Phase 2
N=131
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
Muscular Atrophy, Spinal
Bottom Line
View on ClinicalTrials.gov: NCT02628743 ↗Enrolled (actual)
131
Serious AEs
27.5%
Results posted
Aug 2019
Primary outcome: Primary: Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) — 27.5; 91.6 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Olesoxime (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Dec 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) |
27.5; 91.6 | — |
| SECONDARY Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score |
30.01; -0.06; -0.31; -1.26; -3.32; -4.87 | — |
| SECONDARY Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score |
41.21; 0.31; 0.08; -0.64; -2.96; -4.02 | — |
| SECONDARY Plasma Concentrations of Olesoxime |
129.0; 9475.5; 10021.3; 10257.8; 9665.6; 10351.4 | — |
| SECONDARY Change From Baseline in Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score |
58.69; -0.80; -1.33; -0.72; -0.35; -0.11 | — |
| SECONDARY Change From Baseline in Caregiver PedsQL Generic Core Scales Version 4.0 Score |
55.58; -0.85; -1.86; -0.67; -1.64; -0.33 | — |
| SECONDARY Change From Baseline in PedsQL Neuromuscular Module Version 3.0 Scale Score |
69.57; -0.98; -0.86; -1.08; -1.18; -1.41 | — |
| SECONDARY Change From Baseline in Caregiver PedsQL Neuromuscular Module Version 3.0 Scale Score |
59.44; 0.13; -0.11; 0.65; 1.21; -0.07 | — |
| SECONDARY Change From Baseline in EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score - Total Score |
0.0471; 0.0084; -0.0164; 0.0168; 0.0081; -0.0259 | — |
| SECONDARY Change From Baseline in Caregiver Proxy EQ-5D-5L Questionnaire Index Score - Total Score |
0.0429; 0.0652; 0.0203; 0.0696; 0.0699; 0.0455 | — |
| SECONDARY Change From Baseline in EQ-5D-5L Visual Analogue Scale (EQ-5D-5L VAS) Score |
69.4; 1.3; 0.2; 1.3; 5.4; 0.6 | — |
| SECONDARY Change From Baseline in Caregiver Proxy EQ-5D-5L VAS Score |
72.7; -0.3; 0.8; 0.4; 5.7; 0.5 | — |
| SECONDARY Number of Subjects Employed Assessed Using the Work Productivity and Activity Impairment Questionnaire: Caregiver (WPAI:CG) Questionnaire |
56; 66; 58; 62; 56; 39 | — |
| SECONDARY Change From Baseline in Hours Actually Worked and Work Hours Missed Assessed Using WPAI:CG Questionnaire |
2.0; 2.0; 1.9; 0.9; 3.2; 1.1 | — |
| SECONDARY Change From Baseline in Work Time Missed, Impairment While Working, Overall Work Impairment and Activity Impairment Assessed Using WPAI:CG Questionnaire Score |
6.7; 7.0; 0.8; 6.2; 6.7; 4.7 | — |
| SECONDARY Change From Baseline in Degree Patient Caregiving Affected Productivity and Activities Using WPAI:CG Questionnaire |
29.3; -3.0; -1.5; -3.5; 0.7; -6.9 | — |
| SECONDARY Change From Baseline in Short-Form 36 (SF-36) Physical Composite Scores (PCS) and Mental Composite Scores (MCS): Caregiver |
48.07; -0.24; 0.27; -0.34; -0.48; 1.68 | — |
| SECONDARY Change From Baseline in SF-36 Domain Scores: Caregiver |
44.51; 1.73; 2.03; 1.25; 2.39; 6.02 | — |
| SECONDARY Change From Baseline in Revised Utility Index Score (SF-6D_R2): Caregiver |
0.70; -0.01; -0.01; -0.01; 0.00; 0.01 | — |
| SECONDARY SMA Independence Scale (SMAIS) Score: Patient |
71.7; 74.8 | — |
| SECONDARY SMA Independence Scale (SMAIS) Score: Caregiver |
60.1; 58.0 | — |
Summary
The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of the following two clinical studies: TRO19622 CL E Q 1115-1 (open-label Phase Ib, multicenter, single- and multiple- dose study) or TRO19622 CL E Q 1275-1 (NCT01302600, Phase II/III, adaptive, parallel-group, double blind, randomized, placebo-controlled, multicenter, multinational study).
Eligibility Criteria
Inclusion Criteria
- Participation in the previous studies (TRO19622 CL E Q 1115-1 or TRO19622 CL E Q 1275-1)
- For women of childbearing potential: agreement to use an acceptable birth control method during the treatment period and for at least 28 days after the last dose of olesoxime
Exclusion Criteria
- Female participants who are pregnant or lactating, or intending to become pregnant during the study
- Participants who, in the opinion of the investigator, are not suitable to participate in this open-label study
- Participants who have developed study drug hypersensitivity to olesoxime or one of the formulation excipients, including sesame oil
- Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening
- Concomitant or previous participation in a survival motor neuron 2 (SMN2) targeting antisense oligonucleotide study within 6 months prior to screening
- History of human immunodeficiency virus infection, history of Hepatitis B infection within the past year, history of Hepatitis C infection which has not been adequately treated
- History of illicit drug or alcohol abuse within 12 months prior to screening, in the investigator's judgment
- Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in and completion of the study
- History or presence of an abnormal electrocardiogram (ECG) that is clinically significant in the investigator's opinion
Data sourced from ClinicalTrials.gov (NCT02628743). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.