8 active not recruiting, 4 completed, 3 recruiting, 1 enrolling by invitation. 12 Phase 2, 5 Phase 3, 2 Phase 1.
Follow vosoritide: new trials by email Literature Review of vosoritide trials Research Report on vosoritide
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 |
| 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 |
| 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 |
| 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 |
| Vosoritide for Short Stature in Turner Syndrome Vosoritide - Turner Syndrome, Short Stature | Phase 2 | Unknown | Roopa Kanakatti Shankar, MBBS, MS | 20 | 2024-04-12 | 2024-06-24 |
| Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI Vosoritide Injection [Voxzogo] - MPS IVA, MPS VI | Phase 1 / Phase 2 | Active not recruiting | University of California, San Francisco | 6 | 2023-09-25 | 2026-03-10 |
| 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 |
| Vosoritide for Selected Genetic Causes of Short Stature Vosoritide - Short Stature | Phase 2 | Active not recruiting | Andrew Dauber | 56 | 2020-08-04 | 2026-03-18 |
| 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 |
| 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 |
| 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 Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia BMN 111 - Achondroplasia | Phase 2 | Completed | BioMarin Pharmaceutical | 35 | 2014-01-13 | 2021-01-15 |
| A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers BMN 111, Normal Saline - Achondroplasia | Phase 1 | Completed | BioMarin Pharmaceutical | 74 | 2012-02-01 | 2012-06-11 |
Dwarfism (11) · Bone Diseases (10) · Achondroplasia (9) · Musculoskeletal Diseases (8) · Bone Diseases, Developmental (6) · Genetic Diseases, Inborn (5) · Osteochondrodysplasias (5) · Hypochondroplasia (4) · Noonan Syndrome (2) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1) · Turner Syndrome (1)
BioMarin Pharmaceutical (14) · Andrew Dauber (1) · Roopa Kanakatti Shankar, MBBS, MS (1) · University of California, San Francisco (1)
United States (16) · Australia (13) · United Kingdom (11) · Japan (8) · France (7) · Germany (7) · Italy (6) · Spain (5) · Canada (4) · South Korea (2) · Turkey (Türkiye) (2) · Poland (1) · Romania (1)
Andrew Dauber, MS MMSc (1) · Ellen B Fung, PhD (1) · Roopa Kanakatti Shankar, MBBS, MS (1)
PMID 38649657, PMID 31269546, PMID 34431071, PMID 39305160, PMID 39740666, PMID 33761804, PMID 37984383, PMID 38813446, PMID 41783511, PMID 42713641
2015 (2), 2016 (1), 2017 (1)