A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Acho… (NCT06164951) | Clinical Trial Compass
CompletedPhase 3
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
United States114 participantsStarted 2023-11-10
Plain-language summary
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the QED-sponsored study PROPEL (QBGJ398-001).
Who can participate
Age range3 Years – 17 Years
SexALL
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Inclusion criteria
✓. Subject must be 3 to \<18 years of age at screening with growth potential defined as annualized height velocity of \>1.5 cm/year over a period of at least 6 months of participation in the PROPEL observational study (QBGJ398-001), pubertal Tanner stage ≤4, and bone age ≤13 years in females and ≤15 years in males.
✓. Subjects who have a diagnosis of ACH that has been documented clinically and confirmed by genetic testing.
✓. Subjects must have completed at least 26 weeks in the PROPEL (QBGJ398-001) study before screening.
✓. Subjects are able to swallow oral medication.
✓. Subjects and parent(s), legal guardian(s), or caregivers are willing and able to comply with study visits and study procedures.
✓. Subjects are ambulatory and able to stand without assistance.
✓. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
✓. If sexually active, subjects, whether male or female, must be willing to use a highly effective method of contraception while taking study drug and for 3 months after the last dose of study drug.
Exclusion criteria
✕. Subjects who have hypochondroplasia or short stature condition other than ACH.
✕. Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
What they're measuring
1
Change from baseline (BL) in annualized height velocity (cm/year)
. 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 and/or would require treatment with a prohibited medication, and/or would place the subject at high risk for poor treatment compliance or for not completing the study.
✕. 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 or short stature.
✕. Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid therapy (ie, \>15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (ie, 2.5-10 mg/kg/day of hydrocortisone or equivalent) for over 3 weeks within 6 months of the screening visit (low-dose local preparations including inhaled steroid for asthma, intranasal sprays for allergies, and topical steroids are allowed).