COURAGE-ALS OLE
Research type
Research Study
Full title
A Phase 3, Open-Label Extension of COURAGE-ALS(CY 5031)
IRAS ID
1006770
Contact name
Bonnie Charpentier
Contact email
Sponsor organisation
Cytokinetics Inc
Eudract number
2021-004727-33
Clinicaltrials.gov Identifier
Research summary
Research Summary
Reldesemtiv is being investigated as a potential new therapy for the improvement of skeletal muscle function in conditions associated with muscle weakness or fatigue such as Amyotrophic Lateral Sclerosis (“ALS”). The main purpose of this research study is to evaluate the safety and tolerability of reldesemtiv. By safety, we mean the collection of information about whether there are any negative symptoms and/or side effects that may result from taking reldesemtiv. By tolerability, we mean how well a patient tolerates the unexpected medical problems that may arise while taking the study drug.Patients will participate in the study will be about 52 weeks (week 48 weeks of dosing, 4 weeks for follow-up). During this time, there will be up to six clinic visits and eight telephone visits.
Patients who have ALS and you have completed dosing in the COURAGE-ALS study (CY 5031) will be able to participate in this trial.
This trial will take place in a hospital setting.
Participants will have medical history reviewed, physical and neurological examinations, vital signs, blood sampling, pregnancy testing (if applicable), interview with ALSFRS-r (This is a questionnaire-based scale that measures and tracks changes in 12 aspects of a person’s physical function over time. It will be verbally administered by a trained evaluator who may ask additional questions to clarify your responses. ), Review of patient use of breathing devices, wheelchairs, feeding tubes, and speech aids and reviews of adverse events and concomitant medication.
Summary of Results
"Based on interim analysis results of the Phase 3 parent trial CY 5031 which met the criteria for futility in participants with ALS, CY 5032 was prematurely terminated on 31 March 2023, and all sites were asked to notify participants to discontinue study treatment on this date. As no
future clinical development or commercialization of reldesemtiv is planned, this abbreviated CSR summarizes the available safety results, as outlined in the abbreviated Safety Analaysis Plan.
Seventy-one participants who were previously enrolled in the double-blind placebo-controlled CY 5031 parent trial received at least one dose of reldesemtiv in the CY 5032 open-label extension trial. The mean duration of exposure to reldesemtiv was 95.4 days, with a range of 1 to
240 days.
Treatment with reldesemtiv was well tolerated and no participant discontinued study drug for safety reasons. TEAEs that were reported during the trial were generally indicative of progression of the underlying ALS and its common comorbidities and complications.
TESAEs were reported for 11 participants (15.5%), none of which were considered by the investigator as related to study drug. The causes of death in all five participants who died during the trial could be attributed to the progressive nature of ALS.
No participant had a concurrent ALT or AST level > 3×ULN and a total bilirubin > 2×ULN at any visit.
In conclusion, reldesemtiv was generally well tolerated, with an AE profile that is expected in a patient population with ALS and also consistent with the reldesemtiv safety profile."REC name
London - South East Research Ethics Committee
REC reference
22/LO/0908
Date of REC Opinion
30 Mar 2023
REC opinion
Further Information Favourable Opinion