NCT06991361 · Leslie Kean
Ex Vivo-Expanded Regulatory T Cells Plus Low-Dose Interleukin-2 for Steroid-Refractory Chronic GVHD
(EVENT)
What this study is about
Hematopoietic Stem Cell Transplant (HCT) has been used to treat children and adults who have leukemia, lymphoma and other cancers of blood and immune cells since the 1970s. For many of these forms of cancer, HCT works well. However, HCT can cause serious, sometimes life-threatening complications. One of the most serious and common complications of HCT is graft-versus-host disease (GVHD).
View original scientific description
Hematopoietic Stem Cell Transplant (HCT) has been used to treat children and adults who have leukemia, lymphoma and other cancers of blood and immune cells since the 1970s. For many of these forms of cancer, HCT works well. However, HCT can cause serious, sometimes life-threatening complications. One of the most serious and common complications of HCT is graft-versus-host disease (GVHD). GVHD can appear early after the transplant, usually about 3 to 4 weeks after the transplant is given. This is called acute GVHD (aGVHD), because it usually happens over a couple of days. If successfully treated, acute GVHD quickly goes away. Sometimes GVHD happens months after the transplant. Then it is called chronic GVHD (cGVHD), because it happens gradually and goes away slowly. The investigators are doing this study to see if one dose of Ex vivo-Expanded Regulatory T cells (EVE-Treg) can be used together with the daily Interleukin 2 (IL-2) to treat cGVHD, that has not responded to steroid treatment or low-dose IL-2.
Interventions
BIOLOGICAL
EVE-Treg
Ex Vivo-Expanded (EVE) Regulatory T cells.
Primary outcome measures
Safety of EVE-Treg
Time frame: 5 weeks
Safety of EVE-Treg, in combination with physician-prescribed daily low-dose IL-2, for the treatment of chronic GVHD refractory to steroids and low-dose IL-2. Safety will be based on adverse events at each Dose Level of EVE-Treg
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Recipient of 7-8/8 HLA-matched allogeneic hematopoietic stem cell transplantation or recipient of haploidentical allogenic hematopoietic stem cell transplantation.
- Age 2 and up.
- Must have steroid-refractory chronic GVHD that is still active despite at least 4 weeks of treatment with low-dose subcutaneous (SC) IL-2.
- Stable dose of glucocorticoids for 2 weeks prior to enrollment
- no addition or subtraction of other immunosuppressive medications for 4 weeks prior to enrollment.
- Must have adequate organ and marrow function.
- Ability to understand and willingness to sign a written informed consent form.
- Donor who is willing and cleared to donate starting material for manufacture of EVE-Treg.
Exclusion criteria
- Recipient of umbilical cord blood stem cell graft.
- Ongoing prednisone requirement greater than 1 mg/kg/day (or equivalent).
- Karnofsky/Lansky performance score less than 40%.
- Concurrent use of methotrexate, azathioprine, or a calcineurin-inhibitor plus sirolimus.
- Other investigational agents within 4 weeks prior to enrollment.
- Participants with post-transplant exposure to donor lymphocyte infusion, or T-cell or IL-2 targeted medications within 100 days prior to enrollment.
- Participants with new immunosupprssive medication, extra-corporeal photopheresis or rituximab therapy initiated int he 4 weeks prior to enrollment.
- Participants with active malignant relapse or recrudescence of their prior hematologic disorder.
- Uncontrolled intercurrent illness unrelated to cGVHD.
- Participants with psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant patients are excluded.
Where
- Boston, Massachusetts
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Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 23, 2026 · Source of record for eligibility and locations