Open Label Extension of BMN 111 in children with achondroplasia
Research type
Research Study
Full title
A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia
IRAS ID
199226
Contact name
Melita Irving
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2015-004004-30
Duration of Study in the UK
6 years, 2 months, 1 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 might 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.
The purpose of the study is to continue to learn about the long-term effects BMN 111 (the study medication) has on participants, their growth, and their ACH symptoms. Children of both sexes, under the age of 18, may be able to take part in this extension study if they have received 2 years of BMN 111 medication on the previous 111-202 study. The participant will be given one single injection at the same dose of medication received upon completion of the previous study. This will occur daily, at approximately the same time each day. The approved caregiver will continue to administer the study medication between clinic visits. Home health care is not required, however this service can be provided. The participant will be asked to visit the clinic once every 13 weeks, totalling 22 visits over the course of 5 years.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
16/EM/0241
Date of REC Opinion
8 Aug 2016
REC opinion
Further Information Favourable Opinion