Trial Outcomes & Findings for Study to Investigate the Efficacy and Safety of FAB122 (Daily Oral Edaravone) in Patients With Amyotrophic Lateral Sclerosis (NCT NCT05178810)

NCT ID: NCT05178810

Last Updated: 2025-03-18

Results Overview

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 313 participants
• Primary outcome timeframe: 48 weeks
• Results posted on: 2025-03-18

Participant Flow

From the 313 randomized participants, 9 patients were excluded from the analysis and 2 were randomized by mistake and not treated. So a total of 302 participants are considered in the analysis.

Participant milestones

Measure	FAB122 FAB122: Daily dose 100 mg	Placebo Placebo: Daily dose
Overall Study STARTED	205	97
Overall Study COMPLETED	155	71
Overall Study NOT COMPLETED	50	26

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Investigate the Efficacy and Safety of FAB122 (Daily Oral Edaravone) in Patients With Amyotrophic Lateral Sclerosis

Baseline characteristics by cohort

Measure	FAB122 n=205 Participants FAB122: Daily dose 100 mg	Placebo n=97 Participants Placebo: Daily dose	Total n=302 Participants Total of all reporting groups
Age, Continuous	59.3 years STANDARD_DEVIATION 10.5 • n=5 Participants	59.3 years STANDARD_DEVIATION 10 • n=7 Participants	59.3 years STANDARD_DEVIATION 10.3 • n=5 Participants
Sex: Female, Male Female	82 Participants n=5 Participants	35 Participants n=7 Participants	117 Participants n=5 Participants
Sex: Female, Male Male	123 Participants n=5 Participants	62 Participants n=7 Participants	185 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	185 Participants n=5 Participants	90 Participants n=7 Participants	275 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	18 Participants n=5 Participants	6 Participants n=7 Participants	24 Participants n=5 Participants
Region of Enrollment Europe	205 participants n=5 Participants	97 participants n=7 Participants	302 participants n=5 Participants

PRIMARY outcome

Timeframe: 48 weeks

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

Outcome measures

Measure	FAB122 n=171 Participants FAB122: Daily dose 100 mg	Placebo n=76 Participants Placebo: Daily dose	FAB122 48 Weeks FAB122: Daily dose 100 mg	Placebo 48 Weeks Placebo: Daily dose	FAB122 100mg 72 Weeks FAB122: Daily dose 100 mg	Placebo 72 Weeks Placebo: Daily dose
Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score After 48 Weeks.	-11.2 units on a scale Standard Deviation 7.9	-10.8 units on a scale Standard Deviation 8.4	—	—	—	—

SECONDARY outcome

Timeframe: 48 weeks and 72 weeks

The CAFS (Combined assessment of function and survival) combined information on survival time and ALSFRS-R (ALS Functional Rating Scale-Revised) scores. For this endpoint, each subject's outcome was ranked: the worst subject outcomes received the lowest rank numbers such that a higher CAFS score indicates a better outcome.

CAFS rankings are computed using:

Survival data: Patients who die earlier are ranked lower than those who survive longer.

ALSFRS-R scores: For patients with the same survival duration, ranks are determined by their functional decline (change in ALSFRS-R from baseline).

For this study the range could go from 1 to 302. Better Outcome: Higher CAFS rank, indicating prolonged survival and/or less functional decline.

Worse Outcome: Lower CAFS rank, indicating shorter survival and/or greater functional decline.

Outcome measures

Measure	FAB122 n=67 Participants FAB122: Daily dose 100 mg	Placebo n=33 Participants Placebo: Daily dose	FAB122 48 Weeks n=205 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=97 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks FAB122: Daily dose 100 mg	Placebo 72 Weeks Placebo: Daily dose
Combined Assessment of Function and Survival (CAFS) at 48 and 72 Weeks.	48.7 score on a scale Standard Deviation 29.41	54.15 score on a scale Standard Deviation 28.83	153.31 score on a scale Standard Deviation 86.2	147.68 score on a scale Standard Deviation 92.38	—	—

SECONDARY outcome

Timeframe: 72 weeks

Results are based on the overall survival over 72 weeks of treatment, the result is the survival probability estimated over 72 weeks.

Survival probability is calculated considering time to death, tracheostomy or initiation of non-invasive ventilation for more than 20 hours a day for more than 10 consecutive days, over 72 weeks.

Outcome measures

Measure	FAB122 n=67 Participants FAB122: Daily dose 100 mg	Placebo n=33 Participants Placebo: Daily dose	FAB122 48 Weeks FAB122: Daily dose 100 mg	Placebo 48 Weeks Placebo: Daily dose	FAB122 100mg 72 Weeks FAB122: Daily dose 100 mg	Placebo 72 Weeks Placebo: Daily dose
Survival Probability	0.728 Survival probability Interval 0.599 to 0.821	0.870 Survival probability Interval 0.688 to 0.949	—	—	—	—

SECONDARY outcome

Timeframe: 24 weeks, 72 weeks

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

Outcome measures

Measure	FAB122 n=193 Participants FAB122: Daily dose 100 mg	Placebo n=93 Participants Placebo: Daily dose	FAB122 48 Weeks n=43 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=24 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks FAB122: Daily dose 100 mg	Placebo 72 Weeks Placebo: Daily dose
Change From Baseline in ALSFRS-R Score After 24 and 72* Weeks	-6.0 units on a scale Standard Deviation 5.4	-5.4 units on a scale Standard Deviation 5.1	-15.2 units on a scale Standard Deviation 10.5	-12.6 units on a scale Standard Deviation 8.2	—	—

SECONDARY outcome

Timeframe: 24, 48, 72 weeks

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

For this outcome, measures on the ALSFRS-R were used to determine the slope of the decrease.

Outcome measures

Measure	FAB122 n=205 Participants FAB122: Daily dose 100 mg	Placebo n=97 Participants Placebo: Daily dose	FAB122 48 Weeks n=205 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=97 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=67 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=33 Participants Placebo: Daily dose
The Slope of the Decrease in ALSFRS-R Score Over Time at 24, 48 and 72* Weeks;	-0.2981 units on a scale/week Standard Error 0.0182	-0.2445 units on a scale/week Standard Error 0.265	-0.2874 units on a scale/week Standard Error 0.0143	-0.2767 units on a scale/week Standard Error 0.0206	-0.3003 units on a scale/week Standard Error 0.0223	-0.2531 units on a scale/week Standard Error 0.0313

SECONDARY outcome

Timeframe: 24, 48 and 72 weeks

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

In this outcome, the Bulbar function (as part of ALSFRS-R) is evaluated, it is related to Speech, Salivation and Swallowing. The maximum score on Bulbar function is 12, and the minimum is 0. Higher score better outcome.

Outcome measures

Measure	FAB122 n=193 Participants FAB122: Daily dose 100 mg	Placebo n=93 Participants Placebo: Daily dose	FAB122 48 Weeks n=171 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=76 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=43 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=24 Participants Placebo: Daily dose
Change From Baseline in ALSFRS-R Score on Bulbar Function (Question 1-3 of the ALSFRS-R) After 24, 48 and 72* Weeks;	-1.1 units on a scale Standard Deviation 1.7	-1.2 units on a scale Standard Deviation 1.6	-2.1 units on a scale Standard Deviation 2.5	-2.2 units on a scale Standard Deviation 2.8	-3.1 units on a scale Standard Deviation 3.5	-2.7 units on a scale Standard Deviation 2.9

SECONDARY outcome

Timeframe: 24, 48 and 72 weeks

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

In this outcome, the fine motor function (as part of ALSFRS-R) is evaluated, it is related to Handwriting, Eating and Cutting food, Dressing and hygiene. The maximum score on fine motor function is 12, and the minimum is 0. Higher score better outcome.

Outcome measures

Measure	FAB122 n=192 Participants FAB122: Daily dose 100 mg	Placebo n=93 Participants Placebo: Daily dose	FAB122 48 Weeks n=171 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=76 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=43 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=24 Participants Placebo: Daily dose
Change From Baseline ALS Functional Rating Scale - Revised Score - Fine Motor Function	-2.0 units on a scale Standard Deviation 2.1	-1.7 units on a scale Standard Deviation 2.0	-3.8 units on a scale Standard Deviation 2.9	-3.3 units on a scale Standard Deviation 2.5	-4.7 units on a scale Standard Deviation 3.1	-4.2 units on a scale Standard Deviation 3.3

SECONDARY outcome

Timeframe: 24, 48, 72 weeks

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

In this outcome, the gross motor function (as part of ALSFRS-R) is evaluated, it is related to Climbing stairs, Walking, Rising from a chair. The maximum score on gross motor function is 12, and the minimum is 0. Higher score better outcome.

Outcome measures

Measure	FAB122 n=193 Participants FAB122: Daily dose 100 mg	Placebo n=93 Participants Placebo: Daily dose	FAB122 48 Weeks n=171 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=76 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=43 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=24 Participants Placebo: Daily dose
Change From Baseline ALS Functional Rating Scale - Revised Score - Gross Motor Function	-2.0 units on a scale Standard Deviation 1.9	-1.7 units on a scale Standard Deviation 1.8	-3.4 units on a scale Standard Deviation 2.7	-3.3 units on a scale Standard Deviation 2.2	-4.5 units on a scale Standard Deviation 3.1	-3.8 units on a scale Standard Deviation 2.4

SECONDARY outcome

Timeframe: 24, 48, 72 weeks

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

In this outcome, the respiratory function (as part of ALSFRS-R) is evaluated, it is related to Dyspnea, Orthopnea, Breathing insufficiency. The maximum score on respiratory function is 12, and the minimum is 0. Higher score better outcome.

Outcome measures

Measure	FAB122 n=193 Participants FAB122: Daily dose 100 mg	Placebo n=93 Participants Placebo: Daily dose	FAB122 48 Weeks n=171 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=76 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=43 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=24 Participants Placebo: Daily dose
Change From Baseline ALS Functional Rating Scale - Revised Score - Respiratory Function	-0.9 units on a scale Standard Deviation 2.2	-0.8 units on a scale Standard Deviation 2.1	-1.9 units on a scale Standard Deviation 3.2	-2.0 units on a scale Standard Deviation 3.3	-2.8 units on a scale Standard Deviation 4.2	-2.0 units on a scale Standard Deviation 2.8

SECONDARY outcome

Timeframe: 72 weeks (+/-1 week visit window)

Population: The first 100 patients in the study were planned to complete a 72 weeks follow up. This analysis is done over this population.

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

For this outcome, the time a 3, 6, 9 and 12 points change or death from baseline in ALSFRS-R.

Outcome measures

Measure	FAB122 n=67 Participants FAB122: Daily dose 100 mg	Placebo n=33 Participants Placebo: Daily dose	FAB122 48 Weeks FAB122: Daily dose 100 mg	Placebo 48 Weeks Placebo: Daily dose	FAB122 100mg 72 Weeks FAB122: Daily dose 100 mg	Placebo 72 Weeks Placebo: Daily dose
Time to a 3, 6, 9 and 12 Points Change or Death From Baseline in ALSFRS-R Score Over 72* Weeks; Time to 3 points change or death from baseline in ALSFRS-R score	85 days Interval 56.0 to 169.0	114 days Interval 84.0 to 169.0	—	—	—	—
Time to a 3, 6, 9 and 12 Points Change or Death From Baseline in ALSFRS-R Score Over 72* Weeks; Time to 6 points change or death from baseline in ALSFRS-R score	194 days Interval 113.0 to 286.0	226 days Interval 142.0 to 315.0	—	—	—	—
Time to a 3, 6, 9 and 12 Points Change or Death From Baseline in ALSFRS-R Score Over 72* Weeks; Time to 9 points change or death from baseline in ALSFRS-R score	308 days Interval 189.0 to 504.0	334 days Interval 197.0 to 510.0	—	—	—	—
Time to a 3, 6, 9 and 12 Points Change or Death From Baseline in ALSFRS-R Score Over 72* Weeks; Time to 12 points change or death from baseline in ALSFRS-R score	351 days Interval 223.0 to 511.0	438 days Interval 250.0 to 508.0	—	—	—	—

SECONDARY outcome

Timeframe: 72 weeks

The King's staging system is a simple clinical staging system, which defines 4 stages of ALS. The 1st 3 stages are defined by functional involvement of a region: bulbar, upper limbs, and lower limbs. The number of regions involved gives the stage. Stage 4 is reached if swallowing (4A) or respiratory (4B) difficulty is severe enough to require intervention.

The outcome of this measure shows the number of patients which staging decline of 1 point or more or No decline.

Outcome measures

Measure	FAB122 n=39 Participants FAB122: Daily dose 100 mg	Placebo n=24 Participants Placebo: Daily dose	FAB122 48 Weeks FAB122: Daily dose 100 mg	Placebo 48 Weeks Placebo: Daily dose	FAB122 100mg 72 Weeks FAB122: Daily dose 100 mg	Placebo 72 Weeks Placebo: Daily dose
Change in Clinical Staging (King's Staging System and MiToS) Over 72 Weeks Decline >= 1	22 Participants	12 Participants	—	—	—	—
Change in Clinical Staging (King's Staging System and MiToS) Over 72 Weeks No decline	17 Participants	12 Participants	—	—	—	—

SECONDARY outcome

Timeframe: 24, 48 and 72* weeks;

Outcome measures

Measure	FAB122 n=205 Participants FAB122: Daily dose 100 mg	Placebo n=97 Participants Placebo: Daily dose	FAB122 48 Weeks n=205 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=97 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=67 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=33 Participants Placebo: Daily dose
Overall Survival: Proportion of Subjects Alive (Survival Rate) After 24, 48 and 72* Weeks;	0.980 proportion of participants Interval 0.947 to 0.992	0.990 proportion of participants Interval 0.928 to 0.999	0.933 proportion of participants Interval 0.888 to 0.961	0.933 proportion of participants Interval 0.857 to 0.97	0.791 proportion of participants Interval 0.667 to 0.873	0.870 proportion of participants Interval 0.688 to 0.949

SECONDARY outcome

Timeframe: 24, 48 and 72* weeks

Outcome measures

Measure	FAB122 n=205 Participants FAB122: Daily dose 100 mg	Placebo n=97 Participants Placebo: Daily dose	FAB122 48 Weeks n=205 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=97 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=67 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=33 Participants Placebo: Daily dose
Proportion of Subjects Alive and no Tracheostomy, or no Initiation of Non-invasive Ventilation for More Than 20 Hours a Day for More Than 10 Consecutive Days After 24, 48 and 72* Weeks	0.970 proportion of participants Interval 0.935 to 0.986	0.990 proportion of participants Interval 0.928 to 0.999	0.903 proportion of participants Interval 0.853 to 0.937	0.890 proportion of participants Interval 0.804 to 0.939	0.728 proportion of participants Interval 0.599 to 0.821	0.870 proportion of participants Interval 0.688 to 0.949

SECONDARY outcome

Timeframe: 24, 48 and 72* weeks

Outcome measures

Measure	FAB122 n=170 Participants FAB122: Daily dose 100 mg	Placebo n=86 Participants Placebo: Daily dose	FAB122 48 Weeks n=129 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=59 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=33 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=20 Participants Placebo: Daily dose
Change From Baseline in Slow Vital Capacity (SVC, Liters) at 24, 48 and 72* Weeks;	-0.4 liters Standard Deviation 0.5	-0.5 liters Standard Deviation 0.7	-0.7 liters Standard Deviation 0.7	-0.7 liters Standard Deviation 0.7	-1 liters Standard Deviation 1	-1 liters Standard Deviation 0.7

SECONDARY outcome

Timeframe: 24, 48 and 72* weeks

HHD is a procedure for quantitative strength testing performed in the upper and lower extremities bilaterally. The overall mega score are derived as z-scores of average muscle HHD assessments, percent changes from Baseline are used to derive individual muscle scores.

Muscle strength is expressed as the percent change from baseline: (post-baselinevalue-baselinevalue)/baselinevalue×100(post-baseline value - baseline value) / baseline value \times 100(post-baselinevalue-baselinevalue)/baselinevalue×100. If the baseline value is zero, the data is considered missing.

The HDD mega-score averages strength across four muscle locations. It is calculated by averaging the non-missing transformed values. Maximum muscle strength is then standardized using a z-score, based on data from a healthy population.

Z-score of 0 represents the healthy population mean. Negative z-score value means worse outcome.

Outcome measures

Measure	FAB122 n=135 Participants FAB122: Daily dose 100 mg	Placebo n=62 Participants Placebo: Daily dose	FAB122 48 Weeks n=109 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=49 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=19 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=16 Participants Placebo: Daily dose
Change From Baseline in the Overall Mega Score for the Hand-held Dynamometer (HHD) at 24, 48 and 72* Weeks.	-0.4 Z-Score Standard Deviation 0.7	-0.4 Z-Score Standard Deviation 0.6	-0.7 Z-Score Standard Deviation 1.1	-0.8 Z-Score Standard Deviation 0.9	-1.2 Z-Score Standard Deviation 1.2	-1.4 Z-Score Standard Deviation 0.8

SECONDARY outcome

Timeframe: 24, 48 and 72* weeks

ALS Assessment Questionnaire-40-Item. The ALSAQ-40 is specifically used to measure the subjective wellbeing of patients with ALS. There are 40 items/questions in the long form, the ALSAQ-40, with 5 discrete scales: physical mobility (10 items), activities of daily living and independence (10 items), eating and drinking (3 items), communication (7 items), and emotional reactions (10 items). Range is from 0 to 100 scale, where 0 indicates the best quality of life and 100 the worst.

Outcome measures

Measure	FAB122 n=176 Participants FAB122: Daily dose 100 mg	Placebo n=83 Participants Placebo: Daily dose	FAB122 48 Weeks n=155 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=67 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=40 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=23 Participants Placebo: Daily dose
Change From Baseline in the Total Score on the ALS Assessment Questionnaire-40-Item (ALSAQ-40) Form at 24, 48 and 72* Weeks;	11.2 units on a scale Standard Deviation 12.8	8.9 units on a scale Standard Deviation 12.3	22.7 units on a scale Standard Deviation 16.8	19.9 units on a scale Standard Deviation 17.5	27.1 units on a scale Standard Deviation 18.4	22.5 units on a scale Standard Deviation 19.5

SECONDARY outcome

Timeframe: 24, 48 and 72* weeks

European Quality of Life 5 levels and 5 dimensions is a generic questionnaire of health-related quality of life.

5 Domains: Mobility, Self-care, Usual activities, Pain/Discomfort and Anxiety/Depression.

Scale range is from 0 to 100, being 0 the worst and 100 the best.

Outcome measures

Measure	FAB122 n=205 Participants FAB122: Daily dose 100 mg	Placebo n=97 Participants Placebo: Daily dose	FAB122 48 Weeks n=205 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=97 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=67 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=33 Participants Placebo: Daily dose
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Mobility - Level 4	49 units on a scale Standard Deviation 23.9	27 units on a scale Standard Deviation 27.8	49 units on a scale Standard Deviation 23.9	21 units on a scale Standard Deviation 21.6	14 units on a scale Standard Deviation 6.8	7 units on a scale Standard Deviation 7.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Anxiety/Depression - Level 1	82 units on a scale Standard Deviation 40	39 units on a scale Standard Deviation 40.2	57 units on a scale Standard Deviation 27.8	29 units on a scale Standard Deviation 29.9	12 units on a scale Standard Deviation 5.9	7 units on a scale Standard Deviation 7.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Anxiety/Depression - Level 2	53 units on a scale Standard Deviation 25.9	20 units on a scale Standard Deviation 20.6	52 units on a scale Standard Deviation 25.4	15 units on a scale Standard Deviation 15.5	12 units on a scale Standard Deviation 5.9	7 units on a scale Standard Deviation 7.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Anxiety/Depression - Level 3	39 units on a scale Standard Deviation 19	26 units on a scale Standard Deviation 26.8	38 units on a scale Standard Deviation 18.5	21 units on a scale Standard Deviation 21.6	11 units on a scale Standard Deviation 5.4	6 units on a scale Standard Deviation 6.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Anxiety/Depression - Level 4	12 units on a scale Standard Deviation 5.9	3 units on a scale Standard Deviation 3.1	12 units on a scale Standard Deviation 5.9	4 units on a scale Standard Deviation 4.1	6 units on a scale Standard Deviation 2.9	4 units on a scale Standard Deviation 4.1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Anxiety/Depression - Level 5	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0	3 units on a scale Standard Deviation 1.5	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Pain/Discomfort - Level 1	66 units on a scale Standard Deviation 32.2	33 units on a scale Standard Deviation 34	57 units on a scale Standard Deviation 27.8	27 units on a scale Standard Deviation 27.8	13 units on a scale Standard Deviation 6.3	8 units on a scale Standard Deviation 8.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Pain/Discomfort - Level 2	61 units on a scale Standard Deviation 29.8	26 units on a scale Standard Deviation 26.8	47 units on a scale Standard Deviation 22.9	19 units on a scale Standard Deviation 19.6	10 units on a scale Standard Deviation 4.9	8 units on a scale Standard Deviation 8.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Pain/Discomfort - Level 3	48 units on a scale Standard Deviation 23.4	22 units on a scale Standard Deviation 22.7	39 units on a scale Standard Deviation 19	19 units on a scale Standard Deviation 19.6	12 units on a scale Standard Deviation 5.9	6 units on a scale Standard Deviation 6.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Pain/Discomfort - Level 4	9 units on a scale Standard Deviation 4.4	7 units on a scale Standard Deviation 7.2	14 units on a scale Standard Deviation 6.8	4 units on a scale Standard Deviation 4.1	6 units on a scale Standard Deviation 2.9	1 units on a scale Standard Deviation 1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Pain/Discomfort - Level 5	2 units on a scale Standard Deviation 1	0 units on a scale Standard Deviation 0	5 units on a scale Standard Deviation 2.4	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0	1 units on a scale Standard Deviation 1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Usual Activities - Level 1	21 units on a scale Standard Deviation 10.2	10 units on a scale Standard Deviation 10.3	10 units on a scale Standard Deviation 4.9	4 units on a scale Standard Deviation 4.1	1 units on a scale Standard Deviation 0.5	1 units on a scale Standard Deviation 1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Usual Activities - Level 2	35 units on a scale Standard Deviation 17.1	8 units on a scale Standard Deviation 8.2	22 units on a scale Standard Deviation 10.7	4 units on a scale Standard Deviation 4.1	4 units on a scale Standard Deviation 2	2 units on a scale Standard Deviation 2.1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Usual Activities - Level 3	50 units on a scale Standard Deviation 24.4	31 units on a scale Standard Deviation 32	30 units on a scale Standard Deviation 14.6	22 units on a scale Standard Deviation 22.7	5 units on a scale Standard Deviation 2.4	5 units on a scale Standard Deviation 5.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Usual Activities - Level 4	41 units on a scale Standard Deviation 20	22 units on a scale Standard Deviation 22.7	42 units on a scale Standard Deviation 20.5	22 units on a scale Standard Deviation 22.7	13 units on a scale Standard Deviation 6.3	5 units on a scale Standard Deviation 5.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Usual Activities - Level 5	40 units on a scale Standard Deviation 19.5	16 units on a scale Standard Deviation 16.5	57 units on a scale Standard Deviation 27.8	17 units on a scale Standard Deviation 17.5	18 units on a scale Standard Deviation 8.8	11 units on a scale Standard Deviation 11.3
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Self-care - Level 1	36 units on a scale Standard Deviation 17.6	11 units on a scale Standard Deviation 11.3	16 units on a scale Standard Deviation 7.8	5 units on a scale Standard Deviation 5.2	3 units on a scale Standard Deviation 1.5	1 units on a scale Standard Deviation 1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Self-care - Level 2	34 units on a scale Standard Deviation 16.6	17 units on a scale Standard Deviation 17.5	25 units on a scale Standard Deviation 12.2	6 units on a scale Standard Deviation 6.2	3 units on a scale Standard Deviation 1.5	2 units on a scale Standard Deviation 2.1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Self-care - Level 3	42 units on a scale Standard Deviation 20.5	29 units on a scale Standard Deviation 29.9	26 units on a scale Standard Deviation 12.7	19 units on a scale Standard Deviation 19.6	5 units on a scale Standard Deviation 2.4	2 units on a scale Standard Deviation 2.1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Self-care - Level 4	24 units on a scale Standard Deviation 11.7	11 units on a scale Standard Deviation 11.3	26 units on a scale Standard Deviation 12.7	15 units on a scale Standard Deviation 15.5	6 units on a scale Standard Deviation 2.9	5 units on a scale Standard Deviation 5.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Self-care - Level 5	51 units on a scale Standard Deviation 24.9	20 units on a scale Standard Deviation 20.6	69 units on a scale Standard Deviation 33.7	24 units on a scale Standard Deviation 24.7	24 units on a scale Standard Deviation 11.7	14 units on a scale Standard Deviation 14.4
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Mobility - Level 1	29 units on a scale Standard Deviation 14.1	17 units on a scale Standard Deviation 17.5	16 units on a scale Standard Deviation 7.8	9 units on a scale Standard Deviation 9.3	3 units on a scale Standard Deviation 1.5	5 units on a scale Standard Deviation 5.2
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Mobility - Level 2	35 units on a scale Standard Deviation 17.1	13 units on a scale Standard Deviation 13.4	21 units on a scale Standard Deviation 10.2	6 units on a scale Standard Deviation 6.2	3 units on a scale Standard Deviation 1.5	1 units on a scale Standard Deviation 1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Mobility - Level 3	43 units on a scale Standard Deviation 21	19 units on a scale Standard Deviation 19.6	29 units on a scale Standard Deviation 14.1	14 units on a scale Standard Deviation 14.4	6 units on a scale Standard Deviation 2.9	2 units on a scale Standard Deviation 2.1
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks. Mobility - Level 5	31 units on a scale Standard Deviation 15.1	12 units on a scale Standard Deviation 12.4	47 units on a scale Standard Deviation 22.9	19 units on a scale Standard Deviation 19.6	15 units on a scale Standard Deviation 7.3	9 units on a scale Standard Deviation 9.3

SECONDARY outcome

Timeframe: 24, 48 and 72* weeks

The Health-Related Quality of Life (HR-QoL) is a questionnaire using a Visual Analog Scale (VAS) ranging from 0 (bad) to 100 (very good).

Outcome measures

Measure	FAB122 n=182 Participants FAB122: Daily dose 100 mg	Placebo n=85 Participants Placebo: Daily dose	FAB122 48 Weeks n=160 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=67 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=41 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=23 Participants Placebo: Daily dose
Change From Baseline in Visual Analogue Scale (VAS) Score at 24, 48 and 72* Weeks.	-10.2 units on a scale Standard Deviation 19.4	-7.2 units on a scale Standard Deviation 23.6	-15.7 units on a scale Standard Deviation 20.2	-15.6 units on a scale Standard Deviation 24.9	-25.1 units on a scale Standard Deviation 21.3	-15.1 units on a scale Standard Deviation 22.3

SECONDARY outcome

Timeframe: 72 weeks

The ECAS (Edinburgh Cognitive and behavioural ALS Screen) is a brief multidomain assessment originally designed for people with ALS.

Total Score Range: 0 to 136 points. Higher scores indicate better cognitive function.

For ALS-Specific (Assesses cognitive domains most often affected in ALS) Score Range: 0 to 100 points. Higher scores indicate better cognitive function.

And ALS Non-Specific (Evaluates broader cognitive abilities unrelated to ALS pathology) Score Range: 0 to 36 points. Higher scores indicate better cognitive function.

Outcome measures

Measure	FAB122 n=179 Participants FAB122: Daily dose 100 mg	Placebo n=88 Participants Placebo: Daily dose	FAB122 48 Weeks n=147 Participants FAB122: Daily dose 100 mg	Placebo 48 Weeks n=62 Participants Placebo: Daily dose	FAB122 100mg 72 Weeks n=35 Participants FAB122: Daily dose 100 mg	Placebo 72 Weeks n=22 Participants Placebo: Daily dose
Proportion of Subjects With a Change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and Behavioural ALS Screen) Total Score; ALS Specific - Change <8	154 Participants	69 Participants	128 Participants	45 Participants	31 Participants	18 Participants
Proportion of Subjects With a Change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and Behavioural ALS Screen) Total Score; ALS Specific - Change >=8	25 Participants	19 Participants	19 Participants	17 Participants	4 Participants	4 Participants
Proportion of Subjects With a Change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and Behavioural ALS Screen) Total Score; ALS non-Specific - Change <4	153 Participants	67 Participants	112 Participants	46 Participants	29 Participants	15 Participants
Proportion of Subjects With a Change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and Behavioural ALS Screen) Total Score; ALS non-Specific - Change >=4	26 Participants	21 Participants	35 Participants	16 Participants	6 Participants	7 Participants
Proportion of Subjects With a Change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and Behavioural ALS Screen) Total Score; ECAS Total Score - Change <9	152 Participants	70 Participants	121 Participants	44 Participants	29 Participants	19 Participants
Proportion of Subjects With a Change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and Behavioural ALS Screen) Total Score; ECAS Total Score - Change >=9	27 Participants	18 Participants	26 Participants	18 Participants	6 Participants	3 Participants

Adverse Events

FAB122

Serious events: 62 serious events

Other events: 180 other events

Deaths: 20 deaths

Placebo

Serious events: 28 serious events

Other events: 82 other events

Deaths: 7 deaths

Serious adverse events

Measure	FAB122 n=205 participants at risk FAB122: Daily dose 100 mg	Placebo n=97 participants at risk Placebo: Daily dose
Respiratory, thoracic and mediastinal disorders Respiratory failure	9.3% 19/205 • Number of events 19 • 72 weeks	9.3% 9/97 • Number of events 9 • 72 weeks
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	1.5% 3/205 • Number of events 3 • 72 weeks	2.1% 2/97 • Number of events 2 • 72 weeks
Respiratory, thoracic and mediastinal disorders Respiratory disorder	0.98% 2/205 • Number of events 2 • 72 weeks	2.1% 2/97 • Number of events 2 • 72 weeks
Infections and infestations Pneumonia	2.9% 6/205 • Number of events 7 • 72 weeks	2.1% 2/97 • Number of events 2 • 72 weeks
Infections and infestations COVID-19	2.0% 4/205 • Number of events 4 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Injury, poisoning and procedural complications Fall	1.5% 3/205 • Number of events 3 • 72 weeks	0.00% 0/97 • 72 weeks
Cardiac disorders Cardiac arrest	1.5% 3/205 • Number of events 3 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Cardiac disorders Myocardial infarction	1.5% 3/205 • Number of events 3 • 72 weeks	0.00% 0/97 • 72 weeks
Gastrointestinal disorders Dysphagia	2.0% 4/205 • Number of events 4 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
General disorders Death	1.5% 3/205 • Number of events 3 • 72 weeks	0.00% 0/97 • 72 weeks
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.49% 1/205 • Number of events 1 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Respiratory, thoracic and mediastinal disorders Lung disorder	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Infections and infestations Pneumonia aspiration	0.98% 2/205 • Number of events 2 • 72 weeks	0.00% 0/97 • 72 weeks
Infections and infestations Lower respiratory tract infection	0.49% 1/205 • Number of events 1 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Infections and infestations Urinary tract infection	0.49% 1/205 • Number of events 1 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Infections and infestations Bronchitis	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Infections and infestations Post procedural infection	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Infections and infestations Septic shock	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Infections and infestations Urosepsis	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Infections and infestations Respiratory tract infection	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Injury, poisoning and procedural complications Femural neck fracture	0.49% 1/205 • Number of events 1 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Injury, poisoning and procedural complications Femur fracture	0.49% 1/205 • Number of events 1 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Injury, poisoning and procedural complications Concussion	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Injury, poisoning and procedural complications Fibula fracture	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Injury, poisoning and procedural complications Head injury	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Injury, poisoning and procedural complications Joint dislocation	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Injury, poisoning and procedural complications Humerus fracture	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Injury, poisoning and procedural complications Rib fracture	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Injury, poisoning and procedural complications Subdural haematoma	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Cardiac disorders Acute coronary syndrome	0.49% 1/205 • Number of events 1 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Cardiac disorders Cardiac failure	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Cardiac disorders Myocardial ischaemia	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Cardiac disorders Acute myocardial infarction	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Gastrointestinal disorders Intestinal haemorrhage	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Gastrointestinal disorders Intestinal obstruction	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Gastrointestinal disorders Nausea	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Gastrointestinal disorders Vomiting	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
General disorders Pyrexia	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
General disorders Brain death	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Nervous system disorders Cerebral haematoma	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Nervous system disorders Cerebral haemorrhage	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Nervous system disorders Hypoxic ischaemic encephalopathy	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Investigations Weight decrease	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Investigations Oxygen saturation decreased	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Malignant melanoma	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Thyroid cancer	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Psychiatric disorders Acute psychosis	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Psychiatric disorders Panic attack	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Vascular disorders Hypertensive crisis	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Vascular disorders Deep vein thrombosis	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Hepatobiliary disorders Cholecystitis	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Hepatobiliary disorders Cholelithasis	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Metabolism and nutrition disorders Dehydration	0.00% 0/205 • 72 weeks	1.0% 1/97 • Number of events 1 • 72 weeks
Reproductive system and breast disorders Utherine haemorrhage	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks
Surgical and medical procedures Hospitalisation	0.49% 1/205 • Number of events 1 • 72 weeks	0.00% 0/97 • 72 weeks

Other adverse events

Measure	FAB122 n=205 participants at risk FAB122: Daily dose 100 mg	Placebo n=97 participants at risk Placebo: Daily dose
Infections and infestations COVID-19	18.0% 37/205 • Number of events 38 • 72 weeks	14.4% 14/97 • Number of events 14 • 72 weeks
Infections and infestations Nasopharygitis	9.8% 20/205 • Number of events 29 • 72 weeks	6.2% 6/97 • Number of events 6 • 72 weeks
Infections and infestations Urinary tract infection	7.3% 15/205 • Number of events 18 • 72 weeks	6.2% 6/97 • Number of events 10 • 72 weeks
Infections and infestations Upper respiratory tract infection	7.3% 15/205 • Number of events 15 • 72 weeks	5.2% 5/97 • Number of events 6 • 72 weeks
Gastrointestinal disorders Constipation	12.2% 25/205 • Number of events 25 • 72 weeks	12.4% 12/97 • Number of events 12 • 72 weeks
Gastrointestinal disorders Dysphagia	9.8% 20/205 • Number of events 20 • 72 weeks	8.2% 8/97 • Number of events 8 • 72 weeks
Injury, poisoning and procedural complications Fall	19.5% 40/205 • Number of events 65 • 72 weeks	27.8% 27/97 • Number of events 52 • 72 weeks
Respiratory, thoracic and mediastinal disorders Respiratory failure	15.1% 31/205 • Number of events 35 • 72 weeks	14.4% 14/97 • Number of events 15 • 72 weeks
Investigations Nerve conduction studies abnormal	9.3% 19/205 • Number of events 19 • 72 weeks	9.3% 9/97 • Number of events 9 • 72 weeks
Musculoskeletal and connective tissue disorders Arthralgia	8.3% 17/205 • Number of events 20 • 72 weeks	5.2% 5/97 • Number of events 5 • 72 weeks
Musculoskeletal and connective tissue disorders Back pain	6.3% 13/205 • Number of events 13 • 72 weeks	6.2% 6/97 • Number of events 6 • 72 weeks
Nervous system disorders Headache	6.3% 13/205 • Number of events 13 • 72 weeks	6.2% 6/97 • Number of events 7 • 72 weeks

Additional Information

Clinical Operations Head

Ferrer

Phone: +34 649 442 389

Email: nalbareda@ferrer.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60