A Study of Ranolazine in ALS
NCT Number: NCT06527222
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Interventions: DRUG: Ranolazine|DRUG: Ranolazine|DRUG: Placebo
Sponsor: Jeffrey Statland
Collaborators: ALS Association
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 72
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: QUADRUPLE (PARTICIPANT, CARE_PROVIDER, INVESTIGATOR, OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-12
Completion Date: 2026-12
Locations: University of Kansas Medical Center, Fairway, Kansas, 66205, United States

Summary:
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.

Primary Outcome Measures: Frequency of Treatment-Emergent Adverse Events, 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., Up to 28 weeks|Tolerability of treatment assignment, 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., Up to 28 weeks|Muscle cramp frequency, 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., Up to 28 weeks|Muscle cramp severity, 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., Up to 28 weeks|Muscle cramps impact on quality of life, 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, Up to 28 weeks|Safety Lab Cystatin C, Patient safety measured with lab value Cystatin C in mg/L., Up to 28 weeks|Safety Lab Estimated Glomerular Filtration Rate (eGFR), Patient safety measured with lab value eGFR in mL/min/1.73., Up to 28 weeks|Safety Lab Alanine Transaminase (ALT), Patient safety measured with lab value ALT in IU/L., Up to 28 weeks|Safety Lab Aspartate Transferase (AST), Patient safety measured with lab value AST in IU/L., Up to 28 weeks|Safety Lab Alkaline Phosphatase (ALP), Patient safety measured with lab value ALP in IU/L., Up to 28 weeks|Safety Lab Total Bilirubin, Patient safety measured with lab value total bilirubin in mg/dL., Up to 28 weeks
Secondary Outcome Measures: Muscle strength, Change in muscle strength over time as measured by hand grip and hand-held dynamometry (HHD) in pounds., Up to 28 weeks|ALS Functional Rating Scale-Revised (ALSFRS-R), 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., Up to 28 weeks|Forced Vital Capacity (FVC), Change in respiratory function as measured by Forced Vital Capacity (FVC) in liters., Up to 28 weeks|Serum neurofilament light, 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., Up to 28 weeks|Lymphocyte Mitochondrial Function, 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, Up to 28 weeks