20 completed, 4 active not recruiting, 4 terminated, 1 enrolling by invitation. 31 Phase 3, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 31 | Completed 20, Active not recruiting 4, Terminated 4, Enrolling by invitation 1, Recruiting 1, Withdrawn 1 |
| Phase 2 | 3 | Completed 1, Recruiting 1, Withdrawn 1 |
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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 |
|---|---|---|---|---|---|---|
| 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 |
| Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia Vosoritide - Hypochondroplasia | Phase 3 | Enrolling by invitation | BioMarin Pharmaceutical | 140 | 2025-06-20 | 2026-09-09 |
| ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 3 | Terminated | Inozyme Pharma | 2 | 2025-03-26 | 2026-09-29 |
| Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Vosoritide, Placebo - Hypochondroplasia | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 80 | 2024-06-17 | 2026-01-20 |
| Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A Valoctocogene roxaparvovec - Hemophilia A | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 6 | 2023-12-25 | 2026-03-19 |
| The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency INZ-701, Control Arm (Conventional Therapy) - Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 3 | Terminated | Inozyme Pharma | 27 | 2023-11-05 | 2026-10-01 |
| Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria Pegvaliase, Diet Only - Phenylketonuria (PKU) | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 55 | 2022-06-17 | 2026-07-21 |
| Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A valoctocogene roxaparvovec - Hemophilia A | Phase 3 | Completed | BioMarin Pharmaceutical | 22 | 2020-12-08 | 2025-10-08 |
| Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU Pegvaliase - Phenylketonuria (PKU) | Phase 3 | Completed | BioMarin Pharmaceutical | 37 | 2018-09-13 | 2022-02-17 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg Valoctocogene Roxaparvovec - Hemophilia A | Phase 3 | Completed | BioMarin Pharmaceutical | 1 | 2018-03-14 | 2023-10-03 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) valoctocogene roxaparvovec - Hemophilia A | Phase 3 | Completed | BioMarin Pharmaceutical | 144 | 2017-12-19 | 2025-03-25 |
| An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia BMN 111 - Achondroplasia | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 119 | 2017-12-12 | 2026-03-13 |
| A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia BMN 111, Placebo - Achondroplasia | Phase 3 | Completed | BioMarin Pharmaceutical | 121 | 2016-12-12 | 2022-03-02 |
| BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) BMN 701 - Late-onset Pompe Disease | Phase 3 | Terminated | BioMarin Pharmaceutical | 24 | 2014-04-01 | 2018-06-14 |
| Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU BMN165 20mg/day, BMN165 40mg/day, Placebo - Phenylketonuria (PKU) | Phase 3 | Completed | BioMarin Pharmaceutical | 215 | 2013-07-29 | 2021-05-21 |
| BMN 110 Phase 3B in Australian Patients BMN 110 - Mucopolysaccharidosis IVA (Morquio A Syndrome) | Phase 3 | Completed | BioMarin Pharmaceutical | 13 | 2013-07-01 | 2019-09-26 |
| A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects Drisapersen - Muscular Dystrophies | Phase 3 | Terminated | BioMarin Pharmaceutical | 53 | 2013-05-01 | 2018-06-14 |
| An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 BMN 165 - Phenylketonuria | Phase 3 | Completed | BioMarin Pharmaceutical | 261 | 2013-05-01 | 2019-02-26 |
| Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - Weekly, BMN 110 - Every Other Week - Mucopolysaccharidosis IV A, Morquio A Syndrome | Phase 3 | Completed | BioMarin Pharmaceutical | 173 | 2011-07-01 | 2021-07-21 |
| Kuvan® in Phenylketonuria Patients Less Than 4 Years Old Kuvan®, Phenylalanine (Phe)-restricted diet - Phenylketonuria | Phase 3 | Completed | BioMarin Pharmaceutical | 56 | 2011-06-01 | 2017-09-15 |
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Phenylketonurias (11) · Genetic Diseases, Inborn (8) · Blood Coagulation Disorders (4) · Blood Coagulation Disorders, Inherited (4) · Bone Diseases (4) · Coagulation Protein Disorders (4) · Hematologic Diseases (4) · Hemophilia A (4) · Hemorrhagic Disorders (4) · Hemostatic Disorders (4) · Musculoskeletal Diseases (4) · Osteochondrodysplasias (4) · Achondroplasia (3) · Bone Diseases, Developmental (3) · Dwarfism (3)
sapropterin (9) · vosoritide (5) · Valoctocogene Roxaparvovec (4) · pegvaliase (4) · Chondroitinsulfatases (2) · GALNS protein, human (2) · N-Acetylgalactosamine-4-Sulfatase (2) · phenoptin (2) · Chondroitinases and Chondroitin Lyases (1) · Dextrans (1) · Enzyme Replacement Therapy (1) · Galns protein, mouse (1) · PRO051 (1) · Phenylalanine (1)
United States (27) · United Kingdom (12) · Germany (10) · Australia (9) · Canada (9) · Italy (9) · France (8) · Japan (8) · Spain (7) · Turkey (Türkiye) (6) · Brazil (5) · Netherlands (4) · Saudi Arabia (4) · South Korea (4) · Taiwan (4)
Debra Lounsbury (2) · Alex Dorenbaum, MD (1) · Eric He, MD (1) · Glen R Elliott, PhD, MD (1) · Ignacio Alvarez, MD (1) · Joshua Lilienstein, M.D. (1) · Markus Merilainen, MD (1) · Suyash Prasad, MD (1)
PMID 40411344, PMID 33874971, PMID 34826353, PMID 38649657, PMID 39305160, PMID 39740666, PMID 17693179, PMID 19261295, PMID 21646032, PMID 25487082, PMID 27955919, PMID 28274234, PMID 28535791, PMID 29248359, PMID 32915950, PMID 34344399, PMID 34431071, PMID 35294811, PMID 35879931, PMID 36812433, PMID 37263523, PMID 38616241, PMID 39024543, PMID 40986187, PMID 42713641
2017 (3), 2009 (1), 2015 (1)