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Clinical Trials in Spain / NCT06873035
Enrolling by invitation Phase 2/3

An Interventional Study of Infigratinib in Children With Hypochondroplasia

NCT06873035 · tracked via the Priya Life Science Spain tracker
Phase
Phase 2/3
Started
2025-04-22
Last updated
2026-07-16

Condition(s) studied

Hypochondroplasia

Investigational drug(s) / intervention(s)

infigratinib 0.128 mg/kg/day →infigratinib 0.25 mg/kg/day →

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

Study summary

ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses, who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).

Eligibility

Sex
ALL
Min age
3 Years
Max age
18 Years
Healthy volunteers
No
Key Inclusion Criteria: * Participants must have completed at least 26 weeks and still be on the observational study (QBGJ398-004). * 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. Key Exclusion Criteria: * Participants who have ACH or a short stature condition other than HCH. * 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.

Primary outcome measure(s)

Trial sites (24)

FacilityCityRegionStatus
UCSF Benioff Children's Hospital Oakland California
Childrens Hospital Colorado Aurora Colorado
Children's National Hospital Washington D.C. District of Columbia
Johns Hopkins School of Medicine Baltimore Maryland
University of Missouri Columbia Missouri
Cincinnati Children's Hospital Medical Center Cincinnati Ohio
Vanderbilt University Medical Center Nashville Tennessee
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic Madison Wisconsin
Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne Parkville Victoria
London Health Services Centre - Children's Hospital of Western Ontario London Ontario
Children's Hospital of Eastern Ontario Research Institute Ottawa Ontario
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine Montreal Quebec
Hôpital Femme Mère Enfant Bron France
Hôpital Universitaire Necker-Enfants Malades Paris France
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants Toulouse France
Haukeland University Hospital Bergen Norway
Paediatric Clinical Research Unit at Oslo University Hospital Oslo Norway
Hospital Pediátrico de Coimbra Coimbra Portugal
KK Women's and Children's Hospital Singapore Singapore
Unidad de Cirugía Artroscopica, Hopsital MIKS Vitoria-Gasteiz Spain
Astrid Lindgren Children's Hospital Solna Sweden
Manchester University Manchester England
Sheffield Children's Hospital Sheffield England
Glasgow Clinical Research Facility, Queen Elizabeth University Hospital Glasgow Scotland

More QED Therapeutics, a BridgeBio company trials in Spain

Other trials for the same condition

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 NCT06873035 on ClinicalTrials.gov ↗ ← All trials in Spain