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MB-dNPM1-TCR.1 in Relapsed/Refractory AML

ConditionLeukemia, Myeloid, Acute
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorMiltenyi Biomedicine GmbH

About this trial

The goal of this Phase I/II, single arm, prospective, open label, dose escalation trial is to assess safety, feasibility and efficacy of ex vivo expanded autologous T cells genetically modified to express a T cell receptor (TCR) specific for dNPM1 peptides restricted to human leukocyte antigen (HLA) A\*02:01 in patients with relapsed or refractory AML.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age ≥ 18 years

Patients must be able to understand and be willing to give signed informed consent

No morphological CR or extramedullary AML after at least two courses of intensive chemotherapy, decitabine or other standard therapy or

MRD positive after at least two courses of intensive chemotherapy or other standard therapy and not eligible for allogeneic stem cell transplantation or

Disqualifiers

Pregnant or breast feeding women.

Active infection with HIV-1, HIV-2, HBV, HCV, HTLV-I, HTLV-II, SARS-CoV-2 or Treponema Pallidum.

Any clinically significant, advanced or unstable disease or inadequate main organ function that may put the patient at increased risk for severe complications of trial participation at the discretion of the investigator.

Unwillingness or inability to comply with procedures required in this clinical trial protocol.

Trial design

Design model

Single group

Treatments tested in this trial

  • MB-dNPM1-TCR.1

    Biological/Vaccine

    T Cell Receptor (TCR) T cell therapy

Treatment groups

29 Participants
are divided into 1 treatment group
Group A: MB-dNPM1-TCR.1Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Primary endpoint Phase I

Maximum tolerated dose (MTD), as identified by a Bayesian Optimal Interval (BOIN) design at a target toxicity rate of 30%, with toxicity defined as patients experiencing dose limiting toxicity (DLT) until day 28 (week 4) after infusion of MB-dNPM1-TCR.1.

Time frame
day 28
2

Primary endpoint Phase II

BOR rate to the treatment with MB-dNPM1-TCR.1 assessed at any time within the first 3 months (12 weeks) after infusion as described above.

Time frame
week 12

Secondary outcomes

1

Persistence

Percentage and total number of MB-dNPM1-TCR+ cells in peripheral blood and/or bone marrow over time.

Time frame
from day 6 to week 96
2

Best objective response (BOR)

Best objective response (BOR) during 12 weeks after infusion of MB-dNPM1-TCR.1. BOR is defined in relation to disease activity before thawing the leukapheresis for manufacturing (day -18 and -15).

Time frame
week 12
3

Overall survival (OS)

Overall survival (OS) defined as the time between the date of infusion of MB-dNPM1-TCR.1 and the date of death from any cause.

Time frame
up to week 96
4

Progression-free survival (PFS)

Progression-free survival (PFS) defined as the time between the date of infusion of MB-dNPM1-TCR.1 and the date of objective disease progression or death from any cause whichever occurs first.

Time frame
up to week 96

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Miltenyi Biomedicine GmbH

Lead sponsor

Source ClinicalTrials.gov
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