Immunotherapy combo shows promise for Post-Transplant leukemia relapse
NCT ID NCT03600155
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 1 trial tested two immunotherapy drugs, nivolumab and ipilimumab, in 29 patients whose acute myeloid leukemia or myelodysplastic syndrome came back or did not respond after a donor stem cell transplant. The goal was to find the safest dose and see if the drugs could help the immune system fight the cancer. The study was completed, but results are not yet reported.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- nivolumab and ipilimumab (immunotherapy drugs)
- What this could lead to
- If it works, this could offer a new treatment option for people whose leukemia or MDS returns after a stem cell transplant.
- What could go wrong
- This is a very early, small phase 1 trial focused on safety and dosing, not effectiveness. The drugs can cause serious immune-related side effects, and the results may not apply to all patients.
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
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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29 people
The number who actually took part.
- Started
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Oct 2018
- Finished
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Dec 2024
- 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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18 years 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: * Patients with evidence of relapsed or refractory AML or MDS following allogeneic stem cell transplantation * Patients must have received preparative regimens to include either busulfan- or melphalan-based regimens * Patient must have achieved myeloid engraftment as defined by an absolute neutrophil count \>= 500 micro/L on 3 consecutive days * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 * Total bilirubin =\< 2 times upper limit of normal (x ULN) (=\< 3 x ULN if considered to be due to Gilbert's syndrome) * Aspartate aminotransferase or alanine aminotransferase =\< 2.5 x ULN * Serum creatinine =\< 2 x ULN or glomerular filtration rate (GFR) \>= 50 * Patients must provide written informed consent * The interval from the infusion of stem cells to time of initiation of nivolumab or ipilimumab will be at least 6 weeks (42 days) * Females must be surgically or biologically sterile or postmenopausal (amenorrheic for at least 12 months) or if of childbearing potential, must have a negative serum or urine pregnancy test within 72 hours before the start of the treatment * Women of childbearing potential must agree to use an adequate method of contraception during the study and until 3 months after the last treatment. Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study until 3 months after the last treatment Exclusion Criteria: * Patients with known allergy or hypersensitivity to nivolumab or ipilimumab or any of their components * Patients with acute GVHD \> grade 2 at any time during the post-transplant course * Patients with a known history of severe interstitial lung disease or severe pneumonitis or active pneumonitis that is uncontrolled in the opinion of the treating physician * Patients with a known history of any of the following autoimmune diseases are excluded: * Patients with a history of inflammatory bowel disease (including Crohn's disease and ulcerative colitis) * Patients with a history of rheumatoid arthritis, systemic progressive sclerosis (scleroderma), systemic lupus erythematosus, autoimmune vasculitis (e.g., Wegener's granulomatosis) * Patients with solid organ allografts (such as renal transplant) are excluded * Ongoing immunosuppressive therapy for the treatment of GVHD. Patients receiving GVHD prophylaxis will be allowed on this study * Patients with symptomatic central nervous system (CNS) leukemia at the time of evaluation or patients with poorly controlled CNS leukemia * Active and uncontrolled disease/(active uncontrolled infection, uncontrolled hypertension despite adequate medical therapy, active and uncontrolled congestive heart failure New York Heart Association \[NYHA\] class III/IV, clinically significant and uncontrolled arrhythmia) as judged by the treating physician * Patients with known human immunodeficiency virus seropositivity will be excluded * Known to be positive for hepatitis B by surface antigen expression. Known to have active hepatitis C infection (positive by polymerase chain reaction or on antiviral therapy for hepatitis C within the last 6 months) * Any other medical, psychological, or social condition that may interfere with study participation or compliance, or compromise patient safety in the opinion of the investigator * Patients unwilling or unable to comply with the protocol * Pregnant or breastfeeding
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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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M D Anderson Cancer Center
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Platelet-Boosting drug help control a rare bone marrow disorder?
- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?
- Can a Nine-Week group program help blood cancer patients grow through trauma?
- A nationwide effort to map a rare blood Cancer's toll
- Can blood tests predict transplant complications?