Safety and Efficacy of BMN 111 in Children with Achondroplasia OLE
Research type
Research Study
Full title
A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
IRAS ID
249622
Contact name
Melita Irving
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2017-002404-28
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
111299, IND Number
Duration of Study in the UK
6 years, 11 months, 20 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. 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 regulation. BMN 111 has been made in the lab with the intention that it might work like CNP, but may stay in the human body longer. Currently, there are no specific medicines approved that increase the height potential of a child with ACH however surgical limb-lengthening can be used for those who wish to increase final height potential.This study (111-302) will allow for long-term assessment of the effect of daily BNM 111 administration on safety, tolerability, growth velocity, height, and body proportions in participants who have completed 1 year of placebo or BNM 111 treatment in the ongoing Phase 3 111-301 study.
In 111-302, all participants will receive BMN 111. Therefore, depending on treatment assignment in 111-301, placebo or BMN-111, some participants will receive a total of at least 5 to 6 years of BMN 111 treatment, 111-301 plus 111-302 study duration. If at the end of 260 weeks (5 years) the participant has not reached near-final adult height, they will remain on this study for more than 5 years.
Participants will visit the clinic at least 17 times during the initial 5-year period, with the potential of more visits every 26 weeks (up to week 312, and then every 52 weeks) until near-adult height is reached.
The participants will be given one single injection of medication daily. An approved caregiver will administer the study medication between clinic visits.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
18/YH/0368
Date of REC Opinion
12 Oct 2018
REC opinion
Favourable Opinion