Observational Study in Children with Hypochondroplasia - ACCEL
Research type
Research Study
Full title
Prospective Clinical Assessment Study in Children with Hypochondroplasia: ACCEL
IRAS ID
339669
Contact name
Melita Irving
Contact email
Sponsor organisation
QED Therapeutics, Inc.
Clinicaltrials.gov Identifier
141059, IND
Duration of Study in the UK
1 years, 9 months, 1 days
Research summary
This is a prospective, non-interventional clinical assessment study to collect data on children aged 2.5 to <17 years with hypochondroplasia (HCH), which will contribute to the characterisation of the natural history of children with HCH.
HCH is a rare genetic (inherited) bone disorder with the prevalence estimated at approximately 1:15,000 to 1:40,000. At present, there is no specific treatment for HCH and its current management focuses on the prevention and treatment of its complications. With the absence of approved targeted therapies, the current approach to management primarily involves supportive care, which fails to adequately address the concerns of the affected individuals. Therefore, there is a high unmet need for effective treatment in this patient population.
The purpose of this study is to contribute to the characterisation of the natural history of children with HCH who have short stature (as defined for the study) and to collect baseline data from these children prior to potential enrollment in a QED-sponsored interventional study.
The study is planned to involve about 150 participants from 2.5 to <17 years old at approximately 30 study sites, worldwide, who have HCH. As part of the study, participants will be assessed at the screening/baseline visit, 3 and 6 months later and every 6 months after that for a maximum of 2 years.
During participation in the study, participants will undergo some of the following procedures and tests: physical exams, assessment of vital signs, blood tests, ECG test, Echocardiogram, growth measurement etc.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
24/EM/0161
Date of REC Opinion
2 Sep 2024
REC opinion
Further Information Favourable Opinion