Amyotrophic Lateral Sclerosis
Conditions
Brief summary
A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis
Interventions
DRUGFAB122
FAB122 Daily dose 100 mg
Sponsors
Ferrer Internacional S.A.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Masking description
All subjects were treated with FAB122 in ADOREXT study, arms from parent study ADORE have been analyzed.
Intervention model description
For ADOREXT study, arms from parent study ADORE have been analyzed. Data presented for the FAB122 study group correspond to subjects receiving FAB122 in the ADORE study as well as 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).
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. who completed the full study period in the main ADORE study (FAB122-CT-2001); 2. whom the investigator has no concern and judges tolerable for initiating or continuing treatment with FAB122 from a risk and benefit point of view; 3. a female subject should not be able to become pregnant up to 30 days after the last dose of FAB122 and needs to meet at least one of the following criteria: * female who is of reproductive potential and has a negative pregnancy test at baseline and is non-lactating. * female subject who is not of reproductive potential is eligible without requiring the use of contraception 4. a male patient must: * agree he will not donate sperm during the period he will be using FAB122, AND use a condom during sexual intercourse with pregnant or non-pregnant women of childbearing potential
Exclusion criteria
1. Patient who has a medical condition or personal circumstances which, in the opinion of the investigator, will make initiation or continuation of treatment with FAB122 not tolerable for them from a risk and benefit point of view. 2. Patient who discontinued study drug prematurely in the double-blind phase of the study (ADORE Study) for safety reasons. 3. Patient who has received any other investigational drug within the period between last visit of the main study and first visit of the extension study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Experiencing Treatment Emergent Adverse Events | approximately 44 weeks | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The Secondary Efficacy Objective to Evaluate the Effect of Treatment With FAB122 Based on Change From Baseline in ALSFRS-R Until End of Study | 45 weeks | Change 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. |
Countries
Spain
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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). | 135 |
| Placebo 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). | 66 |
| Total | 201 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 0 |
| Overall Study | Death | 16 | 9 |
| Overall Study | Disease progression | 1 | 0 |
| Overall Study | Lost to Follow-up | 0 | 1 |
| Overall Study | other causes | 2 | 1 |
| Overall Study | Withdrawal by Subject | 5 | 1 |
Baseline characteristics
| Characteristic | Total | Placebo | FAB122 |
|---|---|---|---|
| Age, Customized Mean (SD) | 58.5 years STANDARD_DEVIATION 10.5 | 58.5 years STANDARD_DEVIATION 10.5 | 58.4 years STANDARD_DEVIATION 10.6 |
| BMI | 25.35 kg/m^2 STANDARD_DEVIATION 3.8 | 24.76 kg/m^2 STANDARD_DEVIATION 3.23 | 25.64 kg/m^2 STANDARD_DEVIATION 4.03 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 44 Participants | 16 Participants | 28 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 142 Participants | 48 Participants | 94 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 15 Participants | 2 Participants | 13 Participants |
| Height | 170 CM STANDARD_DEVIATION 9.6 | 169 CM STANDARD_DEVIATION 9.8 | 170.5 CM STANDARD_DEVIATION 9.5 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 18 Participants | 3 Participants | 15 Participants |
| Race (NIH/OMB) White | 182 Participants | 63 Participants | 119 Participants |
| Region of Enrollment Europe | 201 participants | 66 participants | 135 participants |
| Sex: Female, Male Female | 87 Participants | 30 Participants | 57 Participants |
| Sex: Female, Male Male | 114 Participants | 36 Participants | 78 Participants |
| Weight | 73.62 KG STANDARD_DEVIATION 14.43 | 71.07 KG STANDARD_DEVIATION 13.32 | 74.87 KG STANDARD_DEVIATION 14.83 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 16 / 135 | 9 / 66 |
| other Total, other adverse events | 77 / 135 | 41 / 66 |
| serious Total, serious adverse events | 29 / 135 | 15 / 66 |
Outcome results
Primary
Number of Participants Experiencing Treatment Emergent Adverse Events
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.
Time frame: approximately 44 weeks
Population: 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).
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | Any TEAE | 77 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | Any Serious AE | 29 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | Any Serious TEAE | 29 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | Any TEAE leading to treatment discontinuation | 7 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | Any AE leading to death | 16 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by relationship - Unrelated | 75 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by relationship - Related | 6 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by Severity - Mild | 52 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by Severity - Moderate | 32 Participants |
| FAB122 | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by Severity - Severe | 24 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by Severity - Mild | 31 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | Any TEAE | 41 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by relationship - Unrelated | 40 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | Any Serious AE | 15 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by Severity - Severe | 10 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | Any Serious TEAE | 15 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by relationship - Related | 3 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | Any TEAE leading to treatment discontinuation | 5 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | TEAE by Severity - Moderate | 17 Participants |
| Placebo | Number of Participants Experiencing Treatment Emergent Adverse Events | Any AE leading to death | 9 Participants |
Secondary
The Secondary Efficacy Objective to Evaluate the Effect of Treatment With FAB122 Based on Change From Baseline in ALSFRS-R Until End of Study
Change 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.
Time frame: 45 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 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 |
| Placebo | The Secondary Efficacy Objective to Evaluate the Effect of Treatment With FAB122 Based on Change From Baseline in ALSFRS-R Until End of Study | -3.8 units on a scale | Standard Deviation 5 |