Clinical Trial Finder

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.

ACCEL 2/3 is a Phase 2/3 study that comprises of 2 portions. The Phase 2 portion is an open-label, portion in children with HCH aged 5 to 11 years old followed by a Phase 3 portion which is double-blind, placebo-controlled in children with HCH aged >3 years old to <18 years old.

Arms

Experimental: Phase 2 Cohort 1

infigratinib (0.128 mg/kg/day)

Experimental: Phase 2 Cohort 2

infigratinib (0.25 mg/kg/day)

Interventions

Drug: - infigratinib 0.128 mg/kg/day

Oral infigratinib 0.128 mg/kg/day

Drug: - infigratinib 0.25 mg/kg/day

Oral infigratinib 0.25 mg/kg/day