Open-Label, Long-Term, Extension Study of Infigratinib in Children with Achondroplasia PROPEL OLE
Research type
Research Study
Full title
Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL OLE
IRAS ID
1004032
Contact name
Jack Winnike
Contact email
Sponsor organisation
QED Therapeutics, Inc.
Eudract number
2021-001855-15
Clinicaltrials.gov Identifier
Research summary
This study is investigating the medication named infigratinib phosphate in participants with achondroplasia (ACH).
Achondroplasia is a rare genetic (inherited) bone disorder that occurs in one in 15,000 to 40,000 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. 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 long-term safety, tolerability, and efficacy of Infigratinib.
The study is planned to involve 230 participants from 3 to 18 years old who have ACH and who previously participated in an interventional 2 study with infigratinib. Additionally, up to 50 participants who have never received infigratinib before might be included (treatment naïve).
Participants in this study will be required to attend study visits approximately every 3 months during the first year followed by visits every 6 months through to completion of the study. During this period participants will receive a daily dose of infigratinib until they have completed the study.
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
Wales REC 2
REC reference
21/WA/0290
Date of REC Opinion
26 Oct 2021
REC opinion
Further Information Favourable Opinion