Trial · NCT02628743
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
NCT02628743, Phase 2, Completed, Hoffmann-La Roche; results first posted 2019-08-09
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA). Trials, record NCT02628743. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02628743
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
- NCT number
- NCT02628743
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 131 participants, actual
- Lead sponsor
- Hoffmann-La Roche
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Olesoxime
- First posted
- 2015-12-11
- Last updated
- 2019-08-09
- Other registries' IDs
- eudract:2015-001589-25
- Results first posted
- 2019-08-09
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) | Baseline up to approximately 3 years | |
| Secondary | Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The MFM scale evaluated motor function in three dimensions. D1 evaluates functions related to standing and transfer, D2 evaluates axial and proximal function in supine and sitting position on mat and chair and D3 evaluates distal motor function. The scoring of each task uses a 4-point Likert scale based on the participant's maximal abilities without assistance: 0, cannot initiate the task or maintain the starting position; 1, performs the task partially; 2, performs the task incompletely or imperfectly (with compensatory/uncontrolled movements or slowness); and 3, performs the task fully and "normally". The scores are summed to yield a total score expressed as the percentage of the maximum possible score (the one obtained with no physical impairment); the lower the total score, the more severe the impairment. |
| Secondary | Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The MFM scale evaluated motor function in three dimensions. D1 evaluates functions related to standing and transfer, D2 evaluates axial and proximal function in supine and sitting position on mat and chair and D3 evaluates distal motor function. The scoring of each task uses a 4-point Likert scale based on the participant's maximal abilities without assistance: 0, cannot initiate the task or maintain the starting position; 1, performs the task partially; 2, performs the task incompletely or imperfectly (with compensatory/uncontrolled movements or slowness); and 3, performs the task fully and "normally". The scores are summed to yield a total score expressed as the percentage of the maximum possible score (the one obtained with no physical impairment); the lower the total score, the more severe the impairment. |
| Secondary | Plasma Concentrations of Olesoxime | Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104 and 130 | Values are reported separately for QD and BID doses. Dose increase occurred after Week 104. |
| Secondary | Change From Baseline in Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The PedsQL Generic Core Scale includes 23 items using self-report and/or parent report (ages 5+). The instrument covers physical, emotional, social and school functioning. Scale items are linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, and 4 = 0) so that higher scores indicate better health related quality of life. |
| Secondary | Change From Baseline in Caregiver PedsQL Generic Core Scales Version 4.0 Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The PedsQL Generic Core Scale includes 23 items. The instrument covers physical, emotional, social and school functioning. Scale items are linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, and 4 = 0) so that higher scores indicate better health related quality of life. Questionnaire was completed by the caregiver. |
| Secondary | Change From Baseline in PedsQL Neuromuscular Module Version 3.0 Scale Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The PedsQL Neuromuscular Module (Version 3.0) includes 25 items using self-report (ages 5 - 18) and/or parent report (ages 5 -18). The instrument covers problems related to neuromuscular disease, communication and family resources. Scale items are linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, and 4 = 0) so that higher scores indicate better health related quality of life. |
| Secondary | Change From Baseline in Caregiver PedsQL Neuromuscular Module Version 3.0 Scale Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The PedsQL Neuromuscular Module (Version 3.0) includes 25 items using self-report (ages 5 - 18) and/or parent report (ages 5 -18). The instrument covers problems related to neuromuscular disease, communication and family resources. Scale items are linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, and 4 = 0) so that higher scores indicate better health related quality of life. Questionnaire was completed by the caregiver. |
| Secondary | Change From Baseline in EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score - Total Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The EQ-5D-5L is a self-reported health status questionnaire that consists of six questions used to calculate a health utility score for use in health economic analysis. There are two components to the EQ-5D-5L: a five-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Overall scores range from 0 to 1, with low scores representing a higher level of dysfunction. |
| Secondary | Change From Baseline in Caregiver Proxy EQ-5D-5L Questionnaire Index Score - Total Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | The EQ-5D-5L is a self-reported health status questionnaire that consists of six questions used to calculate a health utility score for use in health economic analysis. There are two components to the EQ-5D-5L: a five-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Overall scores range from 0 to 1, with low scores representing a higher level of dysfunction. The questionnaire was completed by the caregiver. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) | Baseline up to approximately 3 years | Safety population included all patients who received at least one dose of study medication. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | Intent-to-Treat Population (ITT) includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM. | percentage of maximum score | MEAN |
| SECONDARY | Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | ITT population includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM | percentage of maximum score | MEAN |
| SECONDARY | Plasma Concentrations of Olesoxime | Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104 and 130 | Included participants in the safety population that received at least one dose of the study drug and had measurable concentration values. | ng/mL | MEAN |
| SECONDARY | Change From Baseline in Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | Intent-to-Treat Population (ITT) includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM. | score on scale | MEAN |
| SECONDARY | Change From Baseline in Caregiver PedsQL Generic Core Scales Version 4.0 Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | Intent-to-Treat Population (ITT) includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM. Scale scores were not computed if more than 50% of items in the scale were missing. | score on scale | MEAN |
| SECONDARY | Change From Baseline in PedsQL Neuromuscular Module Version 3.0 Scale Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | Intent-to-Treat Population (ITT) includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM. Scale scores were not computed if more than 50% of items in the scale were missing. | score on scale | MEAN |
| SECONDARY | Change From Baseline in Caregiver PedsQL Neuromuscular Module Version 3.0 Scale Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | Intent-to-Treat Population (ITT) includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM. Scale scores were not computed if more than 50% of items in the scale were missing. | score on scale | MEAN |
| SECONDARY | Change From Baseline in EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score - Total Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | Intent-to-Treat Population (ITT) includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM. The number analyzed represents participants who completed the questionnaire. | score on scale | MEAN |
| SECONDARY | Change From Baseline in Caregiver Proxy EQ-5D-5L Questionnaire Index Score - Total Score | Baseline (Week 1), Weeks 26, 52, 78, 104 and 130 | Intent-to-Treat Population (ITT) includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM. The number analyzed represents participants who completed the questionnaire. | score on scale | MEAN |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group286
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) | Olesoxime | SAEs | 27.5 | percentage of participants | |
| Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) | Olesoxime | AEs | 91.6 | percentage of participants | |
| Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Olesoxime | Baseline | 30.01 | Standard Deviation 12.77 | percentage of maximum score |
| Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Olesoxime | Week 26 | -0.06 | Standard Deviation 4.75 | percentage of maximum score |
| Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Olesoxime | Week 52 | -0.31 | Standard Deviation 4.61 | percentage of maximum score |
| Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Olesoxime | Week 78 | -1.26 | Standard Deviation 5.64 | percentage of maximum score |
| Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Olesoxime | Week 104 | -3.32 | Standard Deviation 7.08 | percentage of maximum score |
| Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score | Olesoxime | Week 130 | -4.87 | Standard Deviation 9.91 | percentage of maximum score |
| Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score | Olesoxime | Baseline | 41.21 | Standard Deviation 12.81 | percentage of maximum score |
| Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score | Olesoxime | Week 26 | 0.31 | Standard Deviation 4.35 | percentage of maximum score |
1 to 10 of 286Source ClinicalTrials.gov
See all 286 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Olesoxime | 131 | |
| Overall Study | COMPLETED | Olesoxime | 0 | |
| Overall Study | NOT COMPLETED | Olesoxime | 131 |
Source ClinicalTrials.gov
Adverse events62
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | ABDOMINAL PAIN UPPER | Gastrointestinal disorders | Olesoxime | 8 | 131 | 12 |
| other | CONSTIPATION | Gastrointestinal disorders | Olesoxime | 9 | 131 | 13 |
| other | DIARRHOEA | Gastrointestinal disorders | Olesoxime | 20 | 131 | 34 |
| other | NAUSEA | Gastrointestinal disorders | Olesoxime | 9 | 131 | 13 |
| other | VOMITING | Gastrointestinal disorders | Olesoxime | 23 | 131 | 43 |
| other | PYREXIA | General disorders | Olesoxime | 28 | 131 | 40 |
| other | BRONCHITIS | Infections and infestations | Olesoxime | 15 | 131 | 22 |
| other | GASTROENTERITIS | Infections and infestations | Olesoxime | 13 | 131 | 18 |
| other | INFLUENZA | Infections and infestations | Olesoxime | 13 | 131 | 14 |
| other | NASOPHARYNGITIS | Infections and infestations | Olesoxime | 30 | 131 | 57 |
1 to 10 of 62Source ClinicalTrials.gov
See all 62 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Olesoxime | 0 | 131 |
| Total, other adverse events | Olesoxime | 105 | 131 |
| Total, serious adverse events | Olesoxime | 36 | 131 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36288197 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02628743
GET https://conductscience.org/api/trials/v1/NCT02628743/registered?page=2