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01
Overview
Phase 2, open-label, single-arm trial of olesoxime in spinal muscular atrophy (SMA), sponsored by Roche. Primary endpoint: Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) at 3 years. Started Jan 2016; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
Olesoxime
Olesoxime 10 mg/kg BID
03
Who it enrols
Patients with SMA who previously participated in studies TRO19622 CL E Q 1115-1 or TRO19622 CL E Q 1275-1.
04
Endpoints
Primary
Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
Secondary
Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score
Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score
Plasma Concentrations of Olesoxime
Change From Baseline in Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score
Change From Baseline in EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score - Total Score
Change From Baseline in Work Time Missed, Impairment While Working, Overall Work Impairment and Activity Impairment Assessed Using WPAI:CG Questionnaire Score
Change From Baseline in Short-Form 36 (SF-36) Physical Composite Scores (PCS) and Mental Composite Scores (MCS): Caregiver
SMA Independence Scale (SMAIS) Score: Patient
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