New cell therapy aims to stop transplant complications in blood cancer patients
NCT ID NCT04328714
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested whether a special type of cell (interferon gamma-primed mesenchymal stromal cells) could be safely given to prevent graft-versus-host disease, a common and serious complication after bone marrow transplant. Only 4 patients with acute leukemia or myelodysplastic syndrome were enrolled before the study was stopped. The main goals were to see if the cells could be prepared and delivered safely, and to find the best dose.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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4 people
The number who actually took part.
- Started
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Dec 2021
- Finished
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Jul 2022
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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1 year and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * All transplant patients who undergo HCT with a myeloablative (MA) or Fludarabine/Melphalan (RIC) conditioning regimen and a HLA A- B- C- DR-matched unrelated donor as treatment for hematologic malignancy or MDS. * Age ≥ 1 year at the time that the informed consent document is signed. * Patients with acute leukemia must be in complete remission (defined as an M1 marrow -\<5% blasts- no evidence of extramedullary disease. Complete remissions without platelet recovery (CRp) will be considered remissions. * Planned GVHD prophylaxis with a calcineurin inhibitor and methotrexate per institutional standards. * Subject or parent/guardian must sign an informed consent document, and if appropriate, children must sign an assent document. Exclusion Criteria: * Patients who are to receive a non-myeloablative conditioning regimen. * Patients receiving another investigational drug for acute GVHD prevention during the conditioning regimen or a planned investigational drug for the first year after transplant (there are no restrictions on GVHD treatment). * Any medical or psychological condition or situation deemed by the Investigators to put the patient at increased risk of complications or non-compliance. * Patient with a secondary malignancy who would be otherwise eligible for study, but for whom remission from the primary disease cannot be conclusively confirmed or for whom the chance of relapse of the primary disease is significant. * Pregnancy (positive serum b-HCG) or breastfeeding. * Estimated glomerular filtration rate (GFR) of \< 50 mL/min/1.73m2. * Cardiac ejection fraction \< 50 (using M-Mode if assessment is done by Echocardiogram) * T bilirubin \> 2 × upper limit of normal or alanine aminotransferase (ALT) \> 4 × upper limit of normal or aspartate aminotransferase (AST) \> 4 x upper limit of normal unresolved veno-occlusive disease * Pulmonary disease with forced vital capacity (FVC), forced expiratory volume (FEV1) or diffusing capacity for carbon monoxide (DLCO) parameters \<45% predicted (corrected for hemoglobin) or requiring supplemental oxygen. Children who are developmentally unable to perform pulmonary function testing will be assessed solely on their need for supplemental oxygen. * Karnofsky performance score or Lansky Play-Performance Scale score \<80 * Human leukocyte antigen (HLA) antibody screen positive for HLA antibodies specific against the MSC products.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's Healthcare of Atlanta at Egleston
Atlanta, Georgia, 30322, United States
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Emory University
Atlanta, Georgia, 30322, United States
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Winship Cancer Institute of Emory University
Atlanta, Georgia, 30322, United States
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Other studies related to the condition(s) this trial covers.
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