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

Ferrer

Status and phase

Completed

Phase 3

Conditions

Amyotrophic Lateral Sclerosis

Treatments

Drug: FAB122

Drug: Placebo

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT05178810

FAB122-CT-2001

Details and patient eligibility

About

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.

Enrollment

313 patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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;