Study Purpose:
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 Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
FAB122, Placebo
Placebo:
Yes
Phase:
Phase 3
Study Chair(s)/Principal Investigator(s):
N/A
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Study Sponsor:
Ferrer Internacional S.A.
Estimated Enrollment:
300
Estimated Study Start Date:
10 / 18 / 2021
Estimated Study Completion Date:
06 / 20 / 2024
Posting Last Modified Date:
01 / 11 / 2023
Date Study Added to neals.org:
01 / 05 / 2022
Minimum Age:
18 Years
Maximum Age:
80 Years
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;
University Hospitals Leuven
Leuven
Belgium
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
CHRU de Lille - Hôpital Roger Salengro
Lille
France
Universitätsmedizin Berlin
Berlin
Germany
Universitätsklinikum Carl Gustav Carus
Dresden
Germany
Hannover Medical School
Hannover
Germany
Universitätsklinikum Ulm
Ulm
Germany
Trinity College Dublin/Beaumont Hospital
Dublin
Ireland
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
University of Torino - Rita Levi Montalcini Department of Neuroscience
Milan
Italy
Centro Clinico NEMO
Milan
Italy
Azienda Ospedaliera Universitaria Cagliari
Cagliari
Italy
University of Milan Medical School
Milan
Italy
UMC Utrecht
Utrecht
Netherlands
Centrum Medyczne Neuromed
Bydgoszcz
Poland
Linden Medical Centre
Kraków
Poland
City Clinic SP. z o. o.
Warsaw
Poland
Centro Hospitalar Universitário Lisboa-Norte
Lisboa
Portugal
Hospital Clínico Universitario de Santiago de Compostela
Santiago De Compostela
Spain
Hospital Universitario y Politécnico La Fe
Valencia
Spain
Hospital Virgen del Rocio
Sevilla
Spain
Hospital San Rafael
Madrid
Spain
Hospital Regional Universitario Málaga
Málaga
Spain
Hospital Universitario La Paz-Carlos III
Madrid
Spain
Hospital Universitario de Basurto
Bilbao
Spain
Hospital Universitari de Bellvitge
Barcelona
Spain
Karolinska Institutet
Estocolmo
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