Amyotrophic Lateral Sclerosis
Conditions
Keywords
Amyotrophic Lateral Sclerosis, TRO19622, Trophos
Brief summary
The purpose of the assay is to assess the safety and the efficacy of TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS, as compared to placebo, assessed by the 18-month survival rate.
Detailed description
A stand alone treatment with TRO19622 is not acceptable for ethical reasons. Riluzole is an approved and widely used ALS treatment in the European community, in Japan and in the USA. Therefore, in this study, TRO19622 will be assessed as add-on to riluzole in patients suffering from ALS. At the start of the study, patients will be randomized to one of two groups : TRO19622 (330 mg QD or placebo (once a day). Each treatment will be administered for 18 months under double-blind conditions. The product under evaluation will be administered to patients receiving the standard of care for ALS, including riluzole. Riluzole dosage (50 mg bid) must be stable and well tolerated for at least one month prior to inclusion into the study. After the double-blind period, open-label administration of TRO19622 will be allowed for safety and survival assessments and until efficacy results are available. A separate open-label protocol will be written 6 months after the randomization of the last patient into the study.
Interventions
DRUGOlesoxime
2 capsules of TRO19622 (330mg) once a day with the noon meal as add-on therapy to riluzol 50mg bid
DRUGPlacebo Comparator
2 capsules of Placebo once a day with the noon meal as add-on therapy to riluzole 50mg bid
DRUGRiluzole
Riluzole given as add-on therapy 50mg bid
Sponsors
European Commission
Hoffmann-La Roche
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Patients with sporadic or familial Amyotrophic Lateral Sclerosis * Patients with a clinical diagnosis of laboratory-supported probable, probable, or definite ALS according to the modified El Escorial criteria8. * Have signed an Informed Consent to participate to the trial before any study related procedure has taken place. * Be of age \>18 (exclusive) and \< 80 years (inclusive). * If a female, not lactating, has a negative pregnancy test and agrees to use an effective method of birth control. * Onset of ALS Symptoms (weakness) for more than 6 months (inclusive) and less than 36 months(inclusive). * Slow vital capacity (SVC), measured three times, one of the measure being \>/= 70% of that predicted. * Treated with riluzole at the stable dose of 50 mg bid for at least 30 days before enrolment.
Exclusion criteria
* Tracheostomy, invasive ventilation, or non invasive positive pressure ventilation (NIPPV). * Gastrostomy. * Evidence of major psychiatric disorder or clinically evident dementia. * Diagnosis of a neurodegenerative disease in addition to ALS. * Have a current medication that could interfere with TRO19622 pharmacokinetics: tamoxifene. * Have current medications that could interfere with TRO19622 absorption such as ezetimibe, bile salts chelators (cholesteramine), fibrates, phytosterols, niacin (vitamin B3),fish oils. Have a current medication of lipid lowering agents other than statins. * Known hypersensitivity to any component of the study drug. * Patients with known intolerance or contra-indication to riluzole. * Have a recent history (within the previous 6 months) or current evidence of alcohol or drug abuse. * Have concurrent unstable disease involving any system eg, carcinoma other than basal cell carcinoma, any cardiac dysrhythmia, myocardial infarction, clinical or ECG signs of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms of Coronary Artery Disease, or any other condition that in the opinion of the Investigator would make the patient unsuitable for study participation. . In Germany: Have any cardiac dysrhythmia, myocardial infarction, clinical or ECG signs of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms of Coronary Artery Disease or any cardiovascular illness known or identified at the screening or inclusion visits, or have concurrent unstable disease involving any system eg, carcinoma other than basal cell carcinoma or any other condition that in the opinion of the Investigator would make the patient unsuitable for study participation. * Having a baseline QTc (Bazett) \> 450 msec for males and \> 470 msec for females. * Patients with known hepatitis B/C or HIV positive serology. * Be pregnant female or lactating. * Have renal impairment defined as blood creatinine \> 1:5 X upper limit of normal. * Have hepatic impairment and/or liver enzymes (ALAT or ASAT) \> 3 X ULN. * Hemostasis disorders or current treatment with oral anticoagulants. * Be possibly dependent on the Investigator or the Sponsor (eg, including, but not limited to, affiliated employee). * Participated in any other investigational drug or therapy study with a non approved medication, within the previous 3 months. * Patients without Social Security Insurance (France).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival Rate at 18 Months | From the date of randomization until the date of death or last follow-up censored at 18 months (548 days) | Overall survival was defined from the date of randomization until the date of death (event) or last known alive date (censored). If the death date was after 18 months, the participant was censored at 18 months (548 days). Participants still alive at or after 18 months were censored at 18 months/ 548 days. All data over the 18-month follow-up period after randomization, and participant survival status at the 18-month follow-up visit for participants who withdrew prematurely from the study for reasons other than death were included. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Inclusion, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 | The ALSFRS-R is an ordinal rating scale (0 through 4) used to determine the ALS participant's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability). |
| Percentage of Participants With a Global ALS FRS-R Score of <30 or Death | Month 18 (548 days) | Percentage of participants with a global ALS FRS-R score of \< 30 or death was estimated using the Kaplan-Meier method in the ITT, with a two-tailed log-rank, both stratified by site of onset (bulbar or spinal) and non-stratified. The ALSFRS-R is an ordinal rating scale (0 through 4) used to determine the ALS participant's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability). |
| Slow Vital Capacity (SVC) Percent Predicted | Baseline, Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 | SVC as a percent of the predicted value was evaluated and reported. |
| Percentage of Participants With Failure Over 18 Months | From randomization to the time of the first event to consider at 18 months (548 days) | Time to failure was defined as the time from randomization to the time of the first event to consider (Tracheostomy, invasive ventilation \[IV\] or non invasive ventilation \[NIV\]) |
| Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Inclusion, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 | MMT score involved the examination of 30 items. These 30 items are scored from 0 (no trace of contraction) to 5 (normal power at first try). The global score is the sum of the item scores and can range from 0 to 150. Higher score indicates some power. |
| The Single-Item Mc Gill Quality of Life Scale | Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 | The single-item McGill quality of life scale evaluated the following question Considering all parts of my life - physical, emotional, social, spiritual, and financial - over the past two (2) days, the quality of my life has been…as a score of 1 to 10 on a visual analog scale where 0 is very bad and 10 is excellent. |
| Percentage of Participants With SVC Percent Predicted <70% or Had Died Over 18 Months | Month 18 (548 days) | — |
Countries
Belgium, France, Germany, Spain, United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Olesoxime 2 capsules of TRO19622 (330mg) once a day with the noon meal as add-on therapy to riluzole 50mg bid.
Olesoxime: 2 capsules of TRO19622 (330mg) once a day with the noon meal as add-on therapy to riluzole 50mg bid.
Riluzole: Riluzole given as add-on therapy 50mg bid. | 259 |
| Placebo Comparator 2 capsules of Placebo once a day with the noon meal as add-on therapy to riluzole 50mg bid Placebo Comparator: 2 capsules of Placebo once a day with the noon meal as add-on therapy to riluzole 50mg bid Riluzole: Riluzole given as add-on therapy 50mg bid | 253 |
| Total | 512 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 9 | 10 |
| Overall Study | Death | 58 | 65 |
| Overall Study | Lack of efficacy, logistics, unwell | 9 | 10 |
| Overall Study | Lost to Follow-up | 1 | 3 |
| Overall Study | Non-compliance with IMP | 1 | 1 |
| Overall Study | Protocol Violation | 1 | 0 |
| Overall Study | Withdrawal by Subject | 33 | 25 |
Baseline characteristics
| Characteristic | Olesoxime | Placebo Comparator | Total |
|---|---|---|---|
| Age, Continuous | 57.3 years STANDARD_DEVIATION 11.2 | 55.7 years STANDARD_DEVIATION 11.2 | 56.5 years STANDARD_DEVIATION 11.2 |
| Region of Enrollment Belgium | 13 Participants | 12 Participants | 25 Participants |
| Region of Enrollment France | 113 Participants | 108 Participants | 221 Participants |
| Region of Enrollment Germany | 73 Participants | 74 Participants | 147 Participants |
| Region of Enrollment Spain | 33 Participants | 34 Participants | 67 Participants |
| Region of Enrollment United Kingdom | 27 Participants | 25 Participants | 52 Participants |
| Sex: Female, Male Female | 92 Participants | 89 Participants | 181 Participants |
| Sex: Female, Male Male | 167 Participants | 164 Participants | 331 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 79 / 259 | 80 / 253 |
| other Total, other adverse events | 98 / 259 | 101 / 253 |
| serious Total, serious adverse events | 68 / 259 | 65 / 253 |
Outcome results
Primary
Overall Survival Rate at 18 Months
Overall survival was defined from the date of randomization until the date of death (event) or last known alive date (censored). If the death date was after 18 months, the participant was censored at 18 months (548 days). Participants still alive at or after 18 months were censored at 18 months/ 548 days. All data over the 18-month follow-up period after randomization, and participant survival status at the 18-month follow-up visit for participants who withdrew prematurely from the study for reasons other than death were included.
Time frame: From the date of randomization until the date of death or last follow-up censored at 18 months (548 days)
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Olesoxime | Overall Survival Rate at 18 Months | 67.5 percentage of partcipants |
| Placebo Comparator | Overall Survival Rate at 18 Months | 69.4 percentage of partcipants |
p-value: 0.71Stratified Log-Rank Test
Secondary
Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R)
The ALSFRS-R is an ordinal rating scale (0 through 4) used to determine the ALS participant's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability).
Time frame: Inclusion, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status. Number analyzed indicates number of participants who were evaluated for the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 1 | 38.2 score on a scale | Standard Deviation 5.46 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 9 | 32.7 score on a scale | Standard Deviation 7.72 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 3 | 36.4 score on a scale | Standard Deviation 6.29 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 12 | 30.5 score on a scale | Standard Deviation 8.34 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 2 | 37.6 score on a scale | Standard Deviation 5.64 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 15 | 28.6 score on a scale | Standard Deviation 8.56 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 6 | 34.3 score on a scale | Standard Deviation 7.11 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 18 | 27.0 score on a scale | Standard Deviation 9.38 |
| Olesoxime | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Inclusion | 39.1 score on a scale | Standard Deviation 4.78 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 18 | 26.3 score on a scale | Standard Deviation 9.13 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Inclusion | 38.2 score on a scale | Standard Deviation 5.25 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 1 | 37.2 score on a scale | Standard Deviation 5.59 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 2 | 36.7 score on a scale | Standard Deviation 6.02 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 3 | 35.3 score on a scale | Standard Deviation 6.62 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 6 | 33.1 score on a scale | Standard Deviation 7.62 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 9 | 30.4 score on a scale | Standard Deviation 8.23 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 12 | 28.8 score on a scale | Standard Deviation 8.47 |
| Placebo Comparator | Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | Month 15 | 27.6 score on a scale | Standard Deviation 8.92 |
Secondary
Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups
MMT score involved the examination of 30 items. These 30 items are scored from 0 (no trace of contraction) to 5 (normal power at first try). The global score is the sum of the item scores and can range from 0 to 150. Higher score indicates some power.
Time frame: Inclusion, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status. Number analyzed indicates number of participants who were evaluated for the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Olesoxime | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 6 | 117 score on a scale | Standard Deviation 24.4 |
| Olesoxime | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 12 | 106 score on a scale | Standard Deviation 29.7 |
| Olesoxime | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 3 | 121 score on a scale | Standard Deviation 22.8 |
| Olesoxime | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 15 | 101 score on a scale | Standard Deviation 32 |
| Olesoxime | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 9 | 112 score on a scale | Standard Deviation 27.1 |
| Olesoxime | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 18 | 95.2 score on a scale | Standard Deviation 33.7 |
| Olesoxime | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Inclusion | 128 score on a scale | Standard Deviation 18 |
| Placebo Comparator | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 18 | 91.8 score on a scale | Standard Deviation 34.7 |
| Placebo Comparator | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Inclusion | 126 score on a scale | Standard Deviation 18.8 |
| Placebo Comparator | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 3 | 120 score on a scale | Standard Deviation 22 |
| Placebo Comparator | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 6 | 114 score on a scale | Standard Deviation 24.6 |
| Placebo Comparator | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 9 | 109 score on a scale | Standard Deviation 27.1 |
| Placebo Comparator | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 12 | 103 score on a scale | Standard Deviation 29.6 |
| Placebo Comparator | Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups | Month 15 | 99.2 score on a scale | Standard Deviation 31.2 |
Secondary
Percentage of Participants With a Global ALS FRS-R Score of <30 or Death
Percentage of participants with a global ALS FRS-R score of \< 30 or death was estimated using the Kaplan-Meier method in the ITT, with a two-tailed log-rank, both stratified by site of onset (bulbar or spinal) and non-stratified. The ALSFRS-R is an ordinal rating scale (0 through 4) used to determine the ALS participant's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability).
Time frame: Month 18 (548 days)
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Olesoxime | Percentage of Participants With a Global ALS FRS-R Score of <30 or Death | 28.2 percentage of participants |
| Placebo Comparator | Percentage of Participants With a Global ALS FRS-R Score of <30 or Death | 24.9 percentage of participants |
p-value: 0.21Stratified Log-Rank Test
p-value: 0.2Non-stratified Log-Rank Test
Secondary
Percentage of Participants With Failure Over 18 Months
Time to failure was defined as the time from randomization to the time of the first event to consider (Tracheostomy, invasive ventilation \[IV\] or non invasive ventilation \[NIV\])
Time frame: From randomization to the time of the first event to consider at 18 months (548 days)
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Olesoxime | Percentage of Participants With Failure Over 18 Months | 67.1 percentage of participants |
| Placebo Comparator | Percentage of Participants With Failure Over 18 Months | 65.5 percentage of participants |
p-value: 0.83Stratified Log-Rank Test
p-value: 0.73Non-stratified Log-Rank Test
Secondary
Percentage of Participants With SVC Percent Predicted <70% or Had Died Over 18 Months
Time frame: Month 18 (548 days)
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status. Number analyzed indicates number of participants who were evaluated for specified analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Olesoxime | Percentage of Participants With SVC Percent Predicted <70% or Had Died Over 18 Months | 28.9 percentage of participants |
| Placebo Comparator | Percentage of Participants With SVC Percent Predicted <70% or Had Died Over 18 Months | 31.9 percentage of participants |
p-value: 0.56Stratified Log-Rank Test
Secondary
Slow Vital Capacity (SVC) Percent Predicted
SVC as a percent of the predicted value was evaluated and reported.
Time frame: Baseline, Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status. Number analyzed indicates number of participants who were evaluated at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Baseline | 93.1 percentage (%) | Standard Deviation 14.6 |
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Month 1 | 89.7 percentage (%) | Standard Deviation 17.8 |
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Month 3 | 84.8 percentage (%) | Standard Deviation 20.4 |
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Month 6 | 80.7 percentage (%) | Standard Deviation 22.9 |
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Month 9 | 77.9 percentage (%) | Standard Deviation 24.3 |
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Month 12 | 74.5 percentage (%) | Standard Deviation 25.4 |
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Month 15 | 70.5 percentage (%) | Standard Deviation 28.8 |
| Olesoxime | Slow Vital Capacity (SVC) Percent Predicted | Month 18 | 69.0 percentage (%) | Standard Deviation 27.6 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Month 18 | 67.1 percentage (%) | Standard Deviation 25.5 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Baseline | 93.1 percentage (%) | Standard Deviation 15.4 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Month 9 | 75.5 percentage (%) | Standard Deviation 24.7 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Month 1 | 89.7 percentage (%) | Standard Deviation 17.6 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Month 15 | 71.4 percentage (%) | Standard Deviation 27.1 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Month 3 | 85.7 percentage (%) | Standard Deviation 19.7 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Month 12 | 71.3 percentage (%) | Standard Deviation 29 |
| Placebo Comparator | Slow Vital Capacity (SVC) Percent Predicted | Month 6 | 80.5 percentage (%) | Standard Deviation 22.5 |
Secondary
The Single-Item Mc Gill Quality of Life Scale
The single-item McGill quality of life scale evaluated the following question Considering all parts of my life - physical, emotional, social, spiritual, and financial - over the past two (2) days, the quality of my life has been…as a score of 1 to 10 on a visual analog scale where 0 is very bad and 10 is excellent.
Time frame: Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18
Population: ITT population included all randomized participants irrespective of study medication administration and eligibility status. Number analyzed indicates number of participants who were evaluated for the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Inclusion | 6.51 score on a scale | Standard Deviation 1.65 |
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Month 1 | 6.17 score on a scale | Standard Deviation 1.73 |
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Month 3 | 5.83 score on a scale | Standard Deviation 1.98 |
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Month 6 | 5.55 score on a scale | Standard Deviation 2.12 |
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Month 9 | 5.30 score on a scale | Standard Deviation 2.14 |
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Month 12 | 4.97 score on a scale | Standard Deviation 2.11 |
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Month 15 | 4.96 score on a scale | Standard Deviation 2.26 |
| Olesoxime | The Single-Item Mc Gill Quality of Life Scale | Month 18 | 4.77 score on a scale | Standard Deviation 2.29 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Month 18 | 5.01 score on a scale | Standard Deviation 2.16 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Inclusion | 6.47 score on a scale | Standard Deviation 1.65 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Month 9 | 5.25 score on a scale | Standard Deviation 2 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Month 1 | 6.27 score on a scale | Standard Deviation 1.78 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Month 15 | 4.90 score on a scale | Standard Deviation 2.13 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Month 3 | 5.75 score on a scale | Standard Deviation 1.89 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Month 12 | 5.10 score on a scale | Standard Deviation 2.02 |
| Placebo Comparator | The Single-Item Mc Gill Quality of Life Scale | Month 6 | 5.50 score on a scale | Standard Deviation 2 |