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

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:

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:

NCT05178810

Neals Affiliated?

No

Coordinating Center Contact Information

Ferrer MedInfo / email hidden; JavaScript is required / +34 609 850 565

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:

10 / 13 / 2022

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 | Recruiting

Philip Van Damme

Leuven
Belgium

CHU de Limoges - Hôpital Dupuytren | Recruiting

Phillippe Couratier

Limoges
France

Centre Hospitalo-Universitaire La Timone | Recruiting

Shahram Attarian

Marseille
France

CHU de Montpellier | Recruiting

Elisa De La Cruz

Montpellier
France

CHU Nice | Recruiting

Marie-Helene Soriani

Nice
France

Hôpital de la Salpêtrière | Recruiting

Francois Salachas

Paris
France

CHRU de Tours | Recruiting

Philippe Corcia

Tours
France

CHRU de Lille - Hôpital Roger Salengro | Recruiting

Véronique Danel Brunaud

Lille
France

Universitätsmedizin Berlin | Recruiting

Thomas Meyer

Berlin
Germany

Universitätsklinikum Carl Gustav Carus | Recruiting

René Gunther

Dresden
Germany

Hannover Medical School | Recruiting

Susanne Petri

Hannover
Germany

Universitätsklinikum Ulm | Recruiting

Albert Ludolph

Ulm
Germany

Trinity College Dublin/Beaumont Hospital | Recruiting

Orla Hardiman

Dublin
Ireland

Azienda Ospedaliero Universitaria Di Modena | Recruiting

Jessica Mandrioli

Modena
Italy

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

Francesca Trojsi

Napoli
Italy

University of Padua - Azienda Ospedaliera di Padova | Recruiting

Gianni Soraru

Padua
Italy

University of Torino - Rita Levi Montalcini Department of Neuroscience | Recruiting

Andrea Calvo

Milan
Italy

Centro Clinico NEMO | Recruiting

Valeria Sansone

Milan
Italy

Azienda Ospedaliera Universitaria Cagliari | Recruiting

Giuseppe Borghero

Cagliari
Italy

University of Milan Medical School | Recruiting

Vincenzo Silani

Milan
Italy

UMC Utrecht | Recruiting

Leonard van den Berg

Utrecht
Netherlands

Centrum Medyczne Neuromed | Recruiting

Pawel Lisewski

Bydgoszcz
Poland

Linden Medical Centre | Recruiting

Jakub Antczak

Kraków
Poland

City Clinic SP. z o. o. | Recruiting

Magdalena Kuzma-Kozakiewicz

Warsaw
Poland

Centro Hospitalar Universitário Lisboa-Norte | Recruiting

Mamede De Carvalho

Lisboa
Portugal

Hospital Clínico Universitario de Santiago de Compostela | Recruiting

Julio Pardo Fernandez

Santiago De Compostela
Spain

Hospital Universitario y Politécnico La Fe | Recruiting

Juan Francisco Vázquez Costa

Valencia
Spain

Hospital Virgen del Rocio | Recruiting

Carmen Paradas

Sevilla
Spain

Hospital San Rafael | Recruiting

Jesus Mora

Madrid
Spain

Hospital Regional Universitario Málaga | Recruiting

Virginia Reyes Garrido

Málaga
Spain

Hospital Universitario La Paz-Carlos III | Recruiting

Javier Mascias

Madrid
Spain

Hospital Universitario de Basurto | Recruiting

Luis Varona

Bilbao
Spain

Hospital Universitari de Bellvitge | Recruiting

Monica Povedano

Barcelona
Spain

Karolinska Institutet | Recruiting

Caroline Ingre

Estocolmo
Sweden

King's College London | Recruiting

Ammar Al-Chalabi

London
United Kingdom

Manchester MND care centre | Recruiting

John Ealing

Manchester
United Kingdom

John Radcliffe Hospital | Recruiting

Martin Turner

Oxford
United Kingdom

Sheffield Teaching Hospitals NHS Foundation Trust | Recruiting

Christopher McDermott

Sheffield
United Kingdom