Achondroplasia
Conditions
Interventions
Once daily subcutaneous administration of Voxzogo 15 mcg/kg was continued until the final height (defined as epiphyseal closure confirmed by imaging and AGV < 1.5 cm/year) was reached, or for 5 years
Sponsors
BioMarin Pharmaceutical Inc.(ICCC:EPS Corporation)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Must have completed Study 111-301 2. Female >= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study 3. If sexually active, willing to use a highly effective method of contraception while participating in the study 4. Are willing and able to perform all study procedures as physically possible
Exclusion criteria
Exclusion criteria: 1. Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study 2. Have a clinically significant finding or arrhythmia on Baseline ECG that indicates abnormal cardiac function 3. Evidence of decreased growth velocity (<1.5 cm/year) or of growth plate closure through bilateral lower extremity X-rays 4. Require any investigational agent prior to completion of study period 5. Current therapy with medications known to alter renal function 6. Pregnant or breastfeeding or plan to become pregnant during study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluate the long-term safety, tolerability, and efficacy for growth in children with ACH treated with BMN 111 | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Evaluate the pharmacokinetics (PK) of BMN 111 2. Evaluate immunogenicity of BMN 111 and assess impact on safety, PK, and efficacy measures 3. Evaluate body proportion ratios of the extremities 4. Evaluate effect of BMN 111 on bone morphology/quality by X-ray and dual X-ray absorptiometry (DXA) 5. Evaluate changes in health-related quality of life as measured by the Quality of Life in Short Stature Youth (QoLISSY) and the PedsQL questionnaires 6. Evaluate changes in functional independence as measured by the Wee-FIM clinician-reported outcome 7. Evaluate change from baseline in bone metabolism biomarkers | — |
Countries
Europe, Japan, North America, Oceania
Contacts
Public ContactEPS Corporation jRCT Inquiry Center
EPS Corporation
Outcome results
None listed