Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis

NCT ID: NCT03472950

Last Updated: 2024-12-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-06-11
• Study Completion Date: 2022-12-09

Brief Summary

The purpose of this research study is to evaluate the safety and effectiveness of Ranolazine, and how well it is tolerated in patients with Amyotrophic Lateral Sclerosis (ALS). Ranolazine is an FDA approved drug that is used for decreasing chest pain.

Detailed Description

Amyotrophic Lateral Sclerosis (ALS) is a progressive debilitating and fatal neurodegenerative disease involving the motor neurons in the primary motor cortex, corticospinal tracts, brainstem and spinal cord with 5,000 newly diagnosed patients per year in the USA. There is a pressing need for additional therapies, as the only two FDA-approved drugs for ALS, riluzole and edaravone, showed prolongation of median survival of only two to three months and only a modest benefit in daily functioning, respectively. The ability to identify FDA approved drugs which can be repurposed to ALS, and which may slow disease progression, alleviate symptoms, or prolong survival will have an immediate positive impact of the lives of patients with ALS and their family members. Hypothesis: Ranolazine, an FDA approved drug for angina which inhibits the late Na+ current and intracellular Ca2+ accumulation may be neuroprotective in ALS by reducing neuronal hyperexcitability, may slow disease progression and reduce cramp frequency.

Conditions

ALS

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ranolazine 500mg

Participants will take Ranolazine 500mg twice daily for up to 4 weeks.

Group Type: EXPERIMENTAL

Ranolazine 500 MG

Intervention Type: DRUG

Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).

Ranolazine 1000mg

Participants will take Ranolazine 1000mg twice daily for up to 4 weeks.

Group Type: EXPERIMENTAL

Ranolazine 1000 MG

Intervention Type: DRUG

Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).

Interventions

Ranolazine 500 MG

Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).

Intervention Type: DRUG

Ranolazine 1000 MG

Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Patients with clinically definite, probable, laboratory supported probable, or possible ALS per revised El Escorial criteria
• Cramp frequency greater than 4 cramps per week during 2 week run in
• ALS functional rating scale-revised (ALSFRS-R) score of greater than 24
• Able to lie on back for study procedures

Exclusion Criteria

• Tracheostomy invasive ventilation, or use of non-invasive ventilation greater than 12 hours per day
• Pregnant or lactating
• Participation in a prior experimental drug trial less than 30 days prior to screening
• Patients taking ranolazine
• Patients taking medications which are contraindicated for use with ranolazine such as strong CYP3 inhibitors (ketoconazole, clarithromycin, nelfinavir), and CYP3 inducers (rifampin, phenobarbital)
• Patients with clinically significant medical comorbidities (hepatic, renal, cardiac, etc)
• Patients with baseline QT interval prolongation on Electrocardiography (ECG)
• Patients pre-disposed to secondary QT prolongation for other health conditions like family history of congenital long QT syndrome, heart failure, bradycardia, or cardiomyopathies

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gilead Sciences

INDUSTRY

Sponsor Role: collaborator

University of Kansas Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Jeffrey Statland

Assistant Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Jeffrey Statland, MD

Role: PRINCIPAL_INVESTIGATOR

University of Kansas Medical Center

Locations

University of Kansas Medical Center

Kansas City, Kansas, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

STUDY00141491

Identifier Type: -

Identifier Source: org_study_id