This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.
Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation.
Age 0 to 32 months (2 years and 8 months) at screening.
Signed informed consent, which must be obtained from each participant's parent(s) or legal guardian.
Parent(s)/Guardian(s) willing and able to attend all study visits and comply with all study requirements.
Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth.
Gestational age at birth <37 weeks and/or birth weight <2500 grams.
Gastroesophageal reflux disease requiring prolonged treatment (>1 week) with prohibited medications.
Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery.
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Treatment groupsSee each treatment group below.