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

Cytokinetics

Status and phase: Completed; Phase 3

Conditions: Amyotrophic Lateral Sclerosis (ALS)

Treatments: Drug: tirasemtiv

Study type: Interventional

Funder types: Industry

Identifiers

NCT02936635

CY 4033

2016-002629-13 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).

Full description

Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.

Enrollment: 280 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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280 participants in 2 patient groups

Delayed Start Treatment

Experimental group

Description:

The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.

Treatment:

Drug: tirasemtiv

Early Start Treatment

Experimental group

Description:

The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033.

Treatment:

Drug: tirasemtiv