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A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02628743
Recruitment Status : Completed
First Posted : December 11, 2015
Results First Posted : August 9, 2019
Last Update Posted : August 9, 2019
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Muscular Atrophy, Spinal
Intervention Drug: Olesoxime
Enrollment 131
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Olesoxime
Hide Arm/Group Description Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Period Title: Overall Study
Started 131
Completed 0
Not Completed 131
Reason Not Completed
Not Available             3
Withdrawal by Subject             50
Study Terminated by Sponsor             76
Physician Decision             2
Arm/Group Title Olesoxime
Hide Arm/Group Description Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Baseline Participants 131
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 131 participants
14.7  (5.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 131 participants
Female
66
  50.4%
Male
65
  49.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 131 participants
American Indian or Alaska Native
0
   0.0%
Asian
3
   2.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   0.8%
White
94
  71.8%
More than one race
0
   0.0%
Unknown or Not Reported
33
  25.2%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 131 participants
Hispanic or Latino 1
Not Hispanic or Latino 89
Not Stated 29
Unknown 12
1.Primary Outcome
Title Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
Hide Description [Not Specified]
Time Frame Baseline up to approximately 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included all patients who received at least one dose of study medication.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 131
Measure Type: Number
Unit of Measure: percentage of participants
SAEs 27.5
AEs 91.6
2.Secondary Outcome
Title Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 127
Mean (Standard Deviation)
Unit of Measure: percentage of maximum score
Baseline Number Analyzed 127 participants
30.01  (12.77)
Week 26 Number Analyzed 127 participants
-0.06  (4.75)
Week 52 Number Analyzed 111 participants
-0.31  (4.61)
Week 78 Number Analyzed 112 participants
-1.26  (5.64)
Week 104 Number Analyzed 99 participants
-3.32  (7.08)
Week 130 Number Analyzed 64 participants
-4.87  (9.91)
3.Secondary Outcome
Title Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population includes all participants who received at least one dose of study medication and have at least one post-baseline assessment of MFM
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 127
Mean (Standard Deviation)
Unit of Measure: percentage of maximum score
Baseline Number Analyzed 127 participants
41.21  (12.81)
Week 26 Number Analyzed 127 participants
0.31  (4.35)
Week 52 Number Analyzed 111 participants
0.08  (4.08)
Week 78 Number Analyzed 112 participants
-0.64  (5.28)
Week 104 Number Analyzed 99 participants
-2.96  (7.09)
Week 130 Number Analyzed 64 participants
-4.02  (9.48)
4.Secondary Outcome
Title Plasma Concentrations of Olesoxime
Hide Description Values are reported separately for QD and BID doses. Dose increase occurred after Week 104.
Time Frame Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
Included participants in the safety population that received at least one dose of the study drug and had measurable concentration values.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 131
Mean (Standard Deviation)
Unit of Measure: ng/mL
Baseline Number Analyzed 128 participants
129.0  (160.2)
Week 13 Number Analyzed 128 participants
9475.5  (3962.5)
Week 26 Number Analyzed 119 participants
10021.3  (4885.6)
Week 39 Number Analyzed 117 participants
10257.8  (4443.7)
Week 52 Number Analyzed 112 participants
9665.6  (4941.8)
Week 78 Number Analyzed 107 participants
10351.4  (5267.3)
Week 104 Number Analyzed 100 participants
8566.4  (5353.1)
Dose Increase Visit (BID) Number Analyzed 19 participants
14797.1  (5773.8)
Week 130 (QD) Number Analyzed 46 participants
6274.6  (6281.9)
Week 130 (BID) Number Analyzed 20 participants
13956.9  (9565.0)
5.Secondary Outcome
Title Change From Baseline in Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 128
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline (Total Score) Number Analyzed 128 participants
58.69  (11.17)
Total Score - Week 26 Number Analyzed 126 participants
-0.80  (9.10)
Total Score - Week 52 Number Analyzed 118 participants
-1.33  (10.52)
Total Score - Week 78 Number Analyzed 109 participants
-0.72  (10.98)
Total Score - Week 104 Number Analyzed 101 participants
-0.35  (11.69)
Total Score - Week 130 Number Analyzed 67 participants
-0.11  (10.83)
Baseline (Physical Score) Number Analyzed 128 participants
28.49  (16.50)
Physical Score - Week 26 Number Analyzed 126 participants
-1.76  (15.99)
Physical Score - Week 52 Number Analyzed 117 participants
-4.48  (17.68)
Physical Score - Week 78 Number Analyzed 109 participants
-3.18  (19.67)
Physical Score - Week 104 Number Analyzed 101 participants
-1.42  (17.87)
Physical Score - Week 130 Number Analyzed 67 participants
-3.23  (16.33)
Baseline (Emotional Score) Number Analyzed 128 participants
74.61  (17.34)
Emotional Score - Week 26 Number Analyzed 126 participants
-1.19  (18.62)
Emotional Score - Week 52 Number Analyzed 118 participants
1.19  (16.27)
Emotional Score - Week 78 Number Analyzed 108 participants
-0.05  (17.23)
Emotional Score - Week 104 Number Analyzed 101 participants
-0.54  (18.37)
Emotional Score - Week 130 Number Analyzed 67 participants
2.31  (18.47)
Baseline (Social Score) Number Analyzed 128 participants
72.77  (18.59)
Social Score - Week 26 Number Analyzed 126 participants
1.43  (14.15)
Social Score - Week 52 Number Analyzed 118 participants
0.93  (15.91)
Social Score - Week 78 Number Analyzed 109 participants
2.39  (17.38)
Social Score - Week 104 Number Analyzed 101 participants
2.38  (18.62)
Social Score - Week 130 Number Analyzed 67 participants
4.10  (16.05)
Baseline (School/Work Score) Number Analyzed 127 participants
77.20  (15.83)
School/Work Score - Week 26 Number Analyzed 125 participants
-1.24  (14.28)
School/Work Score - Week 52 Number Analyzed 117 participants
-1.32  (14.09)
School/Work Score - Week 78 Number Analyzed 107 participants
-0.51  (14.26)
School/Work Score - Week 104 Number Analyzed 100 participants
-1.65  (15.24)
School/Work Score - Week 130 Number Analyzed 66 participants
-2.58  (15.04)
6.Secondary Outcome
Title Change From Baseline in Caregiver PedsQL Generic Core Scales Version 4.0 Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline (Total Score) Number Analyzed 121 participants
55.58  (12.49)
Total Score - Week 26 Number Analyzed 115 participants
-0.85  (13.15)
Total Score - Week 52 Number Analyzed 106 participants
-1.86  (13.12)
Total Score - Week 78 Number Analyzed 103 participants
-0.67  (13.88)
Total Score - Week 104 Number Analyzed 93 participants
-1.64  (17.95)
Total Score - Week 130 Number Analyzed 62 participants
-0.33  (13.41)
Baseline (Physical Score) Number Analyzed 121 participants
28.99  (25.84)
Physical Score - Week 26 Number Analyzed 115 participants
-0.58  (28.66)
Physical Score - Week 52 Number Analyzed 106 participants
-5.10  (25.86)
Physical Score - Week 78 Number Analyzed 103 participants
-2.91  (28.91)
Physical Score - Week 104 Number Analyzed 93 participants
-3.28  (34.80)
Physical Score - Week 130 Number Analyzed 62 participants
-0.56  (30.09)
Baseline (Emotional Score) Number Analyzed 121 participants
67.48  (17.31)
Emotional Score - Week 26 Number Analyzed 115 participants
-2.13  (15.30)
Emotional Score - Week 52 Number Analyzed 106 participants
-0.90  (15.08)
Emotional Score - Week 78 Number Analyzed 103 participants
-0.39  (18.81)
Emotional Score - Week 104 Number Analyzed 93 participants
-0.70  (20.54)
Emotional Score - Week 130 Number Analyzed 62 participants
-0.32  (15.94)
Baseline (Social Score) Number Analyzed 121 participants
68.34  (16.19)
Social Score - Week 26 Number Analyzed 115 participants
-0.42  (16.84)
Social Score - Week 52 Number Analyzed 106 participants
-0.49  (15.92)
Social Score - Week 78 Number Analyzed 103 participants
1.47  (18.40)
Social Score - Week 104 Number Analyzed 93 participants
-0.55  (21.72)
Social Score - Week 130 Number Analyzed 61 participants
1.25  (16.22)
Baseline (School/Work Score) Number Analyzed 120 participants
73.67  (17.69)
School/Work Score - Week 26 Number Analyzed 112 participants
-0.31  (16.69)
School/Work Score - Week 52 Number Analyzed 104 participants
0.53  (18.14)
School/Work Score - Week 78 Number Analyzed 101 participants
0.94  (18.39)
School/Work Score - Week 104 Number Analyzed 91 participants
-0.71  (20.64)
School/Work Score - Week 130 Number Analyzed 59 participants
-1.53  (16.17)
7.Secondary Outcome
Title Change From Baseline in PedsQL Neuromuscular Module Version 3.0 Scale Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 90
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline (Total Score) Number Analyzed 90 participants
69.57  (14.01)
Total Score - Week 26 Number Analyzed 89 participants
-0.98  (10.75)
Total Score - Week 52 Number Analyzed 81 participants
-0.86  (12.81)
Total Score - Week 78 Number Analyzed 79 participants
-1.08  (12.11)
Total Score - Week 104 Number Analyzed 70 participants
-1.18  (13.13)
Total Score - Week 130 Number Analyzed 52 participants
-1.41  (12.61)
Baseline (Neuromuscular) Number Analyzed 90 participants
66.03  (15.39)
Neuromuscular - Week 26 Number Analyzed 89 participants
-1.77  (12.28)
Neuromuscular - Week 52 Number Analyzed 81 participants
-2.22  (14.29)
Neuromuscular - Week 78 Number Analyzed 79 participants
-2.69  (12.82)
Neuromuscular - Week 104 Number Analyzed 70 participants
-1.90  (15.11)
Neuromuscular - Week 130 Number Analyzed 52 participants
-3.14  (14.20)
Baseline (Family Resources Score) Number Analyzed 90 participants
74.39  (20.10)
Family Resources Score - Week 26 Number Analyzed 89 participants
-0.11  (17.35)
Family Resources Score - Week 52 Number Analyzed 80 participants
1.78  (16.07)
Family Resources Score - Week 78 Number Analyzed 79 participants
0.82  (19.22)
Family Resources Score - Week 104 Number Analyzed 70 participants
-1.29  (19.81)
Family Resources Score - Week 130 Number Analyzed 52 participants
-0.38  (16.36)
Baseline (Communication Score) Number Analyzed 90 participants
81.57  (21.14)
Communication Score - Week 26 Number Analyzed 89 participants
2.01  (19.86)
Communication Score - Week 52 Number Analyzed 81 participants
1.44  (24.43)
Communication Score - Week 78 Number Analyzed 79 participants
4.85  (22.19)
Communication Score - Week 104 Number Analyzed 70 participants
2.98  (24.25)
Communication Score - Week 130 Number Analyzed 52 participants
6.57  (25.15)
8.Secondary Outcome
Title Change From Baseline in Caregiver PedsQL Neuromuscular Module Version 3.0 Scale Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 88
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline (Total Score) Number Analyzed 88 participants
59.44  (14.83)
Total Score - Week 26 Number Analyzed 87 participants
0.13  (10.12)
Total Score - Week 52 Number Analyzed 78 participants
-0.11  (12.89)
Total Score - Week 78 Number Analyzed 77 participants
0.65  (13.23)
Total Score - Week 104 Number Analyzed 67 participants
1.21  (14.40)
Total Score - Week 130 Number Analyzed 51 participants
-0.07  (12.86)
Baseline (Neuromuscular) Number Analyzed 88 participants
56.28  (15.70)
Neuromuscular - Week 26 Number Analyzed 87 participants
-0.64  (10.92)
Neuromuscular - Week 52 Number Analyzed 78 participants
-1.33  (13.39)
Neuromuscular - Week 78 Number Analyzed 77 participants
0.05  (13.97)
Neuromuscular - Week 104 Number Analyzed 67 participants
0.54  (15.32)
Neuromuscular - Week 130 Number Analyzed 51 participants
-1.39  (14.65)
Baseline (Family Resources Score) Number Analyzed 88 participants
58.24  (25.08)
Family Resources Score - Week 26 Number Analyzed 86 participants
1.74  (16.57)
Family Resources Score - Week 52 Number Analyzed 77 participants
2.44  (19.04)
Family Resources Score - Week 78 Number Analyzed 77 participants
0.91  (19.31)
Family Resources Score - Week 104 Number Analyzed 67 participants
1.27  (18.63)
Family Resources Score - Week 130 Number Analyzed 50 participants
1.00  (17.26)
Baseline (Communication Score) Number Analyzed 88 participants
79.36  (22.92)
Communication Score - Week 26 Number Analyzed 87 participants
1.92  (20.08)
Communication Score - Week 52 Number Analyzed 78 participants
2.88  (22.31)
Communication Score - Week 78 Number Analyzed 77 participants
3.57  (20.92)
Communication Score - Week 104 Number Analyzed 67 participants
4.85  (24.24)
Communication Score - Week 130 Number Analyzed 51 participants
4.90  (22.74)
9.Secondary Outcome
Title Change From Baseline in EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score - Total Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 74
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline Number Analyzed 74 participants
0.0471  (0.1345)
Week 26 Number Analyzed 72 participants
0.0084  (0.1563)
Week 52 Number Analyzed 68 participants
-0.0164  (0.1191)
Week 78 Number Analyzed 66 participants
0.0168  (0.1910)
Week 104 Number Analyzed 57 participants
0.0081  (0.1701)
Week 130 Number Analyzed 34 participants
-0.0259  (0.1232)
10.Secondary Outcome
Title Change From Baseline in Caregiver Proxy EQ-5D-5L Questionnaire Index Score - Total Score
Hide Description 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.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 54
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline Number Analyzed 54 participants
0.0429  (0.2335)
Week 26 Number Analyzed 50 participants
0.0652  (0.4091)
Week 52 Number Analyzed 47 participants
0.0203  (0.2820)
Week 78 Number Analyzed 47 participants
0.0696  (0.3094)
Week 104 Number Analyzed 39 participants
0.0699  (0.2887)
Week 130 Number Analyzed 31 participants
0.0455  (0.3323)
11.Secondary Outcome
Title Change From Baseline in EQ-5D-5L Visual Analogue Scale (EQ-5D-5L VAS) Score
Hide Description 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. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 75
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline Number Analyzed 75 participants
69.4  (19.7)
Week 26 Number Analyzed 73 participants
1.3  (17.1)
Week 52 Number Analyzed 69 participants
0.2  (17.4)
Week 78 Number Analyzed 67 participants
1.3  (19.9)
Week 104 Number Analyzed 58 participants
5.4  (17.0)
Week 130 Number Analyzed 35 participants
0.6  (18.1)
12.Secondary Outcome
Title Change From Baseline in Caregiver Proxy EQ-5D-5L VAS Score
Hide Description 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. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. Questionnaire was completed by the caregiver.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 54
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline Number Analyzed 54 participants
72.7  (21.4)
Week 26 Number Analyzed 49 participants
-0.3  (27.7)
Week 52 Number Analyzed 47 participants
0.8  (21.2)
Week 78 Number Analyzed 47 participants
0.4  (20.9)
Week 104 Number Analyzed 40 participants
5.7  (24.3)
Week 130 Number Analyzed 31 participants
0.5  (17.9)
13.Secondary Outcome
Title Number of Subjects Employed Assessed Using the Work Productivity and Activity Impairment Questionnaire: Caregiver (WPAI:CG) Questionnaire
Hide Description The WPAI:CG consists of four questions about the effects of Spinal Muscular Atrophy (SMA) on the following: employment status, hours missed due to patient caregiving, hours missed due to other reasons, hours actually worked and two questions that measure the degree to which patient caregiving affected productivity and regular daily activities.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 125
Measure Type: Number
Unit of Measure: participants
Baseline Number Analyzed 121 participants
56
Week 26 Number Analyzed 119 participants
66
Week 52 Number Analyzed 109 participants
58
Week 78 Number Analyzed 107 participants
62
Week 104 Number Analyzed 97 participants
56
Week 130 Number Analyzed 65 participants
39
14.Secondary Outcome
Title Change From Baseline in Hours Actually Worked and Work Hours Missed Assessed Using WPAI:CG Questionnaire
Hide Description The WPAI:CG consists of four questions about the effects of SMA on the following: employment status, hours missed due to patient caregiving (HMC), hours missed due to other reasons (HMO), hours actually worked (HAW) and two questions that measure the degree to which patient caregiving affected productivity and regular daily activities.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 56
Mean (Standard Deviation)
Unit of Measure: hours
Baseline (HMC) Number Analyzed 56 participants
2.0  (3.7)
HMC, Week 26 Number Analyzed 50 participants
2.0  (7.3)
HMC, Week 52 Number Analyzed 44 participants
1.9  (8.2)
HMC, Week 78 Number Analyzed 49 participants
0.9  (5.6)
HMC, Week 104 Number Analyzed 45 participants
3.2  (8.3)
HMC, Week 130 Number Analyzed 35 participants
1.1  (8.1)
Baseline (HMO) Number Analyzed 56 participants
4.8  (7.3)
HMO, Week 26 Number Analyzed 51 participants
-1.5  (7.9)
HMO, Week 52 Number Analyzed 43 participants
-1.5  (9.0)
HMO, Week 78 Number Analyzed 49 participants
-0.1  (8.6)
HMO, Week 104 Number Analyzed 45 participants
0.0  (9.6)
HMO, Week 130 Number Analyzed 35 participants
0.8  (7.8)
Baseline (HAW) Number Analyzed 56 participants
36.0  (47.0)
HAW, Week 26 Number Analyzed 51 participants
-12.0  (46.7)
HAW, Week 52 Number Analyzed 43 participants
-8.3  (49.2)
HAW, Week 78 Number Analyzed 48 participants
-8.6  (49.8)
HAW, Week 104 Number Analyzed 44 participants
1.2  (14.4)
HAW, Week 130 Number Analyzed 35 participants
1.5  (27.5)
15.Secondary Outcome
Title Change From Baseline in Work Time Missed, Impairment While Working, Overall Work Impairment and Activity Impairment Assessed Using WPAI:CG Questionnaire Score
Hide Description The WPAI:CG consists of four questions about the effects of Spinal Muscular Atrophy (SMA) on the following: employment status, hours missed due to patient caregiving, hours missed due to other reasons, hours actually worked and two questions that measure the degree to which patient caregiving affected productivity and regular daily activities. WPAI:CG outcomes are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity. The outcomes are presented for Percent work time missed (WTM), Percent impairment (IMP), Percent overall work impairment (OWI) and Percent activity impairment (AIM).
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: percentage
Baseline (WTM) Number Analyzed 53 participants
6.7  (11.9)
WTM, Week 26 Number Analyzed 44 participants
7.0  (22.3)
WTM, Week 52 Number Analyzed 38 participants
0.8  (15.4)
WTM, Week 78 Number Analyzed 43 participants
6.2  (23.3)
WTM, Week 104 Number Analyzed 39 participants
6.7  (20.1)
WTM, Week 130 Number Analyzed 31 participants
4.7  (24.4)
Baseline (IMP) Number Analyzed 56 participants
29.3  (26.6)
IMP, Week 26 Number Analyzed 50 participants
-3.0  (22.8)
IMP, Week 52 Number Analyzed 46 participants
-1.5  (27.3)
IMP, Week 78 Number Analyzed 49 participants
-3.5  (25.0)
IMP, Week 104 Number Analyzed 45 participants
0.7  (22.8)
IMP, Week 130 Number Analyzed 35 participants
-6.9  (24.7)
Baseline (OWI) Number Analyzed 53 participants
33.8  (28.1)
OWI, Week 26 Number Analyzed 43 participants
-3.6  (25.3)
OWI, Week 52 Number Analyzed 38 participants
-3.3  (25.4)
OWI, Week 78 Number Analyzed 43 participants
-3.0  (31.9)
OWI, Week 104 Number Analyzed 39 participants
3.5  (26.2)
OWI, Week 130 Number Analyzed 31 participants
-3.4  (33.3)
Baseline (AIM) Number Analyzed 121 participants
45.8  (29.3)
AIM, Week 26 Number Analyzed 114 participants
-3.9  (28.7)
AIM, Week 52 Number Analyzed 106 participants
-1.3  (30.6)
AIM, Week 78 Number Analyzed 105 participants
-6.3  (32.6)
AIM, Week 104 Number Analyzed 94 participants
-7.6  (27.8)
AIM, Week 130 Number Analyzed 64 participants
-5.5  (31.7)
16.Secondary Outcome
Title Change From Baseline in Degree Patient Caregiving Affected Productivity and Activities Using WPAI:CG Questionnaire
Hide Description The WPAI:CG consists of four questions about the effects of Spinal Muscular Atrophy (SMA) on the following: employment status, hours missed due to patient caregiving, hours missed due to other reasons, hours actually worked and two questions that measure the degree to which patient caregiving affected productivity and regular daily activities. WPAI:CG outcomes are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: percentage of impairment
Baseline (Productivity) Number Analyzed 56 participants
29.3  (26.6)
Productivity, Week 26 Number Analyzed 50 participants
-3.0  (22.8)
Productivity, Week 52 Number Analyzed 46 participants
-1.5  (27.3)
Productivity, Week 78 Number Analyzed 49 participants
-3.5  (25.0)
Productivity, Week 104 Number Analyzed 45 participants
0.7  (22.8)
Productivity, Week 130 Number Analyzed 35 participants
-6.9  (24.7)
Baseline (Activities) Number Analyzed 121 participants
45.8  (29.3)
Activities, Week 26 Number Analyzed 114 participants
-3.9  (28.7)
Activities, Week 52 Number Analyzed 106 participants
-1.3  (30.6)
Activities, Week 78 Number Analyzed 105 participants
-6.3  (32.6)
Activities, Week 104 Number Analyzed 94 participants
-7.6  (27.8)
Activities, Week 130 Number Analyzed 64 participants
-5.5  (31.7)
17.Secondary Outcome
Title Change From Baseline in Short-Form 36 (SF-36) Physical Composite Scores (PCS) and Mental Composite Scores (MCS): Caregiver
Hide Description The SF-36 was used to assess health-related quality of life at baseline and at on-treatment visits. The SF-36 consisted of 36 questions covering 8 domains (physical functioning, role-functioning physical, bodily pain, general health, vitality, social functioning, role-functioning emotional and mental health), with each domain scoring on a scale 0-100 (a score of 0 = maximum disability and a score of 100 = no disability). The 8 domains are further summarized to 2 distinct higher-ordered clusters: the physical and mental composite t-scores (PCS and MCS). The range for all 8 domains as well as for the composite norm-based t-scores is from 0 to 100 with 100 as best possible health status and 0 as worst health status. Reported here are the Physical Composite Scores (PCS) and Mental Composite Scores (MCS).
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 101
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline (PCS) Number Analyzed 99 participants
48.07  (10.33)
PCS, Week 26 Number Analyzed 95 participants
-0.24  (10.95)
PCS, Week 52 Number Analyzed 87 participants
0.27  (9.73)
PCS, Week 78 Number Analyzed 88 participants
-0.34  (10.87)
PCS, Week 104 Number Analyzed 77 participants
-0.48  (10.30)
PCS, Week 130 Number Analyzed 56 participants
1.68  (12.94)
Baseline (MCS) Number Analyzed 101 participants
49.71  (10.52)
MCS, Week 26 Number Analyzed 97 participants
-1.05  (10.22)
MCS, Week 52 Number Analyzed 89 participants
-1.64  (10.06)
MCS, Week 78 Number Analyzed 90 participants
-0.21  (9.30)
MCS, Week 104 Number Analyzed 79 participants
-0.08  (7.73)
MCS, Week 130 Number Analyzed 57 participants
-1.21  (9.98)
18.Secondary Outcome
Title Change From Baseline in SF-36 Domain Scores: Caregiver
Hide Description The SF-36 was used to assess health-related quality of life at baseline and at on-treatment visits. The SF-36 consisted of 36 questions covering 8 domains (physical functioning, role-functioning physical, bodily pain, general health, vitality, social functioning, role-functioning emotional and mental health), with each domain scoring on a scale 0-100 (a score of 0 = maximum disability and a score of 100 = no disability). The range for all 8 norm-based domains was from 0 to 100 with 100 as best possible health status and 0 as worst health status.
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 101
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline (Physical) Number Analyzed 99 participants
44.51  (15.97)
Physical, Week 26 Number Analyzed 95 participants
1.73  (16.36)
Physical, Week 52 Number Analyzed 87 participants
2.03  (14.23)
Physical, Week 78 Number Analyzed 88 participants
1.25  (16.16)
Physical, Week 104 Number Analyzed 77 participants
2.39  (16.61)
Physical, Week 130 Number Analyzed 56 participants
6.02  (18.87)
Baseline (Role (Physical)) Number Analyzed 101 participants
47.95  (8.83)
Role (physical), Week 26 Number Analyzed 97 participants
-1.18  (10.29)
Role (physical), Week 52 Number Analyzed 89 participants
-0.76  (9.45)
Role (physical), Week 78 Number Analyzed 90 participants
-1.02  (9.82)
Role (physical), Week 104 Number Analyzed 79 participants
-1.05  (8.92)
Role (physical), Week 130 Number Analyzed 57 participants
-0.35  (10.75)
Baseline (Bodily pain) Number Analyzed 101 participants
50.94  (8.77)
Bodily pain, Week 26 Number Analyzed 97 participants
-1.82  (8.75)
Bodily pain, Week 52 Number Analyzed 89 participants
-1.83  (9.04)
Bodily pain, Week 78 Number Analyzed 90 participants
-2.14  (10.55)
Bodily pain, Week 104 Number Analyzed 79 participants
-2.40  (8.44)
Bodily pain, Week 130 Number Analyzed 57 participants
-2.96  (9.82)
Baseline (General health) Number Analyzed 101 participants
50.21  (10.65)
General health, Week 26 Number Analyzed 97 participants
-0.63  (10.59)
General health, Week 52 Number Analyzed 89 participants
-0.75  (9.64)
General health, Week 78 Number Analyzed 90 participants
-0.22  (10.83)
General health, Week 104 Number Analyzed 79 participants
-1.17  (11.22)
General health, Week 130 Number Analyzed 57 participants
1.07  (11.87)
Baseline (Vitality) Number Analyzed 101 participants
51.28  (9.42)
Vitality, Week 26 Number Analyzed 97 participants
-1.04  (8.48)
Vitality, Week 52 Number Analyzed 89 participants
-1.23  (7.53)
Vitality, Week 78 Number Analyzed 90 participants
-0.03  (9.67)
Vitality, Week 104 Number Analyzed 79 participants
-0.07  (8.16)
Vitality, Week 130 Number Analyzed 57 participants
0.37  (8.57)
Baseline (Social) Number Analyzed 101 participants
48.01  (9.49)
Social, Week 26 Number Analyzed 97 participants
-0.67  (10.60)
Social, Week 52 Number Analyzed 89 participants
-1.24  (11.14)
Social, Week 78 Number Analyzed 90 participants
0.28  (10.29)
Social, Week 104 Number Analyzed 79 participants
-0.95  (9.12)
Social, Week 130 Number Analyzed 57 participants
0.35  (10.46)
Baseline (Role (emotional)) Number Analyzed 101 participants
48.38  (9.58)
Role (emotional), Week 26 Number Analyzed 97 participants
-1.15  (10.59)
Role (emotional), Week 52 Number Analyzed 89 participants
-1.72  (11.38)
Role (emotional), Week 78 Number Analyzed 90 participants
-1.16  (10.58)
Role (emotional), Week 104 Number Analyzed 79 participants
-1.06  (8.03)
Role (emotional), Week 130 Number Analyzed 57 participants
-0.43  (9.87)
Baseline (Mental health) Number Analyzed 101 participants
48.46  (9.39)
Mental health, Week 26 Number Analyzed 97 participants
-0.24  (9.73)
Mental health, Week 52 Number Analyzed 89 participants
-0.50  (9.15)
Mental health, Week 78 Number Analyzed 90 participants
0.07  (7.77)
Mental health, Week 104 Number Analyzed 79 participants
1.32  (7.42)
Mental health, Week 130 Number Analyzed 57 participants
-0.29  (9.21)
19.Secondary Outcome
Title Change From Baseline in Revised Utility Index Score (SF-6D_R2): Caregiver
Hide Description The SF-6D focuses on seven of the eight health domains covered by the SF-36: physical functioning, role participation (combined role-physical and role-emotional), social functioning, bodily pain, mental health, and vitality. SF-6D Health Utility Index (HUI) Score = 0 (worst measured health state) to 1 (best measured health state).
Time Frame Baseline (Week 1), Weeks 26, 52, 78, 104 and 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 98
Mean (Standard Deviation)
Unit of Measure: score on scale
Baseline Number Analyzed 98 participants
0.70  (0.11)
Week 26 Number Analyzed 92 participants
-0.01  (0.12)
Week 52 Number Analyzed 86 participants
-0.01  (0.10)
Week 78 Number Analyzed 87 participants
-0.01  (0.12)
Week 104 Number Analyzed 76 participants
0.00  (0.11)
Week 130 Number Analyzed 54 participants
0.01  (0.13)
20.Secondary Outcome
Title SMA Independence Scale (SMAIS) Score: Patient
Hide Description The SMAIS was developed specifically for SMA in order to assess function-related independence. The SMAIS contains 29 items, assessing the amount of assistance required from another individual to perform daily activities, such as eating or transferring to/from a wheelchair. Each item is scored on a zero to four scale (with an additional option to indicate that an item is non-applicable). Item scores are summed to create the total score. The range of total score is between 0 and 116. Lower scores indicate greater dependence on another individual.
Time Frame Week 104 and Week 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: score on scale
Week 104 Number Analyzed 14 participants
71.7  (23.4)
Week 130 Number Analyzed 12 participants
74.8  (22.5)
21.Secondary Outcome
Title SMA Independence Scale (SMAIS) Score: Caregiver
Hide Description The SMAIS was developed specifically for SMA in order to assess function-related independence. The SMAIS contains 29 items, assessing the amount of assistance required from another individual to perform daily activities, such as eating or transferring to/from a wheelchair. Each item is scored on a zero to four scale (with an additional option to indicate that an item is non-applicable). Item scores are summed to create the total score. The range of total score is between 0 and 116. Lower scores indicate greater dependence on another individual.
Time Frame Week 104 and Week 130
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Olesoxime
Hide Arm/Group Description:
Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
Overall Number of Participants Analyzed 21
Mean (Standard Deviation)
Unit of Measure: score on scale
Week 104 Number Analyzed 20 participants
60.1  (22.2)
Week 130 Number Analyzed 17 participants
58.0  (24.9)
Time Frame Baseline up to approximately 3 years
Adverse Event Reporting Description Safety population included all patients who received at least one dose of study medication.
 
Arm/Group Title Olesoxime
Hide Arm/Group Description Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube.
All-Cause Mortality
Olesoxime
Affected / at Risk (%)
Total   0/131 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Olesoxime
Affected / at Risk (%) # Events
Total   36/131 (27.48%)    
Gastrointestinal disorders   
CONSTIPATION * 1  1/131 (0.76%)  1
VOMITING * 1  1/131 (0.76%)  1
GASTRITIS * 1  1/131 (0.76%)  1
Infections and infestations   
BRONCHITIS * 1  3/131 (2.29%)  5
GASTROENTERITIS * 1  2/131 (1.53%)  2
UPPER RESPIRATORY TRACT INFECTION * 1  2/131 (1.53%)  3
ENCEPHALITIS * 1  1/131 (0.76%)  1
GASTROINTESTINAL INFECTION * 1  1/131 (0.76%)  1
LOWER RESPIRATORY TRACT INFECTION * 1  4/131 (3.05%)  7
LUNG INFECTION * 1  2/131 (1.53%)  2
OSTEOMYELITIS * 1  1/131 (0.76%)  1
PNEUMONIA * 1  10/131 (7.63%)  13
PNEUMONIA VIRAL * 1  1/131 (0.76%)  1
POSTOPERATIVE WOUND INFECTION * 1  1/131 (0.76%)  1
RESPIRATORY TRACT INFECTION * 1  1/131 (0.76%)  3
URINARY TRACT INFECTION * 1  1/131 (0.76%)  1
VIRAL UPPER RESPIRATORY TRACT INFECTION * 1  1/131 (0.76%)  1
Injury, poisoning and procedural complications   
ACCIDENTAL OVERDOSE * 1  1/131 (0.76%)  1
FALL * 1  1/131 (0.76%)  1
FEMORAL NECK FRACTURE * 1  1/131 (0.76%)  1
FEMUR FRACTURE * 1  2/131 (1.53%)  2
HUMERUS FRACTURE * 1  1/131 (0.76%)  1
JOINT DISLOCATION * 1  1/131 (0.76%)  1
POSTOPERATIVE WOUND COMPLICATION * 1  1/131 (0.76%)  1
PROCEDURAL HAEMORRHAGE * 1  1/131 (0.76%)  1
PROCEDURAL PAIN * 1  1/131 (0.76%)  1
PROCEDURAL PNEUMOTHORAX * 1  1/131 (0.76%)  1
THERMAL BURN * 1  1/131 (0.76%)  1
Metabolism and nutrition disorders   
FOOD REFUSAL * 1  1/131 (0.76%)  1
Musculoskeletal and connective tissue disorders   
ARTHRALGIA * 1  1/131 (0.76%)  1
PATHOLOGICAL FRACTURE * 1  1/131 (0.76%)  1
Psychiatric disorders   
ANXIETY * 1  1/131 (0.76%)  1
DEPRESSED MOOD * 1  1/131 (0.76%)  1
Renal and urinary disorders   
NEPHROLITHIASIS * 1  1/131 (0.76%)  1
Reproductive system and breast disorders   
TESTICULAR TORSION * 1  1/131 (0.76%)  1
Respiratory, thoracic and mediastinal disorders   
DYSPNOEA * 1  1/131 (0.76%)  1
LUNG DISORDER * 1  1/131 (0.76%)  1
OBSTRUCTIVE AIRWAYS DISORDER * 1  1/131 (0.76%)  1
RESPIRATORY DISTRESS * 1  1/131 (0.76%)  1
RESPIRATORY FAILURE * 1  2/131 (1.53%)  4
Surgical and medical procedures   
MEDICAL DEVICE REMOVAL * 1  1/131 (0.76%)  1
SKIN GRAFT * 1  1/131 (0.76%)  1
1
Term from vocabulary, MedDRA version 21.1
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Olesoxime
Affected / at Risk (%) # Events
Total   105/131 (80.15%)    
Gastrointestinal disorders   
ABDOMINAL PAIN UPPER * 1  8/131 (6.11%)  12
CONSTIPATION * 1  9/131 (6.87%)  13
DIARRHOEA * 1  20/131 (15.27%)  34
NAUSEA * 1  9/131 (6.87%)  13
VOMITING * 1  23/131 (17.56%)  43
General disorders   
PYREXIA * 1  28/131 (21.37%)  40
Infections and infestations   
BRONCHITIS * 1  15/131 (11.45%)  22
GASTROENTERITIS * 1  13/131 (9.92%)  18
INFLUENZA * 1  13/131 (9.92%)  14
NASOPHARYNGITIS * 1  30/131 (22.90%)  57
PHARYNGITIS * 1  7/131 (5.34%)  9
RHINITIS * 1  10/131 (7.63%)  15
UPPER RESPIRATORY TRACT INFECTION * 1  36/131 (27.48%)  63
Musculoskeletal and connective tissue disorders   
ARTHRALGIA * 1  12/131 (9.16%)  14
BACK PAIN * 1  9/131 (6.87%)  16
MUSCULOSKELETAL PAIN * 1  8/131 (6.11%)  8
Nervous system disorders   
HEADACHE * 1  23/131 (17.56%)  44
Respiratory, thoracic and mediastinal disorders   
COUGH * 1  17/131 (12.98%)  23
OROPHARYNGEAL PAIN * 1  16/131 (12.21%)  29
Skin and subcutaneous tissue disorders   
RASH * 1  8/131 (6.11%)  10
1
Term from vocabulary, MedDRA version 21.1
*
Indicates events were collected by non-systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Communications
Organization: Hoffmann-La Roche
Phone: 800 821-8590
EMail: genentech@druginfo.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02628743     History of Changes
Other Study ID Numbers: BN29854
2015-001589-25 ( EudraCT Number )
First Submitted: December 1, 2015
First Posted: December 11, 2015
Results First Submitted: June 12, 2019
Results First Posted: August 9, 2019
Last Update Posted: August 9, 2019