PROPEL 2
Research type
Research Study
Full title
Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2
IRAS ID
272479
Contact name
Melita Irving
Contact email
Sponsor organisation
QED Therapeutics, Inc.
Eudract number
2019-002954-21
Clinicaltrials.gov Identifier
141059, IND
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
Summary of Research
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. 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 identify the dose to be used in future studies in children with ACH, based on safety and efficacy evaluations.
The study is planned to involve 60 participants from 3 to 11 years old who have ACH and who previously participated in the PROPEL study for at least 6 months. This study consists of 2 parts: the first part is called Dose Escalation with extended Treatment period and will enrol 40 participants; and the second part is called Dose Expansion and will enrol 20 new participants.In the Dose Escalation with extended treatment period the participants will receive treatment for 18 months and will be part of the study for a total of up to 22 months. If a child is enrolled in the Expansion part, then he/she will receive treatment for 12 months and will be part of the study for a total of up to 16 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.Summary of Results
The lay summary is not currently available.REC name
HSC REC A
REC reference
20/NI/0032
Date of REC Opinion
31 Mar 2020
REC opinion
Further Information Favourable Opinion