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Estudio de MP0533 en pacientes con leucemia mieloide aguda o síndrome mielodisplásico en recaída o refractario

22 de septiembre de 2026 actualizado por: Molecular Partners AG

Un estudio de fase 1/2a, primero en humanos, abierto, multicéntrico, de escalada de dosis de MP0533 en pacientes con leucemia mieloide aguda (LMA) o síndrome mielodisplásico (SMD) en recaída/refractaria (R/R)

The purpose of this study is to evaluate the safety, tolerability, and preliminary activity of MP0533 in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Tipo de estudio

Intervencionista

Inscripción (Actual)

80

Fase

  • Fase 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Francia
      • Bordeaux, Francia
        • CHU Bordeaux
      • Paris, Francia, 75010
        • AP-HP Hôpital Saint-Louis
      • Toulouse, Francia
        • IUCT Oncopole
  • Lituania
      • Vilnius, Lituania
        • Vilnius University Hospital Santaros Klinikos
  • Países Bajos
      • Amsterdam, Países Bajos
        • Amsterdam UMC - Locatie VUmc
      • Rotterdam, Países Bajos
        • Erasmus MC
    • Provincie Groningen
      • Groningen, Provincie Groningen, Países Bajos
        • Groningen UMC
  • Suiza
    • Canton of Bern
      • Bern, Canton of Bern, Suiza, 3010
        • Inselspital, Universitaetsspital Bern
    • Canton of Zurich
      • Zurich, Canton of Zurich, Suiza, 8006
        • Universitaetsspital Zuerich

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

18 años y mayores (Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

Inclusion Criteria:

  • Has signed and dated written informed consent prior to performing any study procedure, including screening
  • Diagnosis of relapsed/refractory AML or relapsed/refractory MDS/AML according to the ELN recommendation 2022.
  • Age ≥18 years old on the day of signing informed consent
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 to 2
  • Anticipated life expectancy ≥ 12 weeks by investigator judgement
  • White blood count (WBC) ≤ 15G/L at day of trial drug infusion
  • Adequate renal and hepatic function
  • Is using highly effective contraception, for females of childbearing potential and for men

Exclusion Criteria:

  • Mixed phenotype acute leukemia
  • Patients with favorable AML mutations according to ELN recommendation 2022 and 2024
  • Allogeneic HCT within the last 3 months and/or eligibility for standard 2nd line of targeted therapy, like gilteritinib for FLT3 mutated AML, unless this therapeutic option has already been given and proven ineffective (patient relapsed or resistant to), or contraindicated, or confounding mutations exist, or there is a lack of access to this recommended therapy.
  • More than 2 prior lines of anti-leukemic therapy
  • Active GvHD requiring immune-suppressive therapy
  • Use of immunosuppressive drugs
  • Clinical signs of AML in the central nervous system
  • Major surgery within 28 days prior to start of study medication
  • Other malignancy requiring active therapy, but adjuvant endocrine therapy is allowed
  • Any uncontrolled active infection
  • Treatment with investigational agents or agents targeting CD33, CD123 or CD70 within 4 weeks or five times the half-life of the agent, whichever is longer, prior to start of trial medication
  • Left ventricular ejection fraction of < 50% on echocardiographic exam at screening
  • History or evidence of clinically significant cardiovascular disease
  • Pulmonary disease with clinically relevant hypoxia
  • Active hepatitis
  • Concurrent enrolment in another clinical trial, unless it is an observational (non-interventional) study or it is the follow-up period of an interventional study
  • Known hypersensitivity to any of the excipients of the investigational medicinal product (IMP), i.e. finished MP0533 drug

Dose Expansion Group (Arm B in treatment-naïve patients only):

Inclusion

• Treatment-naïve patients who are eligible to AZA+VEN as standard of care

Dose Escalation and Expansion Groups (Arm B only):

Exclusion

  1. received VEN in prior treatment lines
  2. received strong and/or moderate CYP3A inducers within 7 days before the initiation of AZA/VEN regimen;
  3. Has consumed grapefruit, grapefruit products, Seville oranges or Starfruit within 3 days before the initiation of AZA/VEN regimen;
  4. Has a malabsorption syndrome or other condition that precludes the enteral route of administration of VEN.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Dose escalation (Part 1)

• MP0533 is administered by intravenous infusion
Droga: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 1

MP0533 is administered by intravenous infusion

Otros nombres:
  • Parte 1
Experimental: Dose escalation (Part 2 - Arm A)
  • MP0533 is administered by intravenous infusion
  • Obinutuzumab pretreatment administered
Droga: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A
  • MP0533 is administered by intravenous infusion
  • Obinutuzumab pretreatment administered
Otros nombres:
  • Parte 2 - Arm A
Experimental: Dose escalation (Part 2 - Arm B)
  • MP0533 is administered by intravenous infusion
  • Azacitidine is administered by subcutaneous injection for 7 days per cycle
  • Venetoclax is administered orally for 14 days per cycle
  • Optional Obinutuzumab pretreatment administered
Droga: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) + azacitidine + venetoclax
  • MP0533 is administered by intravenous infusion
  • Azacitidine is administered by subcutaneous injection for 7 days per cycle
  • Venetoclax is administered orally for 14 days per cycle
  • Optional obinutuzumab pretreatment administered
Otros nombres:
  • Parte 2 - Arm B
Experimental: Dose expansion (Arm A)
  • MP0533 is administered by intravenous infusion at densified dosing schedule
  • Obinutuzumab pretreatment administered
Droga: MP0533 with Obinutuzumab pretreatment
  • MP0533 is administered by intravenous infusion at densified dosing schedule
  • Obinutuzumab pretreatment administered
Otros nombres:
  • Brazo A
Experimental: Dose expansion (Arm B relapsed/refractory AML)
  • MP0533 is administered by intravenous infusion
  • Azacitidine is administered by subcutaneous injection for 7 days per cycle
  • Venetoclax is administered orally for 14 days per cycle
  • Optional Obinutuzumab pretreatment administered
Droga: MP0533 + azacitidine + venetoclax with optional Obinutuzumab pretreatment, Arm B in treatment naïve patients
  • MP0533 is administered by intravenous infusion
  • Azacitidine is administered by subcutaneous injection for 7 days per cycle
  • Venetoclax is administered orally for 14 days per cycle
  • Optional obinutuzumab pretreatment administered
Otros nombres:
  • Brazo B en pacientes ingenuos de tratamiento
Experimental: Dose expansion (Arm B in treatment naïve patients)
  • MP0533 is administered by intravenous infusion
  • Azacitidine is administered by subcutaneous injection for 7 days per cycle
  • Venetoclax is administered orally for 14 days per cycle
  • Optional obinutuzumab pretreatment administered
Droga: MP0533 + azacitidine + venetoclax with optional Obinutuzumab pretreatment, Arm B relapsed/refractory AML
  • MP0533 is administered by intravenous infusion
  • Azacitidine is administered by subcutaneous injection for 7 days per cycle
  • Venetoclax is administered orally for 14 days per cycle
  • Optional obinutuzumab pretreatment administered
Otros nombres:
  • Brazo B recaída/AML refractaria

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Phase 1 dose escalation: Recommended Phase 2 Dose Regimen and/or Maximum Tolerated Dose Regimen
Periodo de tiempo: from start of treatment to end of first cycle (day 1 - 28)

Incidence of dose limiting toxicities, assessment of toxicity/safety, pharmacokinetic and efficacy parameters
from start of treatment to end of first cycle (day 1 - 28)
Phase 2 dose extension: Overall Response Rate
Periodo de tiempo: throughout the study (on average 3 months)

Best overall response of complete remission (CR), complete remission with partial hematological recovery (CRh), complete remission with incomplete hematological recovery (CRi), morphologic leukemia-free state (MLFS) and partial remission (PR) according to the European LeukemiaNet (ELN) response criteria 2022
throughout the study (on average 3 months)

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Serum Concentration-time profiles (max. serum)
Periodo de tiempo: throughout the study (on average 1 year)

Determination of PK parameters including (but not limited to) maximum serum concentration (Cmax)
throughout the study (on average 1 year)
Serum Concentration-time profiles (at Cmax (Tmax))
Periodo de tiempo: throughout the study (on average 1 year)

Determination of PK parameters including (but not limited to) time at Cmax (Tmax)
throughout the study (on average 1 year)
Serum Concentration-time profiles (min. serum concentration)
Periodo de tiempo: throughout the study (on average 1 year)

Determination of PK parameters including (but not limited to) minimal serum concentration (Cmin)
throughout the study (on average 1 year)
Area under the concentration-time curve (AUC)
Periodo de tiempo: throughout the study (on average 1 year)

Pharmacokinetic (PK) analysis of MP0533
throughout the study (on average 1 year)
Total Clearance (CL)
Periodo de tiempo: throughout the study (on average 1 year)

PK analysis of MP0533
throughout the study (on average 1 year)
Volume of distribution (Vd)
Periodo de tiempo: throughout the study (on average 1 year)

PK analysis of MP0533
throughout the study (on average 1 year)
Half-life (t1/2)
Periodo de tiempo: throughout the study (on average 1 year)

PK analysis of MP0533
throughout the study (on average 1 year)
Incidence of adverse events (AEs) as a measure of safety
Periodo de tiempo: throughout the study (on average 1 year)

Type, incidence and severity of AEs according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0
throughout the study (on average 1 year)
Event free survival (EFS)
Periodo de tiempo: throughout the study (on average 1 year)

time from the date of first study treatment administration to the date of treatment failure, hematologic relapse from CR/CRh/CRi or death from any cause
throughout the study (on average 1 year)
Duration of response (DoR)
Periodo de tiempo: throughout the study (on average 1 year)

time from the start date of CR, CRh, CRi, MLFS or PR to relapse or death
throughout the study (on average 1 year)
Overall survival (OS)
Periodo de tiempo: throughout the study (up to 3 years)

time from the date of first study treatment administration to the date of death
throughout the study (up to 3 years)
Transfusion-Independence (TI)
Periodo de tiempo: throughout the study (on average 1 year)

portion of subjects who achieved RBC/platelet transfusion independence post baseline
throughout the study (on average 1 year)
Number of patients proceeding to a stem cell transplantation
Periodo de tiempo: throughout the study (on average 1 year)

Number of patients proceeding to a stem cell transplantation
throughout the study (on average 1 year)

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Molecular Partners AG

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

29 de diciembre de 2022

Finalización primaria (Actual)

29 de mayo de 2026

Finalización del estudio (Actual)

14 de septiembre de 2026

Fechas de registro del estudio

Enviado por primera vez

14 de diciembre de 2022

Primero enviado que cumplió con los criterios de control de calidad

4 de enero de 2023

Publicado por primera vez (Actual)

6 de enero de 2023

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

25 de septiembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

22 de septiembre de 2026

Última verificación

1 de septiembre de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • MP0533-CP101
  • 2022-002432-31 (Número EudraCT)
  • 2023-505259-39-00 (Ctis)
  • 2023 (Subvención/contrato del NIH de EE. UU.)

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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