COACH: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children with Achondroplasia

Children with Achondroplasia

AGV (cm/year) (Week 52)., TEAEs.

AGV (cm/year) (Week 52)., AGV (cm/year) (Week 26, 104, 156). Change from baseline in height Z-score (Week 13, 26, 52, 104, 156)., Achondroplasia Child Experience Measures (Week 26, 52, 104, 156). • ACEM-OSM. ACEM-Impact. Achondroplasia Parent Experience Measure (Week 26, 52, 104, 156): APEM-Impact, Change from baseline in the following COAs (Week 26, 52, 104, 156). - QoLISSY. - SF-10. - PGIS. - PGIC. - HCRU. - CGI-S., Change from baseline of investigator reported sleep apnoea, quality of sleep and snoring pattern (Week 26, 52, 104, 156)., Change from baseline in length of long bones (e.g., femur, humerus, tibia, fibula, tibia/fibula ratio), other measurements (mechanical axis lines), and vertebral column (cobb angles, vertebral posterior element morphometry, vertebral body morphometry, spinal height) using standardized radiologic assessment (Week 52, 104, 156)., Change from baseline in body composition including body fat, lean mass, skeletal muscle and bone mineral density using DXA assessment (Week 52, 104, 156)., Change from baseline in number of rises during a 1-MSTST (Week 52, 104, 156). Change from baseline in maximal knee extensor muscle strength (Week 52, 104, 156)., Safety assessments throughout the trial (Vital signs, ECG, safety laboratory tests, physical and skeletal examinations, injection site reactions, and fundoscopy). Bone age based on X-ray (Week 26, 52, 104 and 156)., Plasma concentration of Total CNP, Free CNP and mPEG. Serum concentrations of hGH and lonapegsomatropin., Serum concentrations of IGF-1 and IGFBP3., Detection and characterisation of both antidrug and anti-prodrug antibodies.

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