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

NCT05178810 | Completed Phase 3 Results DMC

• 1 other identifier: FAB122-CT-2001
• Study Type: interventional
• Target: 313
• Locations: 11 countries
• Sites: 38

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• 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.

  48 weeks

Secondary Outcomes (18)

• Combined Assessment of Function and Survival (CAFS) at 48 and 72 Weeks.

  48 weeks and 72 weeks

• Survival Probability

  72 weeks

• Change From Baseline in ALSFRS-R Score After 24 and 72* Weeks

  24 weeks, 72 weeks

• The Slope of the Decrease in ALSFRS-R Score Over Time at 24, 48 and 72* Weeks;

  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;

  24, 48 and 72 weeks

Study Arms (2)

FAB122

EXPERIMENTAL

Drug: FAB122

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

FAB122 DRUG

Daily dose 100 mg

FAB122

Placebo DRUG

Daily dose

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Ferrer Internacional S.A.: lead
• Julius Clinical: collaborator
• Stichting TRICALS Foundation: collaborator

Study Sites (38)

University Hospitals Leuven

Leuven, Belgium

Location

CHRU de Lille - Hôpital Roger Salengro

Lille, France

Location

CHU de Limoges - Hôpital Dupuytren

Limoges, France

Location

Centre Hospitalo-Universitaire La Timone

Marseille, France

Location

CHU de Montpellier

Montpellier, France

Location

CHU Nice

Nice, France

Location

Hôpital de la Salpêtrière

Paris, France

Location

CHRU de Tours

Tours, France

Location

Universitätsmedizin Berlin

Berlin, Germany

Location

Universitätsklinikum Carl Gustav Carus

Dresden, Germany

Location

Hannover Medical School

Hanover, Germany

Location

Universitätsklinikum Ulm

Ulm, Germany

Location

Trinity College Dublin/Beaumont Hospital

Dublin, Ireland

Location

Azienda Ospedaliera Universitaria Cagliari

Cagliari, Italy

Location

Centro Clinico NEMO

Milan, Italy

Location

University of Milan Medical School

Milan, Italy

Location

University of Torino - Rita Levi Montalcini Department of Neuroscience

Milan, Italy

Location

Azienda Ospedaliero Universitaria Di Modena

Modena, Italy

Location

Azienda Ospedaliera Universitaria ( A O U ) dell'Università degli studi della Campania "Luigi Vanvitelli"

Napoli, Italy

Location

University of Padua - Azienda Ospedaliera di Padova

Padua, Italy

Location

UMC Utrecht

Utrecht, Netherlands

Location

Centrum Medyczne Neuromed

Bydgoszcz, Poland

Location

Linden Medical Centre

Krakow, Poland

Location

City Clinic SP. z o. o.

Warsaw, Poland

Location

Centro Hospitalar Universitário Lisboa-Norte

Lisbon, Portugal

Location

Hospital Universitari de Bellvitge

Barcelona, Spain

Location

Hospital Universitario de Basurto

Bilbao, Spain

Location

Hospital San Rafael

Madrid, Spain

Location

Hospital Universitario La Paz-Carlos III

Madrid, Spain

Location

Hospital Regional Universitario Málaga

Málaga, Spain

Location

Hospital Clínico Universitario de Santiago de Compostela

Santiago de Compostela, Spain

Location

Hospital Virgen del Rocio

Seville, Spain

Location

Hospital Universitario y Politécnico La Fe

Valencia, Spain

Location

Karolinska Institutet

Stockholm, Sweden

Location

King's College London

London, United Kingdom

Location

Manchester MND care centre

Manchester, United Kingdom

Location

John Radcliffe Hospital

Oxford, United Kingdom

Location

Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield, United Kingdom

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Clinical Operations Head
• Organization: Ferrer

nalbareda@ferrer.com

+34 649 442 389

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2021
• First Posted: January 5, 2022
• Study Start: October 18, 2021
• Primary Completion: October 26, 2023
• Study Completion: October 26, 2023
• Last Updated: March 18, 2025
• Results First Posted: March 18, 2025

Record last verified: 2025-03

Locations

IE (1); SE (1); PT (1); NL (1); ES (8); BE (1); FR (7); GB (4); DE (4); PL (3); IT (7)