Study of an Innovative Therapy Using CAR-T Cells Targeting IL-1RAP in Patients With High-Risk Myelodysplastic Syndromes (MDS

Sponsor
University Hospital, Grenoble
Study ID
NCT07455500
Status
Not Yet Recruiting

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Conditions

  • Myelodysplastic Syndrome

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Bone marrow and blood sampling — OTHER
    Patients diagnosed with, or suspected of having, low-risk or high-risk myelodysplastic syndrome (MDS) will undergo bone marrow aspirate and peripheral blood sample collection at the time of initial diagnostic evaluation. These procedures will be performed exclusively within the context of routine clinical care, without any additional invasive procedures specifically for research purposes. For patients with high-risk MDS, additional bone marrow and peripheral blood samples may be collected in the event of suspected relapse during active treatment or following allogeneic hematopoietic stem cell transplantation, in accordance with standard-of-care clinical assessments

Study Details

The surface protein IL-1RAP, expressed by leukemic blast cells, represents a target of interest for patients with acute myeloid leukemia (AML). Its restricted and specific expression on leukemic cells makes it a promising target for chimeric antigen receptor T-cell (CAR-T cell) immunotherapy. High-risk myelodysplastic syndromes (MDS) correspond to a pre-leukemic condition characterized by an accumulation of bone marrow blasts. Unfortunately, very few effective treatments are currently available, apart from allogeneic hematopoietic stem cell transplantation, which can only be performed in a limited number of patients. It has been demonstrated that high-risk MDS blasts express IL-1RAP. The project will aim to: * Confirm IL-1RAP expression on primary MDS blast cells. * Measure circulating soluble IL-1RAP in plasma samples from MDS patients. * Investigate the interaction with the microenvironment in relation to IL-1RAP cellular expression. * Evaluate the effect of first-line standard treatment for MDS on IL-1RAP surface expression. * Assess the in vitro efficacy of an IL-1RAP-targeted CAR-T cell on MDS leukemic stem cells. * Assess the in vivo efficacy of an IL-1RAP-targeted CAR-T cell in a humanized murine model of MDS. To successfully conduct this project, it is essential to collect blood and bone marrow samples from high-risk MDS patients This project will require the collection of bone marrow and blood samples from patients with MDS, either newly diagnosed or currently undergoing treatment.

Key Dates

First listed
Mar 6, 2026
Start date
May 31, 2026
Status verified
Mar 2026
Primary completion
Jan 31, 2030
Completion
Jan 31, 2031

Study Design

Enrollment
120 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE

Arms

  • Experimental: MDS patients with low and high risk MDS

Primary Outcome Measure

Validation of IL-1RAP CAR-T Cell Efficacy: [ Time Frame: Baseline through study completion, an average of 1 year ]

Central Contacts

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