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Studie van MP0533 bij patiënten met recidiverende of refractaire acute myeloïde leukemie of myelodysplastisch syndroom

22 september 2026 bijgewerkt door: Molecular Partners AG

Een fase 1/2a, first-in-human, open-label, multicenter, dosisescalatieonderzoek van MP0533 bij patiënten met recidiverende/refractaire (R/R) acute myeloïde leukemie (AML) of myelodysplastisch syndroom (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)

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

80

Fase

  • Fase 1

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

18 jaar en ouder (Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Sequentiële toewijzing
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Dose escalation (Part 1)

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

MP0533 is administered by intravenous infusion

Andere namen:
  • Deel 1
Experimenteel: Dose escalation (Part 2 - Arm A)
  • MP0533 is administered by intravenous infusion
  • Obinutuzumab pretreatment administered
Geneesmiddel: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A
  • MP0533 is administered by intravenous infusion
  • Obinutuzumab pretreatment administered
Andere namen:
  • Deel 2 - ARM A
Experimenteel: 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
Geneesmiddel: 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
Andere namen:
  • Deel 2 - ARM B
Experimenteel: Dose expansion (Arm A)
  • MP0533 is administered by intravenous infusion at densified dosing schedule
  • Obinutuzumab pretreatment administered
Geneesmiddel: MP0533 with Obinutuzumab pretreatment
  • MP0533 is administered by intravenous infusion at densified dosing schedule
  • Obinutuzumab pretreatment administered
Andere namen:
  • Arm A
Experimenteel: 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
Geneesmiddel: 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
Andere namen:
  • ARM B In behandeling naïeve patiënten
Experimenteel: 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
Geneesmiddel: 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
Andere namen:
  • ARM B Rested/Refracty AML

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Phase 1 dose escalation: Recommended Phase 2 Dose Regimen and/or Maximum Tolerated Dose Regimen
Tijdsspanne: 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
Tijdsspanne: 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)

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Serum Concentration-time profiles (max. serum)
Tijdsspanne: 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))
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: throughout the study (on average 1 year)

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

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

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

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

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Molecular Partners AG

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

29 december 2022

Primaire voltooiing (Werkelijk)

29 mei 2026

Studie voltooiing (Werkelijk)

14 september 2026

Studieregistratiedata

Eerst ingediend

14 december 2022

Eerst ingediend dat voldeed aan de QC-criteria

4 januari 2023

Eerst geplaatst (Werkelijk)

6 januari 2023

Updates van studierecords

Laatste update geplaatst (Werkelijk)

25 september 2026

Laatste update ingediend die voldeed aan QC-criteria

22 september 2026

Laatst geverifieerd

1 september 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • MP0533-CP101
  • 2022-002432-31 (EudraCT-nummer)
  • 2023-505259-39-00 (Ctis)
  • 2023 (Subsidie/contract van de Amerikaanse NIH)

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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