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NCT Number: NCT06527222

A Study of Ranolazine in ALS

The purpose of this study is to evaluate safety, effect on cramps, function and quality of life of ranolazine versus placebo for the treatment of ALS.

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Key information

Conditions

Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of California, San Francisco, San Francisco, California, United States

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About this study

A prospective, multi center, double-blind, placebo-controlled, parallel group study of 2 doses of ranolazine (500 mg and 1000 mg twice daily) compared to placebo in patients with ALS.

Approximately 72 adults with ALS will be enrolled into the study in the United States at approximately 7 ALS treatment sites.

Participants will take oral ranolazine or placebo twice daily, attend a minimum of 5 onsite research visits, and 4 remote research visits.

The study is estimated to last 28 weeks for each participant.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 18 years or older
  • Diagnosed with clinically definite, possible, probably, or lab-supported probable ALS per revised El Escorial criteria
  • Breathing assessment called forced vital capacity (FVC) greater than or equal to 50%.
  • Able to swallow pills at the start of the study and expected to for the length of the study.
  • If on ALS modifying medications must be on a stable dose at least 30 days.
  • Experiencing 4 or more cramps per week during a 2-week screening period.

Exclusion criteria

  • Disease duration < 5 years
  • Tracheostomy invasive ventilation, or noninvasive ventilation of more than 12 hours/day
  • Pregnant or lactating, adults unable to consent, and prisoners
  • Taking ranolazine or investigational drug or has received an investigational drug within 30 days (or 5 half-lives for drugs, whichever is longer) prior to screening
  • Medically uncontrolled comorbidities (heart, liver, kidney disease)
  • Baseline QTc interval prolongation >450 ms for men/ >470 ms for women, history of long QT syndrome, or medications which prolong the QT interval
  • Participation in an experimental drug trial less than 30 days before screening
  • Patients have to be on a stable dosage of any medications used to treat muscle cramps for ≥30 days or have been off these medications ≥30 days prior to randomization.

Treatment and study plan

Ranolazine

Drug

500mg twice daily

Other names: Ranexa

Placebo

Drug

Ranolazine placebo twice daily

Primary outcomes

  1. Frequency of Treatment-Emergent Adverse Events

    Time frame: Up to 28 weeks

    Patient report and medical records will be used to document adverse events and severe adverse events. Adverse events and severe adverse events will be assessed by the investigator and reported as needed for safety.

  2. Tolerability of treatment assignment

    Time frame: Up to 28 weeks

    Tolerability will be measured by percentage of patients who complete the treatment assignment. Dose limiting toxicities will be determined by individual with an adverse event necessitating stopping.

  3. Muscle cramp frequency

    Time frame: Up to 28 weeks

    Muscle cramp frequency will be measured numerically with a reporting period of 7 days.

    Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire.

  4. Muscle cramp severity

    Time frame: Up to 28 weeks

    Muscle cramp severity will be measured by a score of 1-10 (1 being a very mild muscle cramp and 10 being the most severe cramp you ever experienced). Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire.

  5. Muscle cramps impact on quality of life

    Time frame: Up to 28 weeks

    Effect of muscle cramps on quality of life will be measured with three patient reported yes or no questions (Yes indicating an impact on quality of life or no indicating no impact on quality of life). Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire

  6. Safety Lab Cystatin C

    Time frame: Up to 28 weeks

    Patient safety measured with lab value Cystatin C in mg/L.

  7. Safety Lab Estimated Glomerular Filtration Rate (eGFR)

    Time frame: Up to 28 weeks

    Patient safety measured with lab value eGFR in mL/min/1.73.

  8. Safety Lab Alanine Transaminase (ALT)

    Time frame: Up to 28 weeks

    Patient safety measured with lab value ALT in IU/L.

  9. Safety Lab Aspartate Transferase (AST)

    Time frame: Up to 28 weeks

    Patient safety measured with lab value AST in IU/L.

  10. Safety Lab Alkaline Phosphatase (ALP)

    Time frame: Up to 28 weeks

    Patient safety measured with lab value ALP in IU/L.

  11. Safety Lab Total Bilirubin

    Time frame: Up to 28 weeks

    Patient safety measured with lab value total bilirubin in mg/dL.

Secondary outcomes

  1. Muscle strength

    Time frame: Up to 28 weeks

    Change in muscle strength over time as measured by hand grip and hand-held dynamometry (HHD) in pounds.

  2. ALS Functional Rating Scale-Revised (ALSFRS-R)

    Time frame: Up to 28 weeks

    Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.

  3. Forced Vital Capacity (FVC)

    Time frame: Up to 28 weeks

    Change in respiratory function as measured by Forced Vital Capacity (FVC) in liters.

  4. Serum neurofilament light

    Time frame: Up to 28 weeks

    Changes in serum neurofilament light measured from baseline to end of treatment assignment. Data will be collected to determine differences between placebo and Ranolazine treatment groups at completion of the study.

  5. Lymphocyte Mitochondrial Function

    Time frame: Up to 28 weeks

    Change in mitochondrial function in lymphocytes measured from baseline to the end of treatment assignment. Data will be collected to determine differences between placebo and Ranolazine treatment groups at completion of the study

Study contacts

Contact information is provided by the study sponsor or research team.

Katie Lillig, BS

CONTACT

[email protected]

913-945-9932

Sponsors and collaborators

Lead sponsor

Swathy Chandrashekhar, MBBS

Other

Collaborators

  • ALS Association

Registry information

Official study title

Ranolazine in ALS: Safety, and Effect on Cramps, Function and Quality of Life.

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Jul 30, 2024
Registry last updated
Jun 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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