An Interventional Study of Infigratinib in Children With Hypochondroplasia
NCT ID: NCT06873035
Last Updated: 2025-12-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
PHASE2/PHASE3
24 participants
INTERVENTIONAL
2025-04-22
2027-01-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Phase 2 Cohort 1
infigratinib (0.128 mg/kg/day)
infigratinib 0.128 mg/kg/day
Oral infigratinib 0.128 mg/kg/day
Phase 2 Cohort 2
infigratinib (0.25 mg/kg/day)
infigratinib 0.25 mg/kg/day
Oral infigratinib 0.25 mg/kg/day
Interventions
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infigratinib 0.128 mg/kg/day
Oral infigratinib 0.128 mg/kg/day
infigratinib 0.25 mg/kg/day
Oral infigratinib 0.25 mg/kg/day
Eligibility Criteria
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Inclusion Criteria
* Phase 2 portion: Participants 5-11 years of age (inclusive).
* Phase 3 portion: Participants 3 to \<18 years of age at screening with growth potential
* Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
* Participants are able to swallow oral medication.
* Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
* Participants are ambulatory and able to stand without assistance. Sex and Contraceptive/Barrier Requirements
* Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
* If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception, as relevant, while taking study drug and for 1 month after the last dose of study drug.
* Signed informed consent.
Exclusion Criteria
* Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
* Current evidence of clinically significant corneal or retinal disorder/keratopathy confirmed by ophthalmic examination.
* Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.
* History and/or current evidence of extensive ectopic tissue calcification.
* History of malignancy.
* Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature.
* Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid.
* Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study.
* Participants receiving medications which could increase serum phosphorus and/or calcium concentrations
* Clinically significant abnormality in any laboratory test result at screening.
* Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.
* Allergy to any components of the study drug.
* Concurrent circumstance, disease, or condition that would interfere with study participation.
3 Years
18 Years
ALL
No
Sponsors
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QED Therapeutics, a BridgeBio company
INDUSTRY
Responsible Party
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Locations
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UCSF Benioff Children's Hospital
Oakland, California, United States
Childrens Hospital Colorado
Aurora, Colorado, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
University of Missouri
Columbia, Missouri, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
London Health Services Center - Children's Hospital of Western Ontario
London, Ontario, Canada
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, Canada
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Hôpital Femme Mère Enfant
Bron, France, France
Hôpital Universitaire Necker-Enfants Malades
Paris, France, France
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse, France, France
Haukeland University Hospital
Bergen, Norway, Norway
Paediatric Clinical Research Unit at Osla University Hospital
Oslo, Norway, Norway
Hospital Pediátrico de Coimbra
Coimbra, Portugal, Portugal
KK Women's and Children's Hospital
Singapore, Singapore, Singapore
Hospital Vithas San Jose
Vitoria-Gasteiz, Spain, Spain
Astrid Lindgren Children's Hospital
Solna, Sweden, Sweden
Manchester University
Manchester, United Kingdom, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom, United Kingdom
Countries
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Other Identifiers
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QBGJ398-304
Identifier Type: -
Identifier Source: org_study_id
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