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

Completed · Phase 3 · Has a placebo group

Conditions studied: Achondroplasia

In brief

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.

Key facts

Study ID

NCT03197766

Run by

BioMarin Pharmaceutical

People needed

121

Starts

2016-12-12

Expected to finish

2019-10-30

Last updated by the study team

2022-03-02

Who can join

Age: 5 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Children's Hospital & Research Center Oakland — Oakland, California, United States
• Harbor - UCLA Medical Center — Torrance, California, United States
• Alfred I. duPont Hospital for Children — Wilmington, Delaware, United States
• Emory University — Decatur, Georgia, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• University of Missouri — Columbia, Missouri, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Baylor College of Medicine — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Medical College of Wisconsin, Children's Hospital — Milwaukee, Wisconsin, United States
• The Children's Hospital at Westmead — Westmead, New South Wales, Australia
• Murdoch Children's Research Institute — Parkville, Victoria, Australia
• Otto-von-Guericke Universitaet, Universitaetskinderklinik — Magdeburg, Germany
• Universitätsklinikum Münster — Münster, Germany
• Osaka University Hospital — Osaka, Japan
• Saitama Children's Medical Center — Saitama, Japan
• Tokushima University Hospital — Tokushima, Japan
• Institut Catala de Traumatologica I Medicina de l'Esport — Barcelona, Spain
• Hospital Sant Joan de Deu — Barcelona, Spain
• Hospital Universitario Virgen de la Victoria — Málaga, Spain
• Acibadem University School of Medicine — Istanbul, Turkey (Türkiye)
• Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital — London, United Kingdom
• Sheffield Children's NHS Foundation Trust — Sheffield, United Kingdom

Full record on ClinicalTrials.gov

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