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

Study Purpose:

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.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Jeffrey Statland, MD, University of Kansas Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT03472950

Neals Affiliated?

No

Coordinating Center Contact Information

University of Kansas Medical Center
Laura Herbelin / .(JavaScript must be enabled to view this email address) / 913-588-5095
.(JavaScript must be enabled to view this email address) Kansas City, Kansas 66160 United States

Full Study Summary:

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.

Study Sponsor:

University of Kansas Medical Center

Participant Duration:

Estimated Enrollment:

20

Estimated Study Start Date:

06/11/2018

Estimated Study Completion Date:

06/11/2019

Posting Last Modified Date:

08/06/2018

Date Study Added to alsconsortium.org:

08/06/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    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

  • Site Contact Information

    University of Kansas Medical Center
    Kansas City, Kansas 66160
    United States