BioMarin Pharmaceutical: Achondroplasia trials

The registry lists 14 Achondroplasia studies with BioMarin Pharmaceutical as lead sponsor. 2 are open to participants now and 3 have results posted.

Phase 1 2Phase 2 6Phase 3 3No phase 4(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.

• Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaRecruitingNCT07441876Phase 2/3
• VIrtual STudy in Achondroplasia for the US (VISTA)RecruitingNCT06168201
• 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
• Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy ParticipantsTerminatedNCT05813314Phase 1
• 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
• Lifetime Impact Study for AchondroplasiaCompletedNCT03872531
• Lifetime Impact of Achondroplasia Study in Europe-LIAISECompletedNCT03449368
• 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
• A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult VolunteersCompletedNCT01590446Phase 1