A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Completed · Phase 2 · Has a placebo group

Conditions studied: Achondroplasia

In brief

Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.

Key facts

Study ID

NCT03583697

Run by

BioMarin Pharmaceutical

People needed

75

Starts

2018-06-13

Expected to finish

2022-01-26

Last updated by the study team

2024-06-13

Who can join

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

You may qualify if…

• Diagnosis of achondroplasia (ACH), confirmed by genetic testing. If subjects had previous genetic testing, subjects must have a lab report from a certified laboratory with the study specific mutation documented.
• Age 0 to < 60 months, at study entry (Day 1)
• Cohort 1 and 2 subjects must have at least a 6-month period of pretreatment growth assessment in Study 111 901 immediately before screening, and have one documented measurement of height/body length a minimum of 6 months prior to the screening visit for 111-206. Cohort 3 subjects must have a minimum of 3 months of observation prior to treatment. This observational period can be obtained either (1) via prior enrollment in Study 111-901 or (2) via enrollment in this Study 111 206 for a minimum of 3 months of non-treatment observation prior to commencement of treatment.
• Parent(s) or guardian(s) (and the subjects themselves, if required by local regulations or ethics committee) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure
• Willing and able to perform all study procedures as physically possible
• Parent(s) or caregiver(s) are willing to administer daily injections to the subjects and complete the required training

You may not qualify if…

• Have hypochondroplasia or short-stature condition other than achondroplasia (e.g., trisomy 21, pseudoachondroplasia, etc.)
• Subject weighs < 5.0 kg (Cohort 1 and 2) or < 4.0 kg (Cohort 3)
• Have any of the following:
• Hypothyroidism or hyperthyroidism
• Insulin-requiring diabetes mellitus
• Autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus, juvenile dermatomyositis, scleroderma, etc.)
• Inflammatory bowel disease
• Autonomic neuropathy
• Have a history of any of the following:
• Renal insufficiency defined as serum creatinine > 2 mg/dL
• Chronic anemia or Hgb <10.0 g/dL (based on screening clinical laboratory testing)
• Baseline systolic blood pressure (BP) below age and gender specified normal range or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms e.g., dizziness, fainting) or recurrent symptomatic orthostatic hypotension
• Cardiac or vascular disease, including the following
• Cardiac dysfunction (abnormal echocardiogram determined to be clinically significant by principal investigator PI and medical monitor) at Screening Visit
• Hypertrophic cardiomyopathy
• Pulmonary hypertension
• Congenital heart disease with ongoing cardiac dysfunction
• Cerebrovascular disease
• Aortic insufficiency or other clinically significant valvular dysfunction
• Clinically significant atrial or ventricular arrhythmias
• Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or Fridericia's corrected QT interval (QTc-F) >450 msec on screening ECG
• Have evidence of cervicomedullary compression (CMC) likely to require surgical intervention within 60 days of Screening as determined by the Investigator based on the following assessments
• Physical exam (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
• Polysomnography (e.g., severe central sleep apnea)
• MRI indicating presence of severe CMC or spinal cord damage

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
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Baylor College of Medicine — Houston, Texas, 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
• Osaka University Hospital — Osaka, Japan
• Saitama Children's Medical Center — Saitama, Japan
• Tokushima University Hospital — Tokushima, Japan
• 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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