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

Amyotrophic Lateral Sclerosis Clinical Trial

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Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05178810
• Other study ID #: FAB122-CT-2001
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: October 18, 2021
• Est. completion date: June 20, 2024

Study information

• Verified date: January 2023
• Source: Ferrer Internacional S.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 300
• Est. completion date: June 20, 2024
• Est. primary completion date: June 20, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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;

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• FAB122
Daily dose 100 mg
• Placebo
Daily dose

Locations

Country	Name	City	State
Belgium	University Hospitals Leuven	Leuven
France	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
Germany	Universitätsmedizin Berlin	Berlin
Germany	Universitätsklinikum Carl Gustav Carus	Dresden
Germany	Hannover Medical School	Hannover
Germany	Universitätsklinikum Ulm	Ulm
Ireland	Trinity College Dublin/Beaumont Hospital	Dublin
Italy	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
Netherlands	UMC Utrecht	Utrecht
Poland	Centrum Medyczne Neuromed	Bydgoszcz
Poland	Linden Medical Centre	Kraków
Poland	City Clinic SP. z o. o.	Warsaw
Portugal	Centro Hospitalar Universitário Lisboa-Norte	Lisboa
Spain	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	Sevilla
Spain	Hospital Universitario y Politécnico La Fe	Valencia
Sweden	Karolinska Institutet	Estocolmo
United Kingdom	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

Sponsors (3)

Lead Sponsor	Collaborator
Ferrer Internacional S.A.	Julius Clinical, Stichting TRICALS Foundation

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R)	The total score ranges from 48 (no limitation in daily activities) to 0 (total inability).	48 weeks