for people ages 5-18 (full criteria)
at Oakland, California and other locations
study started



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.

Official Title

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia


This is a Phase 3 randomized, placebo-controlled, double-blind multicenter study with approximately 110 subjects, aged 5 to < 18 years old. Subjects with documented Achondroplasia confirmed by genetic testing will have been enrolled in Study 111-901 for at least a 6-month period immediately before entering into the 111-301 study. Eligible subjects will be randomly assigned to one of two treatment groups: placebo or BMN 111 at 15 μg/kg. The route of administration is subcutaneous injection and the frequency is daily.


Achondroplasia Dwarfism Bone Diseases Bone Diseases, Developmental ACH Natriuretic Peptide, C-Type Musculoskeletal Diseases Natriuretic Agents Physiological Effects of Drugs Skeletal Dysplasias Genetic Diseases, Inborn Osteochondrodysplasias BMN 111


You can join if…

Open to people ages 5-18

  • 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 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
  • 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


  • 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
BioMarin Pharmaceutical
NIH Genetics Home Reference related topics: Achondroplasia
NIH Genetic and Rare Diseases Information Center resources: Achondroplasia
Phase 3
Study Type
Last Updated