Amyotrophic Lateral SclerosisPhase 3NCT05442775
A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
A Phase 3 study testing Reldesemtiv in people with Amyotrophic Lateral Sclerosis, run by Cytokinetics at 41 sites in 9 countries.
Trial summary
The purpose of this study is to assess the long-term safety and tolerability of reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Reldesemtiv
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 71 participants
- Where
- 41 sitesin 9 countries
- Study dates
- 4 Aug 2022to 7 Jun 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able to comprehend and willing to sign an ICF and willing to comply with all study procedures and restrictions for the duration specified in the Schedule of Activities. If non-written consent is given, a Legal Designee of the patient must sign the ICF form.
- Completed dosing in CY 5031
Who cannot join
- Has taken investigational study drug (other than reldesemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
- Presence on Day 1 of any medically significant cardiac, pulmonary, gastrointestinal, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data.
- Use of a strong cytochrome P450 (CYP) 3A4 inhibitor within 7 days prior to first dose of reldesemtiv in CY 5032 or a strong CYP3A4 inducer within 14 days prior to first dose of reldesemtiv in CY 5032
- Use of a medication that is an OCT1/OCT2 substrate within 7 days prior to first dose of reldesemtiv in CY 5032
- Currently participating in another trial, managed access program, open label extension, early access program, or through the right to try act is receiving an investigational drug or received an investigational drug or device within 30 days (or 5 half-lives for drugs, whichever is longer) prior to Day 1. Patients also cannot be taking outside of a clinical trial certain investigational drugs (which includes drugs, supplements, and nutraceuticals) that are currently being studied or have been studied for the treatment of ALS.
Trial locations and contacts
41 sites in 9 countries.
41 sites in 9 countries
United States21 sites
Michigan Medicine
Ann Arbor, Michigan, 48109
University of Colorado Hospital Anschutz Outpatient Pavilion
Aurora, Colorado, 80045
Johns Hopkins Outpatient Center
Baltimore, Maryland, 21287
Atrium Health Neuroscience Institute
Charlotte, North Carolina, 28207
Cleveland Clinic
Cleveland, Ohio, 44195
Texas Neurology, P.A.
Dallas, Texas, 75206
Henry Ford Hospital
Detroit, Michigan, 48202
Virginia Commonwealth University
Henrico, Virginia, 23233
Indiana University IU Health Neuroscience Center
Indianapolis, Indiana, 43202
University of Florida
Jacksonville, Florida, 32209
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224
The University of Kansas Medical Center
Kansas City, Kansas, 66160
Neurology Associates
Lincoln, Nebraska, 68506
Froedtert Hospital - Department of Neurology
Milwaukee, Wisconsin, 53226
Hospital for Special Surgery
New York, New York, 10021
University of California Irvine - ALS & Neuromuscular Center
Orange, California, 92868
St. Joseph's Hospital & Medical Center - Barrow Neurological Institute
Phoenix, Arizona, 85013
California Pacific Medical Center - Forbes Norris MDA/ALS Research Center
San Francisco, California, 94109
SUNY Upstate Medical University Institute for Human Performance
Syracuse, New York, 13210
University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
Tampa, Florida, 33612
George Washington Medical Faculty Associates
Washington D.C., District of Columbia, 20037
Canada9 sites
University of Calgary - Heritage Medical Research Clinic
Calgary, Alberta, T2N 4Z6
University of Alberta
Edmonton, Alberta, T6G1Z1
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, E3B 0C7
McMaster University
Hamilton, Ontario, L8N4K1
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
Ottawa Hospital Research Institute
Ottawa, Ontario, K1Y4E9
CHU de Quebec-Universite Laval
Québec, G1J1Z4
University of Saskatchewan
Saskatoon, Saskatchewan, S7K0M7
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
Australia3 sites
Concord Repatriation General Hospital
Concord, New South Wales, 2139
Royal Brisbane and Women's Hospital, Neurology Department
Herston, Queensland, 4029
The Perron Institute
Nedlands, 6009
Italy2 sites
IRCCS Istituto Auxologico Italiano Ospedale San Luca U.O. Neurologia e Stroke Unit
Milan, 20149
AOU Citta della Salute e della Scienza di Torino Universita degli Studi di Torino P.O. Mollinette - Dipartimento de Neuroscienze "Rita Levi Montalcini"
Torino, 10126
Spain2 sites
Hospital San Rafael
Madrid, 28016
Hospital Universitario y Politecnico La Fe
Valencia, 46026
Netherlands1 site
UMC Utrecht Department of Neurology, ALS Center
Utrecht, 3584
About this study
The purpose of this study is to assess the long-term safety and tolerability of reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS)
Read the detailed description
CY 5032 is an open-label extension (OLE) study of the selective fast skeletal muscle troponin activator, reldesemtiv, in patients with ALS who finished dosing (through Week 48) in CY 5031 (COURAGE-ALS). Approximately 400 patients from the sites that participated in CY 5031 are expected to be enrolled in the open-label extension, CY 5032.
Following enrollment, patients will continue dosing with reldesemtiv, 300 mg twice a day for a 600 mg total daily dose (TDD) for a period of 48 weeks.
At the end of 48 weeks, patients may transition to a reldesemtiv Managed Access Program (MAP) if the treating physician agrees to participate in the program.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Reldesemtiv
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, CK-2127107, Reldesemtiv
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ReldesemtivDrug
Oral tablet
| Study group | Role | What they receive |
|---|---|---|
| Reldesemtiv 300 mg twice daily | Experimental | Patients in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD). Participants who had been down titrated during the parent trail take 150 mg by mouth twice a day. Receives: Drug: Reldesemtiv |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long-term Safety and Tolerability
Incidence of treatment-emergent adverse events
Measured: Baseline to Week 34 (time the study was terminated prematurely)
Secondary outcomes (1)
Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes
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.
Measured: Baseline to Week 32 (last timepoint before study was terminated prematurely)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2022
- Study start4 Aug 2022
- Primary completion7 Jun 2023
- Study completion7 Jun 2023
Why it stopped: The DMC recommended the trial be discontinued due to futility following a planned second interim analysis of the parent trial (CY 5031).
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cytokinetics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Cytokinetics MD, Study chair, Scientific Leadership at Cytokinetics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05442775
- Sponsor's protocol ID
- CY 5032
- Other IDs
- 2021-004727-33
- Status last verified
- Jul 2024
- First submitted
- 16 Jun 2022
- Last updated
- 13 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Shin E, Bhat AG, Ramanathan M. Large Language Models for Clinical Trial Protocol Assessments. Clin Pharmacol Ther. 2026 Feb;119(2):393-402. doi: 10.1002/cpt.70096. Epub 2025 Oct 21.
PubMed 41121745
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
- ClinicalTrials.gov ID
- NCT05442775
- Acronym / sponsor ID
- CY 5032
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, CK-2127107, Reldesemtiv
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The DMC recommended the trial be discontinued due to futility following a planned second interim analysis of the parent trial (CY 5031).
- Enrolment
- 71 participants (actual)
- Lead sponsor
- CytokineticsIndustry (pharma, biotech, devices)
- Study start
- 4 Aug 2022 (actual)
- Primary completion
- 7 Jun 2023 (actual)
- Study completion
- 7 Jun 2023 (actual)
- First posted
- 5 Jul 2022 (actual)
- Results
- Posted 13 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the long-term safety and tolerability of reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS)
Detailed description
CY 5032 is an open-label extension (OLE) study of the selective fast skeletal muscle troponin activator, reldesemtiv, in patients with ALS who finished dosing (through Week 48) in CY 5031 (COURAGE-ALS). Approximately 400 patients from the sites that participated in CY 5031 are expected to be enrolled in the open-label extension, CY 5032.
Following enrollment, patients will continue dosing with reldesemtiv, 300 mg twice a day for a 600 mg total daily dose (TDD) for a period of 48 weeks.
At the end of 48 weeks, patients may transition to a reldesemtiv Managed Access Program (MAP) if the treating physician agrees to participate in the program.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Reldesemtiv 300 mg twice daily | Experimental | Drug: ReldesemtivPatients in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD). Participants who had been down titrated during the parent trail take 150 mg by mouth twice a day. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Able to comprehend and willing to sign an ICF and willing to comply with all study procedures and restrictions for the duration specified in the Schedule of Activities. If non-written consent is given, a Legal Designee of the patient must sign the ICF form.
- Completed dosing in CY 5031
Key exclusion criteria (5 listed)
- Has taken investigational study drug (other than reldesemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
- Presence on Day 1 of any medically significant cardiac, pulmonary, gastrointestinal, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data.
- Use of a strong cytochrome P450 (CYP) 3A4 inhibitor within 7 days prior to first dose of reldesemtiv in CY 5032 or a strong CYP3A4 inducer within 14 days prior to first dose of reldesemtiv in CY 5032
- Use of a medication that is an OCT1/OCT2 substrate within 7 days prior to first dose of reldesemtiv in CY 5032
- Currently participating in another trial, managed access program, open label extension, early access program, or through the right to try act is receiving an investigational drug or received an investigational drug or device within 30 days (or 5 half-lives for drugs, whichever is longer) prior to Day 1. Patients also cannot be taking outside of a clinical trial certain investigational drugs (which includes drugs, supplements, and nutraceuticals) that are currently being studied or have been studied for the treatment of ALS.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ReldesemtivOral tablet | Drug | Reldesemtiv 300 mg twice daily |
Primary outcome
Long-term Safety and Tolerability
Time frame: Baseline to Week 34 (time the study was terminated prematurely)
Incidence of treatment-emergent adverse events
1 secondary outcome
Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes
Time frame: Baseline to Week 32 (last timepoint before study was terminated prematurely)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41121745Shin E, Bhat AG, Ramanathan M. Large Language Models for Clinical Trial Protocol Assessments. Clin Pharmacol Ther. 2026 Feb;119(2):393-402. doi: 10.1002/cpt.70096. Epub 2025 Oct 21.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Cytokinetics MD study chair
Scientific Leadership at Cytokinetics
41 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Reldesemtiv in people with Amyotrophic Lateral Sclerosis, run by Cytokinetics at 41 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 13 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in United States, Canada, Australia, Italy, Spain and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long-term Safety and Tolerability (time frame: Baseline to Week 34 (time the study was terminated prematurely)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05442775, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2024.