Phase 3
Completed N=121
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Source: ClinicalTrials.gov NCT03197766 ↗Enrolled (actual)
121
Serious AEs
5.8%
Results posted
Mar 2022
Primary outcomePrimary: Change From Baseline in Annualized Growth Velocity (AGV) at Week 52 — 1.71; 0.13 cm/year — p=< 0.0001
◆ Published Evidence
Established
▲ Trending
21citations · ~11 / year
Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.
Summary
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
Linked Publications (4)
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Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.
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Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study.
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Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia.
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Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Annualized Growth Velocity (AGV) at Week 52 |
1.71; 0.13 | < 0.0001 sig |
| SECONDARY Change From Baseline in Height Z-score at Week 52 |
0.27; -0.01 | < 0.0001 sig |
| SECONDARY Change From Baseline in Upper to Lower Segment Body Ratio at Week 52 |
-0.03; -0.02 | = 0.506 |
| SECONDARY Summary of Subjects Experiencing Adverse Events (AEs) During Treatment |
59; 60; 3; 4; 53; 51 | — |
Eligibility Criteria
Inclusion Criteria
- Parent(s) or guardian(s) consent
- 5 to 2 mg/dL
- Chronic anemia
- Baseline systolic blood pressure (BP) 450 msec
- Have an unstable condition likely to require surgical intervention during the study (including progressive cervical medullary compression or severe untreated sleep apnea)
- Decreased growth velocity (< 1.5 cm/yr) over a period of 6 months or evidence of growth plate closure (proximal tibia, distal femur)
- Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or treatment greater than 6 months at any time
- Greater than 1 month treatment with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the previous 12 months
- Planned or expected to have limb-lengthening surgery during the study period. Subjects with previous limb- lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae.
- Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex, excluding tooth extraction), during the study period. Subjects with previous bone-related surgery may enroll if surgery occurred at least 6 months prior to the study and healing is complete without sequelae.
- Had a fracture of the long bones or spine within 6 months prior to screening
- History of severe untreated sleep apnea
- New initiation of sleep apnea treatment (e.g. CPAP or sleep apnea-mitigating surgery) in the previous 2 months prior to screening
- History of hip surgery or hip dysplasia atypical for achondroplastic subjects
- History of clinically significant hip injury in the 30 days prior to screening
- History of slipped capital femoral epiphysis or avascular necrosis of the femoral head
- Abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant
- Concurrent disease or condition that would interfere with study participation or safety evaluations, for any reason
- Condition or circumstance that places the subject at high risk for poor treatment compliance or for not completing the study
Data sourced from ClinicalTrials.gov (NCT03197766) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.