98 completed, 28 terminated, 14 active not recruiting, 12 recruiting. 52 Phase 2, 31 Phase 3, 29 Phase 1, 7 Not applicable, 7 Phase 4.
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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 |
|---|---|---|---|---|---|---|
| Immune Modulation During Palynziq® Treatment in Adults (IMPALA) Pegvaliase, Methotrexate - Phenylketonuria | Phase 4 | Recruiting | BioMarin Pharmaceutical | 12 | 2026-05-29 | 2026-06-17 |
| 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 |
| 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 |
| Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants BMN 349, Placebo - Alpha 1-Antitrypsin Deficiency | Phase 1 | Active not recruiting | BioMarin Pharmaceutical | 6 | 2025-02-21 | 2026-05-06 |
| Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq RDD to Palynziq - Phenylketonuria, PKU | Phase 4 | Completed | BioMarin Pharmaceutical | 9 | 2025-02-07 | 2026-10-01 |
| 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 Study to Assess Growth in Children With Idiopathic Short Stature Idiopathic Short Stature | Recruiting | BioMarin Pharmaceutical | 300 | 2024-08-08 | 2026-06-23 | |
| 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 |
| SEAPORT 1: Study of INZ-701 in Adults With Hemodialysis-Dependent End-Stage Kidney Disease INZ-701 - Calciphylaxis | Phase 1 | Completed | Inozyme Pharma | 11 | 2024-02-12 | 2026-08-24 |
| A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) Pegvaliase - Phenylketonuria (PKU) | Recruiting | BioMarin Pharmaceutical | 200 | 2024-01-20 | 2026-03-24 | |
| 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 |
| 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 |
| A Multicenter Multinational Observational Study of Children With Hypochondroplasia Hypochondroplasia | Recruiting | BioMarin Pharmaceutical | 400 | 2023-11-27 | 2025-04-03 | |
| 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 |
| A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria BMN 255, Placebo - Hyperoxaluria, Nonalcoholic Fatty Liver Disease | Phase 1 | Withdrawn | BioMarin Pharmaceutical | 2023-09-26 | 2024-06-11 | |
| The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 1 | Terminated | Inozyme Pharma | 9 | 2023-06-25 | 2026-10-15 |
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Phenylketonurias (56) · Mucopolysaccharidosis IV (21) · Achondroplasia (15) · Genetic Diseases, Inborn (13) · Hemophilia A (12) · Mucopolysaccharidosis VI (11) · Bone Diseases (10) · Dwarfism (10) · Lysosomal Storage Diseases (10) · Muscular Dystrophy, Duchenne (10) · Blood Coagulation Disorders (8) · Blood Coagulation Disorders, Inherited (8) · Coagulation Protein Disorders (8) · Hematologic Diseases (8) · Hemorrhagic Disorders (8)
sapropterin (38) · pegvaliase (16) · vosoritide (14) · GALNS protein, human (10) · Valoctocogene Roxaparvovec (7) · phenoptin (5) · Chondroitinases and Chondroitin Lyases (4) · Enzyme Replacement Therapy (4) · Galns protein, mouse (4) · N-Acetylgalactosamine-4-Sulfatase (4) · cerliponase alfa (4) · talazoparib (4) · PRO051 (3) · Sulfatases (3) · Ascorbic Acid (2)
United States (134) · United Kingdom (54) · Italy (38) · Germany (35) · Australia (29) · France (29) · Spain (20) · Canada (19) · Netherlands (17) · Japan (14) · Turkey (Türkiye) (14) · Belgium (13) · Brazil (13) · Taiwan (11) · South Korea (10)
Celeste Decker, MD (5) · Debra Lounsbury (5) · Don Nwose, MD (3) · Ari Gershman, MD (2) · Desiree White, Ph.D. (2) · Dorothy K. Grange, M.D. (2) · Ece Kucuksayrac, MD (2) · Heather W Saavedra, MS (2) · Nadia Ali, Ph.D. (2) · A. Ferlini, PhD (1)
PMID 38649657, PMID 31269546, PMID 32593547, PMID 34431071, PMID 39740666, PMID 40411344, PMID 29526614, PMID 29688815, PMID 32915950, PMID 33874971, PMID 33980287, PMID 34826353, PMID 35879931, PMID 38101904, PMID 38776275, PMID 39305160, PMID 40986187, PMID 11310367, PMID 17693179, PMID 19261295, PMID 20837663, PMID 20924685, PMID 21428760, PMID 21646032, PMID 21877895, PMID 22315282, PMID 23006929, PMID 23462988, PMID 23608077, PMID 23782126, PMID 23928118, PMID 24043380, PMID 24108527, PMID 24743000, PMID 24764221, PMID 25178307, PMID 25214650, PMID 25487082, PMID 26331768, PMID 27134829, PMID 27339555, PMID 27513937, PMID 27588424, PMID 27955919, PMID 28274234, PMID 28465125, PMID 28535791, PMID 29159458, PMID 29224506, PMID 29248359
2017 (5), 2015 (3), 2009 (2), 2014 (2), 2019 (2), 2016 (1), 2023 (1), R01HD073292 (1), U54NS065768 (1)