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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.

En recrutementAccepte des participants actuellement, selon la fiche du registre.
Contacter cette étude

En bref

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 participants recherchés6 sites1 pays

Catégories

Neurologie74%Endocrinien et métabolique26%

Enregistré dans 1 registre

Une même étude peut être enregistrée dans plusieurs registres. Nous l'affichons une seule fois et renvoyons vers chaque fiche que nous détenons.

Les informations de l'essai sont affichées telles que publiées par le registre, dans leur langue d'origine.

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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.

Résumé

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.

Affections

  • Amyotrophic Lateral Sclerosis

Éligibilité

SexeTous
Âges18 Years – Aucun maximum
Volontaires sainsNon

Éligibilité telle qu'elle figure dans le registre

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.

Éligibilité en énoncés simples

Les critères de cette fiche n'ont pas encore été décomposés en énoncés distincts. Le texte du registre ci-dessus est complet et fait foi.

Conception de l'étude

Type d'étudeInterventionnelle
PhasePhase 2
RépartitionRandomisée
Modèle d'interventionPARALLEL
Objectif principalTREATMENT
InsuQUADRUPLE (4)
Recrutement72 participants recherchés

Promoteur et collaborateurs

  • Swathy Chandrashekhar, MBBS Promoteur
  • ALS Association Collaborateurs

Groupes et interventions

  • 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.

Interventions

  • Médicament Placebo

    Ranolazine placebo twice daily

  • Médicament Ranolazine

    500mg twice daily

  • Médicament Ranolazine

    1000 mg twice daily

Critères de jugement

  1. Critère de jugement principal

    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.

    Délai Up to 28 weeks

  2. Critère de jugement principal

    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.

    Délai Up to 28 weeks

  3. Critère de jugement principal

    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.

    Délai Up to 28 weeks

  4. Critère de jugement principal

    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.

    Délai Up to 28 weeks

  5. Critère de jugement principal

    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

    Délai Up to 28 weeks

  6. Critère de jugement principal

    Safety Lab Cystatin C

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

    Délai Up to 28 weeks

  7. Critère de jugement principal

    Safety Lab Estimated Glomerular Filtration Rate (eGFR)

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

    Délai Up to 28 weeks

  8. Critère de jugement principal

    Safety Lab Alanine Transaminase (ALT)

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

    Délai Up to 28 weeks

  9. Critère de jugement principal

    Safety Lab Aspartate Transferase (AST)

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

    Délai Up to 28 weeks

  10. Critère de jugement principal

    Safety Lab Alkaline Phosphatase (ALP)

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

    Délai Up to 28 weeks

  11. Critère de jugement principal

    Safety Lab Total Bilirubin

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

    Délai Up to 28 weeks

  12. Critère de jugement secondaire

    Muscle strength

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

    Délai Up to 28 weeks

  13. Critère de jugement secondaire

    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.

    Délai Up to 28 weeks

  14. Critère de jugement secondaire

    Forced Vital Capacity (FVC)

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

    Délai Up to 28 weeks

  15. Critère de jugement secondaire

    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.

    Délai Up to 28 weeks

  16. Critère de jugement secondaire

    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

    Délai Up to 28 weeks

Dates

Date de début29 avril 2025 (réelle)
Achèvement principal1 juillet 2028 (estimée)
Achèvement1 juillet 2028 (estimée)
Première publication30 juillet 2024 (réelle)
Dernière mise à jour8 juin 2026
Résultats publiésNon précisé dans la fiche du registre
Statut vérifié pour la dernière foisjuin 2026

Réelle signifie que l'événement a eu lieu. Estimée signifie que le promoteur s'y attend. Les deux ont un sens différent.

Lieux

6 sites sont en recrutement

United States

ÉtablissementVilleÉtat ou régionStatut
University of Missouri Health CareColumbiaMissouriEn recrutement
The Ohio State UniversityColumbusOhioEn recrutement
University of Kansas Medical CenterFairwayKansasEn recrutement
Mayo Clinic FloridaJacksonvilleFloridaEn recrutement
University of California, San FranciscoSan FranciscoCaliforniaEn recrutement
University of Kansas Medical Center: WichitaWichitaKansasEn recrutement

Documents de l'étude

Aucun document n'est lié dans cette fiche du registre.

Évolution au fil du temps

Aucun changement n'a été enregistré depuis que nous avons intégré cette fiche pour la première fois.

Un changement est enregistré chaque fois que le promoteur met à jour la fiche du registre. Le statut, les dates, le recrutement et les sites apparaissent ici au fur et à mesure de leur évolution.

Registre: ClinicalTrials.gov · NCT########

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