111-206- BMN 111 in Infants & Young Children with Achondroplasia (ACH)
Research type
Research Study
Full title
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia, Age 0 to < 60 Months
IRAS ID
243470
Contact name
Melita Irving
Contact email
Sponsor organisation
BioMarin Pharmaceuticals Inc.
Eudract number
2016-003826-18
Clinicaltrials.gov Identifier
111299, IND
Duration of Study in the UK
2 years, 5 months, days
Research summary
Summary of Research
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 for it to work like CNP, but stay in the human body longer.This phase 2 study (111-206) is being conducted to assess the safety and potential benefit of BMN 111 in infants and young children with ACH. Children with ACH have a high incidence of serious medical, respiratory and neurological complications, and currently no specific medicines are approved in the US or EU.
Approximately 70 participants will be enrolled if they meet the eligibility criteria at approximately 10-15 centres worldwide. If the participant has taken part in 111-901) they will be in the study for around 60 weeks (including screening, study medication administration and follow up). If the participant enrols in this study directly, they will be in the study for around 70 weeks to account for the 3-month observational study
Children of both sexes, under the age of 60 months, may be able to take part in this study. The participants will be divided into cohorts based on age and will be given one single injection of medication or placebo daily for up to 52 weeks 3 sentinels will be enrolled in each cohort and the rest of the participants will be randomised 1:1 to receive either BMN 111 or placebo. 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.
Summary of Results
We have requested a delay in the EudraCT result posting requirement for the 111-206, until the variation has been submitted/reviewed – we await EMA acceptance of this proposal before providing the lay summary.REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
18/YH/0240
Date of REC Opinion
12 Sep 2018
REC opinion
Further Information Favourable Opinion