27 completed, 12 terminated, 7 active not recruiting, 3 recruiting. 52 Phase 2, 15 Phase 1, 3 Phase 3.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy BMN 351 - Duchenne Muscular Dystrophy (DMD) | Phase 2 | Enrolling by invitation | BioMarin Pharmaceutical | 18 | 2026-04-20 | 2026-07-24 |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia BMN 333, Vosoritide Injection [Voxzogo] - Achondroplasia | Phase 2 / Phase 3 | Recruiting | BioMarin Pharmaceutical | 160 | 2026-04-20 | 2026-10-22 |
| A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months Vosoritide, Placebo - Hypochondroplasia | Phase 2 | Recruiting | BioMarin Pharmaceutical | 60 | 2025-07-30 | 2026-03-31 |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment Vosoritide Injection - Noonan Syndrome | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 30 | 2024-11-22 | 2026-09-23 |
| A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature Vosoritide Injection, Human Growth Hormone, Placebo - Idiopathic Short Stature | Phase 2 | Recruiting | BioMarin Pharmaceutical | 100 | 2024-10-21 | 2026-05-22 |
| A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy BMN 351 - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 18 | 2024-01-03 | 2026-06-25 |
| A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331 - Hereditary Angioedema, HAE | Phase 1 / Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 44 | 2022-02-15 | 2024-05-16 |
| Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors Valoctocogene roxaparvovec - Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 10 | 2020-12-10 | 2026-07-10 |
| A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia vosoritide - Achondroplasia | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 20 | 2020-10-10 | 2026-03-13 |
| AAV Gene Therapy Study for Subjects with PKU BMN 307 - Phenylketonuria (PKU) | Phase 1 / Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 100 | 2020-09-24 | 2024-12-12 |
| An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. - Achondroplasia | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 73 | 2019-06-12 | 2026-03-13 |
| A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia BMN 111, Placebo - Achondroplasia | Phase 2 | Completed | BioMarin Pharmaceutical | 75 | 2018-06-13 | 2024-06-13 |
| Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 Valoctocogene Roxaparvovec - Hemophilia A, Gene Therapy | Phase 1 / Phase 2 | Terminated | BioMarin Pharmaceutical | 3 | 2018-04-24 | 2025-08-22 |
| Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg - Duchenne Muscular Dystrophy | Phase 2 | Terminated | BioMarin Pharmaceutical | 7 | 2016-04-01 | 2018-01-26 |
| A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) BMN 111 - Achondroplasia | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 30 | 2016-01-26 | 2026-03-13 |
| A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device - Jansky-Bielschowsky Disease, Batten Disease | Phase 2 | Completed | BioMarin Pharmaceutical | 14 | 2016-01-22 | 2025-02-17 |
| Gene Therapy Study in Severe Haemophilia A Patients (270-201) valoctocogene roxaparvovec - Severe Haemophilia A | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 15 | 2015-09-28 | 2025-04-10 |
| Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors Talazoparib Tosylate - Advanced Breast Cancer, HER2/Neu Negative | Phase 2 | Completed | Melinda Telli | 21 | 2015-08-01 | 2023-02-21 |
| An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease BMN 190, Intracerebroventricular (ICV) access device - Jansky-Bielschowsky Disease, Batten Disease | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 23 | 2015-02-01 | 2022-08-24 |
| Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg - Duchenne Muscular Dystrophy | Phase 2 | Terminated | BioMarin Pharmaceutical | 15 | 2014-12-01 | 2017-12-08 |
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Phenylketonurias (10) · Bone Diseases (7) · Muscular Dystrophy, Duchenne (7) · Achondroplasia (6) · Dwarfism (6) · Genetic Diseases, Inborn (5) · Lysosomal Storage Diseases (5) · Mucopolysaccharidosis IV (5) · Musculoskeletal Diseases (5) · Blood Coagulation Disorders (3) · Blood Coagulation Disorders, Inherited (3) · Bone Diseases, Developmental (3) · Coagulation Protein Disorders (3) · Hematologic Diseases (3) · Hemophilia A (3)
sapropterin (13) · vosoritide (9) · GALNS protein, human (5) · pegvaliase (4) · Chondroitinases and Chondroitin Lyases (3) · Enzyme Replacement Therapy (3) · Galns protein, mouse (3) · Sulfatases (3) · Valoctocogene Roxaparvovec (3) · cerliponase alfa (3) · Ascorbic Acid (2) · phenoptin (2) · Human Growth Hormone (1) · N-Acetylgalactosamine-4-Sulfatase (1) · PRO051 (1)
United States (37) · United Kingdom (26) · Italy (16) · Australia (12) · Germany (11) · France (9) · Netherlands (7) · Belgium (5) · Japan (4) · South Korea (4) · Spain (4) · Canada (3) · Sweden (3) · Taiwan (3) · Turkey (Türkiye) (3)
Ari Gershman, MD (2) · Celeste Decker, MD (2) · Debra Lounsbury (2) · Don Nwose, MD (2) · A. Ferlini, PhD (1) · Adam Shaywitz, MD PhD (1) · Celeste Decker, M.D. (1) · David Jacoby (1) · Glen R Elliott, Ph.D., M.D. (1) · Glen R Elliott, PhD, MD (1) · J. J. Verschuuren, MD (1)
PMID 32593547, PMID 38649657, PMID 29526614, PMID 29688815, PMID 31269546, PMID 33980287, PMID 38101904, PMID 38776275, PMID 20837663, PMID 21428760, PMID 22315282, PMID 23782126, PMID 24743000, PMID 26331768, PMID 27588424, PMID 29159458, PMID 29224506, PMID 29973227, PMID 31893514, PMID 32915950, PMID 33761804, PMID 34378280, PMID 34431071, PMID 35411075, PMID 35879931, PMID 36253484, PMID 37984383, PMID 38975624, PMID 40986187, PMID 41783511
2015 (2), 2014 (1), 2016 (1), 2017 (1), 2019 (1), 2023 (1)