Programme d'accès étendu pour SNDX-5613
28 août 2025 mis à jour par: Syndax Pharmaceuticals
Programme d'accès élargi pour SNDX-5613 chez les patients atteints de leucémies aiguës récidivantes/réfractaires avec altérations génétiques associées à la surexpression de 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.
Aperçu de l'étude
Statut
Plus disponible
Les conditions
Intervention / Traitement
Type d'étude
Accès étendu
Type d'accès étendu
- Traitement IND/Protocole
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Lieux d'étude
-
-
Alabama
-
Birmingham, Alabama, États-Unis, 35233
- Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama
-
-
Arizona
-
Phoenix, Arizona, États-Unis, 85016
- Phoenix Children's Hospital
-
-
California
-
Duarte, California, États-Unis, 91010
- City of Hope
-
Irvine, California, États-Unis, 92618
- City of Hope at Orange County Lennar Foundation Cancer Center
-
Los Angeles, California, États-Unis, 90095
- UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment
-
Palo Alto, California, États-Unis, 94304
- Lucile Packard Children's Hospital-Stanford
-
San Diego, California, États-Unis, 92123
- Rady Children's Hospital
-
San Francisco, California, États-Unis, 94158
- University of California, San Francisco
-
-
Colorado
-
Aurora, Colorado, États-Unis, 80045
- Center for Cancer and Blood Disorders, Colorado Children's Hospital
-
Aurora, Colorado, États-Unis, 80045
- HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center
-
-
Florida
-
Orlando, Florida, États-Unis, 32804
- Advent Health Orlando
-
-
Georgia
-
Atlanta, Georgia, États-Unis, 30322
- Children's Healthcare of Atlanta
-
Atlanta, Georgia, États-Unis, 30322
- Winship Cancer Institute at Emory University
-
-
Illinois
-
Chicago, Illinois, États-Unis, 60611
- Lurie Children's Hospital of Chicago
-
Chicago, Illinois, États-Unis, 60637
- University of Chicago Medical Center
-
-
Iowa
-
Iowa City, Iowa, États-Unis, 52242
- University of Iowa Hospitals and Clinics
-
-
Louisiana
-
New Orleans, Louisiana, États-Unis, 70118
- Children's Hospital
-
-
Massachusetts
-
Boston, Massachusetts, États-Unis, 02215
- Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center
-
-
Missouri
-
Kansas City, Missouri, États-Unis, 64108
- Children's Mercy Hospital-Kansas City
-
St Louis, Missouri, États-Unis, 63110
- Siteman Cancer Center - Washington University
-
-
New Jersey
-
Hackensack, New Jersey, États-Unis, 07601
- Hackensack University Medical Center
-
-
New York
-
Long Island City, New York, États-Unis, 10065
- Memorial Sloan Kettering Cancer Center
-
The Bronx, New York, États-Unis, 10467
- Montefiore Medical Center
-
-
North Carolina
-
Durham, North Carolina, États-Unis, 27705
- Duke University Medical Center
-
-
Ohio
-
Cincinnati, Ohio, États-Unis, 45229
- Cincinnati Children's Hospital Medical Center
-
Cincinnati, Ohio, États-Unis, 45267
- Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies
-
Columbus, Ohio, États-Unis, 43210
- OSU Medical Center
-
-
Oregon
-
Portland, Oregon, États-Unis, 97239
- Doernbecher Children's Hospital, Oregon Health & Science University
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, États-Unis, 19104
- Children's Hospital of Philadelphia
-
-
Tennessee
-
Memphis, Tennessee, États-Unis, 38105
- St. Jude Children's Research Hospital
-
-
Texas
-
Houston, Texas, États-Unis, 77030
- MD Anderson Cancer Center
-
Houston, Texas, États-Unis, 77030
- Texas Children's Hospital
-
Houston, Texas, États-Unis, 77030
- Memorial Hermann-Texas Medical Center
-
-
Utah
-
Salt Lake City, Utah, États-Unis, 84112
- Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital
-
-
Washington
-
Seattle, Washington, États-Unis, 98105
- Seattle Children's Research Institute, Seattle Childrens Hospital
-
-
Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
6 mois et plus (Enfant, Adulte, Adulte plus âgé)
Accepte les volontaires sains
N/A
Sexes éligibles pour l'étude
Tout
La description
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:
- R/R leukemia is defined by the presence of ≥5% blasts in the bone marrow and/or persistence or reappearance of peripheral blasts.
- 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.
- Participants with isolated extra-medullary disease are allowed if extramedullary disease was confirmed with biopsy.
- 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.
- 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.
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Les enquêteurs
- Directeur d'études: Medical Director, Syndax Pharmaceuticals
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates d'inscription aux études
Première soumission
15 juin 2023
Première soumission répondant aux critères de contrôle qualité
15 juin 2023
Première publication (Réel)
26 juin 2023
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
5 septembre 2025
Dernière mise à jour soumise répondant aux critères de contrôle qualité
28 août 2025
Dernière vérification
1 août 2025
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- SNDX-5613-0707
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .