Enrollment Open for COURAGE-ALS Trial of Reldesemtiv

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by Patricia Inacio PhD |

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COURAGE-ALS trial

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Patient enrollment has begun for Cytokinetics‘ pivotal Phase 3 clinical trial investigating reldesemtiv for the treatment of people with early amyotrophic lateral sclerosis (ALS).

The global COURAGE-ALS trial (NCT04944784) will enroll approximately 555 adults who are within two years of their first symptom of muscle weakness. Participants must have moderate lung function loss and mild disability, which is defined as an ALS Functional Rating Scale Revised (ALSFRS-R) score of no more than 44 at the time of screening. More information about study contacts and locations will be available here soon.

To enhance patient participation, the trial was designed to include remote clinic visits, home nursing visits, and mobile-app based endpoint measurements. These elements were implemented following feedback from Cytokinetics’ ALS Patient and Caregiver Advisory Council (ALS-PAC), as well as meetings with patients, caregivers, healthcare professionals, advocates, and payors.

Also, at least one patient representative will join the steering committee of COURAGE-ALS, to provide a patient perspective throughout the trial and when interpreting results.

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“We recognize the profound urgency to deliver new treatments to people with ALS and are pleased to open COURAGE-ALS after gathering important input from patients, regulators, advocates and the clinical community,” Fady I. Malik, MD, PhD, executive vice president of research and revelopment at Cytokinetics, said in a press release.

After entering COURAGE-ALS, patients will be assigned randomly to receive either a placebo or 300 mg of oral reldesemtiv twice daily over 24 weeks (nearly six months). Following that part, all participants will receive reldesemtiv for another 24 weeks.

Patients who are receiving standard ALS therapies, such as Radicava (edaravone) and riluzole, can continue doing so during the trial.

The trial’s primary goal is to measure changes in ALSFRS-R over the initial 24 weeks. Secondary goals include total ALSFRS-R scores and time to respiratory insufficiency and survival, as well as changes in respiratory function, quality of life, and muscle strength.

A data monitoring committee will conduct two planned interim analyses. The first is a futility study — meant to determine if the trial can meet its goals — that will take place 12 weeks after one-third or more of the participants are assigned randomly to receive the medication or a placebo. A second analysis also will assess for futility and determine whether the trial needs to enroll more patients.

Continued access to reldesemtiv will be available to patients who complete COURAGE-ALS, as well as those who participated in prior reldesemtiv clinical trials, the company said.

“We are also working toward our goal to provide continued access to reldesemtiv for participants who complete dosing in COURAGE-ALS, as well as to make it available to participants from our previously completed ALS trials,” Malik said.

COURAGE-ALS follows encouraging results from the recently completed FORTITUDE-ALS Phase 2 trial (NCT03160898), which evaluated reldesemtiv in 458 patients diagnosed with early ALS.

Initial results showed that reldesemtiv significantly slowed lung function decline in the overall population. A follow-up analysis revealed that participants with shorter symptom duration saw the greatest benefits, experiencing significant changes in their ALSFRS-R scores compared with those on a placebo.

Reldesemtiv is a fast skeletal muscle troponin activator designed to increase the muscles’ response to weak nerve signals. This is expected to slow the progressive muscle weakness seen in ALS and other neuromuscular diseases.

“As pioneers in muscle biology, we have been pursuing fast skeletal muscle activation for the potential treatment of ALS for over a decade and, based on the results from FORTITUDE-ALS, we believe there is a compelling rationale to advance reldesemtiv into this pivotal Phase 3 clinical trial, as it potentially may add to current standard of care and improve patients’ functional status and overall quality of life,” said Malik.