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Voxzogo 0.56 mg powder and solvent for solution for injection

DRUG8 trials

Sponsors

Biomarin Pharmaceutical Inc.

Conditions

AchondroplasiaHypochondroplasiaIdiopathic Short Stature (ISS)Short Stature Homeobox-Containing Gene DeficiencyTurner Syndromeand Noonan Syndrome

Phase 2

A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia
CompletedCTIS2023-508754-26-00
Biomarin Pharmaceutical Inc.Achondroplasia
Start: 2017-02-10End: 2024-12-19Target: 1Updated: 2024-09-30
A Phase 2, Randomized, Multicenter, Basket Study of Vosoritide in Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome with Inadequate Growth During or After Human Growth Hormone Treatment
RecruitingCTIS2024-515861-33-00
Biomarin Pharmaceutical Inc.and Noonan Syndrome, Short Stature Homeobox-Containing Gene Deficiency, Turner Syndrome
Start: 2025-03-25Target: 40Updated: 2026-01-21
A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
RecruitingCTIS2024-520137-74-00
Biomarin Pharmaceutical Inc.Idiopathic Short Stature (ISS)
Start: 2025-10-10Target: 31Updated: 2025-09-16
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children with Hypochondroplasia, Aged 0 to < 36 Months
RecruitingCTIS2024-512261-14-00
Biomarin Pharmaceutical Inc.Hypochondroplasia
Start: 2025-12-22Target: 30Updated: 2026-01-19
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 versus Vosoritide in Children with Achondroplasia
Not yet recruitingCTIS2025-523811-12-00
Biomarin Pharmaceutical Inc.Achondroplasia
Target: 25Updated: 2026-05-05

Phase 3