The PROPEL Trial V1.0
Research type
Research Study
Full title
PROSPECTIVE CLINICAL ASSESSMENT STUDY IN CHILDREN WITH ACHONDROPLASIA: The PROPEL Trial
IRAS ID
262894
Contact name
Mars Skae
Contact email
Sponsor organisation
QED Therapeutics, Inc
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
This is a Phase 0 prospective clinical assessment study without drug to evaluate children aged 2.5 to 10 years with Achondroplasia. This study is designed to evaluate study participants over a period of time of minimum 6 months and maximum of 2 years (maximum 6 visits in total) prior to potential enrolment in a QED-sponsored interventional study. Study participants will be assessed at baseline, at Month 3, Month 6, and every 6 months thereafter for a maximum of 2 years. Data on growth, disease-related medical events and their management will be collected at every visit. Blood samples for biomarkers of bone growth will be collected at baseline and every\n6 months. Participants will complete study activities when they finish 2 years of participation or enrol in a QED sponsored interventional study to treat Achondroplasia.
REC name
West of Scotland REC 4
REC reference
19/WS/0066
Date of REC Opinion
13 Jun 2019
REC opinion
Further Information Favourable Opinion