Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
29 participants
INTERVENTIONAL
2024-08-01
2028-06-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Phase I: Since this is a first in human trial the primary goal in phase I is to establish the recommended dose of MB-dNPM1-TCR.1 for phase II. We assess the maximum tolerated dose (MTD) with toxicity defined as patients experiencing dose limiting toxicity (DLT) until day 28 after infusion of MB-dNPM1-TCR.1. Therefore a BOIN trial design will be used to guide dose escalation and de-escalation decisions in phase I.
Phase II: The second phase will evaluate the efficacy and safety in patients treated with the recommended dose from phase I. The phase II part follows a Simon's 'minimax' two stage design.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
MB-dNPM1-TCR.1
Biological: MB-dNPM1-TCR.1
MB-dNPM1-TCR.1
T Cell Receptor (TCR) T cell therapy
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
MB-dNPM1-TCR.1
T Cell Receptor (TCR) T cell therapy
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Patients must be able to understand and be willing to give signed informed consent
3. Relapsed or refractory acute myeloid leukemia (last disease staging within 4 weeks prior to screening) without standard treatment options defined as:
* No morphological CR after at least two courses of intensive chemotherapy, decitabine or other standard therapy or
* MRD positive after at least two courses of intensive chemotherapy and not eligible for allogeneic stem cell transplantation or
* Relapsed bone marrow or blood disease after CR after first line treatment and not eligible to undergo allogeneic stem cell transplantation or
* Bone marrow or blood relapse, non-response or MRD positivity after allogeneic stem cell transplantation and not eligible to receive Donor Lymphocyte Infusion (DLI) according to local standards, relapse after DLI.
4. Positive for HLA-A\*02:01 according to genotyping results.
5. AML has NPM1 mutation which is recognized by dNPM1-TCR.1 and for which a specific Q-PCR is available for disease monitoring.
6. Number of circulating WBC above 1x109/L with less than 20% leukemic blasts and at least 0.3x109 T cells/L and 0.03 x 109 CD8+ T cells/L.
7. Life expectancy of at least 3 months.
8. ECOG performance status 0-3.
9. Negative pregnancy test in women of childbearing potential.
10. For fertile men and women, agreement to use highly effective contraceptive methods during the trial.
Exclusion Criteria
2. Active infection with HIV-1, HIV-2, HBV, HCV, HTLV-I, HTLV-II, SARS-CoV-2 or Treponema Pallidum.
3. 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.
4. Use of systemic immune suppression including, but not limited to:
immunosuppressive agents such as cyclosporine or corticosteroids (at an equivalent dose of 0.5 mg prednisone/kg body weight per day, or higher). Inhaled steroid and physiological replacement for adrenal insufficiency are allowed.
5. Unwillingness or inability to comply with procedures required in this clinical trial protocol.
6. Uncontrolled central nervous system (CNS) disease.
7. Uncontrolled life-threatening infections or uncontrolled disseminated intravascular coagulation; however, if these problems resolve, the start of treatment can be initiated on a delayed schedule.
8. Subjects currently on any other IMP (including within the last 30 days before start of treatment).
9. Current use of high dose immunosuppression for immune disorders interfering with T cell function (on discretion of the investigator).
10. Known hypersensitivity against any drug of the mandatory trial procedures.
11. Serum creatinine ≥ 2.0 × ULN or eGFR \< 30 mL/min calculated according to the modified MDRD formula.
12. BMI ≥40
13. Has received vaccination with live vaccines 6 weeks prior to treatment
14. Major surgery less than 30 days before start of treatment.
15. Committal to an institution on judicial or official order.
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Miltenyi Biomedicine GmbH
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
C.J.M. Halkes, Dr
Role: PRINCIPAL_INVESTIGATOR
Leiden University Medical Center
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Leiden University Medical Center
Leiden, , Netherlands
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
C.J.M. Halkes, Dr
Role: primary
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
M-2020-358
Identifier Type: -
Identifier Source: org_study_id