PROPEL 3
Research type
Research Study
Full title
A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age with Achondroplasia: PROPEL 3
IRAS ID
1008314
Contact name
Carol Zoltowski, VMD
Contact email
Sponsor organisation
QED Therapeutics, Inc.
Eudract number
2023-506130-67
Research summary
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in Children 3 with achondroplasia (ACH) who have completed at least 26 weeks (i.e., 6 months) of participation in the PROPEL study (Protocol QBGJ398-001).
Achondroplasia is a rare genetic (inherited) bone disorder that occurs in one in 15,000 to 30,000 live births. At present, there is no specific treatment for achondroplasia and its current management focuses on the prevention and
treatment of its complications.
Infigratinib is an oral medication that is being studied for the treatment of ACH. It has not been used in children with
achondroplasia before. This medication targets the primary cause of the irregular bone growth in ACH. Infigratinib may
improve bone growth in these children, including the growth of long bones. By doing so, it may promote better overall
health in children with ACH.The main objective of the study is to evaluate the efficacy of infigratinib and also if it is safe for use in children 3-<18 years old. The dose is 0.25 mg/kg/day which was the dose selected from the ongoing PROPEL 2 (Protocol QBGJ398-201) study being conducted in children. The dose can be decreased to 0.128 mg/kg/day.
The study is planned to involve about 110 participants from 3 to <18 years old at approximately 30 study sites, worldwide, who have achondroplasia and who previously completed at least 26 weeks in the PROPEL study (Protocol QBGJ398-001).
As part of the study, participants will receive treatment for 12 months and will be part of the study for a total of up to 15 months.
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, growth measurement, dental exam, x-rays, etc.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
23/NE/0219
Date of REC Opinion
20 Dec 2023
REC opinion
Further Information Favourable Opinion