Zum Hauptinhalt springen
xMedica

NCT06527222ClinicalTrials.gov

A Study of Ranolazine in ALS

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

RekrutiertNimmt laut Registereintrag derzeit Teilnehmende auf.
Diese Studie kontaktieren

Kurz gefasst

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.

Phase 272 Teilnehmende gesucht6 Studienzentren1 Land

Kategorien

In 1 Register registriert

Eine Studie kann in mehreren Registern registriert sein. Wir zeigen sie einmal und verlinken jeden Eintrag, den wir haben.

Die Studieninformationen werden so angezeigt, wie sie vom Register veröffentlicht wurden, in ihrer Originalsprache.

Interesse an dieser Studie?

Anmelden oder ein Konto erstellen um Ihr Interesse zu bekunden und diese Studie zu verfolgen.

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.

  • Present: Registered before enrolment beganFirst posted 2024-07-30; recorded start 2025-04-29
  • Present: Has a defined primary outcomeA primary outcome measure is listed in the record
  • Present: The primary outcome states a time frameThe primary outcome measure records a time frame
  • Present: Participants are randomly assignedAllocation is recorded as randomised
  • Present: Has a comparison groupThe record lists a placebo or comparator arm, or more than one study arm
  • Present: Uses blinding (masking)A masking level is recorded in the record
  • Not stated: An ethics committee is not stated in the registry recordThis registry's ingested record has no ethics-committee field
  • Not stated: Regulatory oversight (such as an IND or IDE) is not stated in the registry recordThis registry's ingested record has no regulatory-authorisation field
  • Not stated: 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.
  • Present: No cost to participants is mentioned in the recordChecked the summary, description and eligibility text; no cost-to-participant phrase found
  • Not stated: No other studies from this sponsor are in this databaseCounted from the lead sponsor named in the record (Swathy Chandrashekhar, MBBS)
  • Not stated: 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.

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.

Zusammenfassung

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.

Erkrankungen

  • Amyotrophic Lateral Sclerosis

Eignung

Eignung
GeschlechtAlle
Alter18 YearsKein Höchstalter
Gesunde FreiwilligeNein

Eignung im Wortlaut des Registers

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.

Eignung in einfachen Aussagen

Die Kriterien dieses Eintrags wurden noch nicht in einzelne Aussagen aufgeschlüsselt. Der Registertext oben ist vollständig und ist die maßgebliche Fassung.

Studiendesign

Studiendesign
StudientypInterventionell
PhasePhase 2
ZuteilungRandomisiert
InterventionsmodellPARALLEL
Primäres ZielTREATMENT
VerblindungQUADRUPLE (4)
Teilnahme72 Teilnehmende gesucht

Sponsor und Mitwirkende

  • Swathy Chandrashekhar, MBBS Sponsor
  • ALS Association Mitwirkende

Studienarme und Interventionen

  • 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.

Interventionen

  • Arzneimittel Placebo

    Ranolazine placebo twice daily

  • Arzneimittel Ranolazine

    500mg twice daily

  • Arzneimittel Ranolazine

    1000 mg twice daily

Endpunkte

  1. Primärer Endpunkt

    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.

    Zeitrahmen Up to 28 weeks

  2. Primärer Endpunkt

    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.

    Zeitrahmen Up to 28 weeks

  3. Primärer Endpunkt

    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.

    Zeitrahmen Up to 28 weeks

  4. Primärer Endpunkt

    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.

    Zeitrahmen Up to 28 weeks

  5. Primärer Endpunkt

    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

    Zeitrahmen Up to 28 weeks

  6. Primärer Endpunkt

    Safety Lab Cystatin C

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

    Zeitrahmen Up to 28 weeks

  7. Primärer Endpunkt

    Safety Lab Estimated Glomerular Filtration Rate (eGFR)

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

    Zeitrahmen Up to 28 weeks

  8. Primärer Endpunkt

    Safety Lab Alanine Transaminase (ALT)

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

    Zeitrahmen Up to 28 weeks

  9. Primärer Endpunkt

    Safety Lab Aspartate Transferase (AST)

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

    Zeitrahmen Up to 28 weeks

  10. Primärer Endpunkt

    Safety Lab Alkaline Phosphatase (ALP)

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

    Zeitrahmen Up to 28 weeks

  11. Primärer Endpunkt

    Safety Lab Total Bilirubin

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

    Zeitrahmen Up to 28 weeks

  12. Sekundärer Endpunkt

    Muscle strength

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

    Zeitrahmen Up to 28 weeks

  13. Sekundärer Endpunkt

    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.

    Zeitrahmen Up to 28 weeks

  14. Sekundärer Endpunkt

    Forced Vital Capacity (FVC)

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

    Zeitrahmen Up to 28 weeks

  15. Sekundärer Endpunkt

    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.

    Zeitrahmen Up to 28 weeks

  16. Sekundärer Endpunkt

    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

    Zeitrahmen Up to 28 weeks

Daten

Daten
Startdatum29. April 2025 (tatsächlich)
Primärer Abschluss1. Juli 2028 (geschätzt)
Abschluss1. Juli 2028 (geschätzt)
Erstveröffentlichung30. Juli 2024 (tatsächlich)
Zuletzt aktualisiert8. Juni 2026
Ergebnisse veröffentlichtIm Registereintrag nicht angegeben
Status zuletzt bestätigtJuni 2026

Tatsächlich bedeutet, dass das Ereignis eingetreten ist. Geschätzt bedeutet, dass der Sponsor es erwartet. Die beiden bedeuten Unterschiedliches.

Standorte

6 Studienzentren rekrutieren

United States

United States
EinrichtungStadtBundesland oder RegionStatus
University of Missouri Health CareColumbiaMissouriRekrutiert
The Ohio State UniversityColumbusOhioRekrutiert
University of Kansas Medical CenterFairwayKansasRekrutiert
Mayo Clinic FloridaJacksonvilleFloridaRekrutiert
University of California, San FranciscoSan FranciscoCaliforniaRekrutiert
University of Kansas Medical Center: WichitaWichitaKansasRekrutiert

Studiendokumente

In diesem Registereintrag sind keine Dokumente verlinkt.

Änderungen im Zeitverlauf

Seit wir diesen Eintrag erstmals eingelesen haben, wurden keine Änderungen erfasst.

Eine Änderung wird jedes Mal erfasst, wenn der Sponsor den Registereintrag aktualisiert. Status, Daten, Teilnahme und Studienzentren erscheinen hier, sobald sie sich ändern.