Phase 3
N=121
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Achondroplasia
Bottom Line
View on ClinicalTrials.gov: NCT03197766 ↗Enrolled (actual)
121
Serious AEs
5.8%
Results posted
Mar 2022
Primary outcome: Primary: Change From Baseline in Annualized Growth Velocity (AGV) at Week 52 — 1.71; 0.13 cm/year — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- BMN 111 (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 5+ yrs
- Sex
- All
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- Oct 2019
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 | — |
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.
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). 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.