ADORE (ALS Deceleration with ORal Edaravone) study

• Research type

  Research Study

• Full title

  A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis

• IRAS ID

  288856

• Contact name

  Christopher McDermott

• Sponsor organisation

  Ferrer Internacional, S.A.

• Eudract number

  2020-003376-40

• Clinicaltrials.gov Identifier

  N/A, N/A

• Duration of Study in the UK

  1 years, 7 months, 17 days

• Research summary

  Summary of Research
  Amyotrophic Lateral Sclerosis (ALS) is a very serious and fatal condition clinically characterized by muscle weakness and functional decline. This study is to assess the effect of treatment with 100 mg of FAB122 (edaravone) on disease progression in ALS patients.

  It concerns a multicenter, multinational, double-blind, randomized (2:1), placebo-controlled Phase III study. Approximately 300 subjects will be recruited by hospital sites located in the EU (26 sites), UK (4 sites) and Russia (1 site).

  Subjects will visit the clinic at Screening, Baseline, Week 4, Week 12, and every 12 weeks thereafter. Monthly telephone visits telephone visits are performed in between the visits to the clinic until Week 48.
  
  Blood sampling for FAB122 plasma concentrations will be done in ~90 subjects treated with the study drug. The study will also evaluate the PK interaction between riluzole and FAB122.

  After a subject completed the study (max at 72 weeks), he/she will be offered the possibility to roll over in an open label extension trial in which all subjects will be offered to receive FAB122.

  Summary of Results
  There was no noticeable difference between the FAB122 group and the placebo group in the main measure of ALS symptoms (ALSFRS-R score) after 48 weeks. Other measures at 48 and 72 weeks, including survival time without disease progression, also showed no significant difference between the two groups.
  Additional tests looking at different aspects of ALS symptoms, survival times, lung function, muscle strength, cognitive and behavioral function, and quality of life also did not show any significant differences between the groups.

• REC name

  Yorkshire & The Humber - Sheffield Research Ethics Committee

• REC reference

  21/YH/0144

• Date of REC Opinion

  16 Sep 2021

• REC opinion

  Further Information Favourable Opinion