Safety and Efficacy of BMN 111 in Children with Achondroplasia
Research type
Research Study
Full title
A Phase 3 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia
IRAS ID
206856
Contact name
Melita Irving
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2015-003836-11
Clinicaltrials.gov Identifier
111299, IND Number
Duration of Study in the UK
2 years, 5 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 further confirm the safety and efficacy of BMN 111 (the study medication) has on participants, their growth, and their ACH symptoms for 52 weeks. Children of both sexes, under the age of 16, may be able to take part in this study as long as they took part in study 111-901 for 6 months immediately before this one. The participants will be given one single injection of medication or placebo daily. An 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 a total of 10 times over the course of 52 weeks.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
16/YH/0502
Date of REC Opinion
20 Jan 2017
REC opinion
Further Information Favourable Opinion