A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

About this trial

Last updated 4 years ago

Study ID

111-301

Status

Completed

Type

Interventional

Phase

Phase 3

Placebo

Yes

Accepting

5 to 18 Years

All Sexes

Trial Timing

Ended 7 years ago

What is this trial about?

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.

What are the participation requirements?

Inclusion Criteria

* Parent(s) or guardian(s) consent

* 5 to < 18 years old

* ACH, documented and confirmed by genetic testing

* At least a 6-month period of pretreatment growth assessment in Study 111-901 before study entry

* If sexually active, willing to use a highly effective method of contraception

* Ambulatory and able to stand without assistance

Exclusion Criteria

* Hypochondroplasia or short stature condition other than ACH

* Have any of the following:

* Hypothyroidism or hyperthyroidism

* Insulin-requiring diabetes mellitus

* Autoimmune inflammatory disease

* Inflammatory bowel disease

* Autonomic neuropathy

* History of any of the following:

* Renal insufficiency defined as serum creatinine > 2 mg/dL

* Chronic anemia

* Baseline systolic blood pressure (BP) < 70 millimeters of mercury (mm Hg) or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms ie, dizziness, fainting) or recurrent symptomatic orthostatic hypotension

* Cardiac or vascular disease

* Have a clinically significant finding or arrhythmia on screening electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or Fridericias corrected QTc-F > 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