ION373-CS1: A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

Alexander Disease

Percent change from Baseline to Week 61 in the 10MWT in patients who are in Stratum 1.

Change from Baseline to Week 61 or value at Week 61 for the following: • Patients' self-identified most bothersome symptom (based on a Likert scale for change; all patients) • PedsQL Generic Core Scales (all patients) • Patient Global Impression of Severity (PGIS; all patients) • Patient Global Impression of Change (PGIC; all patients) • Clinical Global Impression of Change (CGIC; all patients), Change from Baseline to Week 61 or value at Week 61 for the following: • Gross Motor Function Measure-88, Dimensions C, D and E (GMFM-88, Dimensions C-E; patients < 5 years old at Screening) or 10MWT (patients ≥ 5 years old at Screening) • 9-Hole Peg Test (9HPT; patients ≥ 8 years old at Screening) • Vineland-3 Motor Skills Domain (patients < 8 years old at Screening) • PedsQL Gastrointestinal Symptoms Scales (all patients), Change from Baseline to Week 61 or value at Week 61 for the following: Vineland-3 Adaptive Behavior Composite (ABC) Score (patients < 18 years old at Screening) • Composite Autonomic Symptom Score 31 (COMPASS-31; patients ≥ 18 years old at Screening) • CSF GFAP levels (all patients) • Clinical Global Impression of Severity (CGIS; all patients), Change from Baseline to Week 61 or value at Week 61 for the following: • Alexander Disease Patient Domain Impression of Severity (AxD-PDIS; all patients) •Alexander Disease Patient Domain Impression of Change (AxD-PDIC; all patients) • Body weight percentile (for patients < 18 years old at Screening) or body weight (for patients ≥ 18 years old at Screening

No related papers found yet.

Italy, Netherlands