To assess the long-term safety and tolerability of reldesemtiv in patients with ALS
ID
Source
Brief title
Condition
- Neuromuscular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The incidence of adverse events (AEs) in the patient population
Secondary outcome
• Time to the first occurrence of respiratory insufficiency (defined as
tracheostomy for any reason or the use of non-invasive ventilation (NIV) for
>=22 hours per day for >=10 consecutive days) or death from date of randomization
in CY 5031 through Week 48 of CY 5032
• Time to the first hospitalization from Day 1 in CY 5031 through Week 48 of CY
5032
• Combined assessment of change in ALSFRS-R total score, time to onset of
respiratory insufficiency, and survival time from baseline of CY 5031 through
Week 48 of CY 5032 and from Week 24 of CY 5031 through Week 48 of CY 5032
• Changes in ALS Functional Rating Scale - Revised (ALSFRS-R) total score from
baseline of CY 5031 through Week 48 of CY 5032 and from Week 24 of CY 5031
through Week 48 of CY 5032
• Slopes of the changes in ALSFRS-R total score from baseline of CY 5031
through Week 48 of CY 5032 and from Week 24 of CY 5031 through Week 48 of CY
5032
Background summary
Reldesemtiv, a fast skeletal muscle troponin activator, is being investigated
as a potential therapy to slow the decline of skeletal muscle function in
patients with ALS. Patients who have completed the Phase 3 clinical trial
(COURAGE-ALS [CY 5031]) of reldesemtiv in patients with ALS may continue to
receive reldesemtiv in the open label extension (OLE), CY 5032. During the last
24 weeks of dosing in CY 5031, all patients receive open-label reldesemtiv;
consequently, all patients eligible for and entering CY 5032 will have already
demonstrated acceptable tolerance of reldesemtiv when they begin dosing. The
OLE permits patients to continue to receive reldesemtiv after completion of CY
5031.
Study objective
To assess the long-term safety and tolerability of reldesemtiv in patients with
ALS
Study design
Open-label Phase-3
Intervention
300 mg reldesemtiv twice daily.
Study burden and risks
2x physical examination
2x neurological examination
6x blood and urine sampling
14x questionnaire taking.
Oyster Point Blvd 350
San Francisco CA 94080
US
Oyster Point Blvd 350
San Francisco CA 94080
US
Listed location countries
Age
Inclusion criteria
• Able to comprehend and willing to sign an Informed Consent Form (ICF). If the
patient is able to comprehend, but non-written consent is given, an impartial
witness must sign the ICF form
• Completed dosing in CY 5031
Exclusion criteria
• Has taken an investigational study drug (other than reldesemtiv) prior to
dosing, within 30 days or five half-lives of the prior agent, whichever is
greater.
When during the study the liver and/or kidney function tests rise to above 3x
ULN, the medication intake will be stopped permanently.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2021-004727-33-NL |
| CCMO | NL81821.041.22 |
| Other | www.clinicaltrials.gov |