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Estudo de MP0533 em pacientes com leucemia mieloide aguda recidivante ou refratária ou síndrome mielodisplásica

22 de setembro de 2026 atualizado por: Molecular Partners AG

Fase 1/2a, primeiro em humanos, aberto, multicêntrico, estudo de escalonamento de dose de MP0533 em pacientes com leucemia mieloide aguda (AML) recidivante/refratária (R/R) ou síndrome mielodisplásica (MDS)

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)

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Tipo de estudo

Intervencional

Inscrição (Real)

80

Estágio

  • Fase 1

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

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

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

18 anos e mais velhos (Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

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.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Não randomizado
  • Modelo Intervencional: Atribuição sequencial
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Dose escalation (Part 1)

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

MP0533 is administered by intravenous infusion

Outros nomes:
  • Parte 1
Experimental: Dose escalation (Part 2 - Arm A)
  • MP0533 is administered by intravenous infusion
  • Obinutuzumab pretreatment administered
Medicamento: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A
  • MP0533 is administered by intravenous infusion
  • Obinutuzumab pretreatment administered
Outros nomes:
  • 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
Medicamento: 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
Outros nomes:
  • Parte 2 - braço B
Experimental: Dose expansion (Arm A)
  • MP0533 is administered by intravenous infusion at densified dosing schedule
  • Obinutuzumab pretreatment administered
Medicamento: MP0533 with Obinutuzumab pretreatment
  • MP0533 is administered by intravenous infusion at densified dosing schedule
  • Obinutuzumab pretreatment administered
Outros nomes:
  • Braço 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
Medicamento: 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
Outros nomes:
  • Braço B em pacientes ingênuos de tratamento
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
Medicamento: 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
Outros nomes:
  • ARM B RECIMADO/REFRATÓRIO AML

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Phase 1 dose escalation: Recommended Phase 2 Dose Regimen and/or Maximum Tolerated Dose Regimen
Prazo: 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
Prazo: 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 resultados secundários

Medida de resultado
Descrição da medida
Prazo
Serum Concentration-time profiles (max. serum)
Prazo: 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))
Prazo: 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)
Prazo: 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)
Prazo: throughout the study (on average 1 year)

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

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

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

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Molecular Partners AG

Publicações e links úteis

A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.

Publicações Gerais

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

29 de dezembro de 2022

Conclusão Primária (Real)

29 de maio de 2026

Conclusão do estudo (Real)

14 de setembro de 2026

Datas de inscrição no estudo

Enviado pela primeira vez

14 de dezembro de 2022

Enviado pela primeira vez que atendeu aos critérios de CQ

4 de janeiro de 2023

Primeira postagem (Real)

6 de janeiro de 2023

Atualizações de registro de estudo

Última Atualização Postada (Real)

25 de setembro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

22 de setembro de 2026

Última verificação

1 de setembro de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • MP0533-CP101
  • 2022-002432-31 (Número EudraCT)
  • 2023-505259-39-00 (Ctis)
  • 2023 (Concessão/Contrato do NIH dos EUA)

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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 .

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