PROPEL I&T
Research type
Research Study
Full title
Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children with Achondroplasia
IRAS ID
1012456
Contact name
Karla Mehl
Contact email
Sponsor organisation
QED Therapeutics, Inc.
Eudract number
2024-518072-31
Research summary
QED Therapeutics, Inc. (the Sponsor) is running a research study to see if a test medicine not yet approved for market, named infigratinib, will help in the treatment of achondroplasia (ACH) and to understand how safe it is for infants and young children with achondroplasia.
Achondroplasia is a rare genetic (inherited) bone disorder that occurs in one in 15,000 to 30,000 children. Currently, only one drug targeting the underlying causes of achondroplasia has been approved. Current management of achondroplasia focuses on the prevention and treatment of its complications.
Infigratinib is an oral medication that has the potential to improve growth of certain bones. By doing so, it may promote better overall health.
Infigratinib can be provided in three formulations for this study, depending on the study portion the child is participating in. In the Single Ascending Dose (SD) portion, infigratinib will be provided as minitablets in three strengths; 0.1mg, 0.5mg, and 1.0mg.
The Phase 2 of the study will be open label, and the aim will be to confirm the doses in the SAD portion are safe and effective. In the Phase 2, infigratinib will be provided as gelatin capsules containing sprinkles called minitablets inside, which will contain the daily dose. The sprinkle capsules will be provided in different strengths in order to allow precise dosing based on the child’s weight.
The Phase 2b portion will be double blind placebo-controlled phase where participants have a 50/50 chance of receiving either placebo or the study drug at a dose based on what was received for their weight during Phase 2.
The Extension portion of the study will be open label, and the aim will be to evaluate the safety and efficacy of infigratinib in participants who completed the Phase 2 or Phase 2b portion of the study. The study will involve about 77 participants aged 0 to less than 3 years old at approximately 13 study sites, worldwide. There will be about 25 children enrolled in the United Kingdom.
Children participating in the SD portion will have approximately 4 visits and a follow up call 2 weeks later. They may receive a second dose with another 4 visits and a follow up call. In the Phase 2 portion there will be a maximum of 14 visits over 52 weeks.
Children under 3 years who complete the 52 weeks will be offered to continue treatment in an Extension portion of this study.
Children over 3 years will be offered to continue treatment in the PROPEL OLE study.REC name
South Central - Berkshire B Research Ethics Committee
REC reference
25/SC/0294
Date of REC Opinion
4 Nov 2025
REC opinion
Further Information Favourable Opinion