ADOREXT
Research type
Research Study
Full title
A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis
IRAS ID
1006908
Contact name
Nuria Albareda
Contact email
Sponsor organisation
Ferrer Internacional, S.A.
Eudract number
2022-003050-32
Clinicaltrials.gov Identifier
Research summary
Research Summary -
The purpose of this open label extension study is to evaluate the long-term safety of FAB122 in patients with ALS. The study will also evaluate the effects of the study drug on the participants overall survival, look at how long it takes for the disease to progress, the participants cognitive function and quality of life.
The study aims to provide longer term access to daily oral edaravone to patients who have demonstrated a good tolerance in the ADORE trial.
The treatment is planned to continue for all subjects until the marketing authorization for FAB122 is received or in case the objectives of the main study are not met (end of OLE study), provided good tolerance and safety is proven.
Participants are required to take the study treatment (fasted) by dissolving (100 mg FAB122 per sachet) in about 100 ml water, once a day.
Subjects will visit the clinic at Baseline (whenever possible, Visit 6 or 8 of the main ADORE study) or a standalone visit and then every 3 months (plus 1 week window) thereafter.
Up to 300 patients maybe enrolled into this study, to be conducted in EU and UK.
Lay Summary -
In Study FAB122-CT-2201, the mean (SD) change from extension baseline in ALSFRS-R total score at EoS was 4.4 (4.8) for the FAB122 group and 3.8 (5.0)for the placebo group. Although no formal statistical test was performed, both groups had comparable values. No significant difference was found between the FAB122 group and the placebo group in survival time using the stratified log-rank test (p=0.8627), nor using an RMST analysis up to EoS, controlling for the stratification variables (p=0.0875). Similarly, no significant difference was found between the FAB122 group and the placebo group in overall survival using the stratified log-rank test (p=0.6739), nor using an RMST analysis up to EoS, controlling for the stratification variables (p=0.3115). To note, time to EoS varied among participants. FAB122 is safe and well tolerated. Safety data from the ADOREXT OLE study are comparable with safety data from the ADORE parent study. Even though no evidence was found to support the efficacy ofFAB122, long-term treatment with FAB122 was safe and well tolerated throughout both the ADORE study and the ADOREXT study
REC name
London - Hampstead Research Ethics Committee
REC reference
22/LO/0905
Date of REC Opinion
8 Sep 2023
REC opinion
Further Information Favourable Opinion