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

NCT ID: NCT05178810

Last Updated: 2025-03-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 313 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-18
• Study Completion Date: 2023-10-26

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

FAB122

Group Type: EXPERIMENTAL

FAB122

Intervention Type: DRUG

Daily dose 100 mg

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Daily dose

Interventions

FAB122

Daily dose 100 mg

Intervention Type: DRUG

Placebo

Daily dose

Intervention Type: DRUG

Eligibility Criteria

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.

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;

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Julius Clinical

INDUSTRY

Sponsor Role: collaborator

Stichting TRICALS Foundation

OTHER

Sponsor Role: collaborator

Ferrer Internacional S.A.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University Hospitals Leuven

Leuven, , Belgium

CHRU de Lille - Hôpital Roger Salengro

Lille, , France

CHU de Limoges - Hôpital Dupuytren

Limoges, , France

Centre Hospitalo-Universitaire La Timone

Marseille, , France

CHU de Montpellier

Montpellier, , France

CHU Nice

Nice, , France

Hôpital de la Salpêtrière

Paris, , France

CHRU de Tours

Tours, , France

Universitätsmedizin Berlin

Berlin, , Germany

Universitätsklinikum Carl Gustav Carus

Dresden, , Germany

Hannover Medical School

Hanover, , Germany

Universitätsklinikum Ulm

Ulm, , Germany

Trinity College Dublin/Beaumont Hospital

Dublin, , Ireland

Azienda Ospedaliera Universitaria Cagliari

Cagliari, , Italy

Centro Clinico NEMO

Milan, , Italy

University of Milan Medical School

Milan, , Italy

University of Torino - Rita Levi Montalcini Department of Neuroscience

Milan, , Italy

Azienda Ospedaliero Universitaria Di Modena

Modena, , Italy

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

Napoli, , Italy

University of Padua - Azienda Ospedaliera di Padova

Padua, , Italy

UMC Utrecht

Utrecht, , Netherlands

Centrum Medyczne Neuromed

Bydgoszcz, , Poland

Linden Medical Centre

Krakow, , Poland

City Clinic SP. z o. o.

Warsaw, , Poland

Centro Hospitalar Universitário Lisboa-Norte

Lisbon, , Portugal

Hospital Universitari de Bellvitge

Barcelona, , Spain

Hospital Universitario de Basurto

Bilbao, , Spain

Hospital San Rafael

Madrid, , Spain

Hospital Universitario La Paz-Carlos III

Madrid, , Spain

Hospital Regional Universitario Málaga

Málaga, , Spain

Hospital Clínico Universitario de Santiago de Compostela

Santiago de Compostela, , Spain

Hospital Virgen del Rocio

Seville, , Spain

Hospital Universitario y Politécnico La Fe

Valencia, , Spain

Karolinska Institutet

Stockholm, , Sweden

King's College London

London, , United Kingdom

Manchester MND care centre

Manchester, , United Kingdom

John Radcliffe Hospital

Oxford, , United Kingdom

Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield, , United Kingdom

Countries

Belgium; France; Germany; Ireland; Italy; Netherlands; Poland; Portugal; Spain; Sweden; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

FAB122-CT-2001

Identifier Type: -

Identifier Source: org_study_id