NCT03197766
Achondroplasia
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
5 Years to 18 Years
No
BMN 111, Placebo
Phase 3
Interventional
121
2016-12-12
2022-03-02
Oakland, California, United States
Torrance, California, United States
Wilmington, Delaware, United States
Decatur, Georgia, United States
Chicago, Illinois, United States
Baltimore, Maryland, United States
Columbia, Missouri, United States
Cincinnati, Ohio, United States
Houston, Texas, United States
Seattle, Washington, United States
Milwaukee, Wisconsin, United States
Westmead, New South Wales, Australia
Parkville, Victoria, Australia
Magdeburg, , Germany
Münster, , Germany
Osaka, , Japan
Saitama, , Japan
Tokushima, , Japan
Barcelona, , Spain
Barcelona, , Spain
Málaga, , Spain
Istanbul, , Turkey
London, , United Kingdom
Sheffield, , United Kingdom
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
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