A Study for Patients Who Completed VITALITY-ALS (CY 4031)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02936635
• Date of registration: 12/10/2016
• Prospective Registration: Yes
• Primary sponsor: Cytokinetics
• Public title: A Study for Patients Who Completed VITALITY-ALS (CY 4031) VIGOR-ALS
• Scientific title: A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
• Date of first enrolment: October 17, 2016
• Target sample size: 280
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02936635
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Portugal, Spain, United Kingdom, United States

Contacts

• Name: Study Director MD Cytokinetics
• Affiliation: Cytokinetics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal
consent is given, a Legal Designee of the patient must sign the ICF form

- Completed participation on study drug and the Follow-Up Visit in the CY 4031 study

- Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree
for the duration of their participation in the study to either:

- Use a condom during sexual intercourse with female partners who are of
childbearing potential AND to have female partners use a highly effective means
of contraception OR

- Abstain from sexual intercourse during participation in the study

- Female patients who are not post-menopausal (= 1 year) or sterilized, must:

- Not be breastfeeding

- Have a negative pregnancy test

- Have no intention to become pregnant during participation in the study AND

- Practice sexual abstinence, defined as refraining from intercourse during the
duration of the study OR if male partners are not vasectomized with a confirmed
zero sperm count, require use of a condom AND use of a highly effective
contraceptive measure

Exclusion Criteria:

- Has a diaphragm pacing system (DPS) at study entry or anticipate DPS placement during
the course of the study

- Has taken an investigational study drug (other than tirasemtiv) prior to dosing,
within 30 days or five half-lives of the prior agent, whichever is greater

- Use of tizanidine and theophylline-containing medications during study participation

- Participation or planning to participate in any form of stem cell therapy for the
treatment of ALS or another investigational drug



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis (ALS)

Intervention(s)

Drug: tirasemtiv

Primary Outcome(s)

Incidence of adverse events (AEs) in patient population [Time Frame: Until end of study, up to 36 months]

Secondary Outcome(s)

Decline in percent predicted Slow Vital Capacity (SVC) from baseline [Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months]

Time to death [Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months]

Slope of the change from baseline in ALSFRS-R [Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033]

Time to first use of assisted ventilation or death [Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months]

Decline in ALS Functional Rating Scale - Revised (ALSFRS-R) score from baseline [Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months]

Time to the first occurrence of respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation (NIV) for =22 hours per day for =10 consecutive days) or death [Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months]

Slope of the change from baseline in percent predicted SVC [Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033]

Secondary ID(s)

2016-002629-13

CY 4033

Source(s) of Monetary Support

Please refer to primary and secondary sponsors