NCT07455500Not yet recruiting
Study of an Innovative Therapy Using CAR-T Cells Targeting IL-1RAP in Patients With High-Risk Myelodysplastic Syndromes (MDS
Role of IL-1RAP in the Pathophysiology of Myelodysplastic Syndromes and Its Use as a Target for Immunotherapy
Source: ClinicalTrials.gov · Last updated 6 Mar 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Study type
- Interventional
- Enrollment
- 120 (estimated)
- First submitted
- 21 Nov 2025
- Start date
- May 2026
- Primary completion date
- Jan 2030
- Lead sponsor
- University Hospital, Grenoble
- Diseases
- Myelodysplastic syndromes
- NCT number
- NCT07455500
What you commit to
- Study type: Interventional
- Planned participants: 120
- From May 2026 to the primary completion date, Jan 2030
- No sites listed in no country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Patient with an active solid tumor or another active hematologic malignancy requiring treatment.
Other
- Patient not diagnosed with MDS or patient diagnosed with acute myeloid leukemia.
- Patient with a contraindication to performing bone marrow aspiration.
- Individuals referred to in Articles L1121-6 to L1121-8 of the French Public Health Code
Who the study is looking for
- Patient with Myelodysplastic Syndrome (MDS)
- Low-risk MDS according to the IPSS-M (20).
- MDS confirmed by bone marrow cytology with a blast percentage between 5% and 19% and/or associated with cytogenetic abnormalities or gene mutations indicating poor prognosis.
- High-risk MDS according to the IPSS-M
- At diagnosis or in cases of relapse/refractoriness to hypomethylating agents or to allogeneic hematopoietic stem cell transplantation.
Sex: All · Does not accept healthy volunteers
This page is information, not medical advice. Discuss any trial with your doctor.