A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Sponsor
BioMarin Pharmaceutical
Study ID
NCT04554940
Phase
PHASE2
Status
Active Not Recruiting

Conditions

  • Achondroplasia

Eligibility Criteria

Sex
ALL
Age
0 Months - 12 Months
Healthy Volunteers
Not accepted

Interventions

  • vosoritide — BIOLOGICAL
    Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Study Details

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery

Key Dates

Start date
Oct 10, 2020
Status verified
Mar 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
20 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Vosoritide + Standard of Care
    Standard of Care treatment for cervicomedullary compression and once daily subcutaneous injection of recommended dose of vosoritide based on weight-band dosing.
  • No Intervention: Standard of Care Alone
    Institutional standard of care monitoring and treatment for cervicomedullary compression

Primary Outcome Measure

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Through Week 260 ]

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