Menin-Inhibitor Targeted Maintenance in AML
Phase 2 Randomized Controlled Study of Revumenib as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation in Patients With KMT2Ar, NPM1m, or NUP98r Acute Myeloid Leukemia (AML)
Visão geral do estudo
Status
Status
Condições
Condições
Intervenção / Tratamento
Intervenção / Tratamento
Descrição detalhada
Tipo de estudo
Tipo de estudo
Inscrição (Estimado)
Inscrição
Estágio
Estágio
- Fase 2
Contactos e Locais
Contato de estudo
Contato de estudo
- Nome: Paul Guo
- Número de telefone: 4583 7634064583
- E-mail: pguo@nmdp.org
Critérios de participação
Critérios de elegibilidade
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
Inclusion Criteria:
Inclusion Criteria:
- Aged ≥18 years at the time of signing informed consent
- Able to provide written informed consent personally or via a legally authorized representative in accordance with applicable regulatory and institutional requirements
- Willing and able to comply with all study procedures and available for the duration of the study
- Diagnosis of acute myeloid leukemia (AML) in complete morphologic remission with one of the following molecular abnormalities:
1. KMT2A-rearranged (KMT2Ar) AML (Excluding KMT2A partial tandem duplication (KMT2A-PTD) 2. NPM1-mutated (NPM1m) AML (Including FLT3-ITD or TKD co-mutation) 3. NUP98-rearranged (NUP98r) AML 5. Planned first allogeneic hematopoietic cell transplantation (allo-HCT) for AML.
6. Transplant Characteristics
- Planned allo-HCT using bone marrow or peripheral blood stem cell graft source.
- Planned reduced-intensity/non-myeloablative conditioning (RIC/NMA) or myeloablative conditioning (MAC), using a conditioning regimen permitted- by the protocol and consistent with standard clinical practice, meeting CIBMTR criteria for conditioning intensity
7. Planned donor:
- HLA-matched related donor (5/6 or 6/6)
- Matched unrelated donor (8/8)
- Mismatched unrelated donor (7/8)
- Haploidentical donor meeting institutional requirements
8. Performance Status:
1. Karnofsky Performance Status ≥70%. 9. Cardiac Function: left ventricular ejection fraction (LVEF) by transthoracic echocardiogram (TTE) or multigated acquisition (MUGA) with no clinical evidence of heart failure: RIC/NMA: ≥50% MAC: ≥5 10. Pulmonary function meeting the following criteria, without supplemental oxygen other than CPAP:
- RIC/NMA: DLCO (corrected for hemoglobin) and FEV1 ≥40% predicted
- MAC: DLCO and FEV1 ≥50% predicted
11. Renal Function: estimated creatinine clearance (CrCl) ≥45mL/min calculated using the Cockcroft-Gault formula or 24-hour urine collection, consistent with standard eligibility criteria for allogeneic HCT recipients.
12. Liver function acceptable per local institutional guidelines for allo-HCT eligibility.
13. Reproductive Status: Willingness to use contraception in accordance with local regulations from first study intervention through the required contraceptive period Willingness to use contraception in accordance with local regulations from first study intervention through the required contraceptive period
Exclusion Criteria:
Disease Status:
a. Evidence of active AML prior to HCT, assessed within 42 days before transplant, defined as any of the following:
- ≥5% bone marrow blasts
- Circulating blasts within 14 days before conditioning
- CNS or other extramedullary disease
- Other active malignancy that, in the investigator's judgment, could interfere with safety or efficacy assessment
- Treatment with non-protocol antileukemic therapy (donor lymphocyte infusion for relapse prophylaxis or treatment will be considered an EFS event)
Cardiac / QT Risk
- Requirement for concomitant medications known to prolong QT/QTc interval, except low-risk agents used as standard supportive care
- Diagnosis or suspicion of Long QT syndrome, or a family history of Long QT syndrome
- Fridericia's corrected QT interval (QTcF) >450 msec.
- History within 6 months of study entry of:
i. Myocardial infarction ii. Unstable angina iii. Congestive heart failure (NYHA Class ≥ II) iv. Life-threatening or uncontrolled arrhythmia v. Cerebrovascular accident or transient ischemic attack
- Chronic respiratory disease requiring continuous supplemental oxygen, or other significant organ dysfunction that would adversely affect study participation.
Active, uncontrolled infection, including any of the following:
- Active, uncontrolled systemic fungal, bacterial, or viral infection within 14 days prior to the start of conditioning
- Any other documented active, uncontrolled infection at the start of conditioning
Chronic viral infections with evidence of active disease, including:
HIV: detectable viral load within 6 months prior to screening
Hepatitis B:
- HBsAg-positive and/or anti-HBc-positive with detectable HBV DNA
- Anti-HBc-positive alone Hepatitis C: positive HCV antibody with detectable HCV RNA
- Planned HCT using cord blood, ex vivo T cell depletion, engineered grafts, or experimental graft sources
Malabsorption syndrome or GI condition that precludes oral administration, including:
- Inability to swallow oral medications
- Prior gastric bypass or severe gastroparesis
- Cirrhosis with Child-Pugh Class B or C
- Pregnant or breastfeeding
- Prior intolerance to menin inhibitor therapy resulting in ≥ Grade 3 treatment-related adverse events
- Any condition, therapy, laboratory abnormality, or allergy to excipients that, in the investigator's judgment, could confound study results, interfere with the participant's ability to comply with study procedures or complete the study, or make participation not in the participant's best interest.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Cuidados de suporte
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Triplo
Número de braços
Armas e Intervenções
Grupo de Participantes / BraçoGrupo de Participantes / Braço |
Intervenção / TratamentoIntervenção / Tratamento |
|---|---|
|
Experimental: Revumenib BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
|
oral tablets
Outros nomes:
|
|
Comparador de Placebo: Placebo BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
|
comprimidos orais
|
O que o estudo está medindo?
Medidas de resultados primários
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Relapse free survival (RFS) in KMT2Ar, NPM1m, and NUP98r AML in the Intent-to-Treat (ITT) population with a minimum of 1 year of follow-up post-randomization.
Prazo: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
RFS is defined as the time from randomization to the date of relapse or the date of death from any cause, whichever comes first.
|
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
Medidas de resultados secundários
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
RFS in the modified ITT (mITT) population
Prazo: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
|
Relapse free survival rate in modified intent to treat population
|
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
|
|
Rate of overall survival (OS) in the ITT population
Prazo: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
Overall survival rate in the intent-to-treat population
|
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
|
Incidence of relapse in the ITT population
Prazo: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
Incidence of relapses in intent-to-treat participant population
|
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
|
Rate of event-free survival (EFS) in the ITT population
Prazo: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
|
Events such as relapse/progression, death from any cause, graft failure, use of donor lymphocyte infusion which have occurred from time from the date of randomization to the date of event occurrence.
|
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
|
|
Rate of non-relapse mortality (NRM) in the ITT population
Prazo: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
Death in participants in the absence of disease progression or relapse
|
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
|
|
Frequency, duration, and severity of Treatment-Emergent Adverse Events (TEAEs), Treatment-Related Adverse Events (TRAEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs) in the Safety Analysis population
Prazo: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization
|
Documentation of number of treatment related of adverse events; their frequencies, duration, and severity
|
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization
|
|
Change from baseline in other observations related to safety for electrocardiograms (ECGs) measuring QT intervals.
Prazo: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
|
Documentation and comparison of abnormal ECGs from baseline measurements in relationship to safety
|
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
|
|
Change from baseline in other observations related to safety for vital signs.
Prazo: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
|
Documentation and comparison of abnormal vital signs from baseline measurements in relationship to safety
|
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
|
|
Change from baseline in other observations related to safety for performance status in the Safety Analysis population
Prazo: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
|
Documentation and comparison of abnormal performance from baseline measurements in relationship to safety
|
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
|
Colaboradores e Investigadores
Patrocinador
Patrocinador
Colaboradores
Colaboradores
Investigadores
Investigadores
- Cadeira de estudo: Steven Devine, M.D, NMDP
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Início do estudo
Conclusão Primária (Estimado)
Conclusão Primária
Conclusão do estudo (Estimado)
Conclusão do estudo
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Primeira postagem
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última Atualização Postada
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
Outros números de identificação do estudo
- MENTAIN
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
produto fabricado e exportado dos EUA
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .