Creapharm Clinical Supplies SAS

The primary objective was to compare the effectiveness of ABX464 with placebo in achieving clinical remission.

Primary objective, Part A plus Part B (induction therapy): To evaluate the efficacy of different doses of SAR443122 in subjects with moderate to severe UC. Secondary objectives, Part A and Part B (induction therapy): 1) To evaluate the effect of SAR443122 on endoscopic improvement in subjects with UC. 2) To evaluate the effect of SAR443122 on clinical remission and clinical response in subjects with UC. 3) To evaluate the effect of SAR443122 on histological improvement in subjects with UC. 4) To evaluate the effect of SAR443122 on histological-endoscopic mucosal improvement (HEMI) in subjects with UC. 5) To evaluate the effect of SAR443122 on disease-specific quality of life. 6) To evaluate the effect of SAR443122 on patient-reported signs and symptoms of ulcerative colitis; To evaluate the pharmacokinetics of SAR443122 in subjects with UC; To evaluate the safety and tolerability of SAR443122 in subjects with moderate to severe UC for 52 weeks.

Primary objective: To determine the effect of avalglucosidase alfa treatment on survival and invasive ventilation-free survival in subjects ≤6 months of age with IOPD after 52 weeks of treatment. Secondary objectives: (1) To determine the effect of avalglucosidase alfa treatment on survival and invasive ventilation-free survival in subjects aged 12 and 18 months, as well as changes in LVM-Z score, AIMS score, length, weight and head circumference Z score, and urinary Hex4 at week 52 in patients with IOPD ≤6 months of age; (2) To determine the safety, tolerability, and immunogenicity of avalglucosidase alfa; (3) To determine the PK characteristics at weeks 12 and 52.