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

March 10, 2025 updated by: Ferrer Internacional S.A.

A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
313

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Leuven, Belgium
        • University Hospitals Leuven
  • France
      • Lille, France
        • CHRU de Lille - Hôpital Roger Salengro
      • Limoges, France
        • CHU de Limoges - Hopital Dupuytren
      • Marseille, France
        • Centre Hospitalo-Universitaire La Timone
      • Montpellier, France
        • CHU de Montpellier
      • Nice, France
        • CHU NICE
      • Paris, France
        • Hôpital de la Salpétrière
      • Tours, France
        • CHRU de Tours
  • Germany
      • Berlin, Germany
        • Universitätsmedizin Berlin
      • Dresden, Germany
        • Universitatsklinikum Carl Gustav Carus
      • Hannover, Germany
        • Hannover Medical School
      • Ulm, Germany
        • Universitätsklinikum Ulm
  • Ireland
      • Dublin, Ireland
        • Trinity College Dublin/Beaumont Hospital
  • Italy
      • Cagliari, Italy
        • Azienda Ospedaliera Universitaria Cagliari
      • Milan, Italy
        • Centro Clinico NeMO
      • Milan, Italy
        • University of Milan Medical School
      • Milan, Italy
        • University of Torino - Rita Levi Montalcini Department of Neuroscience
      • Modena, Italy
        • Azienda Ospedaliero Universitaria di Modena
      • Napoli, Italy
        • Azienda Ospedaliera Universitaria ( A O U ) dell'Università degli studi della Campania "Luigi Vanvitelli"
      • Padua, Italy
        • University of Padua - Azienda Ospedaliera di Padova
  • Netherlands
      • Utrecht, Netherlands
        • UMC Utrecht
  • Poland
      • Bydgoszcz, Poland
        • Centrum Medyczne NEUROMED
      • Kraków, Poland
        • Linden Medical Centre
      • Warsaw, Poland
        • City Clinic SP. z o. o.
  • Portugal
      • Lisboa, Portugal
        • Centro Hospitalar Universitário Lisboa-Norte
  • Spain
      • Barcelona, Spain
        • Hospital Universitari de Bellvitge
      • Bilbao, Spain
        • Hospital Universitario de Basurto
      • Madrid, Spain
        • Hospital San Rafael
      • Madrid, Spain
        • Hospital Universitario La Paz-Carlos III
      • Málaga, Spain
        • Hospital Regional Universitario Malaga
      • Santiago De Compostela, Spain
        • Hospital Clinico Universitario de Santiago de Compostela
      • Sevilla, Spain
        • Hospital Virgen Del Rocio
      • Valencia, Spain
        • Hospital Universitario y Politécnico La Fe
  • Sweden
      • Estocolmo, Sweden
        • Karolinska Institutet
  • United Kingdom
      • London, United Kingdom
        • King's College London
      • Manchester, United Kingdom
        • Manchester MND care centre
      • Oxford, United Kingdom
        • John Radcliffe Hospital
      • Sheffield, United Kingdom
        • Sheffield Teaching Hospitals NHS Foundation Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Main Inclusion Criteria:

  • Age 18 - 80 years (both inclusive), male or female;
  • Diagnosis of definite, probable, probable laboratory supported or possible ALS as based on the El Escorial and the revised Airlie House diagnostic criteria for ALS;

  • 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:

  • Muscle weakness in limbs
  • Speech/swallowing difficulties
  • Respiratory symptoms: dyspnea was noticed
  • SVC equal to or more than 70% of the predicted normal value for gender, height and age at screening visit;
  • Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit;
  • Capable of providing informed consent and complying with trial procedures.

Main Exclusion Criteria:

  • Diagnosis of Primary Lateral Sclerosis;
  • Diagnosis of Frontotemporal Dementia;
  • Diagnosis of other neurodegenerative diseases (e.g. Parkinson disease, Alzheimer disease);
  • Diagnosis of polyneuropathy;
  • Other causes of neuromuscular weakness;
  • Have a significant pulmonary disorder not attributed to ALS and/or require treatment interfering with the evaluation of ALS on respiratory function;
  • Use of intravenous (IV) edaravone within 6 months of the screening visit;
  • Depend on mechanical ventilation (invasive or non-invasive) or require tracheostomy at Screening;
  • Renal impairment as indicated by a creatinine clearance of less than 50 mL/min as calculated by the Cockcroft Gault equation;
  • Subject has a history of clinically significant hepatic disease, hepatitis or biliary tract disease, or subject has a positive screening test for HIV, hepatitis B or C;

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Drug: Placebo
Daily dose
Experimental: FAB122
Drug: FAB122
Daily dose 100 mg

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score After 48 Weeks.
Time Frame: 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.
48 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Combined Assessment of Function and Survival (CAFS) at 48 and 72 Weeks.
Time Frame: 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.
48 weeks and 72 weeks
Survival Probability
Time Frame: 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.
72 weeks
Change From Baseline in ALSFRS-R Score After 24 and 72* Weeks
Time Frame: 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.
24 weeks, 72 weeks
The Slope of the Decrease in ALSFRS-R Score Over Time at 24, 48 and 72* Weeks;
Time Frame: 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.
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;
Time Frame: 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.
24, 48 and 72 weeks
Change From Baseline ALS Functional Rating Scale - Revised Score - Fine Motor Function
Time Frame: 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.
24, 48 and 72 weeks
Change From Baseline ALS Functional Rating Scale - Revised Score - Gross Motor Function
Time Frame: 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.
24, 48, 72 weeks
Change From Baseline ALS Functional Rating Scale - Revised Score - Respiratory Function
Time Frame: 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.
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;
Time Frame: 72 weeks (+/-1 week visit window)

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.
72 weeks (+/-1 week visit window)
Change in Clinical Staging (King's Staging System and MiToS) Over 72 Weeks
Time Frame: 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.
72 weeks
Overall Survival: Proportion of Subjects Alive (Survival Rate) After 24, 48 and 72* Weeks;
Time Frame: 24, 48 and 72* weeks;
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
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
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.
Time Frame: 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.
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;
Time Frame: 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.
24, 48 and 72* weeks
Change From Baseline in EuroQoL - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Score 24, 48 and 72* Weeks.
Time Frame: 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.
24, 48 and 72* weeks
Change From Baseline in Visual Analogue Scale (VAS) Score at 24, 48 and 72* Weeks.
Time Frame: 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).
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;
Time Frame: 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.
72 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ferrer Internacional S.A.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Julius Clinical
Stichting TRICALS Foundation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 18, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 26, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 26, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 16, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 16, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 5, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 10, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • FAB122-CT-2001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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