Experimental immune cell therapy shows promise for tough blood cancers
NCT ID NCT04620681
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested a new treatment for people with myelodysplastic syndrome (MDS) or secondary acute myeloid leukemia (AML) who had not responded to prior therapy. The treatment involved giving specially prepared donor immune cells (with CD8 cells removed to reduce risk) along with standard chemotherapy. The main goal was to find a safe dose and see if the combination could control the disease. Only 19 people took part, so the results are very preliminary.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- CD8-depleted donor immune cells (lymphocytes) plus standard chemotherapy
- What this could lead to
- If successful, this approach could offer a new treatment option for people with MDS or secondary AML who have not responded to standard therapies.
- What could go wrong
- This is an early-phase trial with only 19 participants, so results may not apply broadly. There are risks of infusion reactions, graft-versus-host disease, or lack of benefit.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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19 people
The number who actually took part.
- Started
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Jan 2021
- Finished
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Jul 2024
- Lead sponsor
-
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
-
18 to 79 years
- Sex
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Anyone
- Healthy volunteers
-
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: Myelodysplastic Syndrome (MDS) having failed hypomethylating agent (HMA) therapy cohort: * Age 18-79 years, inclusive * Pathologically confirmed MDS or myelodysplastic/myeloproliferative overlap (MDS/MPN) * IPSS-R score intermediate, high or very high * Must have failed therapy with an HMA (defined as lack of response by International Working Group criteria (1) or intolerance of the drug) Secondary Acute Myeloid Leukemia (sAML): * Pathologically confirmed AML according to World Health Organization (WHO) criteria * Evidence of an antecedent hematologic disorder (AHD) prior to acute leukemia including a known prior diagnosis of MDS, MPN or MDS/MPN or data suggestive of an AHD such as cytopenias, fibrosis, macrocytic anemia, cellular or dysplasia at or prior to the time of diagnosis. If available, MDS-defining karyotypes (-7/del(7q), -5/del(5q), del(13q), del(11q), del(12p), t(12p), del(9q), idic(X)(q13), t(17p) (unbalanced translocations) or i(17q) (ie, loss of 17p), t(11;16)(q23;p13.3), t(3;21)(q26.2;q22.1), t(1;3)(p36.3;q21), t(2;11)(p21;q23), inv(3)(q21q26.2), t(6;9)(p23;q34)) or somatic mutations in multiple genes including p53, TET2, JAK2, CALR, MPL, ASXL1, RUNX1, SRSF2, SF3B1, U2AF1, ZRSR2, ASXL1, EZH2, BCOR, or STAG2 would also confirm eligibility. * Age 60-79 years, inclusive * May be previously untreated For both cohorts: * Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 * Deemed eligible to receive cytotoxic chemotherapy * Creatinine clearance (CrCl)\>50ml/min * Total bilirubin \<2 mg/dL (except for patients with Gilbert's disease), AST and ALT \< 3x ULN * Left Ventricular Ejection Fraction ≥ 50% * Willing and able to participate in study assessments Exclusion Criteria: * Patients who have had systemic chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier. Hydroxyurea during this period may be given as a bridging therapy to maintain disease stability while awaiting treatment. Intrathecal chemotherapy within this time frame is permitted. Intrathecal chemotherapy may be continued during protocol therapy in order to consolidate or maintain a central nervous system (CNS) remission, but not to treat active CNS disease * Acute promyelocytic leukemia, or the presence of t(15;17) * Patients receiving any other investigational agents * Uncontrolled concurrent illness including, but not limited to, ongoing and uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements * Pregnant women are excluded from this study because there is an unknown but potential risk for adverse events in the fetus. Breastfeeding should be discontinued if the mother is treated. These potential risks may also apply to other agents used in this study * Patients who have any debilitating medical or psychiatric illness that would preclude their giving informed consent or their receiving optimal treatment and follow-up * Patients with a poor functional status of ECOG 3-4, or otherwise deemed unfit to tolerate induction chemotherapy. * Patients with blastic transformation of chronic myelogenous leukemia are ineligible * Exposure to a humanized mouse chimeric antibody, as this could sensitize patients to components of the CD8 depletion column that may be present in small amounts in the cell product * Prior allogenic hematopoietic cell transplant
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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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Moffitt Cancer Center
Tampa, Florida, 33612, United States
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Other studies related to the condition(s) this trial covers.
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