BioMarin Pharmaceutical: Dwarfism trials

The registry lists 10 Dwarfism studies with BioMarin Pharmaceutical as lead sponsor. 0 are open to participants now and 3 have results posted.

Phase 2 6Phase 3 3No phase 1(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With AchondroplasiaCompletedNCT03583697Phase 2Results posted
• A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaCompletedNCT03197766Phase 3Results posted
• A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With AchondroplasiaCompletedNCT02055157Phase 2Results posted
• A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentTerminatedNCT06668805Phase 2
• Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaCompletedNCT06455059Phase 3
• A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With AchondroplasiaActive, not recruitingNCT04554940Phase 2
• An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03989947Phase 2
• An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03424018Phase 3
• A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)Active, not recruitingNCT02724228Phase 2
• A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With AchondroplasiaCompletedNCT01603095