An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
- Conditions
- Achondroplasia
- Registration Number
- JPRN-jRCT2080224830
- Lead Sponsor
- BioMarin Pharmaceutical Inc.(ICCC:EPS Corporation)
- Brief Summary
The change from baseline in AGV after the start of treatment with Voxzogo was similar in both the Voxzogo/Voxzogo group and the placebo/Voxzogo group, and improvements from baseline in AGV were consistently seen in patients receiving Voxzogo. Of 119 patients in the safety analysis set, 90 (75.6%) experienced AEs. ADRs occurred in 57 patients (47.9%). No serious or >= Grade 3 ADRs occurred.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 119
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
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method