A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

Completed · Phase 2

Conditions studied: Achondroplasia

In brief

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.

Key facts

Study ID

NCT02055157

Run by

BioMarin Pharmaceutical

People needed

35

Starts

2014-01-13

Expected to finish

2017-10-02

Last updated by the study team

2021-01-15

Who can join

Age: 5 and older, up to 14. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 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
• 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

Where it is running

• Children's Hospital & Research Center Oakland — Oakland, California, United States
• Harbor - UCLA Medical Center — Torrance, California, United States
• Ann and Robert H. Lurie Childrens Hospital of Chicago — Chicago, Illinois, United States
• Johns Hopkins McKusick - Institute of Genetic Medicine — Baltimore, Maryland, United States
• Vanderbilt University — Nashville, Tennessee, United States
• Baylor College of Medicine — Houston, Texas, United States
• Murdoch Children's Research Institute — Parkville, Victoria, Australia
• Institut Necker — Paris, France
• Guys & St. Thomas NHS Foundation Trust Evelina Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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