Trial Outcomes & Findings for A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031) (NCT NCT05442775)
NCT ID: NCT05442775
Last Updated: 2024-08-13
Results Overview
Incidence of treatment-emergent adverse events
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
71 participants
Primary outcome timeframe
Baseline to Week 34 (time the study was terminated prematurely)
Results posted on
2024-08-13
Participant Flow
Participants were enrolled from 41 trial centers in North America, Europe, and Australia. The first participant was enrolled on 04 Aug 2022 and the last participant completed on 07 Jun 2023.
A total of 71 participants who were previously enrolled in the double-blind placebo-controlled CY 5031 parent trial were enrolled in this trial and received at least one dose of reldesemtiv.
Participant milestones
| Measure |
Reldesemtiv
Participants in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD)
Reldesemtiv: Oral tablet
|
|---|---|
|
Overall Study
STARTED
|
71
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
71
|
Reasons for withdrawal
| Measure |
Reldesemtiv
Participants in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD)
Reldesemtiv: Oral tablet
|
|---|---|
|
Overall Study
Trial terminated
|
61
|
|
Overall Study
Lack of Efficacy
|
4
|
|
Overall Study
Withdrawal by Subject
|
4
|
|
Overall Study
Death
|
1
|
|
Overall Study
Participant removed at Sponsor request
|
1
|
Baseline Characteristics
A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
Baseline characteristics by cohort
| Measure |
Reldesemtiv
n=71 Participants
Participants in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD)
Reldesemtiv: Oral tablet
|
|---|---|
|
Age, Continuous
|
61.7 years
STANDARD_DEVIATION 11.62 • n=5 Participants
|
|
Sex: Female, Male
Female
|
31 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
40 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
69 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
65 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
North America
|
53 Participants
n=5 Participants
|
|
Region of Enrollment
Europe
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
8 Participants
n=5 Participants
|
|
Time Since ALS Diagnosis at Screening in CY 5031
|
17.9 months
STANDARD_DEVIATION 4.69 • n=5 Participants
|
|
Percent Predicted Forced Vital Capacity
|
70.6 percent predicted
STANDARD_DEVIATION 22.76 • n=5 Participants
|
|
ALSFRS-R Total Score
|
29.8 scores on a scale
STANDARD_DEVIATION 8.47 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 34 (time the study was terminated prematurely)Population: Safety Analysis Set
Incidence of treatment-emergent adverse events
Outcome measures
| Measure |
Reldesemtiv
n=71 Participants
Participants in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD)
Reldesemtiv: Oral tablet
|
|---|---|
|
Long-term Safety and Tolerability
|
39 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 32 (last timepoint before study was terminated prematurely)Population: Full Analysis Set
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total scores. ALSFRS-R total score range is from 0 to 48. A score of 48 reflects normal function.
Outcome measures
| Measure |
Reldesemtiv
n=71 Participants
Participants in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD)
Reldesemtiv: Oral tablet
|
|---|---|
|
Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes
Week 12
|
29.5 score on a scale
Standard Deviation 9.04
|
|
Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes
Week 24
|
27.4 score on a scale
Standard Deviation 11.86
|
|
Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes
Week 32
|
26.6 score on a scale
Standard Deviation 13.96
|
Adverse Events
Reldesemtiv
Serious events: 11 serious events
Other events: 27 other events
Deaths: 5 deaths
Serious adverse events
| Measure |
Reldesemtiv
n=71 participants at risk
Participants in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD)
Reldesemtiv: Oral tablet
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.2%
3/71 • Number of events 3 • up to 34 weeks (time the study was terminated prematurely)
|
|
Psychiatric disorders
Assisted suicide
|
2.8%
2/71 • Number of events 2 • up to 34 weeks (time the study was terminated prematurely)
|
|
Gastrointestinal disorders
Abdominal pain
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Gastrointestinal disorders
Gastric ulcer haemorrhage
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Gastrointestinal disorders
Lower gastrointestinal haemorrhage
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Gastrointestinal disorders
Pneumoperitoneum
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Infections and infestations
Klebsiella urinary tract infection
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Infections and infestations
Metapneumovirus infection
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Infections and infestations
Pneumonia aspiration
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Infections and infestations
Septic shock
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Infections and infestations
Urinary tract infection
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Cardiac disorders
Stress cardiomyopathy
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
1.4%
1/71 • Number of events 1 • up to 34 weeks (time the study was terminated prematurely)
|
Other adverse events
| Measure |
Reldesemtiv
n=71 participants at risk
Participants in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD)
Reldesemtiv: Oral tablet
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
9.9%
7/71 • Number of events 7 • up to 34 weeks (time the study was terminated prematurely)
|
|
Gastrointestinal disorders
Constipation
|
8.5%
6/71 • Number of events 6 • up to 34 weeks (time the study was terminated prematurely)
|
|
Gastrointestinal disorders
Nausea
|
5.6%
4/71 • Number of events 4 • up to 34 weeks (time the study was terminated prematurely)
|
|
Infections and infestations
Urinary tract infection
|
7.0%
5/71 • Number of events 5 • up to 34 weeks (time the study was terminated prematurely)
|
|
Injury, poisoning and procedural complications
Fall
|
7.0%
5/71 • Number of events 5 • up to 34 weeks (time the study was terminated prematurely)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place