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NCT06527222ClinicalTrials.gov

A Study of Ranolazine in ALS

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

מגייסמקבל משתתפים כעת, לפי רשומת המרשם.
יצירת קשר עם המחקר

בקצרה

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.

שלב 2דרושים 72 משתתפים6 אתריםמדינה אחת

קטגוריות

נוירולוגיה74%אנדוקריני ומטבולי26%

רשום במרשם אחד

מחקר יחיד יכול להיות רשום בכמה מרשמים. אנו מציגים אותו פעם אחת ומקשרים לכל רשומה שברשותנו.

מידע הניסוי מוצג כפי שפורסם על ידי המרשם, בשפת המקור שלו.

מתעניינים במחקר הזה?

התחברות או צרו חשבון כדי לרשום את התעניינותכם ולעקוב אחר המחקר הזה.

How this study is set up

The points below are drawn from the public registry record for this study. Each one cites the field or sentence it came from. A dash (—) means the record does not state something — not that it is missing or wrong. Many well-run studies, especially small ones, leave some of these blank.

  • Registered before enrolment beganFirst posted 2024-07-30; recorded start 2025-04-29
  • Has a defined primary outcomeA primary outcome measure is listed in the record
  • The primary outcome states a time frameThe primary outcome measure records a time frame
  • Participants are randomly assignedAllocation is recorded as randomised
  • Has a comparison groupThe record lists a placebo or comparator arm, or more than one study arm
  • Uses blinding (masking)A masking level is recorded in the record
  • An ethics committee is not stated in the registry recordThis registry's ingested record has no ethics-committee field
  • Regulatory oversight (such as an IND or IDE) is not stated in the registry recordThis registry's ingested record has no regulatory-authorisation field
  • A data monitoring committee is not stated in the registry recordThis registry's ingested record has no data-monitoring-committee fieldA data monitoring committee is not required for many smaller studies, and its absence here is not unusual.
  • No cost to participants is mentioned in the recordChecked the summary, description and eligibility text; no cost-to-participant phrase found
  • No other studies from this sponsor are in this databaseCounted from the lead sponsor named in the record (Swathy Chandrashekhar, MBBS)
  • No posted results from this sponsor are in this database yetBased on the sponsor’s studies in this database
  • The record lists 1 condition.
  • Lead sponsor type recorded as: other.
  • Intervention regulatory context: investigational, within a phased regulatory pathway.

These are facts from the public registry record, not a judgement about the study. Discuss any trial with your own doctor before taking part.

What these facts mean, and what to ask · ruleset v1

Trial stature

Three independent measures of this study, described from its registry record — not a recommendation about it. A rigorous study by investigators nobody has heard of is better evidence than a weak study led by a famous one. How these are scored.

Methodological rigourDEFINITIVE

A definitive-grade design for a phase 2 study, judged from its ClinicalTrials.gov record.

How this score is built
  • Randomised allocation20/20

    Participants are randomly allocated between arms

  • Blinding20/20

    Quadruple-blind

  • Control arm13/15

    Placebo / sham control arm

  • Primary-outcome specificity10/10

    Named primary outcome with a defined time frame

  • Endpoint type3/10

    Surrogate or intermediate endpoint (conservative default)

  • Multi-centre5/8

    Multi-centre: 6 sites

  • Data monitoring committee7/7

    A data monitoring committee is in place

  • Prospective registration5/5

    Registered before the study start date

  • Protocol / SAP posted0/5

    No protocol or SAP posted to the registry

ScaleSMALL

A small study: 72 participants (target), run at 6 sites.

How this score is built
  • Enrolment17/40

    72 participants (target)

  • Site count9/25

    6 sites

  • Country count0/15

    Single country

  • Planned duration8/10

    Planned over about 39 months

  • Sponsor scale0/10

    Swathy Chandrashekhar, MBBS has led 1 trials in our corpus

Investigator standingUNKNOWN

We have no verifiable track record for the investigator named on this trial. That is common for early-career investigators and for records held outside ClinicalTrials.gov — it is not a negative signal.

How this score is built
  • Investigator standing0/100

    No investigator recorded in the registry for this trial

These describe the registry record only, and today we hold ClinicalTrials.gov data. Absent fields lower a score, and absence often reflects registration practice rather than study quality. A high-stature trial is not necessarily safer or a better choice for you — enrolling in a large definitive trial can mean a higher chance of receiving placebo, while a small early-phase study may be the only route to a new therapy.

תקציר

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.

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.

מצבים רפואיים

  • Amyotrophic Lateral Sclerosis

זכאות

מיןהכול
גילאים18 Years – ללא מקסימום
מתנדבים בריאיםלא

זכאות כפי שנכתבה במרשם

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.

זכאות במשפטים פשוטים

הקריטריונים של רשומה זו טרם פורקו למשפטים נפרדים. טקסט המרשם שלמעלה שלם והוא הגרסה המחייבת.

תכנון המחקר

סוג המחקרהתערבותי
שלבשלב 2
הקצאהאקראי
מודל ההתערבותPARALLEL
מטרה ראשיתTREATMENT
הסתרהQUADRUPLE (4)
גיוסדרושים 72 משתתפים

נותן חסות ושותפים

  • Swathy Chandrashekhar, MBBS נותן חסות
  • ALS Association שותפים

זרועות והתערבויות

  • Ranolazine low doseEXPERIMENTAL

    Participants receive Ranolazine 500mg orally twice daily for 24 weeks.

  • Ranolazine high doseEXPERIMENTAL

    Participants receive Ranolazine 1000mg orally twice daily for 24 weeks.

  • PlaceboPLACEBO_COMPARATOR

    Participants receive Ranolazine placebo orally twice daily for 24 weeks.

התערבויות

  • תרופה Placebo

    Ranolazine placebo twice daily

  • תרופה Ranolazine

    500mg twice daily

  • תרופה Ranolazine

    1000 mg twice daily

מדדי תוצאה

  1. מדד תוצאה ראשוני

    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.

    מסגרת זמן Up to 28 weeks

  2. מדד תוצאה ראשוני

    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.

    מסגרת זמן Up to 28 weeks

  3. מדד תוצאה ראשוני

    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.

    מסגרת זמן Up to 28 weeks

  4. מדד תוצאה ראשוני

    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.

    מסגרת זמן Up to 28 weeks

  5. מדד תוצאה ראשוני

    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

    מסגרת זמן Up to 28 weeks

  6. מדד תוצאה ראשוני

    Safety Lab Cystatin C

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

    מסגרת זמן Up to 28 weeks

  7. מדד תוצאה ראשוני

    Safety Lab Estimated Glomerular Filtration Rate (eGFR)

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

    מסגרת זמן Up to 28 weeks

  8. מדד תוצאה ראשוני

    Safety Lab Alanine Transaminase (ALT)

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

    מסגרת זמן Up to 28 weeks

  9. מדד תוצאה ראשוני

    Safety Lab Aspartate Transferase (AST)

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

    מסגרת זמן Up to 28 weeks

  10. מדד תוצאה ראשוני

    Safety Lab Alkaline Phosphatase (ALP)

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

    מסגרת זמן Up to 28 weeks

  11. מדד תוצאה ראשוני

    Safety Lab Total Bilirubin

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

    מסגרת זמן Up to 28 weeks

  12. מדד תוצאה משני

    Muscle strength

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

    מסגרת זמן Up to 28 weeks

  13. מדד תוצאה משני

    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.

    מסגרת זמן Up to 28 weeks

  14. מדד תוצאה משני

    Forced Vital Capacity (FVC)

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

    מסגרת זמן Up to 28 weeks

  15. מדד תוצאה משני

    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.

    מסגרת זמן Up to 28 weeks

  16. מדד תוצאה משני

    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

    מסגרת זמן Up to 28 weeks

תאריכים

תאריך התחלה29 באפריל 2025 (בפועל)
סיום ראשוני1 ביולי 2028 (משוער)
סיום1 ביולי 2028 (משוער)
פורסם לראשונה30 ביולי 2024 (בפועל)
עודכן לאחרונה8 ביוני 2026
התוצאות פורסמולא צוין ברשומת המרשם
המצב אומת לאחרונהיוני 2026

בפועל פירושו שהאירוע התרחש. משוער פירושו שנותן החסות צופה אותו. שתי המשמעויות שונות זו מזו.

מיקומים

6 אתרים מגייסים

United States

מתקןעירמדינה או אזורמצב
University of Missouri Health CareColumbiaMissouriמגייס
The Ohio State UniversityColumbusOhioמגייס
University of Kansas Medical CenterFairwayKansasמגייס
Mayo Clinic FloridaJacksonvilleFloridaמגייס
University of California, San FranciscoSan FranciscoCaliforniaמגייס
University of Kansas Medical Center: WichitaWichitaKansasמגייס

מסמכי המחקר

לא צורפו מסמכים ברשומת מרשם זו.

שינויים לאורך זמן

לא נרשמו שינויים מאז שקלטנו רשומה זו לראשונה.

שינוי נרשם בכל פעם שנותן החסות מעדכן את רשומת המרשם. מצב, תאריכים, גיוס ואתרים מופיעים כאן ככל שהם משתנים.

מרשם: ClinicalTrials.gov · NCT########

https://xmedica.io/trials/ctgov/NCT06527222