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for people ages 5–14 (full criteria)
at Oakland, California and other locations
study started
estimated completion:



This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.

Official Title

A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia


Achondroplasia Dwarfism Bone Diseases, Developmental Bone Diseases Natriuretic Peptide, C-Type


You can join if…

Open to people ages 5–14

  • Parent(s) or guardian(s) are willing and able to provide written, signed informed consent
  • 5 to 14 years old at end of study
  • ACH, documented by clinical grounds, confirmed by genetic testing
  • At least 6-month of pretreatment growth assessment in Study 111-901 before study entry, and one standing height at least 6 months prior to screening for 111-202
  • Negative pregnancy test at the Screening Visit for females ≥ 10 years old or who have begun menses
  • If sexually active, willing to use a highly effective method of contraception while participating in the study
  • Ambulatory, able to stand without assistance
  • Willing and able to perform all study procedures as physically possible
  • Parents/caregivers willing to administer daily injections to the subjects

Additional inclusion Criteria Optional, Open-label Extension Phase:

  • Appropriate written informed consent

You CAN'T join 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
  • Recent acute illness associated with volume dehydration not completely resolved prior to the first dose of study drug
  • Unstable condition requiring surgical intervention during the study
  • Growth plates have fused
  • Have a history of any of the following:
  • Renal insufficiency, defined as creatinine > 2 mg/dl
  • Anemia
  • Baseline systolic BP < 75 mm Hg or recurrent symptomatic hypotension or recurrent symptomatic hypotension, recurrent symptomatic orthostatic hypotension
  • Cardiac or vascular disease, including the following:
  • Cardiac dysfunction (abnormal echocardiogram [ECHO] including left ventricle[LV] mass) at Screening Visit
  • Hypertrophic cardiomyopathy
  • Pulmonary Hypertension
  • Congenital heart disease with ongoing cardiac dysfunction
  • Cerebrovascular disease
  • Aortic insufficiency
  • Clinically significant atrial or ventricular arrhythmias
  • Have an ECG showing any of the following:
  • Right or left atrial enlargement or ventricular hypertrophy
  • PR (period of time from the beginning of atrial depolarization until the beginning of ventricular depolarization) interval > 200 msec
  • QRS (The Q, R, and S heart waves that are measured on an electrocardiogram)interval > 110 msec
  • Corrected QTc-F (Measure of the corrected time between the start of the Q wave and end of the T wave in the heart's electrical cycle) > 450 msec
  • Second- or third-degree atrioventricular block
  • Documented Vitamin D deficiency
  • Require any investigational agent prior to completion of study period
  • Have received another investigational product or investigational medical device within 30 days before the Screening visit
  • Use of any other investigational product or investigational medical device for the treatment of ACH or short stature
  • Current chronic therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers,calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function
  • Treatment with growth hormone, IGF-1 (Insulin-like growth factor), or anabolic steroids in the previous 6 months or long-term treatment (> 3 months) at any time
  • Long-term treatment (> 1 month) with oral corticosteroids
  • Concomitant medication that prolongs the QT/QTc-F interval within 14 days or 5 half-lives, whichever is longer, before the Screening visit
  • Pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study
  • Limb-lengthening or bone-related surgery < 18 months prior to study enrollment
  • Had a fracture of the long bones or spine within 6 months prior to screening (except for fracture of digits or toes)
  • AST (Aspartate Transaminase) or ALT (Alanine Transaminase) at least 3x upper limit of normal (ULN) or total bilirubin at least 2x ULN
  • Evidence of severe sleep apnea requiring surgery or new initiation of CPAP (Continuous positive airway pressure).
  • History of malignancy and chemotherapy/radiation or currently under work-up for suspected malignancy
  • Known hypersensitivity to BMN 111 or its excipients
  • Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study
  • Concurrent disease or condition that would interfere with study participation or safety
  • Have abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant as determined by the PI.
  • Have a history of hip surgery or severe hip dysplasia
  • Have a 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.
  • Are unable to lie flat when in prone position

Additional Exclusion Criteria for Optional, Open-label Extension Phase:

  • Use of restricted therapies during the initial 6 months of the study
  • Permanently discontinued BMN 111 during the initial 6 months of the study


  • Children's Hospital & Research Center Oakland
    Oakland, California, 94609, United States
  • Harbor - UCLA Medical Center
    Torrance, California, 90509, United States


in progress, not accepting new patients
Start Date
Completion Date
BioMarin Pharmaceutical
Phase 2
Study Type
Last Updated
May 19, 2017