111-208 BMN 111 in Children with Achondroplasia (ACH) OLE
Research type
Research Study
Full title
A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
IRAS ID
265089
Contact name
Melita Irving
Contact email
Eudract number
2018-004364-66
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
111299 , IND Number
Duration of Study in the UK
4 years, 8 months, 2 days
Research summary
Achondroplasia (ACH) is the most common form of dwarfism, characterised by disproportionate short stature, where the arms and legs are short in comparison to the rest of the body. There is no approved pharmacological therapy for ACH. BMN 111 is an experimental medicine that is similar to a natural molecule called C-type natriuretic peptide (CNP). CNP is found in the human body and is involved in bone growth regeneration. BMN 111 has been made in the lab with the intention for it to work like CNP, but stay in the human body longer, meaning is has a longer half-life.
This study, 111-208, is a phase 2, open label extension study to 111-206, to evaluate the long-term safety and efficacy of BMN 111 in children with achondroplasia until they reach near adult height. The participants will receive the age appropriate daily dose injection (under the skin) of BMN 111 as determined by the 111-206 study. An approved caregiver will continue to administer the study medication between study visits. This study will allow for long-term assessment of the effect of daily BMN 111 administration on safety, tolerability, growth velocity, height, and body proportions in participants who have completed 1 year of placebo or BMN 111 treatment in study 111-206.
Approximately 70 participants (male and female) will be enrolled across 17 study centres worldwide. Participants will roll over from the 111-206 study and continue in the 111-208 study until near adult height is reached.
Participants will be assessed using, blood, salvia and urine tests, as well as electrocardiograms to look at heart function, with bone scans (DXA) and x-rays to look at bone development. Participants will also be asked to enter a sleep study, up to 5 years of age and have MRI’s of their skull and brain up-to 3 years of age.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
19/YH/0353
Date of REC Opinion
22 Oct 2019
REC opinion
Favourable Opinion