Olutasidenib en pacientes con AML con mutación IDH1 recidivante que han recibido previamente Venetoclax
Estudio piloto de fase 2 de brazo único de olutasidenib en pacientes con LMA con mutación IDH1 recidivante que han recibido previamente venetoclax
This is a prospective, single-arm phase 2 pilot study to assess the response rate of IDH1 mutated relapsed/refractory acute myeloid leukemia (AML) patients who receive olutasidenib after progressing on venetoclax based regimens. Each cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission. Subjects with at least a PR after 6 cycles of treatment will continue treatment as previously described. Subjects without at least a partial response (PR) after 6 cycles of treatment will move to long term follow up.
Descripción general del estudio
Estado
Estado
Condiciones
Condiciones
Intervención / Tratamiento
Intervención / Tratamiento
Tipo de estudio
Tipo de estudio
Inscripción (Estimado)
Inscripción
Fase
Fase
- Fase 2
Contactos y Ubicaciones
Estudio Contacto
Estudio Contacto
- Nombre: Ahran Lee
- Número de teléfono: 14 317-634-5842
- Correo electrónico: alee@hoosiercancer.org
Copia de seguridad de contactos de estudio
- Nombre: Timothy Pardee
- Número de teléfono: MD, PhD
- Correo electrónico: Timothy.Pardee@wfusm.edu
Ubicaciones de estudio
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North Carolina
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Winston-Salem, North Carolina, Estados Unidos, 27157
- Reclutamiento
- Atrium Health Wake Forest Baptist Medical Center
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Investigador principal:
- Timothy Pardee, MD, PhD
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Contacto:
- Elizabeth Parke
- Número de teléfono: 980-442-2000
- Correo electrónico: Elizabeth.Parke@Advocatehealth.org
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Criterios de participación
Criterio de elegibilidad
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Inclusion Criteria:
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Written informed consent and HIPAA authorization for release of personal health information prior to registration. NOTE: HIPAA authorization may be included in the informed consent or obtained separately.
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Age ≥ 18 years at the time of consent.
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ECOG Performance Status of ≤ 2 within 28 days prior to registration.
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Must have histologically or cytologically documented relapsed and/or refractory Acute Myeloid Leukemia (Refractory is defined as failure to achieve a CR after induction chemotherapy or a minimum of two cycles of HMA plus venetoclax)
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Acute Myeloid Leukemia with a documented IDH1 mutation.
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No more than 2 lines of prior therapy. NOTE: One line of therapy must have contained venetoclax.
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Persisting, non-hematologic and non-infectious toxicities from prior treatment must be Grade ≤ 2. NOTE: Documentation of these criteria is required at screening.
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Demonstrate adequate organ function as defined in the table below. All screening labs to be obtained within 14 days prior to registration.
- Renal
- Calculated creatinine clearance (Cockcroft-Gault formula will be used to calculate creatinine clearance) ≥ 30 mL/min
- Hepatic
- Total bilirubin2 ≤ 2× upper limit of normal (ULN); ≤ 3 times ULN in patients with Gilbert Syndrome
- Aspartate aminotransferase (AST) ≤ 5 × ULN
- Alanine aminotransferase (ALT) ≤ 5× ULN
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Participants of childbearing potential must have a negative serum pregnancy test within 7 days prior to initiation of treatment.
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Participants of childbearing potential who are having penile-vaginal intercourse with a person able to father a child must be willing to abstain from penile-vaginal intercourse or must use an effective method(s) of contraception. A participant able to father a child who is having penile-vaginal intercourse with a person of childbearing potential must be willing to abstain from penile-vaginal intercourse or use an effective method(s) of contraception.
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Subjects with known HIV infection may be eligible if they are on effective anti-retroviral therapy with undetectable viral load within 6 months prior to registration. Testing is not required at screening.
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Subjects with known chronic hepatitis B virus (HBV) infection may be eligible if they have an undetectable HBV viral load on suppressive therapy, if indicated. Subjects with a history of hepatitis C virus (HCV) infection may be eligible if they have been treated and cured. Subjects with an HCV infection who are currently on treatment must have an undetectable HCV viral load to be eligible for this trial. Testing is not required at screening.
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As determined by the investigator or protocol designee, ability of the subject to understand and comply with study procedures for the entire length of the study.
Exclusion Criteria:
- Previous exposure to ivosidenib or any IDH1 inhibitor.
- Active infection requiring IV systemic therapy. NOTE: Subjects receiving prophylactic antibiotics (e.g., to prevent a urinary tract infection or chronic obstructive pulmonary disease exacerbation) are eligible for the study.
- Known CNS involvement with AML.
- Previous allogeneic stem cell transplant within 60 days prior to registration.
- Treatment with any investigational drug within 21 days or 2 half-life's prior to registration.
- History of severe allergic anaphylactic reactions to olutasidenib or any of their excipients.
- Pregnant or breastfeeding. NOTE: breast milk cannot be stored for future use while the subject is being treated on study.
- Prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen, per treating physician discretion.
- Concomitant use of known strong (e.g. phenobarbital, enzalutamide, phenytoin, rifampicin, rifabutin, rifapentine, carbamazepine, nevirapine and St John's Wort) or moderate CYP3A inducers (e.g. bosentan, efavirenz, modafinil). The required washout period prior to starting olutasidenib is 2 weeks or 5 half-lives (whichever is longer) prior to initiation of treatment.
- Concomitant use of known sensitive CYP3A substrates (e.g. Midazolam, simvastatin, fluticasone, budesonide). The required washout period prior to starting olutasidenib is 2 weeks or 5 half-lives (whichever is longer) prior to initiation of treatment.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Número de brazos
Armas e Intervenciones
Grupo de participantes/brazoGrupo de participantes/brazo |
Intervención / TratamientoIntervención / Tratamiento |
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Experimental: Olutasidenib and Azacitidine
Each cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission or for whom loss of response is suspected. Subjects with at least a PR after 6 cycles of treatment will continue treatment.. Subjects without at least a PR after 6 cycles of treatment will move to long term follow up. |
Olutasidenib 150 mg orally twice a day Azacitidine 75 mg/m2 subcutaneously or IV (over 10-40 minutes) |
¿Qué mide el estudio?
Medidas de resultado primarias
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Composite complete remission rate
Periodo de tiempo: 2 years
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Composite complete remission (CRc) rate is defined as patients that meet the criteria for CR + CRh + CRi per the modified European LeukemiaNet (mELN) 2022.
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2 years
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Medidas de resultado secundarias
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Overall response rate (ORR)
Periodo de tiempo: 2 years
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ORR is defined as the proportion of patients achieving CR + CRh + CRi + Partial Remission (PR) + morphologic leukemia-free state [MLFS] per ELN 2022. PR is defined as all hematologic criteria of CR; decrease of bone marrow blast percentage to 5% to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50%. MLFS is defined as bone marrow blasts < 5%, absence of circulating blasts, and no hematologic recovery. |
2 years
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Overall survival
Periodo de tiempo: 2 years
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OS is defined as the time from registration to death from any cause or last known follow-up. |
2 years
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Duration of response (DoR)
Periodo de tiempo: 2 years
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DoR is defined as the time between the first achieved response (CR, CRh, CRi, PR, MLFS) and the earliest evidence of relapse (blasts ≥ 5%) per ELN 2022 or death or date of last follow up. |
2 years
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Time to hematologic improvement (hemoglobin)
Periodo de tiempo: 2 years
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Improvement of Hgb is defined as the time it takes for an individual's hemoglobin level to increase by ≥ 1g/dL from Cycle 1 Day 1 without transfusion |
2 years
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Time to hematologic improvement (platelet count)
Periodo de tiempo: 2 years
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Improvement of PLT is defined as the time it takes for an individual's platelet count to increase by ≥ 50 x 10^9/L from Cycle 1 Day 1 without transfusion |
2 years
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Time to hematologic improvement (neutrophil count)
Periodo de tiempo: 2 years
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Improvement of ANC is defined as the time it takes for an individual's neutrophil count to increase to ≥ 1,500 cells/µL for 3 days. |
2 years
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Rate of transfusion independence
Periodo de tiempo: 2 years
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The rate of transfusion independence is defined as the absence of red blood cell (RBC) transfusions for ≥ 8 weeks following the start of treatment |
2 years
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Rate of allogeneic transplant
Periodo de tiempo: 2 years
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The rate of allogeneic transplant is defined as the proportion of patients who undergo allogeneic transplant following response to olutasidenib. |
2 years
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Colaboradores e Investigadores
Patrocinador
Patrocinador
Colaboradores
Colaboradores
Investigadores
Investigadores
- Investigador principal: Timothy Pardee, MD, PhD, Atrium Health Wake Forest Baptist
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
Inicio del estudio
Finalización primaria (Estimado)
Finalización primaria
Finalización del estudio (Estimado)
Finalización del estudio
Fechas de registro del estudio
Enviado por primera vez
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Publicado por primera vez
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización publicada
Última actualización enviada que cumplió con los criterios de control de calidad
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Neoplasias
- Neoplasias por tipo histológico
- Enfermedades hematológicas
- Leucemia Mieloide
- Leucemia
- Enfermedades hemic y linfáticas
- Leucemia Mieloide Aguda
- Químicos orgánicos
- Compuestos heterocíclicos, 1 anillo
- Compuestos heterocíclicos
- Ácidos nucleicos, nucleótidos y nucleósidos
- Citidina
- Nucleósidos de pirimidina
- Pirimidinas
- Compuestos AZA
- Nucleósidos
- Ribonucleósidos
- Azacitidina
- olutasidenib
Otros números de identificación del estudio
Otros números de identificación del estudio
- HCRN-AML24-687
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
producto fabricado y exportado desde los EE. UU.
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