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AzTrials

LeukemiaPhase 1NCT05673057

Study of MP0533 in Patients Acute Myeloid Leukemia or Myelodysplastic Syndrome

A Phase 1 study testing MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 1, MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A and MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) + azacitidine + venetoclax in people with Leukemia and Myeloid, run by Molecular Partners AG at 9 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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) Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Leukemia, Myeloid, Acute
What is being tested?
MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 1, MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A and MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) + azacitidine + venetoclax
Could I receive a placebo?
No, everyone receives an active treatment
How many people
80 participants
Where
9 sitesin 4 countries
Study dates
29 Dec 2022to 14 Sep 2026
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • Adequate renal and hepatic function
  • Is using highly effective contraception, for females of childbearing potential and for men
  • Treatment-naïve patients who are eligible to AZA+VEN as standard of care
  • Dose Escalation and Expansion Groups (Arm B only):

Who cannot join

  • 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
Show all 22Show fewer
  • 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):
  • received VEN in prior treatment lines
  • received strong and/or moderate CYP3A inducers within 7 days before the initiation of AZA/VEN regimen;
  • Has consumed grapefruit, grapefruit products, Seville oranges or Starfruit within 3 days before the initiation of AZA/VEN regimen;
  • Has a malabsorption syndrome or other condition that precludes the enteral route of administration of VEN.

Trial locations and contacts

9 sites in 4 countries.

9 sites in 4 countries

France3 sites

  • CHU Bordeaux

    Bordeaux

  • AP-HP Hôpital Saint-Louis

    Paris, 75010

  • IUCT Oncopole

    Toulouse

Other trials in France

Netherlands3 sites

  • Amsterdam UMC - Locatie VUmc

    Amsterdam

  • Groningen UMC

    Groningen, Provincie Groningen

  • Erasmus MC

    Rotterdam

Other trials in Netherlands

Switzerland2 sites

  • Inselspital, Universitaetsspital Bern

    Bern, Canton of Bern, 3010

  • Universitaetsspital Zuerich

    Zurich, Canton of Zurich, 8006

Other trials in Switzerland

Lithuania1 site

  • Vilnius University Hospital Santaros Klinikos

    Vilnius

Other trials in Lithuania

About this study

Official title: A Phase 1/2a, First-in-human, Open-label, Multicenter, Dose Escalation Study of MP0533 in Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (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)

Being tested
MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 1, MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A, MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) + azacitidine + venetoclax, MP0533 with Obinutuzumab pretreatment, MP0533 + azacitidine + venetoclax with optional Obinutuzumab pretreatment, Arm B relapsed/refractory AML
Keywords
DARPin, CD33, CD123, CD70, T-cell/CD3 engager, multispecific
Medical subject headings
Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 1Drug

    Also called Part 1

    MP0533 is administered by intravenous infusion

  • MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm ADrug

    Also called Part 2 - Arm A

    • MP0533 is administered by intravenous infusion
    • Obinutuzumab pretreatment administered

  • MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) + azacitidine + venetoclaxDrug

    Also called 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

  • MP0533 with Obinutuzumab pretreatmentDrug

    Also called Arm A

    • MP0533 is administered by intravenous infusion at densified dosing schedule
    • Obinutuzumab pretreatment administered

  • MP0533 + azacitidine + venetoclax with optional Obinutuzumab pretreatment, Arm B relapsed/refractory AMLDrug

    Also called 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

  • MP0533 + azacitidine + venetoclax with optional Obinutuzumab pretreatment, Arm B in treatment naïve patientsDrug

    Also called 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
Study groups in NCT05673057
Study groupRoleWhat they receive
Dose escalation (Part 1)Experimental• MP0533 is administered by intravenous infusion
Receives: Drug: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 1
Dose escalation (Part 2 - Arm A)Experimental* MP0533 is administered by intravenous infusion * Obinutuzumab pretreatment administered
Receives: Drug: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A
Dose escalation (Part 2 - Arm B)Experimental* 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
Receives: Drug: MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) + azacitidine + venetoclax
Dose expansion (Arm A)Experimental* MP0533 is administered by intravenous infusion at densified dosing schedule * Obinutuzumab pretreatment administered
Receives: Drug: MP0533 with Obinutuzumab pretreatment
Dose expansion (Arm B relapsed/refractory AML)Experimental* 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
Receives: Drug: MP0533 + azacitidine + venetoclax with optional Obinutuzumab pretreatment, Arm B in treatment naïve patients
Dose expansion (Arm B in treatment naïve patients)Experimental* 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
Receives: Drug: MP0533 + azacitidine + venetoclax with optional Obinutuzumab pretreatment, Arm B relapsed/refractory AML

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 1 dose escalation: Recommended Phase 2 Dose Regimen and/or Maximum Tolerated Dose Regimen

    Incidence of dose limiting toxicities, assessment of toxicity/safety, pharmacokinetic and efficacy parameters

    Measured: from start of treatment to end of first cycle (day 1 - 28)

  2. Phase 2 dose extension: Overall Response Rate

    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

    Measured: throughout the study (on average 3 months)

Secondary outcomes (13)

  1. Serum Concentration-time profiles (max. serum)

    Determination of PK parameters including (but not limited to) maximum serum concentration (Cmax)

    Measured: throughout the study (on average 1 year)

  2. Serum Concentration-time profiles (at Cmax (Tmax))

    Determination of PK parameters including (but not limited to) time at Cmax (Tmax)

    Measured: throughout the study (on average 1 year)

  3. Serum Concentration-time profiles (min. serum concentration)

    Determination of PK parameters including (but not limited to) minimal serum concentration (Cmin)

    Measured: throughout the study (on average 1 year)

  4. Area under the concentration-time curve (AUC)

    Pharmacokinetic (PK) analysis of MP0533

    Measured: throughout the study (on average 1 year)

  5. Total Clearance (CL)

    PK analysis of MP0533

    Measured: throughout the study (on average 1 year)

  6. Volume of distribution (Vd)

    PK analysis of MP0533

    Measured: throughout the study (on average 1 year)

  7. Half-life (t1/2)

    PK analysis of MP0533

    Measured: throughout the study (on average 1 year)

  8. Incidence of adverse events (AEs) as a measure of safety

    Type, incidence and severity of AEs according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0

    Measured: throughout the study (on average 1 year)

  9. Event free survival (EFS)

    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

    Measured: throughout the study (on average 1 year)

  10. Duration of response (DoR)

    time from the start date of CR, CRh, CRi, MLFS or PR to relapse or death

    Measured: throughout the study (on average 1 year)

  11. Overall survival (OS)

    time from the date of first study treatment administration to the date of death

    Measured: throughout the study (up to 3 years)

  12. Transfusion-Independence (TI)

    portion of subjects who achieved RBC/platelet transfusion independence post baseline

    Measured: throughout the study (on average 1 year)

  13. Number of patients proceeding to a stem cell transplantation

    Number of patients proceeding to a stem cell transplantation

    Measured: throughout the study (on average 1 year)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Jan 2023
  2. Study start29 Dec 2022
  3. Primary completion29 May 2026
  4. Study completion14 Sep 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
80 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05673057
Sponsor's protocol ID
MP0533-CP101
Other IDs
2023-505259-39-00, 2022-002432-31, 2023
Status last verified
Sep 2026
First submitted
14 Dec 2022
Last updated
25 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Bianchi M, Reichen C, Croset A, Fischer S, Eggenschwiler A, Grubler Y, Marpakwar R, Looser T, Spitzli P, Herzog C, Villemagne D, Schiegg D, Abduli L, Iss C, Neculcea A, Franchini M, Lekishvili T, Ragusa S, Zitt C, Kaufmann Y, Auge A, Hanggi M, Ali W, Frasconi TM, Wullschleger S, Schlegel I, Matzner M, Luthi U, Schlereth B, Dawson KM, Kirkin V, Ochsenbein AF, Grimm S, Reschke N, Riether C, Steiner D, Leupin N, Goubier A. The CD33xCD123xCD70 Multispecific CD3-Engaging DARPin MP0533 Induces Selective T Cell-Mediated Killing of AML Leukemic Stem Cells. Cancer Immunol Res. 2024 Jul 2;12(7):921-943. doi: 10.1158/2326-6066.CIR-23-0692.

    PubMed 38683145

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 1, MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) monotherapy, Part 2-Arm A and MP0533 (multispecific DARPin CD3 Engager Targeting CD33, CD123 and CD70) + azacitidine + venetoclax in people with Leukemia and Myeloid, run by Molecular Partners AG at 9 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 25 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in France, Netherlands, Switzerland, Lithuania. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1 dose escalation: Recommended Phase 2 Dose Regimen and/or Maximum Tolerated Dose Regimen (time frame: from start of treatment to end of first cycle (day 1 - 28)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05673057, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2026.

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