Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Condition(s) studied
Investigational drug(s) / intervention(s)
Infigratinib: Infigratinib minitablets or sprinkle capsules to be administered by mouth. In participants that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to 0.25 mg/kg/day.
Infigratinib: Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the participants naïve to Infigratinib will be 0.25 mg/kg/day.
Study summary
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who previously completed a QED-sponsored interventional study, and in additional participants who are naïve to infigratinib treatment. Quality of Life assessments for this participant population will also be evaluated. Treatment-naïve participants must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study if the sponsor deems additional long-term safety and/or efficacy data are necessary, and participants are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.
Eligibility
Primary outcome measure(s)
- Incidence of treatment emergent adverse events (TEAE) and serious TEAE — 10 years
- Changes over time in height Z-score in relation to ACH and general pediatric population tables — 10 years
Trial sites (31)
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF Benioff Children's Hospital, Oakland | Oakland | California | |
| Children's Hospital Colorado | Aurora | Colorado | |
| Nemours Alfred I. Dupont Hospital for Children | Wilmington | Delaware | |
| Johns Hopkins University | Baltimore | Maryland | |
| University of Missouri | Columbia | Missouri | |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | |
| Vanderbilt University Medical Center | Nashville | Tennessee | |
| University Hospital and UW Health Clinics | Madison | Wisconsin | |
| Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan | Ciudad Autonoma Buenos Aires | Buenos Aires | |
| Murdoch Children's Hospital | Parkville | Victoria | |
| Stollery Children's Hospital | Edmonton | Alberta | |
| Children's Hospital - London Health Sciences Centre | London | Ontario | |
| Children's Hospital of Eastern Ontario | Ottawa | Ontario | |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | |
| Hopital Femme Mere Enfant | Lyon | France | |
| Hopital Necker-Enfants Malades | Paris | France | |
| Hopital des Enfants | Toulouse | France | |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy | |
| Policlinico A. Gemelli IRCCS | Roma | Italy | |
| Haukeland University Hospital | Bergen | Norway | |
| Oslo University Hospital | Oslo | Norway | |
| KK Women's and Children's Hospital | Singapore | Singapore | |
| Hospital Universitario La Paz | Madrid | Spain | |
| Hospital Universitario Virgen de la Victoria | Málaga | Spain | |
| Hospital Vithas San José | Vitoria-Gasteiz | Álava | |
| Birmingham Women's and Children's NHS Foundation Trust | Birmingham | United Kingdom | |
| University Hospitals Bristol and Weston NHS Foundation Trust | Bristol | United Kingdom | |
| Queen Elizabeth University Hospital | Glasgow | United Kingdom | |
| St. Thomas' Hospital | London | United Kingdom | |
| Manchester University Children's Hospital | Manchester | United Kingdom | |
| Sheffield Children's Hospital | Sheffield | United Kingdom |
More QED Therapeutics, a BridgeBio company trials in the UK
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05145010 on ClinicalTrials.gov ↗ ← All trials in the UK