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

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.

Condition

  • ALS

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Ranolazine 500mg 		Participants will take Ranolazine 500mg twice daily for up to 4 weeks.
  • Drug: 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).
Experimental
Ranolazine 1000mg 		Participants will take Ranolazine 1000mg twice daily for up to 4 weeks.
  • 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).

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66160

More Details

Status
Recruiting
Sponsor
University of Kansas Medical Center

Study Contact

Sherri Anderson
913-945-9936
sanderson10@kumc.edu

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.