14actual | — | 2025-04-10 |
| A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia ANCT04560933observationalAssess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A | — | Completed | Procedure: Blood sample collection | Hemophilia A | 186 | 2023-09-20actual | — | 2024-04-24 |
| Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy ParticipantsNCT05813314Maximum observed drug concentration (Cmax)Randomized · Open-label · Treatment | Phase 1 | Terminatedsponsor's stated reason: The BMN 111-104 study has terminated as further optimization of the approach to achieve bio equivalence is required. The study termination was not due to any patient safety concerns. | Combination product: BMN 111 administration via Injector Pen · Drug: BMN 111 administration via vial and syringe | Achondroplasia | 36 | 2023-06-14actual | — | 2023-10-12 |
| Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia ANCT04323098results2024-04-04Change From Baseline in FVIII Activity as Measured by Chromogenic Substrate Assay at Week 52.Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Biological: valoctocogene roxaparvovec | Hemophilia A | 22 | 2023-01-27actual | — | 2025-10-08 |
| Kuvan®'s Effect on the Cognition of Children With PhenylketonuriaNCT01965912Mean Full Scale Intelligence Quotient (FSIQ) Score of the Wechsler Intelligence Scale for Children (WISC)-IVOpen-label · Treatment | Phase 4 | Completed | Drug: Kuvan® | Phenylketonuria | 34 | 2023-01-04actual | — | 2023-04-24 |
| A Prospective Clinical Study of Phenylketonuria (PKU)NCT04452513observationalChange in Plasma Phe Levels | — | Completed | — | Phenylketonurias | 32 | 2022-06-02actual | — | 2023-03-14 |
| A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 DiseaseNCT02678689results2025-02-17Motor Language (ML) Scale: Rate of Decline in the 0 to 6-point ML Score.Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Biological: BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) · Device: Intracerebroventricular access device | Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder | 14 | 2022-04-20actual | — | 2025-02-17 |
| A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With AchondroplasiaNCT03583697results2024-06-13Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: BMN 111, Placebo | Achondroplasia | 75 | 2022-01-26actual | — | 2024-06-13 |
| Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 DeficiencyNCT01016392observationalIncidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs) | — | Completed | — | Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency | 627 | 2021-05-31actual | — | 2022-04-15 |
| A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With AchondroplasiaNCT01603095observationalCollection of consistent growth measurements | — | Completed | — | Achondroplasia | 363 | Feb 2021actual | — | 2021-04-13 |
| Lifetime Impact Study for AchondroplasiaNCT03872531observationalHealthcare resource use | — | Completed | — | Achondroplasia | 173 | 2021-02-06actual | — | 2021-04-05 |
| PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryNCT00778206observationalObservational Data Only | — | Completed | Drug: Kuvan | Phenylketonuria, Hyperphenylalaninaemia | 1,887 | 2021-01-29actual | — | 2022-10-12 |
| Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKUNCT03694353results2022-02-17Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Pegvaliase | Phenylketonuria (PKU) | 37 | 2021-01-13actual | — | 2022-02-17 |
| An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 DiseaseNCT02485899results2022-08-24Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Biological: BMN 190 · Device: Intracerebroventricular (ICV) access device | Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder | 23 | 2020-12-10actual | — | 2022-08-24 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)NCT03370913results2023-11-28Change From Baseline in Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in EEP.Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Biological: valoctocogene roxaparvovec | Hemophilia A | 144 | 2020-11-16actual | — | 2025-03-25 |
| Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)NCT00214773observationalTo further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase. | — | Completed | — | Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) | 237 | 2020-05-01actual | — | 2021-04-05 |
| Lifetime Impact of Achondroplasia Study in Europe-LIAISENCT03449368observationalHealthcare resource use | — | Completed | — | Achondroplasia | 196 | 2020-04-29actual | — | 2021-04-05 |
| A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaNCT03197766results2022-03-02Change From Baseline in Annualized Growth Velocity (AGV) at Week 52Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: BMN 111, Placebo | Achondroplasia | 121 | 2019-10-30actual | — | 2022-03-02 |
| A Non-Interventional Interview Study of Phenylketonuria (PKU) AdultsNCT03505125observationalPKU Symptom Survey | — | Completed | — | Phenylketonuria (PKU) | 63 | 2019-07-31actual | — | 2020-01-14 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kgNCT03392974results2021-10-08Change of the Median Factor VIII (FVIII) ActivityOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Biological: Valoctocogene Roxaparvovec | Hemophilia A | 1 | 2019-05-22actual | — | 2023-10-03 |
| Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)NCT00924703results2021-10-12Change From Baseline in the Blood Phenylalanine (Phe) ConcentrationOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: rAvPAL-PEG | Phenylketonuria | 68 | 2019-01-31actual | — | 2021-10-12 |
| Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKUNCT00838435results2020-12-22Full-Scale Intelligence Quotient (FSIQ) ScoreOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: sapropterin dihydrochloride | Phenylketonuria | 95 | 2018-09-14actual | — | 2020-12-22 |
| Retrospective Observational Safety Effectiveness With Kuvan in hpANCT03864029observationalDiagnosis on BH4 deficiency based on medical history - laboratory testing results indicating sub-type (% in each sub-type) • Incidents and severity of AE reported (% of incident / % of each severity) | — | Completed | Drug: KUVAN | Tetrahydrobiopterin Deficiency | 26 | 2018-03-09actual | — | 2019-03-07 |
| A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With AchondroplasiaNCT02055157results2021-01-15Overall Summary of Adverse Events During Initial 6-Month PeriodNon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: BMN 111 | Achondroplasia | 35 | 2017-10-02actual | — | 2021-01-15 |
| A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302NCT02468570results2020-06-04observationalChange From Baseline in Rapid Visual Processing (RVP) Mean Response Latency (CANTAB Task)Statistical Analysis Plan ↗ · Study Protocol ↗ | — | Completed | Other: Administration of CANTAB and Subject Global Assessment | Phenylketonuria | 9 | 2017-02-01actual | — | 2020-06-04 |
| A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.NCT01753804observational6 minute walk distance | — | Terminated | Other: Observational study | Duchenne Muscular Dystrophy | 269 | 2016-10-01actual | — | 2017-12-08 |
| 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 AdministrationNCT01910649Acute phase: Safety dataOpen-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD. | Drug: Drisapersen | Muscular Dystrophies | 12 | Sep 2016actual | — | 2016-11-07 |
| A Prospective, Noninterventional, Observational Study of Late-Onset Pompe DiseaseNCT02221362observationalTo evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease | — | Terminated | — | Late-onset Pompe Patients Untreated or Treated With rhGAA | 30 | Sep 2016actual | — | 2016-10-31 |
| Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)NCT02958202Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosingNon-randomized · Open-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD. | Drug: BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg | Duchenne Muscular Dystrophy | 7 | Sep 2016actual | — | 2018-01-26 |
| BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)NCT01924845results2018-06-14Percent Predicted Maximum Inspiratory Pressure (MIP)Open-label · Treatment | Phase 3 | Terminatedsponsor's stated reason: Sponsor decision | Drug: BMN 701 | Late-onset Pompe Disease | 24 | 2016-09-12actual | — | 2018-06-14 |
| Extension Study for Patients Who Have Participated in a BMN 701 StudyNCT01435772results2018-05-22Number of Participants With a Positive Anti-BMN 701 AntibodyNon-randomized · Open-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. | Biological: BMN 701 | Pompe Disease | 21 | 2016-09-09actual | — | 2018-05-22 |
| Phase IIb Study of PRO045 in Subjects With Duchenne Muscular DystrophyNCT01826474Change from baseline in 6 minute walk testNon-randomized · Open-label · Treatment | Phase 1/2 | Terminated | Drug: PRO045, 0.15 mg/kg/week, PRO045, 1.0 mg/kg/week, PRO045, 3.0 mg/kg/week, PRO045, 6.0 mg/kg/week, PRO045, 9.0 mg/kg/week, PRO045, selected dose | Duchenne Muscular Dystrophy | 15 | 2016-08-31actual | — | 2017-12-08 |
| A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)NCT01957059Change from baseline in 6 minute walk testNon-randomized · Open-label · Treatment | Phase 1/2 | Terminated | Drug: Regimen Selection Phase Group 2, Regimen Selection Phase Group 3, Treatment Phase Group 4, Regimen Selection Phase Group 1 (COMPLETED), Dosing Extension | Duchenne Muscular Dystrophy | 9 | 2016-08-03actual | — | 2017-12-08 |
| BMN 110 Phase 3B in Australian PatientsNCT01966029Safety AnalysisOpen-label · Treatment | Phase 3 | Completed | Drug: BMN 110 | Mucopolysaccharidosis IVA (Morquio A Syndrome) | 13 | Jul 2016actual | — | 2019-09-26 |
| Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)NCT02329769Efficacy of PRO044 (composite of several measures)Non-randomized · Open-label · Treatment | Phase 2 | Terminated | Drug: PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg | Duchenne Muscular Dystrophy | 15 | 2016-07-01actual | — | 2017-12-08 |
| A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)NCT02191917results2023-01-09observationalMaximal Inspiratory Pressure (MIP) | — | Terminatedsponsor's stated reason: Study was terminated early due to a company decision not based on safety concerns | Procedure: Respiratory muscle strength measurements by different techniques. | Late-onset Pompe Disease | 8 | 2016-06-22actual | — | 2023-01-09 |
| Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)NCT01415427results2017-05-19Change From Baseline in 6-minute Walk (6MW) Test - ITTRandomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: BMN 110 - Weekly, BMN 110 - Every Other Week | Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA | 173 | 2016-06-16actual | — | 2021-07-21 |
| A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian SubjectsNCT01803412Incidence and severity of Adverse Events (AEs)Non-randomized · Open-label · Treatment | Phase 3 | Terminated | Drug: Drisapersen, Drisapersen, Drisapersen | Muscular Dystrophies | 53 | 2016-06-01actual | — | 2018-06-14 |
| Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)NCT01515956results2017-07-06To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug InfusionOpen-label · Treatment | Phase 2 | Completed | Drug: BMN 110 | Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA | 15 | Feb 2016actual | — | 2017-08-10 |
| Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKUNCT01889862results2019-07-12Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2Randomized · Triple-masked · Treatment | Phase 3 | Completed | Drug: BMN165 20mg/day, BMN165 40mg/day, Placebo | Phenylketonuria (PKU) | 215 | 2016-01-13actual | — | 2021-05-21 |
| 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) DiseaseNCT01907087results2018-06-11Motor-Language (ML) Scale Score During 300 mg Dosing PeriodOpen-label · Treatment | Phase 1/2 | Completed | Biological: BMN 190 | Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease | 24 | Nov 2015actual | — | 2019-03-08 |
| An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165NCT01819727results2018-09-10Number of Participants With Hypersensitivity Adverse ReactionRandomized · Open-label · Treatment | Phase 3 | Completed | Drug: BMN 165 | Phenylketonuria | 261 | 2015-11-25actual | — | 2019-02-26 |
| Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKUNCT00925054results2019-02-26Blood Phenylalanine ConcentrationsNon-randomized · Open-label · Treatment | Phase 2 | Completed | Drug: rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg, rAvPAL-PEG 0.03 mg/kg, rAvPAL-PEG 0.1 mg/kg | Phenylketonuria | 40 | Jun 2015actual | — | 2019-02-26 |
| Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)NCT01212744results2019-02-26Blood Phenylalanine ConcentrationOpen-label · Treatment | Phase 2 | Completed | Drug: rAvPAL-PEG | Phenylketonuria | 16 | Apr 2015actual | — | 2019-02-26 |
| Safety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeNCT01609062results2016-02-01Safety EvaluationRandomized · Triple-masked · Treatment | Phase 2 | Terminated | Drug: BMN 110, BMN 110 | Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA | 25 | Nov 2014actual | — | 2016-02-01 |
| Sapropterin on Cognitive Abilities in Young Adults With PhenylketonuriaNCT01977820results2016-02-12Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to DiscontinuationRandomized · Double-masked · Treatment | Phase 2 | Terminatedsponsor's stated reason: Recruitment challenges | Drug: Sapropterin, Placebo | Phenylketonuria | 2 | Nov 2014actual | — | 2018-02-22 |
| Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited AmbulationNCT01697319results2016-01-12Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)Open-label · Treatment | Phase 2 | Terminated | Drug: BMN 110 | Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA | 16 | Oct 2014actual | — | 2016-01-12 |
| A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)NCT00787995observationalEndurance | — | Terminated | — | MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome | 353 | Jul 2014actual | — | 2014-12-08 |
| A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)NCT01242111results2015-09-30Safety EvaluationOpen-label · Treatment | Phase 1/2 | Terminated | Drug: BMN 110 | MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome | 20 | Jul 2014actual | — | 2015-09-30 |
| Kuvan® in Phenylketonuria Patients Less Than 4 Years OldNCT01376908results2016-05-12Dietary Phenylalanine (Phe) Tolerance at Week 26Randomized · Open-label · Treatment | Phase 3 | Completed | Drug: Kuvan® · Other: Phenylalanine (Phe)-restricted diet | Phenylketonuria | 56 | Jul 2014actual | — | 2017-09-15 |
| A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 WeeksNCT01560286results2019-02-27Blood Phenylalanine ConcentrationOpen-label · Treatment | Phase 2 | Completed | Biological: BMN 165 (rAvPAL-PEG) | Phenylketonuria | 24 | Sep 2013actual | — | 2019-08-06 |
| Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)NCT01037309results2015-04-09Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein ExtractsNon-randomized · Open-label · Treatment | Phase 1/2 | Completed | Drug: PRO044 SC, PRO044 IV | Duchenne Muscular Dystrophy | 18 | May 2013actual | — | 2018-10-16 |
| Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) PatientsNCT01114737results2016-02-01Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 13Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Sapropterin dihydrochloride, Placebo | Phenylketonuria | 206 | Mar 2013actual | — | 2016-02-01 |
| Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe DiseaseNCT01230801results2018-06-11Number of Participants With Adverse EventsOpen-label · Treatment | Phase 1/2 | Completed | Biological: BMN 701 | Pompe Disease | 22 | 2013-03-06actual | — | 2018-06-11 |
| Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02NCT01387854observationalLinear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03 | — | Completed | — | MPS VI | 59 | Oct 2012actual | — | 2014-04-02 |
| A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)NCT01275066results2014-04-15Change From Baseline in Endurance as Measured by the 6-minute Walk TestRandomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: BMN 110 Weekly, Placebo, BMN 110 Every Other Week | MPS IV A | 177 | Aug 2012actual | — | 2014-07-07 |
| A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult VolunteersNCT01590446Safety based on vitals signsRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: BMN 111, Normal Saline | Achondroplasia | 74 | Jun 2012actual | — | 2012-06-11 |
| A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVANCT00884949results2014-06-30Subject Incidence of Treatment Emergent AEsNon-randomized · Open-label · Treatment | Phase 1/2 | Completed | Drug: BMN 110 | MPS IV A | 20 | Feb 2011actual | — | 2014-06-30 |
| Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006NCT00332189results2012-11-16Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study.Non-randomized · Open-label · Treatment | Phase 3 | Completed | Drug: sapropterin dihydrochloride | Phenylketonuria | 111 | Aug 2009actual | — | 2012-11-16 |
| Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 DeficiencyNCT00355264results2020-09-25Blood Phenylalanine(Phe) Levels Measured at Specified TimepointsOpen-label · Treatment | Phase 2 | Completed | Drug: Phenoptin | Tetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric | 12 | Jun 2009actual | — | 2020-09-25 |
| A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VINCT00299000results2010-10-15Change in HeightRandomized · Open-label · Prevention | Phase 4 | Completed | Drug: Naglazyme | Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome | 4 | Apr 2009actual | — | 2011-07-22 |
| Safety and Tolerability Study of rAvPAL-PEG to Treat PhenylketonuriaNCT00634660• To assess the incidence of treatment-emergent adverse events of a single, subcutaneous (SC) injections of rAvPAL-PEG in subjects with PKU.Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: rAvPAL-PEG | Phenylketonuria | 25 | Apr 2009actual | — | 2017-02-01 |
| A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult SubjectsNCT00789568To determine if a single supratherapeutic dose of sapropterin or a single therapeutic dose of sapropterin has an effect on cardiac repolarization compared with placebo as a change from baseline measured by the subject specific QT correction formula(QTci)Randomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: sapropterin dihydrochloride, Moxifloxacin, Moxifloxacin placebo | Phenylketonuria | 56 | Jan 2009actual | — | 2021-07-23 |
| A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial DiseaseNCT00403494results2021-01-07Change in Peak Walking Time (PWT) From BaselineRandomized · Triple-masked · Treatment | Phase 2 | Completed | Other: Placebo · Drug: Sapropterin Dihydrochloride | Intermittent Claudication | 190 | Nov 2008actual | — | 2021-01-07 |
| A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial DysfunctionNCT00532844results2021-05-17Area Under the Curve (AUC0-12hrs) of Plasma BH4 ConcentrationRandomized · Open-label · Treatment | Phase 2 | Completed | Drug: Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C | Endothelial Dysfunction | 52 | Nov 2008actual | — | 2021-05-17 |
| A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell DiseaseNCT00445978results2021-01-28Number of Subjects With Treatment Emergent Adverse Events (TEAEs)Open-label · Treatment | Phase 2 | Completed | Drug: Sapropterin Dihydrochloride | Sickle Cell Disease | 32 | Aug 2008actual | — | 2021-02-25 |
| A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic HypertensionNCT00325962To compare oral 6R-BH4 to placebo with respect to change from baseline in arterial systolic blood pressure after 8 weeks of treatment in subjects with poorly controlled hypertension.Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: 6R-BH4 (sapropterin dihydrochloride) | Hypertension | 84 | Dec 2006actual | — | 2009-07-21 |
| Study of rhASB in Patients With Mucopolysaccharidosis VINCT00104234results2009-03-0612-Minute Walk TestNon-randomized · Open-label · Treatment | Phase 3 | Completed | Drug: N-acetylgalactosamine 4-sulfatase, Placebo/rhASB | Mucopolysaccharidosis VI | 39 | Oct 2006actual | — | 2010-02-02 |
| Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VINCT00048620Lysosomal storage diseaseRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: N-acetylgalactosamine 4-sulfatase | Mucopolysaccharidosis VI | — | — | — | 2006-11-03 |
| Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VINCT0004871112-minute walk testNon-randomized · Open-label · Treatment | Phase 2 | Completed | Drug: N-acetylgalactosamine 4-sulfatase | Mucopolysaccharidosis VI | — | — | — | 2009-08-25 |
| Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VINCT00067470results2009-08-17Change From Baseline in 12-minute Walk Test at 24 WeeksRandomized · Double-masked · Treatment | Phase 3 | Completed | Drug: Placebo, N-acetylgalactosamine 4-sulfatase | Mucopolysaccharidosis VI | 39 | — | — | 2009-12-24 |
| Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsNCT00104247results2009-04-16Change in Blood Phenylalanine Levels From Baseline to Week 6.Randomized · Double-masked · Treatment | Phase 3 | Completed | Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | Phenylketonurias | 89 | — | — | 2014-07-23 |
| Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With PhenylketonuriaNCT00104260Evaluate the degree and frequency of response to Phenoptin™, as demonstrated by a reduction in blood Phe level among subjects with PKU who have elevated Phe levelsNon-randomized · Open-label · Treatment | Phase 2 | Completed | Drug: sapropterin dihydrochloride | Phenylketonurias | 700 | — | — | 2007-04-09 |
| A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine LevelsNCT00225615Primary objective:Non-randomized · Open-label · Treatment | Phase 3 | Completed | Drug: sapropterin dihydrochloride | Phenylketonurias | 100 | — | — | 2009-08-20 |
| Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted DietNCT00272792results2009-07-09Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With PhenylketonuriaRandomized · Double-masked · Treatment | Phase 3 | Completed | Drug: Sapropterin Dihydrochloride, Placebo | Phenylketonurias | 45 | — | — | 2015-08-13 |
| Sapropterin Expanded Access ProgramNCT00484991expanded access | — | Approved for marketing | Drug: Sapropterin dihydrochloride | Phenylketonuria | — | — | — | 2008-04-15 |
| BMN 110 US Expanded Access ProgramNCT01858103expanded access | — | Approved for marketing | Drug: BMN 110 | Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA | — | — | — | 2014-04-02 |
| Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical ConditionsNCT02365376observationalcontinuous collection of donated study subject samples | — | Unknown status | — | Rare Genetic Disorders | — | — | — | 2015-02-18 |
| Extension Study of Drisapersen in DMD SubjectsNCT02636686expanded access | — | No longer available | Drug: Drisapersen | Duchenne Muscular Dystrophy | — | — | — | 2018-01-24 |
| A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 DiseaseNCT02963350expanded access | — | Approved for marketing | Drug: BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1) | CLN2 Disease | — | — | — | 2017-07-24 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19