A Study of Ranolazine in ALS
ConditionAmyotrophic Lateral Sclerosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorSwathy Chandrashekhar, MBBS
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
Ranolazine
Drug500mg twice daily
Ranolazine
Drug1000 mg twice daily
Placebo
DrugRanolazine placebo twice daily
Treatment groups
72 Participants
are divided into 3 treatment groupsGroup A: Ranolazine low doseExperimental treatment 1 intervention
Group B: Ranolazine high doseExperimental treatment 1 intervention
Group C: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
Up to 28 weeks2
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.
Time frame
Up to 28 weeks3
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.
Time frame
Up to 28 weeks4
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.
Time frame
Up to 28 weeksSecondary outcomes
1
Muscle strength
Change in muscle strength over time as measured by hand grip and hand-held dynamometry (HHD) in pounds.
Time frame
Up to 28 weeks2
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.
Time frame
Up to 28 weeks3
Forced Vital Capacity (FVC)
Change in respiratory function as measured by Forced Vital Capacity (FVC) in liters.
Time frame
Up to 28 weeks4
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.
Time frame
Up to 28 weeksSponsors and contacts
Click on the lead sponsor to view all of their trials.
Swathy Chandrashekhar, MBBS
Lead sponsor
University of California, San Francisco
Sponsor institution
ALS Association
Collaborator
Source ClinicalTrials.gov