A Phase 3 interventional study of FAB122 and Placebo in Amyotrophic Lateral Sclerosis, sponsored by Ferrer Internacional S.A.. Completed at 38 sites in 11 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2025-03-18.
Sponsored by Ferrer Internacional S.A. · Phase 3, Interventional, and Treatment
Multicenter, multinational, double-blind, randomized (2:1), placebo-controlled Phase III study to investigate the efficacy and safety of 100 mg FAB122 once daily as oral formulation in ALS patients.
717 studies on the registry are indexed under Motor Neuron Disease; 137 are open to participants now.
This study's enrollment of 313 is above the median of 35 across 461 interventional studies indexed under Motor Neuron Disease.
Browse Motor Neuron Disease studies →Ferrer Internacional S.A. is the lead sponsor of 21 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Main Inclusion Criteria:
Onset of first symptoms* no longer than 24 months prior to randomization;
*Date of onset is the date the patient reported one or more of the following symptoms:
Main Exclusion Criteria:
Drug: FAB122
Drug: Placebo
Daily dose 100 mg
Daily dose
Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score After 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.
Time frame: 48 weeks
Combined Assessment of Function and Survival (CAFS) at 48 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.
Time frame: 48 weeks and 72 weeks
Survival Probability
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.
Time frame: 72 weeks
Change From Baseline in ALSFRS-R Score After 24 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.
Time frame: 24 weeks, 72 weeks
The Slope of the Decrease in ALSFRS-R Score Over Time at 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. For this outcome, measures on the ALSFRS-R were used to determine the slope of the decrease.
Time frame: 24, 48, 72 weeks
Change From Baseline in ALSFRS-R Score on Bulbar Function (Question 1-3 of the ALSFRS-R) After 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.
Time frame: 24, 48 and 72 weeks
Change From Baseline ALS Functional Rating Scale - Revised Score - Fine Motor Function
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.
Time frame: 24, 48 and 72 weeks
Change From Baseline ALS Functional Rating Scale - Revised Score - Gross Motor Function
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.
Time frame: 24, 48, 72 weeks
Change From Baseline ALS Functional Rating Scale - Revised Score - Respiratory Function
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.
Time frame: 24, 48, 72 weeks
Time to a 3, 6, 9 and 12 Points Change or Death From Baseline in ALSFRS-R Score Over 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, the time a 3, 6, 9 and 12 points change or death from baseline in ALSFRS-R.
Time frame: 72 weeks (+/-1 week visit window)
Change in Clinical Staging (King's Staging System and MiToS) Over 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.
Time frame: 72 weeks
Overall Survival: Proportion of Subjects Alive (Survival Rate) After 24, 48 and 72* Weeks;
Time frame: 24, 48 and 72* weeks;
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
Time frame: 24, 48 and 72* weeks
Change From Baseline in Slow Vital Capacity (SVC, Liters) at 24, 48 and 72* Weeks;
Time frame: 24, 48 and 72* weeks
Change From Baseline in the Overall Mega Score for the Hand-held Dynamometer (HHD) at 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.
Time frame: 24, 48 and 72* weeks
Change From Baseline in the Total Score on the ALS Assessment Questionnaire-40-Item (ALSAQ-40) Form at 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.
Time frame: 24, 48 and 72* weeks
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 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.
Time frame: 24, 48 and 72* weeks
Change From Baseline in Visual Analogue Scale (VAS) Score at 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).
Time frame: 24, 48 and 72* weeks
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;
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.
Time frame: 72 weeks
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.
| Milestone | FAB122 | Placebo |
|---|---|---|
| Started | 205 | 97 |
| Completed | 155 | 71 |
| Not completed | 50 | 26 |
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.
| units on a scale | FAB122 | Placebo |
|---|---|---|
| Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score After 48 Weeks. | -11.2 ± 7.9 | -10.8 ± 8.4 |
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.
| score on a scale | FAB122 72 Weeks | Placebo 72 Weeks | FAB122 48 Weeks | Placebo 48 Weeks |
|---|---|---|---|---|
| Combined Assessment of Function and Survival (CAFS) at 48 and 72 Weeks. | 48.7 ± 29.41 | 54.15 ± 28.83 | 153.31 ± 86.2 | 147.68 ± 92.38 |
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.
| Survival probability | FAB122 | Placebo |
|---|---|---|
| Survival Probability | 0.728 (0.599 to 0.821) | 0.870 (0.688 to 0.949) |
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.
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|
| Change From Baseline in ALSFRS-R Score After 24 and 72* Weeks | -6.0 ± 5.4 | -5.4 ± 5.1 | -15.2 ± 10.5 | -12.6 ± 8.2 |
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.
| units on a scale/week | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| The Slope of the Decrease in ALSFRS-R Score Over Time at 24, 48 and 72* Weeks; | -0.2981 ± 0.0182 | -0.2445 ± 0.265 | -0.2874 ± 0.0143 | -0.2767 ± 0.0206 | -0.3003 ± 0.0223 | -0.2531 ± 0.0313 |
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.
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| 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 ± 1.7 | -1.2 ± 1.6 | -2.1 ± 2.5 | -2.2 ± 2.8 | -3.1 ± 3.5 | -2.7 ± 2.9 |
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.
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Change From Baseline ALS Functional Rating Scale - Revised Score - Fine Motor Function | -2.0 ± 2.1 | -1.7 ± 2.0 | -3.8 ± 2.9 | -3.3 ± 2.5 | -4.7 ± 3.1 | -4.2 ± 3.3 |
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.
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Change From Baseline ALS Functional Rating Scale - Revised Score - Gross Motor Function | -2.0 ± 1.9 | -1.7 ± 1.8 | -3.4 ± 2.7 | -3.3 ± 2.2 | -4.5 ± 3.1 | -3.8 ± 2.4 |
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.
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Change From Baseline ALS Functional Rating Scale - Revised Score - Respiratory Function | -0.9 ± 2.2 | -0.8 ± 2.1 | -1.9 ± 3.2 | -2.0 ± 3.3 | -2.8 ± 4.2 | -2.0 ± 2.8 |
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.
| days | FAB122 | Placebo |
|---|---|---|
| Time to 3 points change or death from baseline in ALSFRS-R score | 85 (56 to 169) | 114 (84 to 169) |
| Time to 6 points change or death from baseline in ALSFRS-R score | 194 (113 to 286) | 226 (142 to 315) |
| Time to 9 points change or death from baseline in ALSFRS-R score | 308 (189 to 504) | 334 (197 to 510) |
| Time to 12 points change or death from baseline in ALSFRS-R score | 351 (223 to 511) | 438 (250 to 508) |
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.
| Participants | FAB122 | Placebo |
|---|---|---|
| Decline >= 1 | 22 | 12 |
| No decline | 17 | 12 |
| proportion of participants | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Overall Survival: Proportion of Subjects Alive (Survival Rate) After 24, 48 and 72* Weeks; | 0.980 (0.947 to 0.992) | 0.990 (0.928 to 0.999) | 0.933 (0.888 to 0.961) | 0.933 (0.857 to 0.970) | 0.791 (0.667 to 0.873) | 0.870 (0.688 to 0.949) |
| proportion of participants | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| 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 (0.935 to 0.986) | 0.990 (0.928 to 0.999) | 0.903 (0.853 to 0.937) | 0.890 (0.804 to 0.939) | 0.728 (0.599 to 0.821) | 0.870 (0.688 to 0.949) |
| liters | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Change From Baseline in Slow Vital Capacity (SVC, Liters) at 24, 48 and 72* Weeks; | -0.4 ± 0.5 | -0.5 ± 0.7 | -0.7 ± 0.7 | -0.7 ± 0.7 | -1 ± 1 | -1 ± 0.7 |
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.
| Z-Score | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Change From Baseline in the Overall Mega Score for the Hand-held Dynamometer (HHD) at 24, 48 and 72* Weeks. | -0.4 ± 0.7 | -0.4 ± 0.6 | -0.7 ± 1.1 | -0.8 ± 0.9 | -1.2 ± 1.2 | -1.4 ± 0.8 |
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.
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| 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 ± 12.8 | 8.9 ± 12.3 | 22.7 ± 16.8 | 19.9 ± 17.5 | 27.1 ± 18.4 | 22.5 ± 19.5 |
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.
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Anxiety/Depression - Level 1 | 82 ± 40 | 39 ± 40.2 | 57 ± 27.8 | 29 ± 29.9 | 12 ± 5.9 | 7 ± 7.2 |
| Anxiety/Depression - Level 2 | 53 ± 25.9 | 20 ± 20.6 | 52 ± 25.4 | 15 ± 15.5 | 12 ± 5.9 | 7 ± 7.2 |
| Anxiety/Depression - Level 3 | 39 ± 19 | 26 ± 26.8 | 38 ± 18.5 | 21 ± 21.6 | 11 ± 5.4 | 6 ± 6.2 |
| Anxiety/Depression - Level 4 | 12 ± 5.9 | 3 ± 3.1 | 12 ± 5.9 | 4 ± 4.1 | 6 ± 2.9 | 4 ± 4.1 |
| Anxiety/Depression - Level 5 | 0 ± 0 | 0 ± 0 | 3 ± 1.5 | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Pain/Discomfort - Level 1 | 66 ± 32.2 | 33 ± 34 | 57 ± 27.8 | 27 ± 27.8 | 13 ± 6.3 | 8 ± 8.2 |
| Pain/Discomfort - Level 2 | 61 ± 29.8 | 26 ± 26.8 | 47 ± 22.9 | 19 ± 19.6 | 10 ± 4.9 | 8 ± 8.2 |
| Pain/Discomfort - Level 3 | 48 ± 23.4 | 22 ± 22.7 | 39 ± 19 | 19 ± 19.6 | 12 ± 5.9 | 6 ± 6.2 |
| Pain/Discomfort - Level 4 | 9 ± 4.4 | 7 ± 7.2 | 14 ± 6.8 | 4 ± 4.1 | 6 ± 2.9 | 1 ± 1 |
| Pain/Discomfort - Level 5 | 2 ± 1 | 0 ± 0 | 5 ± 2.4 | 0 ± 0 | 0 ± 0 | 1 ± 1 |
| Usual Activities - Level 1 | 21 ± 10.2 | 10 ± 10.3 | 10 ± 4.9 | 4 ± 4.1 | 1 ± 0.5 | 1 ± 1 |
| Usual Activities - Level 2 | 35 ± 17.1 | 8 ± 8.2 | 22 ± 10.7 | 4 ± 4.1 | 4 ± 2 | 2 ± 2.1 |
| Usual Activities - Level 3 | 50 ± 24.4 | 31 ± 32 | 30 ± 14.6 | 22 ± 22.7 | 5 ± 2.4 | 5 ± 5.2 |
| Usual Activities - Level 4 | 41 ± 20 | 22 ± 22.7 | 42 ± 20.5 | 22 ± 22.7 | 13 ± 6.3 | 5 ± 5.2 |
| Usual Activities - Level 5 | 40 ± 19.5 | 16 ± 16.5 | 57 ± 27.8 | 17 ± 17.5 | 18 ± 8.8 | 11 ± 11.3 |
| Self-care - Level 1 | 36 ± 17.6 | 11 ± 11.3 | 16 ± 7.8 | 5 ± 5.2 | 3 ± 1.5 | 1 ± 1 |
| Self-care - Level 2 | 34 ± 16.6 | 17 ± 17.5 | 25 ± 12.2 | 6 ± 6.2 | 3 ± 1.5 | 2 ± 2.1 |
| Self-care - Level 3 | 42 ± 20.5 | 29 ± 29.9 | 26 ± 12.7 | 19 ± 19.6 | 5 ± 2.4 | 2 ± 2.1 |
| Self-care - Level 4 | 24 ± 11.7 | 11 ± 11.3 | 26 ± 12.7 | 15 ± 15.5 | 6 ± 2.9 | 5 ± 5.2 |
| Self-care - Level 5 | 51 ± 24.9 | 20 ± 20.6 | 69 ± 33.7 | 24 ± 24.7 | 24 ± 11.7 | 14 ± 14.4 |
| Mobility - Level 1 | 29 ± 14.1 | 17 ± 17.5 | 16 ± 7.8 | 9 ± 9.3 | 3 ± 1.5 | 5 ± 5.2 |
| Mobility - Level 2 | 35 ± 17.1 | 13 ± 13.4 | 21 ± 10.2 | 6 ± 6.2 | 3 ± 1.5 | 1 ± 1 |
| Mobility - Level 3 | 43 ± 21 | 19 ± 19.6 | 29 ± 14.1 | 14 ± 14.4 | 6 ± 2.9 | 2 ± 2.1 |
| Mobility - Level 4 | 49 ± 23.9 | 27 ± 27.8 | 49 ± 23.9 | 21 ± 21.6 | 14 ± 6.8 | 7 ± 7.2 |
| Mobility - Level 5 | 31 ± 15.1 | 12 ± 12.4 | 47 ± 22.9 | 19 ± 19.6 | 15 ± 7.3 | 9 ± 9.3 |
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).
| units on a scale | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| Change From Baseline in Visual Analogue Scale (VAS) Score at 24, 48 and 72* Weeks. | -10.2 ± 19.4 | -7.2 ± 23.6 | -15.7 ± 20.2 | -15.6 ± 24.9 | -25.1 ± 21.3 | -15.1 ± 22.3 |
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.
| Participants | FAB122 100mg 24 Weeks | Placebo 24 Weeks | FAB122 100mg 48 Weeks | Placebo 48 Weeks | FAB122 100mg 72 Weeks | Placebo 72 Weeks |
|---|---|---|---|---|---|---|
| ALS Specific - Change <8 | 154 | 69 | 128 | 45 | 31 | 18 |
| ALS Specific - Change >=8 | 25 | 19 | 19 | 17 | 4 | 4 |
| ALS non-Specific - Change <4 | 153 | 67 | 112 | 46 | 29 | 15 |
| ALS non-Specific - Change >=4 | 26 | 21 | 35 | 16 | 6 | 7 |
| ECAS Total Score - Change <9 | 152 | 70 | 121 | 44 | 29 | 19 |
| ECAS Total Score - Change >=9 | 27 | 18 | 26 | 18 | 6 | 3 |
Collected over 72 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| FAB122 | 20/205 (9.8%) | 62/205 (30.2%) | 180/205 (87.8%) |
| Placebo | 7/97 (7.2%) | 28/97 (28.9%) | 82/97 (84.5%) |
| Event | FAB122 | Placebo |
|---|---|---|
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 19/205 | 9/97 |
| PneumoniaInfections and infestations | 6/205 | 2/97 |
| Pulmonary embolismRespiratory, thoracic and mediastinal disorders | 3/205 | 2/97 |
| Respiratory disorderRespiratory, thoracic and mediastinal disorders | 2/205 | 2/97 |
| COVID-19Infections and infestations | 4/205 | 1/97 |
| DysphagiaGastrointestinal disorders | 4/205 | 1/97 |
| FallInjury, poisoning and procedural complications | 3/205 | 0/97 |
| Cardiac arrestCardiac disorders | 3/205 | 1/97 |
| Myocardial infarctionCardiac disorders | 3/205 | 0/97 |
| DeathGeneral disorders | 3/205 | 0/97 |
| Event | FAB122 | Placebo |
|---|---|---|
| FallInjury, poisoning and procedural complications | 40/205 | 27/97 |
| COVID-19Infections and infestations | 37/205 | 14/97 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 31/205 | 14/97 |
| ConstipationGastrointestinal disorders | 25/205 | 12/97 |
| NasopharygitisInfections and infestations | 20/205 | 6/97 |
| DysphagiaGastrointestinal disorders | 20/205 | 8/97 |
| Nerve conduction studies abnormalInvestigations | 19/205 | 9/97 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 17/205 | 5/97 |
| Urinary tract infectionInfections and infestations | 15/205 | 6/97 |
| Upper respiratory tract infectionInfections and infestations | 15/205 | 5/97 |
| Age, Continuous(years) | FAB122 | Placebo | Total |
|---|---|---|---|
| Mean | 59.3 ± 10.5 | 59.3 ± 10 | 59.3 ± 10.3 |
| Sex: Female, Male(Participants) | FAB122 | Placebo | Total |
|---|---|---|---|
| Female | 82 | 35 | 117 |
| Male | 123 | 62 | 185 |
| Race (NIH/OMB)(Participants) | FAB122 | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 2 | 0 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 1 |
| White | 185 | 90 | 275 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 18 | 6 | 24 |
| Region of Enrollment(participants) | FAB122 | Placebo | Total |
|---|---|---|---|
| Europe | 205 | 97 | 302 |
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