Enrolling by invitation NCT07393373
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Infigratinib.
- Who it may be relevant to
- Registry conditions: Hypochondroplasia. Basic parameters: 3 years — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, France, Norway +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Hypochondroplasia: ACCEL OLE
Overview
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.
Interventions
- Drug Infigratinib
Infigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3.
Primary outcome measures
- Incidence of treatment emergent adverse events (TEAE) and serious TEAE [Time frame: 10 years]
- Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts [Time frame: 10 years]
Secondary outcome measures (12)
- Changes over time in AHV Z-score [Time frame: 10 years]
- Changes over time in body proportions [Time frame: 10 years]
- Changes over time in weight Z-score [Time frame: 10 years]
- Changes overtime in BMI [Time frame: 10 years]
- Age of puberty onset and time to Tanner stage ≥4 [Time frame: 10 years]
- Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans [Time frame: 10 years]
- Changes over time in bone morphology/density by x-ray and DXA [Time frame: 10 years]
- Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test) [Time frame: 10 years]
- Change in attention assessed by age-appropriate computerized tests (Identification Test) [Time frame: 10 years]
- Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test) [Time frame: 10 years]
- Change in working memory assessed by age-appropriate computerized tests (One Back Test) [Time frame: 10 years]
- Changes over time in severity of epilepsy measured by frequency and adverse event grading [Time frame: 10 years]
Eligibility criteria
Inclusion criteria
- Inclusion Criteria for Participants Rolling Over from ACCEL 2/3
- Pediatric participants with HCH who have completed ACCEL 2/3
- Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche
Exclusion criteria
- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3
- Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations
- Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication
- Participants who prematurely discontinued ACCEL 2/3
- Participants who have reached final height or near final height
- Current participation in an ongoing clinical study with a sponsor other than QED
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 8 centers
- UCSF Benioff Children's Hospital — Oakland
- Childrens Hospital Colorado — Aurora
- Children's National Hospital — Washington D.C.
- Johns Hopkins School of Medicine — Baltimore
- University of Missouri — Columbia
- Cincinnati Children's Hospital Medical Center — Cincinnati
- Vanderbilt University Medical Center — Nashville
- University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic — Madison
Canada · 3 centers
- London Health Sciences Centre - Children's Hospital of Western Ontario — London
- Children's Hospital of Eastern Ontario Research Institute — Ottawa
- Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine — Montreal
France · 3 centers
- Hôpital Femme Mère Enfant — Bron
- Hôpital Universitaire Necker-Enfants Malades — Paris
- Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants — Toulouse
United Kingdom · 3 centers
- Manchester University — Manchester
- Sheffield Children's Hospital — Sheffield
- Glasgow Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow
Norway · 2 centers
- Haukeland University Hospital — Bergen
- Paediatric Clinical Research Unit at Oslo University Hospital — Oslo
Australia · 1 center
- Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne — Parkville
Portugal · 1 center
- Hospital Pediátrico de Coimbra — Coimbra
Singapore · 1 center
- KK Women's and Children's Hospital — Singapore
Spain · 1 center
- Unidad de Cirugía Artroscopica, Hopsital MIKS — Vitoria-Gasteiz
Sweden · 1 center
- Astrid Lindgren Children's Hospital — Solna
Identifiers
NCT: NCT07393373 · QBGJ398-205