A multicentre, non-interventional study to evaluate long-term safety in patients with achondroplasia treated with Voxzogo® (vosoritide) (BMN 111-603)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  Voxzogo
• Medical condition to be studied:
  Osteochondrodysplasia

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  330

Study design details

• Main study objective:
  To evaluate the long-term impact of treatment with Voxzogo on adverse bone-related safety events in subjects with ACH in a real-world setting.
• Outcomes:
  Incidence of new bone-related safety events of interest.
• Data analysis plan:
  The primary endpoint will include the exposure-adjusted incidence rate (EAIR, derived as the number of new events divided by the total exposure person-time at risk) of all new bone-related safety events of interest which have not previously been observed in the subject, which emerge during the course of the study. When calculating the exposure and person-time follow-up of events of interest, the exposure time period for each subject begins with the start of treatment index date and continues until the occurrence of the protocol-defined safety event, permanent discontinuation of treatment, death, or loss to follow-up. The induction period for risk with exposure is assumed to be immediate with exposure.