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Clinical Trials in the UK / NCT04554940
Active, not recruiting Phase 2

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

NCT04554940 · tracked via the Priya Life Science UK tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 2
Started
2020-10-10
Last updated
2026-03-13

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

vosoritide

vosoritide: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Study summary

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery

Eligibility

Sex
ALL
Min age
0 Months
Max age
12 Months
Healthy volunteers
No
Inclusion Criteria: * Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research related procedure. * Have ACH, documented by genetic testing. * Are willing and able to perform all study procedures as physically possible. * Age 0 to ≤ 12 months, at study entry (Day 1). Given that any potential impact of vosoritide therapy on the foramen magnum is dependent on treating as early as possible and as long as possible while the synchondroses at the base of the skull are still open. For subjects \> 6 months of age at enrollment, a discussion between the investigator and the Medical Monitor should occur with the goal of limiting the number of subjects in the range of \> 6 months to ≤ 12 months of age. * Parent(s) or caregiver(s) are willing to administer daily injections to the subject and complete the required training. * Have evidence of CMC that "may" require surgical intervention Exclusion Criteria: * Have hypochondroplasia or short-stature condition other than achondroplasia (eg, trisomy 21, pseudoachondroplasia, etc). * Have CMC that either does not require surgical intervention (for example foramen magnum narrowing with preservation of the cerebrospinal fluid space) or does require immediate surgical intervention . * Have any of the following: Untreated congenital hypothyroidism or maternal history of hyperthyroidism, Insulin-requiring neonatal diabetes mellitus, Autoimmune inflammatory disease, Inflammatory bowel disease, Autonomic neuropathy. * Have a history of any of the following:Renal insufficiency, Chronic anemia,Baseline systolic blood pressure below age and gender specified normal range or recurrent symptomatic hypotension (defined as episodes of low blood pressure generally accompanied by symptoms eg, pallor, cyanosis, irritability, poor feeding) and Cardiac or vascular disease. * Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or QTc-F ≥ 450 msec on screening ECG. * Have been treated with growth hormone, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time. * Have ever had prior cervicomedullary decompression surgery. * Have had a fracture of the long bones or spine within 6 months prior to Screening.

Primary outcome measure(s)

Trial sites (3)

FacilityCityRegionStatus
Murdoch Children's Research Institute Parkville Victoria
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital London United Kingdom
Sheffield Children's NHS Foundation Trust Sheffield United Kingdom

More BioMarin Pharmaceutical trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04554940 on ClinicalTrials.gov ↗ ← All trials in the UK