OBS16647
Research type
Research Study
Full title
OBS16647 - a longitudal study to evaluate growth measurements and digital biomarkers in paediatric participants with achondroplasia.
IRAS ID
322181
Contact name
Melita Irving
Contact email
Sponsor organisation
Sanofi
Clinicaltrials.gov Identifier
151799, IND; U1111-1267-9080, WHO
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Achondroplasia is a genetic condition that people are born with. In most patients, it is caused by a mutation (change)
in a gene called “FGFR3.” The mutation affects bone growth of the skull, spine, arms, and legs, resulting in short
stature, difficulty walking, and often frequent ear infections and back pain. More serious effects may include enlarged
tonsils, breathing problems while sleeping, called “sleep apnea,” or enlarged ventricles in the brain, all of which may
require surgery.
The purpose of this study is to collect information about the child’s condition and related history to obtain a good
understanding about the rate at which children with achondroplasia grow, their activity levels, gait, and breathing
during sleep, without any study medications.
During this study no study drug will be prescribed outside of the normal medical care for your child’s condition. The
child may be eligible for participation in an interventional drug trial after completion of this longitudinal study. If that is
the case, the growth data obtained from children participating in this longitudinal study will be used to define baseline
growth for the interventional drug trial. The study will be conducted in participants between the ages of birth to 10 years
with achondroplasia.
It will last approximately 24 weeks but for participants under the age of 3 years at the
time of enrollment into this study and who plan to enroll into an eligible interventional drug trial, a study duration of 12
weeks is allowed because they are younger and stand to benefit more from a study drug. It is planned to include
approximately 50 participants and be conducted in North America, Europe, Asia, and Australia.REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
23/WM/0010
Date of REC Opinion
27 Jun 2023
REC opinion
Further Information Favourable Opinion