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

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ClinicalTrials.gov Identifier: NCT03472950
Recruitment Status : Recruiting
First Posted : March 21, 2018
Last Update Posted : June 13, 2019
Sponsor:
Collaborator:
Gilead Sciences
Information provided by (Responsible Party):
Jeffrey Statland, University of Kansas Medical Center

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.

Condition or disease Intervention/treatment Phase
ALS Drug: Ranolazine 500 MG Drug: Ranolazine 1000 MG Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis
Actual Study Start Date : June 11, 2018
Estimated Primary Completion Date : October 1, 2019
Estimated Study Completion Date : November 1, 2019


Arm Intervention/treatment
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).




Primary Outcome Measures :
  1. Dose limiting toxicities (DLT) [ Time Frame: Up to Week 12 ]
    Measured as any drug-related serious adverse event, or drug-related adverse event necessitating study withdrawal. If a dose has less than 33% DLTs it will be considered tolerable.


Secondary Outcome Measures :
  1. Cramp Questionnaire [ Time Frame: Baseline, Weeks 2, 6, and 8 ]
    The cramp questionnaire asks if a person has experienced cramps in last week, how many total, whether they occur daily, how long they last on average (seconds - minutes), locations (body region). Responses measure average severity on a scale from 1-9. A score of 1 being mild and score of 9 being worst ever experienced.

  2. Fasciculation frequency on muscle ultrasound [ Time Frame: Baseline, Weeks 2 and 6 ]
    Count of fasciculation frequency in bilateral biceps, tibialis anterior, and gastrocnemius over 30 seconds

  3. Cramp potential duration [ Time Frame: Baseline, Weeks 2 and 6 ]
    Abductor hallucis brevis measured on EMG after supramaximal stimulation of posterior tibial nerve at 2 and 5 Hz



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03472950


Contacts
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Contact: Sherri Anderson 913-945-9936 sanderson10@kumc.edu

Locations
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United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Sponsors and Collaborators
University of Kansas Medical Center
Gilead Sciences
Investigators
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Principal Investigator: Jeffrey Statland, MD University of Kansas Medical Center

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Responsible Party: Jeffrey Statland, Assistant Professor, University of Kansas Medical Center
ClinicalTrials.gov Identifier: NCT03472950     History of Changes
Other Study ID Numbers: STUDY00141491
First Posted: March 21, 2018    Key Record Dates
Last Update Posted: June 13, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Amyotrophic Lateral Sclerosis
Ranolazine
Motor Neuron Disease
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Sodium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action