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Programma di accesso esteso per SNDX-5613

28 agosto 2025 aggiornato da: Syndax Pharmaceuticals

Programma di accesso ampliato per SNDX-5613 in pazienti con leucemie acute recidivanti/refrattarie con alterazioni genetiche associate a sovraespressione di HOXA

This expanded access program will provide an investigational treatment option in a controlled clinical setting for participants who are not otherwise eligible to participate in other Syndax-sponsored clinical studies and have no approved treatment options.

Panoramica dello studio

Stato

Non più disponibile

Condizioni

Intervento / Trattamento

Tipo di studio

Accesso esteso

Tipo di accesso espanso

  • Trattamento IND/Protocollo

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

  • Stati Uniti
    • Alabama
      • Birmingham, Alabama, Stati Uniti, 35233
        • Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama
    • Arizona
      • Phoenix, Arizona, Stati Uniti, 85016
        • Phoenix Children's Hospital
    • California
      • Duarte, California, Stati Uniti, 91010
        • City of Hope
      • Irvine, California, Stati Uniti, 92618
        • City of Hope at Orange County Lennar Foundation Cancer Center
      • Los Angeles, California, Stati Uniti, 90095
        • UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment
      • Palo Alto, California, Stati Uniti, 94304
        • Lucile Packard Children's Hospital-Stanford
      • San Diego, California, Stati Uniti, 92123
        • Rady Children's Hospital
      • San Francisco, California, Stati Uniti, 94158
        • University of California, San Francisco
    • Colorado
      • Aurora, Colorado, Stati Uniti, 80045
        • Center for Cancer and Blood Disorders, Colorado Children's Hospital
      • Aurora, Colorado, Stati Uniti, 80045
        • HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center
    • Florida
      • Orlando, Florida, Stati Uniti, 32804
        • Advent Health Orlando
    • Georgia
      • Atlanta, Georgia, Stati Uniti, 30322
        • Children's Healthcare of Atlanta
      • Atlanta, Georgia, Stati Uniti, 30322
        • Winship Cancer Institute at Emory University
    • Illinois
      • Chicago, Illinois, Stati Uniti, 60611
        • Lurie Children's Hospital of Chicago
      • Chicago, Illinois, Stati Uniti, 60637
        • University of Chicago Medical Center
    • Iowa
      • Iowa City, Iowa, Stati Uniti, 52242
        • University of Iowa Hospitals and Clinics
    • Louisiana
      • New Orleans, Louisiana, Stati Uniti, 70118
        • Children's Hospital
    • Massachusetts
      • Boston, Massachusetts, Stati Uniti, 02215
        • Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center
    • Missouri
      • Kansas City, Missouri, Stati Uniti, 64108
        • Children's Mercy Hospital-Kansas City
      • St Louis, Missouri, Stati Uniti, 63110
        • Siteman Cancer Center - Washington University
    • New Jersey
      • Hackensack, New Jersey, Stati Uniti, 07601
        • Hackensack University Medical Center
    • New York
      • Long Island City, New York, Stati Uniti, 10065
        • Memorial Sloan Kettering Cancer Center
      • The Bronx, New York, Stati Uniti, 10467
        • Montefiore Medical Center
    • North Carolina
      • Durham, North Carolina, Stati Uniti, 27705
        • Duke University Medical Center
    • Ohio
      • Cincinnati, Ohio, Stati Uniti, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cincinnati, Ohio, Stati Uniti, 45267
        • Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies
      • Columbus, Ohio, Stati Uniti, 43210
        • OSU Medical Center
    • Oregon
      • Portland, Oregon, Stati Uniti, 97239
        • Doernbecher Children's Hospital, Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, Stati Uniti, 19104
        • Children's Hospital of Philadelphia
    • Tennessee
      • Memphis, Tennessee, Stati Uniti, 38105
        • St. Jude Children's Research Hospital
    • Texas
      • Houston, Texas, Stati Uniti, 77030
        • MD Anderson Cancer Center
      • Houston, Texas, Stati Uniti, 77030
        • Texas Children's Hospital
      • Houston, Texas, Stati Uniti, 77030
        • Memorial Hermann-Texas Medical Center
    • Utah
      • Salt Lake City, Utah, Stati Uniti, 84112
        • Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital
    • Washington
      • Seattle, Washington, Stati Uniti, 98105
        • Seattle Children's Research Institute, Seattle Childrens Hospital

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

6 mesi e precedenti (Bambino, Adulto, Adulto più anziano)

Accetta volontari sani

N/A

Sessi ammissibili allo studio

Tutto

Descrizione

Key Inclusion Criteria:

  • Male or female participant aged ≥6 months.

  • Not eligible for participation in an ongoing clinical study and have no approved treatment options.

  • Participant or participant's health care proxy is able and willing to provide written informed consent and able to follow study instructions.

  • Relapsed or refractory (R/R) acute leukemia, as defined by standardized criteria, after standard of care therapy, including but not limited to 1 or 2 cycles of intensive chemotherapy, or venetoclax combinations:

    1. R/R leukemia is defined by the presence of ≥5% blasts in the bone marrow and/or persistence or reappearance of peripheral blasts.
    2. Participants with persistent leukemia after initial therapy or recurrence of leukemia at any time after achieving a response during or after the course of treatment (including allogeneic hematopoietic stem cell transplant [HSCT]) are eligible.
    3. Participants with isolated extra-medullary disease are allowed if extramedullary disease was confirmed with biopsy.
    4. Participants previously treated on a revumenib clinical study who are entering the expanded access program for post-transplant maintenance because they are not eligible to receive revumenib on study or because the study is closed are not required to meet the R/R status.
    5. Participants who underwent HSCT and are eligible to resume treatment with revumenib will be dosed with the last revumenib tolerated dose before transplant.

  • Acute leukemia harboring a lysine (K) methyltransferase 2A gene rearrangement (KMT2Ar), nucleoporin 98 rearrangement, nucleophosmin 1 mutation (or mutated) or any other genetic alteration with overexpression of HOXA genes predicted to potentially respond to menin inhibitors.

    • Note: As revumenib is now approved in the United States, only participants with a KMT2Ar who are not included in the United States prescribing information indication or cannot be accurately dosed (within a 20% margin) with commercial supply and require use of oral solution will be allowed into the study.

  • Adequate liver, renal, and cardiac function.

  • Use of highly effective methods of contraception are required for females and males of childbearing potential from the time of enrollment through 120 days following the last study intervention dose.

For participants currently being treated with revumenib in a Syndax-sponsored clinical study or Syndax investigator-sponsored trial, the following criteria must be met:

  • In the opinion of the Investigator, participant demonstrated acceptable benefit from and tolerability of the study intervention.
  • Participant is considered compliant with study intervention and procedures.
  • Participant does not meet any criteria for study intervention discontinuation.
  • Investigator and participant agree to continue study intervention treatment.
  • Participant continues to experience clinical benefit.

Key Exclusion Criteria:

  • Evidence of uncontrolled infection.
  • Pregnant or nursing women.
  • Cardiac or gastrointestinal disease.
  • Graft-Versus-Host Disease (GVHD): Signs or symptoms of acute or chronic GVHD >Grade 1 within 4 weeks of enrollment. All transplant participants must have been off all systemic immunosuppressive therapy and calcineurin inhibitors for at least 1 week before enrollment, with the exception of steroids.
  • History of or any concurrent condition, therapy, laboratory abnormality, or allergy to excipients that, in the Investigator's opinion, either may interfere with the participant's participation or results in the conclusion that it is not in the best interest of the participant to participate.
  • Participants receiving other antileukemic therapy within 14 days of start of study drug and who have not recovered from previous adverse reactions.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Syndax Pharmaceuticals

Investigatori

  • Direttore dello studio: Medical Director, Syndax Pharmaceuticals

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Date di iscrizione allo studio

Primo inviato

15 giugno 2023

Primo inviato che soddisfa i criteri di controllo qualità

15 giugno 2023

Primo Inserito (Effettivo)

26 giugno 2023

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

5 settembre 2025

Ultimo aggiornamento inviato che soddisfa i criteri QC

28 agosto 2025

Ultimo verificato

1 agosto 2025

Maggiori informazioni

Termini relativi a questo studio

Parole chiave

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • SNDX-5613-0707

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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