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

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ClinicalTrials.gov Identifier: NCT02628743

Recruitment Status : Completed

First Posted : December 11, 2015

Results First Posted : August 9, 2019

Last Update Posted : August 9, 2019

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Hoffmann-La Roche

Study Type	Interventional
Study Design	Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition	Muscular Atrophy, Spinal
Intervention	Drug: Olesoxime
Enrollment	131

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Olesoxime
Arm/Group Description	Participants received a dose of 10 ...
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
Other	3
Withdrawal by Subject	50
Study Terminated by Sponsor	76
Physician Decision	2

Baseline Characteristics

Arm/Group Title		Olesoxime
Arm/Group Description		Participants received a dose of 10 ...
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
Baseline Analysis Population Description		[Not Specified]
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

Outcome Measures

1.Primary Outcome


Title	Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
Description	[Not Specified]
Description	[Not Specified]
Time Frame	Baseline up to approximately 3 years

Outcome Measure Data

Analysis Population Description

Safety population included all patients who received at least one dose of study medication.


Arm/Group Title	Olesoxime
Arm/Group Description:	Participants received a dose of 10 ...
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
Description	The MFM scale evaluated motor function in three dimensions. D1 evaluat...
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

Outcome Measure Data

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
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		30.01 (12.77)
Week 26	Number Analyzed	127 participants
Week 26		-0.06 (4.75)
Week 52	Number Analyzed	111 participants
Week 52		-0.31 (4.61)
Week 78	Number Analyzed	112 participants
Week 78		-1.26 (5.64)
Week 104	Number Analyzed	99 participants
Week 104		-3.32 (7.08)
Week 130	Number Analyzed	64 participants
Week 130		-4.87 (9.91)

3.Secondary Outcome


Title	Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score
Description	The MFM scale evaluated motor function in three dimensions. D1 evaluat...
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

Outcome Measure Data

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
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		41.21 (12.81)
Week 26	Number Analyzed	127 participants
Week 26		0.31 (4.35)
Week 52	Number Analyzed	111 participants
Week 52		0.08 (4.08)
Week 78	Number Analyzed	112 participants
Week 78		-0.64 (5.28)
Week 104	Number Analyzed	99 participants
Week 104		-2.96 (7.09)
Week 130	Number Analyzed	64 participants
Week 130		-4.02 (9.48)

4.Secondary Outcome


Title	Plasma Concentrations of Olesoxime
Description	Values are reported separately for QD and BID doses. Dose increase occ...
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

Outcome Measure Data

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
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		129.0 (160.2)
Week 13	Number Analyzed	128 participants
Week 13		9475.5 (3962.5)
Week 26	Number Analyzed	119 participants
Week 26		10021.3 (4885.6)
Week 39	Number Analyzed	117 participants
Week 39		10257.8 (4443.7)
Week 52	Number Analyzed	112 participants
Week 52		9665.6 (4941.8)
Week 78	Number Analyzed	107 participants
Week 78		10351.4 (5267.3)
Week 104	Number Analyzed	100 participants
Week 104		8566.4 (5353.1)
Dose Increase Visit (BID)	Number Analyzed	19 participants
Dose Increase Visit (BID)		14797.1 (5773.8)
Week 130 (QD)	Number Analyzed	46 participants
Week 130 (QD)		6274.6 (6281.9)
Week 130 (BID)	Number Analyzed	20 participants
Week 130 (BID)		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
Description	The PedsQL Generic Core Scale includes 23 items using self-report and/...
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

Outcome Measure Data

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
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (Total Score)		58.69 (11.17)
Total Score - Week 26	Number Analyzed	126 participants
Total Score - Week 26		-0.80 (9.10)
Total Score - Week 52	Number Analyzed	118 participants
Total Score - Week 52		-1.33 (10.52)
Total Score - Week 78	Number Analyzed	109 participants
Total Score - Week 78		-0.72 (10.98)
Total Score - Week 104	Number Analyzed	101 participants
Total Score - Week 104		-0.35 (11.69)
Total Score - Week 130	Number Analyzed	67 participants
Total Score - Week 130		-0.11 (10.83)
Baseline (Physical Score)	Number Analyzed	128 participants
Baseline (Physical Score)		28.49 (16.50)
Physical Score - Week 26	Number Analyzed	126 participants
Physical Score - Week 26		-1.76 (15.99)
Physical Score - Week 52	Number Analyzed	117 participants
Physical Score - Week 52		-4.48 (17.68)
Physical Score - Week 78	Number Analyzed	109 participants
Physical Score - Week 78		-3.18 (19.67)
Physical Score - Week 104	Number Analyzed	101 participants
Physical Score - Week 104		-1.42 (17.87)
Physical Score - Week 130	Number Analyzed	67 participants
Physical Score - Week 130		-3.23 (16.33)
Baseline (Emotional Score)	Number Analyzed	128 participants
Baseline (Emotional Score)		74.61 (17.34)
Emotional Score - Week 26	Number Analyzed	126 participants
Emotional Score - Week 26		-1.19 (18.62)
Emotional Score - Week 52	Number Analyzed	118 participants
Emotional Score - Week 52		1.19 (16.27)
Emotional Score - Week 78	Number Analyzed	108 participants
Emotional Score - Week 78		-0.05 (17.23)
Emotional Score - Week 104	Number Analyzed	101 participants
Emotional Score - Week 104		-0.54 (18.37)
Emotional Score - Week 130	Number Analyzed	67 participants
Emotional Score - Week 130		2.31 (18.47)
Baseline (Social Score)	Number Analyzed	128 participants
Baseline (Social Score)		72.77 (18.59)
Social Score - Week 26	Number Analyzed	126 participants
Social Score - Week 26		1.43 (14.15)
Social Score - Week 52	Number Analyzed	118 participants
Social Score - Week 52		0.93 (15.91)
Social Score - Week 78	Number Analyzed	109 participants
Social Score - Week 78		2.39 (17.38)
Social Score - Week 104	Number Analyzed	101 participants
Social Score - Week 104		2.38 (18.62)
Social Score - Week 130	Number Analyzed	67 participants
Social Score - Week 130		4.10 (16.05)
Baseline (School/Work Score)	Number Analyzed	127 participants
Baseline (School/Work Score)		77.20 (15.83)
School/Work Score - Week 26	Number Analyzed	125 participants
School/Work Score - Week 26		-1.24 (14.28)
School/Work Score - Week 52	Number Analyzed	117 participants
School/Work Score - Week 52		-1.32 (14.09)
School/Work Score - Week 78	Number Analyzed	107 participants
School/Work Score - Week 78		-0.51 (14.26)
School/Work Score - Week 104	Number Analyzed	100 participants
School/Work Score - Week 104		-1.65 (15.24)
School/Work Score - Week 130	Number Analyzed	66 participants
School/Work Score - Week 130		-2.58 (15.04)

6.Secondary Outcome


Title	Change From Baseline in Caregiver PedsQL Generic Core Scales Version 4.0 Score
Description	The PedsQL Generic Core Scale includes 23 items. The instrument covers...
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

Outcome Measure Data

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
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (Total Score)		55.58 (12.49)
Total Score - Week 26	Number Analyzed	115 participants
Total Score - Week 26		-0.85 (13.15)
Total Score - Week 52	Number Analyzed	106 participants
Total Score - Week 52		-1.86 (13.12)
Total Score - Week 78	Number Analyzed	103 participants
Total Score - Week 78		-0.67 (13.88)
Total Score - Week 104	Number Analyzed	93 participants
Total Score - Week 104		-1.64 (17.95)
Total Score - Week 130	Number Analyzed	62 participants
Total Score - Week 130		-0.33 (13.41)
Baseline (Physical Score)	Number Analyzed	121 participants
Baseline (Physical Score)		28.99 (25.84)
Physical Score - Week 26	Number Analyzed	115 participants
Physical Score - Week 26		-0.58 (28.66)
Physical Score - Week 52	Number Analyzed	106 participants
Physical Score - Week 52		-5.10 (25.86)
Physical Score - Week 78	Number Analyzed	103 participants
Physical Score - Week 78		-2.91 (28.91)
Physical Score - Week 104	Number Analyzed	93 participants
Physical Score - Week 104		-3.28 (34.80)
Physical Score - Week 130	Number Analyzed	62 participants
Physical Score - Week 130		-0.56 (30.09)
Baseline (Emotional Score)	Number Analyzed	121 participants
Baseline (Emotional Score)		67.48 (17.31)
Emotional Score - Week 26	Number Analyzed	115 participants
Emotional Score - Week 26		-2.13 (15.30)
Emotional Score - Week 52	Number Analyzed	106 participants
Emotional Score - Week 52		-0.90 (15.08)
Emotional Score - Week 78	Number Analyzed	103 participants
Emotional Score - Week 78		-0.39 (18.81)
Emotional Score - Week 104	Number Analyzed	93 participants
Emotional Score - Week 104		-0.70 (20.54)
Emotional Score - Week 130	Number Analyzed	62 participants
Emotional Score - Week 130		-0.32 (15.94)
Baseline (Social Score)	Number Analyzed	121 participants
Baseline (Social Score)		68.34 (16.19)
Social Score - Week 26	Number Analyzed	115 participants
Social Score - Week 26		-0.42 (16.84)
Social Score - Week 52	Number Analyzed	106 participants
Social Score - Week 52		-0.49 (15.92)
Social Score - Week 78	Number Analyzed	103 participants
Social Score - Week 78		1.47 (18.40)
Social Score - Week 104	Number Analyzed	93 participants
Social Score - Week 104		-0.55 (21.72)
Social Score - Week 130	Number Analyzed	61 participants
Social Score - Week 130		1.25 (16.22)
Baseline (School/Work Score)	Number Analyzed	120 participants
Baseline (School/Work Score)		73.67 (17.69)
School/Work Score - Week 26	Number Analyzed	112 participants
School/Work Score - Week 26		-0.31 (16.69)
School/Work Score - Week 52	Number Analyzed	104 participants
School/Work Score - Week 52		0.53 (18.14)
School/Work Score - Week 78	Number Analyzed	101 participants
School/Work Score - Week 78		0.94 (18.39)
School/Work Score - Week 104	Number Analyzed	91 participants
School/Work Score - Week 104		-0.71 (20.64)
School/Work Score - Week 130	Number Analyzed	59 participants
School/Work Score - Week 130		-1.53 (16.17)

7.Secondary Outcome


Title	Change From Baseline in PedsQL Neuromuscular Module Version 3.0 Scale Score
Description	The PedsQL Neuromuscular Module (Version 3.0) includes 25 items using ...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (Total Score)		69.57 (14.01)
Total Score - Week 26	Number Analyzed	89 participants
Total Score - Week 26		-0.98 (10.75)
Total Score - Week 52	Number Analyzed	81 participants
Total Score - Week 52		-0.86 (12.81)
Total Score - Week 78	Number Analyzed	79 participants
Total Score - Week 78		-1.08 (12.11)
Total Score - Week 104	Number Analyzed	70 participants
Total Score - Week 104		-1.18 (13.13)
Total Score - Week 130	Number Analyzed	52 participants
Total Score - Week 130		-1.41 (12.61)
Baseline (Neuromuscular)	Number Analyzed	90 participants
Baseline (Neuromuscular)		66.03 (15.39)
Neuromuscular - Week 26	Number Analyzed	89 participants
Neuromuscular - Week 26		-1.77 (12.28)
Neuromuscular - Week 52	Number Analyzed	81 participants
Neuromuscular - Week 52		-2.22 (14.29)
Neuromuscular - Week 78	Number Analyzed	79 participants
Neuromuscular - Week 78		-2.69 (12.82)
Neuromuscular - Week 104	Number Analyzed	70 participants
Neuromuscular - Week 104		-1.90 (15.11)
Neuromuscular - Week 130	Number Analyzed	52 participants
Neuromuscular - Week 130		-3.14 (14.20)
Baseline (Family Resources Score)	Number Analyzed	90 participants
Baseline (Family Resources Score)		74.39 (20.10)
Family Resources Score - Week 26	Number Analyzed	89 participants
Family Resources Score - Week 26		-0.11 (17.35)
Family Resources Score - Week 52	Number Analyzed	80 participants
Family Resources Score - Week 52		1.78 (16.07)
Family Resources Score - Week 78	Number Analyzed	79 participants
Family Resources Score - Week 78		0.82 (19.22)
Family Resources Score - Week 104	Number Analyzed	70 participants
Family Resources Score - Week 104		-1.29 (19.81)
Family Resources Score - Week 130	Number Analyzed	52 participants
Family Resources Score - Week 130		-0.38 (16.36)
Baseline (Communication Score)	Number Analyzed	90 participants
Baseline (Communication Score)		81.57 (21.14)
Communication Score - Week 26	Number Analyzed	89 participants
Communication Score - Week 26		2.01 (19.86)
Communication Score - Week 52	Number Analyzed	81 participants
Communication Score - Week 52		1.44 (24.43)
Communication Score - Week 78	Number Analyzed	79 participants
Communication Score - Week 78		4.85 (22.19)
Communication Score - Week 104	Number Analyzed	70 participants
Communication Score - Week 104		2.98 (24.25)
Communication Score - Week 130	Number Analyzed	52 participants
Communication Score - Week 130		6.57 (25.15)

8.Secondary Outcome


Title	Change From Baseline in Caregiver PedsQL Neuromuscular Module Version 3.0 Scale Score
Description	The PedsQL Neuromuscular Module (Version 3.0) includes 25 items using ...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (Total Score)		59.44 (14.83)
Total Score - Week 26	Number Analyzed	87 participants
Total Score - Week 26		0.13 (10.12)
Total Score - Week 52	Number Analyzed	78 participants
Total Score - Week 52		-0.11 (12.89)
Total Score - Week 78	Number Analyzed	77 participants
Total Score - Week 78		0.65 (13.23)
Total Score - Week 104	Number Analyzed	67 participants
Total Score - Week 104		1.21 (14.40)
Total Score - Week 130	Number Analyzed	51 participants
Total Score - Week 130		-0.07 (12.86)
Baseline (Neuromuscular)	Number Analyzed	88 participants
Baseline (Neuromuscular)		56.28 (15.70)
Neuromuscular - Week 26	Number Analyzed	87 participants
Neuromuscular - Week 26		-0.64 (10.92)
Neuromuscular - Week 52	Number Analyzed	78 participants
Neuromuscular - Week 52		-1.33 (13.39)
Neuromuscular - Week 78	Number Analyzed	77 participants
Neuromuscular - Week 78		0.05 (13.97)
Neuromuscular - Week 104	Number Analyzed	67 participants
Neuromuscular - Week 104		0.54 (15.32)
Neuromuscular - Week 130	Number Analyzed	51 participants
Neuromuscular - Week 130		-1.39 (14.65)
Baseline (Family Resources Score)	Number Analyzed	88 participants
Baseline (Family Resources Score)		58.24 (25.08)
Family Resources Score - Week 26	Number Analyzed	86 participants
Family Resources Score - Week 26		1.74 (16.57)
Family Resources Score - Week 52	Number Analyzed	77 participants
Family Resources Score - Week 52		2.44 (19.04)
Family Resources Score - Week 78	Number Analyzed	77 participants
Family Resources Score - Week 78		0.91 (19.31)
Family Resources Score - Week 104	Number Analyzed	67 participants
Family Resources Score - Week 104		1.27 (18.63)
Family Resources Score - Week 130	Number Analyzed	50 participants
Family Resources Score - Week 130		1.00 (17.26)
Baseline (Communication Score)	Number Analyzed	88 participants
Baseline (Communication Score)		79.36 (22.92)
Communication Score - Week 26	Number Analyzed	87 participants
Communication Score - Week 26		1.92 (20.08)
Communication Score - Week 52	Number Analyzed	78 participants
Communication Score - Week 52		2.88 (22.31)
Communication Score - Week 78	Number Analyzed	77 participants
Communication Score - Week 78		3.57 (20.92)
Communication Score - Week 104	Number Analyzed	67 participants
Communication Score - Week 104		4.85 (24.24)
Communication Score - Week 130	Number Analyzed	51 participants
Communication Score - Week 130		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
Description	The EQ-5D-5L is a self-reported health status questionnaire that consi...
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

Outcome Measure Data

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
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		0.0471 (0.1345)
Week 26	Number Analyzed	72 participants
Week 26		0.0084 (0.1563)
Week 52	Number Analyzed	68 participants
Week 52		-0.0164 (0.1191)
Week 78	Number Analyzed	66 participants
Week 78		0.0168 (0.1910)
Week 104	Number Analyzed	57 participants
Week 104		0.0081 (0.1701)
Week 130	Number Analyzed	34 participants
Week 130		-0.0259 (0.1232)

10.Secondary Outcome


Title	Change From Baseline in Caregiver Proxy EQ-5D-5L Questionnaire Index Score - Total Score
Description	The EQ-5D-5L is a self-reported health status questionnaire that consi...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		0.0429 (0.2335)
Week 26	Number Analyzed	50 participants
Week 26		0.0652 (0.4091)
Week 52	Number Analyzed	47 participants
Week 52		0.0203 (0.2820)
Week 78	Number Analyzed	47 participants
Week 78		0.0696 (0.3094)
Week 104	Number Analyzed	39 participants
Week 104		0.0699 (0.2887)
Week 130	Number Analyzed	31 participants
Week 130		0.0455 (0.3323)

11.Secondary Outcome


Title	Change From Baseline in EQ-5D-5L Visual Analogue Scale (EQ-5D-5L VAS) Score
Description	The EQ-5D-5L is a self-reported health status questionnaire that consi...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		69.4 (19.7)
Week 26	Number Analyzed	73 participants
Week 26		1.3 (17.1)
Week 52	Number Analyzed	69 participants
Week 52		0.2 (17.4)
Week 78	Number Analyzed	67 participants
Week 78		1.3 (19.9)
Week 104	Number Analyzed	58 participants
Week 104		5.4 (17.0)
Week 130	Number Analyzed	35 participants
Week 130		0.6 (18.1)

12.Secondary Outcome


Title	Change From Baseline in Caregiver Proxy EQ-5D-5L VAS Score
Description	The EQ-5D-5L is a self-reported health status questionnaire that consi...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		72.7 (21.4)
Week 26	Number Analyzed	49 participants
Week 26		-0.3 (27.7)
Week 52	Number Analyzed	47 participants
Week 52		0.8 (21.2)
Week 78	Number Analyzed	47 participants
Week 78		0.4 (20.9)
Week 104	Number Analyzed	40 participants
Week 104		5.7 (24.3)
Week 130	Number Analyzed	31 participants
Week 130		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
Description	The WPAI:CG consists of four questions about the effects of Spinal Mus...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		56
Week 26	Number Analyzed	119 participants
Week 26		66
Week 52	Number Analyzed	109 participants
Week 52		58
Week 78	Number Analyzed	107 participants
Week 78		62
Week 104	Number Analyzed	97 participants
Week 104		56
Week 130	Number Analyzed	65 participants
Week 130		39

14.Secondary Outcome


Title	Change From Baseline in Hours Actually Worked and Work Hours Missed Assessed Using WPAI:CG Questionnaire
Description	The WPAI:CG consists of four questions about the effects of SMA on the...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (HMC)		2.0 (3.7)
HMC, Week 26	Number Analyzed	50 participants
HMC, Week 26		2.0 (7.3)
HMC, Week 52	Number Analyzed	44 participants
HMC, Week 52		1.9 (8.2)
HMC, Week 78	Number Analyzed	49 participants
HMC, Week 78		0.9 (5.6)
HMC, Week 104	Number Analyzed	45 participants
HMC, Week 104		3.2 (8.3)
HMC, Week 130	Number Analyzed	35 participants
HMC, Week 130		1.1 (8.1)
Baseline (HMO)	Number Analyzed	56 participants
Baseline (HMO)		4.8 (7.3)
HMO, Week 26	Number Analyzed	51 participants
HMO, Week 26		-1.5 (7.9)
HMO, Week 52	Number Analyzed	43 participants
HMO, Week 52		-1.5 (9.0)
HMO, Week 78	Number Analyzed	49 participants
HMO, Week 78		-0.1 (8.6)
HMO, Week 104	Number Analyzed	45 participants
HMO, Week 104		0.0 (9.6)
HMO, Week 130	Number Analyzed	35 participants
HMO, Week 130		0.8 (7.8)
Baseline (HAW)	Number Analyzed	56 participants
Baseline (HAW)		36.0 (47.0)
HAW, Week 26	Number Analyzed	51 participants
HAW, Week 26		-12.0 (46.7)
HAW, Week 52	Number Analyzed	43 participants
HAW, Week 52		-8.3 (49.2)
HAW, Week 78	Number Analyzed	48 participants
HAW, Week 78		-8.6 (49.8)
HAW, Week 104	Number Analyzed	44 participants
HAW, Week 104		1.2 (14.4)
HAW, Week 130	Number Analyzed	35 participants
HAW, Week 130		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
Description	The WPAI:CG consists of four questions about the effects of Spinal Mus...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (WTM)		6.7 (11.9)
WTM, Week 26	Number Analyzed	44 participants
WTM, Week 26		7.0 (22.3)
WTM, Week 52	Number Analyzed	38 participants
WTM, Week 52		0.8 (15.4)
WTM, Week 78	Number Analyzed	43 participants
WTM, Week 78		6.2 (23.3)
WTM, Week 104	Number Analyzed	39 participants
WTM, Week 104		6.7 (20.1)
WTM, Week 130	Number Analyzed	31 participants
WTM, Week 130		4.7 (24.4)
Baseline (IMP)	Number Analyzed	56 participants
Baseline (IMP)		29.3 (26.6)
IMP, Week 26	Number Analyzed	50 participants
IMP, Week 26		-3.0 (22.8)
IMP, Week 52	Number Analyzed	46 participants
IMP, Week 52		-1.5 (27.3)
IMP, Week 78	Number Analyzed	49 participants
IMP, Week 78		-3.5 (25.0)
IMP, Week 104	Number Analyzed	45 participants
IMP, Week 104		0.7 (22.8)
IMP, Week 130	Number Analyzed	35 participants
IMP, Week 130		-6.9 (24.7)
Baseline (OWI)	Number Analyzed	53 participants
Baseline (OWI)		33.8 (28.1)
OWI, Week 26	Number Analyzed	43 participants
OWI, Week 26		-3.6 (25.3)
OWI, Week 52	Number Analyzed	38 participants
OWI, Week 52		-3.3 (25.4)
OWI, Week 78	Number Analyzed	43 participants
OWI, Week 78		-3.0 (31.9)
OWI, Week 104	Number Analyzed	39 participants
OWI, Week 104		3.5 (26.2)
OWI, Week 130	Number Analyzed	31 participants
OWI, Week 130		-3.4 (33.3)
Baseline (AIM)	Number Analyzed	121 participants
Baseline (AIM)		45.8 (29.3)
AIM, Week 26	Number Analyzed	114 participants
AIM, Week 26		-3.9 (28.7)
AIM, Week 52	Number Analyzed	106 participants
AIM, Week 52		-1.3 (30.6)
AIM, Week 78	Number Analyzed	105 participants
AIM, Week 78		-6.3 (32.6)
AIM, Week 104	Number Analyzed	94 participants
AIM, Week 104		-7.6 (27.8)
AIM, Week 130	Number Analyzed	64 participants
AIM, Week 130		-5.5 (31.7)

16.Secondary Outcome


Title	Change From Baseline in Degree Patient Caregiving Affected Productivity and Activities Using WPAI:CG Questionnaire
Description	The WPAI:CG consists of four questions about the effects of Spinal Mus...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (Productivity)		29.3 (26.6)
Productivity, Week 26	Number Analyzed	50 participants
Productivity, Week 26		-3.0 (22.8)
Productivity, Week 52	Number Analyzed	46 participants
Productivity, Week 52		-1.5 (27.3)
Productivity, Week 78	Number Analyzed	49 participants
Productivity, Week 78		-3.5 (25.0)
Productivity, Week 104	Number Analyzed	45 participants
Productivity, Week 104		0.7 (22.8)
Productivity, Week 130	Number Analyzed	35 participants
Productivity, Week 130		-6.9 (24.7)
Baseline (Activities)	Number Analyzed	121 participants
Baseline (Activities)		45.8 (29.3)
Activities, Week 26	Number Analyzed	114 participants
Activities, Week 26		-3.9 (28.7)
Activities, Week 52	Number Analyzed	106 participants
Activities, Week 52		-1.3 (30.6)
Activities, Week 78	Number Analyzed	105 participants
Activities, Week 78		-6.3 (32.6)
Activities, Week 104	Number Analyzed	94 participants
Activities, Week 104		-7.6 (27.8)
Activities, Week 130	Number Analyzed	64 participants
Activities, Week 130		-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
Description	The SF-36 was used to assess health-related quality of life at baselin...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (PCS)		48.07 (10.33)
PCS, Week 26	Number Analyzed	95 participants
PCS, Week 26		-0.24 (10.95)
PCS, Week 52	Number Analyzed	87 participants
PCS, Week 52		0.27 (9.73)
PCS, Week 78	Number Analyzed	88 participants
PCS, Week 78		-0.34 (10.87)
PCS, Week 104	Number Analyzed	77 participants
PCS, Week 104		-0.48 (10.30)
PCS, Week 130	Number Analyzed	56 participants
PCS, Week 130		1.68 (12.94)
Baseline (MCS)	Number Analyzed	101 participants
Baseline (MCS)		49.71 (10.52)
MCS, Week 26	Number Analyzed	97 participants
MCS, Week 26		-1.05 (10.22)
MCS, Week 52	Number Analyzed	89 participants
MCS, Week 52		-1.64 (10.06)
MCS, Week 78	Number Analyzed	90 participants
MCS, Week 78		-0.21 (9.30)
MCS, Week 104	Number Analyzed	79 participants
MCS, Week 104		-0.08 (7.73)
MCS, Week 130	Number Analyzed	57 participants
MCS, Week 130		-1.21 (9.98)

18.Secondary Outcome


Title	Change From Baseline in SF-36 Domain Scores: Caregiver
Description	The SF-36 was used to assess health-related quality of life at baselin...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline (Physical)		44.51 (15.97)
Physical, Week 26	Number Analyzed	95 participants
Physical, Week 26		1.73 (16.36)
Physical, Week 52	Number Analyzed	87 participants
Physical, Week 52		2.03 (14.23)
Physical, Week 78	Number Analyzed	88 participants
Physical, Week 78		1.25 (16.16)
Physical, Week 104	Number Analyzed	77 participants
Physical, Week 104		2.39 (16.61)
Physical, Week 130	Number Analyzed	56 participants
Physical, Week 130		6.02 (18.87)
Baseline (Role (Physical))	Number Analyzed	101 participants
Baseline (Role (Physical))		47.95 (8.83)
Role (physical), Week 26	Number Analyzed	97 participants
Role (physical), Week 26		-1.18 (10.29)
Role (physical), Week 52	Number Analyzed	89 participants
Role (physical), Week 52		-0.76 (9.45)
Role (physical), Week 78	Number Analyzed	90 participants
Role (physical), Week 78		-1.02 (9.82)
Role (physical), Week 104	Number Analyzed	79 participants
Role (physical), Week 104		-1.05 (8.92)
Role (physical), Week 130	Number Analyzed	57 participants
Role (physical), Week 130		-0.35 (10.75)
Baseline (Bodily pain)	Number Analyzed	101 participants
Baseline (Bodily pain)		50.94 (8.77)
Bodily pain, Week 26	Number Analyzed	97 participants
Bodily pain, Week 26		-1.82 (8.75)
Bodily pain, Week 52	Number Analyzed	89 participants
Bodily pain, Week 52		-1.83 (9.04)
Bodily pain, Week 78	Number Analyzed	90 participants
Bodily pain, Week 78		-2.14 (10.55)
Bodily pain, Week 104	Number Analyzed	79 participants
Bodily pain, Week 104		-2.40 (8.44)
Bodily pain, Week 130	Number Analyzed	57 participants
Bodily pain, Week 130		-2.96 (9.82)
Baseline (General health)	Number Analyzed	101 participants
Baseline (General health)		50.21 (10.65)
General health, Week 26	Number Analyzed	97 participants
General health, Week 26		-0.63 (10.59)
General health, Week 52	Number Analyzed	89 participants
General health, Week 52		-0.75 (9.64)
General health, Week 78	Number Analyzed	90 participants
General health, Week 78		-0.22 (10.83)
General health, Week 104	Number Analyzed	79 participants
General health, Week 104		-1.17 (11.22)
General health, Week 130	Number Analyzed	57 participants
General health, Week 130		1.07 (11.87)
Baseline (Vitality)	Number Analyzed	101 participants
Baseline (Vitality)		51.28 (9.42)
Vitality, Week 26	Number Analyzed	97 participants
Vitality, Week 26		-1.04 (8.48)
Vitality, Week 52	Number Analyzed	89 participants
Vitality, Week 52		-1.23 (7.53)
Vitality, Week 78	Number Analyzed	90 participants
Vitality, Week 78		-0.03 (9.67)
Vitality, Week 104	Number Analyzed	79 participants
Vitality, Week 104		-0.07 (8.16)
Vitality, Week 130	Number Analyzed	57 participants
Vitality, Week 130		0.37 (8.57)
Baseline (Social)	Number Analyzed	101 participants
Baseline (Social)		48.01 (9.49)
Social, Week 26	Number Analyzed	97 participants
Social, Week 26		-0.67 (10.60)
Social, Week 52	Number Analyzed	89 participants
Social, Week 52		-1.24 (11.14)
Social, Week 78	Number Analyzed	90 participants
Social, Week 78		0.28 (10.29)
Social, Week 104	Number Analyzed	79 participants
Social, Week 104		-0.95 (9.12)
Social, Week 130	Number Analyzed	57 participants
Social, Week 130		0.35 (10.46)
Baseline (Role (emotional))	Number Analyzed	101 participants
Baseline (Role (emotional))		48.38 (9.58)
Role (emotional), Week 26	Number Analyzed	97 participants
Role (emotional), Week 26		-1.15 (10.59)
Role (emotional), Week 52	Number Analyzed	89 participants
Role (emotional), Week 52		-1.72 (11.38)
Role (emotional), Week 78	Number Analyzed	90 participants
Role (emotional), Week 78		-1.16 (10.58)
Role (emotional), Week 104	Number Analyzed	79 participants
Role (emotional), Week 104		-1.06 (8.03)
Role (emotional), Week 130	Number Analyzed	57 participants
Role (emotional), Week 130		-0.43 (9.87)
Baseline (Mental health)	Number Analyzed	101 participants
Baseline (Mental health)		48.46 (9.39)
Mental health, Week 26	Number Analyzed	97 participants
Mental health, Week 26		-0.24 (9.73)
Mental health, Week 52	Number Analyzed	89 participants
Mental health, Week 52		-0.50 (9.15)
Mental health, Week 78	Number Analyzed	90 participants
Mental health, Week 78		0.07 (7.77)
Mental health, Week 104	Number Analyzed	79 participants
Mental health, Week 104		1.32 (7.42)
Mental health, Week 130	Number Analyzed	57 participants
Mental health, Week 130		-0.29 (9.21)

19.Secondary Outcome


Title	Change From Baseline in Revised Utility Index Score (SF-6D_R2): Caregiver
Description	The SF-6D focuses on seven of the eight health domains covered by the ...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Baseline		0.70 (0.11)
Week 26	Number Analyzed	92 participants
Week 26		-0.01 (0.12)
Week 52	Number Analyzed	86 participants
Week 52		-0.01 (0.10)
Week 78	Number Analyzed	87 participants
Week 78		-0.01 (0.12)
Week 104	Number Analyzed	76 participants
Week 104		0.00 (0.11)
Week 130	Number Analyzed	54 participants
Week 130		0.01 (0.13)

20.Secondary Outcome


Title	SMA Independence Scale (SMAIS) Score: Patient
Description	The SMAIS was developed specifically for SMA in order to assess functi...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Week 104		71.7 (23.4)
Week 130	Number Analyzed	12 participants
Week 130		74.8 (22.5)

21.Secondary Outcome


Title	SMA Independence Scale (SMAIS) Score: Caregiver
Description	The SMAIS was developed specifically for SMA in order to assess functi...
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

Outcome Measure Data

Analysis Population Description


Arm/Group Title		Olesoxime
Arm/Group Description:		Participants received a dose of 10 ...
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
Week 104		60.1 (22.2)
Week 130	Number Analyzed	17 participants
Week 130		58.0 (24.9)

Adverse Events

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
Arm/Group Description	Participants received a dose of 10 ...
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%)
Serious Adverse Events 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
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

Limitations and Caveats

[Not Specified]

More Information

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
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

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