Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL OLE

Achondroplasia

Incidence of treatment emergent adverse events (TEAE) and serious TEAE, Changes over time in height Z-score in relation to ACH and non-ACH growth charts.

Change over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non-ACH growth charts., Changes over time in body proportions, Changes over time in weight Z-score, Changes over time in BMI, Age of puberty onset and time to Tanner stage ≥4., Changes over time in number of episodes of otitis media per year., Changes over time in number of episodes and/or severity of sleep apnea., Changes over time in range of motion (elbow)., Changes over time in skeletal abnormalities of the lower extremities and spine, Changes in health-related quality of life (HRQoL) as assessed by Pediatric Quality of Life Inventory (PedsQL), Changes in health-related quality of life (HRQoL) as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY), Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS), Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM), Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S), Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C), Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview, Changes in cognitive functions assessed by age-appropriate computerized tests

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