Ireland
--:--IST
Enrolling by invitation Phase 2

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

NCT05145010 · tracked via the Priya Life Science UK tracker
Phase
Phase 2
Started
2021-12-06
Last updated
2026-10-26

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

Infigratinib →Infigratinib →

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

Sex
ALL
Min age
3 Years
Max age
18 Years
Healthy volunteers
No
Key Rollover Participants Inclusion Criteria: 1. Pediatric participants ≥3 years old with ACH who have completed a previous QED-sponsored interventional study with infigratinib. 2. Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. 3. Participants are able to swallow oral medication. 4. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. 5. If sexually active, participant must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug. 6. The PI, or a person designated by the PI, will obtain written informed consent from each participant's parent(s), legal guardian(s), or caregiver(s) and the participant's assent, when applicable, before any study-specific activity is performed. Key Rollover Participants Exclusion Criteria: 1. Participant has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations. 2. Participants who developed a medical condition that will require the initiation of treatment with a prohibited medication. 3. Participants prematurely discontinued a prior QED-sponsored interventional study with infigratinib 4. Current participation in an ongoing clinical study with a sponsor other than QED 5. Participants that have reached final height or near final height. Key Inclusion Criteria for Treatment Naïve Participants: 1. Participants must be 3 to \<18 years of age at screening and have growth potential. 2. Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. 3. Participants are able to swallow oral medication. 4. Participants who have a diagnosis of ACH, documented clinically and confirmed by genetic testing. 5. Participants from PROPEL Study (Protocol QBGJ398-001) only: Must have completed at least a 6-month period of growth assessment in PROPEL prior to study entry. 6. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. 7. If sexually active, participant must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug. 8. The PI, or a person designated by the PI, will obtain written informed consent from each participant's parent(s), legal guardian(s), or caregiver(s) and the participant's assent, when applicable, before any study-specific activity is performed. Key Exclusion Criteria for Treatment Naïve Participants: 1. Participants who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature). 2. Participants who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the participant or would interfere with study participation or safety evaluations. 3. Participants who have a history of malignancy. 4. Participants who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4. 5. Participants who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible. 6. Participants who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months. 7. Participants who have significant abnormality in screening laboratory results. 8. Participants who have had a fracture within 12 months of screening.

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)

FacilityCityRegionStatus
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
Official registry record

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