Trial Outcomes & Findings for Study to Investigate the Long-term Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis (NCT NCT05866926)
NCT ID: NCT05866926
Last Updated: 2025-03-04
Results Overview
To evaluate the long-term safety of FAB122 in patients with ALS by assessing Number of Participants experiencing Treatment Emergent Adverse Events, evaluating nature and severity. The study duration for these subjects, and therefore the duration of FAB122 treatment, was variable depending on the subject's start date, ranging from 3 to approximately 44 weeks.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
201 participants
Primary outcome timeframe
approximately 44 weeks
Results posted on
2025-03-04
Participant Flow
Participant milestones
| Measure |
FAB122
Drug: FAB122(ADORE)-FAB122(ADOREXT)
FAB122: FAB122 Daily dose 100 mg Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as the ADOREXT study (FAB122-FAB122).
|
Placebo
Drug: Placebo(ADORE)-FAB122(ADOREXT)
FAB122: FAB122 Daily dose 100 mg Data presented for the FAB122 study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
|
|---|---|---|
|
Overall Study
STARTED
|
135
|
66
|
|
Overall Study
COMPLETED
|
110
|
54
|
|
Overall Study
NOT COMPLETED
|
25
|
12
|
Reasons for withdrawal
| Measure |
FAB122
Drug: FAB122(ADORE)-FAB122(ADOREXT)
FAB122: FAB122 Daily dose 100 mg Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as the ADOREXT study (FAB122-FAB122).
|
Placebo
Drug: Placebo(ADORE)-FAB122(ADOREXT)
FAB122: FAB122 Daily dose 100 mg Data presented for the FAB122 study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
|
|---|---|---|
|
Overall Study
Death
|
16
|
9
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
5
|
1
|
|
Overall Study
Disease progression
|
1
|
0
|
|
Overall Study
other causes
|
2
|
1
|
Baseline Characteristics
Study to Investigate the Long-term Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis
Baseline characteristics by cohort
| Measure |
FAB122
n=135 Participants
Drug: FAB122(ADORE)-FAB122(ADOREXT) FAB122: FAB122 Daily dose 100 mg Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as the ADOREXT study (FAB122-FAB122).
|
Placebo
n=66 Participants
Drug: Placebo(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg Data presented for the Placebo study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
|
Total
n=201 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
Mean (SD)
|
58.4 years
STANDARD_DEVIATION 10.6 • n=5 Participants
|
58.5 years
STANDARD_DEVIATION 10.5 • n=7 Participants
|
58.5 years
STANDARD_DEVIATION 10.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
57 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
87 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
78 Participants
n=5 Participants
|
36 Participants
n=7 Participants
|
114 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
28 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
44 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
94 Participants
n=5 Participants
|
48 Participants
n=7 Participants
|
142 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
13 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
15 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
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 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
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
119 Participants
n=5 Participants
|
63 Participants
n=7 Participants
|
182 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
|
15 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Region of Enrollment
Europe
|
135 participants
n=5 Participants
|
66 participants
n=7 Participants
|
201 participants
n=5 Participants
|
|
BMI
|
25.64 kg/m^2
STANDARD_DEVIATION 4.03 • n=5 Participants
|
24.76 kg/m^2
STANDARD_DEVIATION 3.23 • n=7 Participants
|
25.35 kg/m^2
STANDARD_DEVIATION 3.8 • n=5 Participants
|
|
Weight
|
74.87 KG
STANDARD_DEVIATION 14.83 • n=5 Participants
|
71.07 KG
STANDARD_DEVIATION 13.32 • n=7 Participants
|
73.62 KG
STANDARD_DEVIATION 14.43 • n=5 Participants
|
|
Height
|
170.5 CM
STANDARD_DEVIATION 9.5 • n=5 Participants
|
169 CM
STANDARD_DEVIATION 9.8 • n=7 Participants
|
170 CM
STANDARD_DEVIATION 9.6 • n=5 Participants
|
PRIMARY outcome
Timeframe: approximately 44 weeksPopulation: Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as in the ADOREXT study (FAB122-FAB122). Data presented for the Placebo study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
To evaluate the long-term safety of FAB122 in patients with ALS by assessing Number of Participants experiencing Treatment Emergent Adverse Events, evaluating nature and severity. The study duration for these subjects, and therefore the duration of FAB122 treatment, was variable depending on the subject's start date, ranging from 3 to approximately 44 weeks.
Outcome measures
| Measure |
FAB122
n=135 Participants
Drug: FAB122(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as in the ADOREXT study (FAB122-FAB122).
|
Placebo
n=66 Participants
Drug: Placebo(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the Placebo study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
|
|---|---|---|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
Any TEAE
|
77 Participants
|
41 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
Any Serious AE
|
29 Participants
|
15 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
Any Serious TEAE
|
29 Participants
|
15 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
TEAE by relationship - Related
|
6 Participants
|
3 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
TEAE by Severity - Severe
|
24 Participants
|
10 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
Any TEAE leading to treatment discontinuation
|
7 Participants
|
5 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
Any AE leading to death
|
16 Participants
|
9 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
TEAE by relationship - Unrelated
|
75 Participants
|
40 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
TEAE by Severity - Mild
|
52 Participants
|
31 Participants
|
|
Number of Participants Experiencing Treatment Emergent Adverse Events
TEAE by Severity - Moderate
|
32 Participants
|
17 Participants
|
SECONDARY outcome
Timeframe: 45 weeksChange from baseline in ALSFRS-R total score until the end of the study. 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=135 Participants
Drug: FAB122(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as in the ADOREXT study (FAB122-FAB122).
|
Placebo
n=66 Participants
Drug: Placebo(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the Placebo study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
|
|---|---|---|
|
The Secondary Efficacy Objective to Evaluate the Effect of Treatment With FAB122 Based on Change From Baseline in ALSFRS-R Until End of Study
|
-4.4 units on a scale
Standard Deviation 4.8
|
-3.8 units on a scale
Standard Deviation 5
|
Adverse Events
FAB122
Serious events: 29 serious events
Other events: 77 other events
Deaths: 16 deaths
Placebo
Serious events: 15 serious events
Other events: 41 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
FAB122
n=135 participants at risk
Drug: FAB122(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as in the ADOREXT study (FAB122-FAB122).
|
Placebo
n=66 participants at risk
Drug: Placebo(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the Placebo study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
7.4%
10/135 • Number of events 10 • 45 weeks
|
6.1%
4/66 • Number of events 4 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dysponea
|
1.5%
2/135 • Number of events 2 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Chronic respiratory failure
|
1.5%
2/135 • Number of events 2 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Infections and infestations
Pneumonia
|
3.0%
4/135 • Number of events 4 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Infections and infestations
Pneumonia aspiration
|
1.5%
2/135 • Number of events 2 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Infections and infestations
COVID-19
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Infections and infestations
Lower respiratory track infection
|
0.00%
0/135 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Cardiac disorders
Cardiac arrest
|
1.5%
2/135 • Number of events 2 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
0.00%
0/135 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
General disorders
Death
|
0.00%
0/135 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Nervous system disorders
Amyotrophic Lateral Sclerosis
|
0.00%
0/135 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Surgical and medical procedures
Hospitalization
|
0.00%
0/135 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Vascular disorders
Hypertensive crisis
|
0.00%
0/135 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Accute respiratory failure
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Lung disorder
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Infections and infestations
Tooth infection
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Cardiac disorders
Cardiac failure
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Cardiac disorders
Myocardial infarction
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Investigations
Lypoprotein increased
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Investigations
Weight decreased
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Nervous system disorders
Cognitive disorder
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Psychiatric disorders
Apathy
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
Other adverse events
| Measure |
FAB122
n=135 participants at risk
Drug: FAB122(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as in the ADOREXT study (FAB122-FAB122).
|
Placebo
n=66 participants at risk
Drug: Placebo(ADORE)-FAB122(ADOREXT) FAB122 Daily dose 100 mg
Data presented for the Placebo study group correspond to subjects receiving Placebo in the ADORE study and FAB122 in the ADOREXT study (Placebo-FAB122).
|
|---|---|---|
|
Infections and infestations
Urinary track infectious
|
8.1%
11/135 • Number of events 14 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Infections and infestations
Pneumonia
|
4.4%
6/135 • Number of events 7 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Infections and infestations
COVID-19
|
3.0%
4/135 • Number of events 4 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
2.2%
3/135 • Number of events 3 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Infections and infestations
Influenza
|
0.00%
0/135 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Infections and infestations
Lower respiratory track infection
|
0.00%
0/135 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/135 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Gastrointestinal disorders
Dysphagia
|
5.2%
7/135 • Number of events 7 • 45 weeks
|
4.5%
3/66 • Number of events 3 • 45 weeks
|
|
Gastrointestinal disorders
Constipation
|
3.0%
4/135 • Number of events 4 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Gastrointestinal disorders
Nausea
|
2.2%
3/135 • Number of events 3 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
4.5%
3/66 • Number of events 3 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
8.1%
11/135 • Number of events 11 • 45 weeks
|
6.1%
4/66 • Number of events 4 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.2%
3/135 • Number of events 3 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.5%
2/135 • Number of events 2 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
3.7%
5/135 • Number of events 6 • 45 weeks
|
9.1%
6/66 • Number of events 14 • 45 weeks
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/135 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Injury, poisoning and procedural complications
Skin laceration
|
0.00%
0/135 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Nervous system disorders
Syncope
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
General disorders
Fatigue
|
3.0%
4/135 • Number of events 4 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
General disorders
Death
|
0.00%
0/135 • 45 weeks
|
3.0%
2/66 • Number of events 2 • 45 weeks
|
|
Investigations
Gamma-glutamyltransferase increased
|
2.2%
3/135 • Number of events 3 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Psychiatric disorders
Insomnia
|
2.2%
3/135 • Number of events 3 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
|
Renal and urinary disorders
Renal Colic
|
0.74%
1/135 • Number of events 1 • 45 weeks
|
4.5%
3/66 • Number of events 3 • 45 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.2%
3/135 • Number of events 3 • 45 weeks
|
0.00%
0/66 • 45 weeks
|
|
Vascular disorders
Hypertension
|
2.2%
3/135 • Number of events 3 • 45 weeks
|
1.5%
1/66 • Number of events 1 • 45 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60