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NCT02996474

Pembrolizumab and Decitabine for Refractory or Relapsed Acute Myeloid Leukemia

Completed Phase 1, PHASE2 Results posted Last updated 28 December 2022
What this trial tests

Phase 1, PHASE2 trial testing Pembrolizumab in Relapsed Acute Myeloid Leukemia in 10 participants. Completed in 2 April 2019.

Timeline
16 December 2016
Primary endpoint
2 April 2019
2 April 2019

Quick facts

Lead sponsorNational Heart, Lung, and Blood Institute (NHLBI)
PhasePhase 1, PHASE2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment10
Start date16 December 2016
Primary completion2 April 2019
Estimated completion2 April 2019
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

Adults 18 to 99, any sex, with Relapsed Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Feasibility of a Novel Combination of Pembrolizumab and Decitabine in Relapsed/Refractory Acute Myeloid Leukemia Participants Primary · 24 weeks

The number of patients who could be treated with a novel combination of Pembrolizumab and Decitabine in relapsed/refractory Acute Myeloid Leukemia participants without being removed from the protocol due to treatment related toxicities.

GroupValue95% CI
Pembrolizumab and Decitabine for Treatment of Acute Myeloid Leukemia10
Median Days to First Response From Start of Study to Initial Achievement of First Response (mCR, mCRi, CR, CRi, PR, or SD) Secondary · At the end of each cycle 2 (week 6), 4 (week 12), 6 (week 18), 8 (up to week 24)

Time to first response (days) is time from start of study to first response (mCR, mCRi, CR, CRi, PR, or SD) in relapsed/refractory acute myeloid leukemia (AML) participants. Measurable residual disease (MRD) negative complete remission is no evidence of residual disease, \< 5% blasts, no Auer rods and may be with mCR or without count recovery (ANC ≥ 1,000/mcl and platelet count ≥ 100,000/mcl) as mCRi. Morphologic complete remission (CR) is ANC ≥ 1,000/mcl, platelet count ≥ 100,000/mcl, \< 5% blasts, no Auer rods, no evidence of disease; Morphologic complete remission with incomplete blood cou

GroupValue95% CI
Pembrolizumab and Decitabine for Treatment of Acute Myeloid Leukemia4237 – 49
Median Days to Best Response From Start of Study to Initial Achievement of Best Response (by the Order of mCR, mCRi, CR, CRi, PR, or SD) Secondary · At the end of each cycle 2 (week 6), 4 (week 12), 6 (week 18), 8 (up to week 24)

Time to Best Response is defined as time (days) from start of study to initial achievement of best response. Best response is defined by the order of mCR, mCRi, CR, CRi, PR, or SD. Measurable residual disease (MRD) negative complete remission is no evidence of residual disease, \< 5% blasts, no Auer rods and may be with mCR or without count recovery (ANC ≥ 1,000/mcl and platelet count ≥ 100,000/mcl) as mCRi. Morphologic complete remission (CR) is ANC ≥ 1,000/mcl, platelet count ≥ 100,000/mcl, \< 5% blasts, no Auer rods, no evidence of disease; Morphologic complete remission with incomplete blo

GroupValue95% CI
Pembrolizumab and Decitabine for Treatment of Acute Myeloid Leukemia4239 – 159
Median Duration (Days) of Response From Initial Achievement of Response to Loss of Response (mCR, mCRi, CR, CRi, PR, or SD) Secondary · At the end of each cycle 2 (week 6), 4 (week 12), 6 (week 18), 8 (up to week 24)

To determine duration (days) of response as defined as time from initial achievement of response to loss of response (mCR, mCRi, CR, CRi, PR, or SD). Measurable residual disease (MRD) negative complete remission is no evidence of residual disease, \< 5% blasts, no Auer rods and may be with mCR or without count recovery (ANC ≥ 1,000/mcl and platelet count ≥ 100,000/mcl) as mCRi. Morphologic complete remission (CR) is ANC ≥ 1,000/mcl, platelet count ≥ 100,000/mcl, \< 5% blasts, no Auer rods, no evidence of disease; Morphologic complete remission with incomplete blood count recovery (CRi) is sam

GroupValue95% CI
Pembrolizumab and Decitabine for Treatment of Acute Myeloid Leukemia14812 – 337
Median Duration (Days) of Best Response From Initial Achievement of Best Response to Loss of Best Response (by the Order of mCR, mCRi, CR, CRi, PR, or SD) Secondary · At the end of each cycle 2 (week 6), 4 (week 12), 6 (week 18), 8 (up to week 24)

Duration of Best Response is time (days) from initial achievement of best response to loss of best response. Best response is defined by the order of mCR, mCRi, CR, CRi, PR, or SD. Measurable residual disease (MRD) negative complete remission is no evidence of residual disease, \< 5% blasts, no Auer rods and may be with mCR or without count recovery (ANC ≥ 1,000/mcl and platelet count ≥ 100,000/mcl) as mCRi. Morphologic complete remission (CR) is ANC ≥ 1,000/mcl, platelet count ≥ 100,000/mcl, \< 5% blasts, no Auer rods, no evidence of disease; Morphologic complete remission with incomplete blo

GroupValue95% CI
Pembrolizumab and Decitabine for Treatment of Acute Myeloid Leukemia14812 – 316
Overall Survival Secondary · from enrollment until date of death, assessed up to 24 weeks

Number of participants overall survival is defined as death from any cause

GroupValue95% CI
Pembrolizumab and Decitabine for Treatment of Acute Myeloid Leukemia0

Adverse events — posted to ClinicalTrials.gov

Time frame: 24 weeks. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Pembrolizumab and Decitabine for Treatment of Acute Myeloid Leukemia
Serious: 7/10 (70%)
Deaths: 0/10

Serious adverse events (12 terms)

ReactionSystemPembrolizumab and Decitabi…
Febrile neutropeniaBlood and lymphatic system disorders
PainGeneral disorders
SepsisInfections and infestations
HypokalaemiaMetabolism and nutrition disorders
DiarrhoeaGastrointestinal disorders
Wound infection bacterialInfections and infestations
Blood culture positiveInvestigations
HypophosphataemiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
HeadacheNervous system disorders
EpistaxisRespiratory, thoracic and mediastinal disorders
HypotensionVascular disorders
Other adverse events (148 terms — click to expand)

ReactionSystemPembrolizumab and Decitabi…
Platelet count decreasedInvestigations
FatigueGeneral disorders
Alanine aminotransferase increasedInvestigations
HypotensionVascular disorders
ConstipationGastrointestinal disorders
Activated partial thromboplastin time prolongedInvestigations
DyspnoeaCardiac disorders
HyperglycaemiaEndocrine disorders
HypoalbuminaemiaHepatobiliary disorders
Aspartate aminotransferase increasedInvestigations
Body temperature increasedInvestigations
Weight increasedInvestigations
PetechiaeBlood and lymphatic system disorders
Localised oedemaCardiac disorders
NauseaGastrointestinal disorders
Oral painGastrointestinal disorders
Decreased appetiteGeneral disorders
LethargyGeneral disorders
MalaiseGeneral disorders
PainGeneral disorders
Imaging procedure abnormalInvestigations
HypocalcaemiaMetabolism and nutrition disorders
HypokalaemiaMetabolism and nutrition disorders
HeadacheNervous system disorders
Increased tendency to bruiseBlood and lymphatic system disorders
Pericardial effusionCardiac disorders
Abdominal distensionGastrointestinal disorders
DyspepsiaGastrointestinal disorders
Non-cardiac chest painGeneral disorders
Lymphocyte count decreasedInvestigations
Neutrophil count decreasedInvestigations
HypernatraemiaMetabolism and nutrition disorders
HyponatraemiaMetabolism and nutrition disorders
HypophosphataemiaMetabolism and nutrition disorders
Neck painMusculoskeletal and connective tissue disorders
AnxietyPsychiatric disorders
AtelectasisRespiratory, thoracic and mediastinal disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders
Dry skinSkin and subcutaneous tissue disorders
PruritusSkin and subcutaneous tissue disorders

Most-reported serious reactions: Febrile neutropenia, Pain, Sepsis, Hypokalaemia, Diarrhoea, Wound infection bacterial, Blood culture positive, Hypophosphataemia.

Data from ClinicalTrials.gov NCT02996474 adverse events section.

Sponsor's own description

Background: Acute myeloid leukemia (AML) is a cancer of the white blood cells. It is fatal if not treated. Treatment for AML that has not responded to treatment (refractory) or has returned after treatment (relapsed) often do not work. Researchers want to see if an immunotherapy drug, combined with a less intense chemotherapy, may be able to help. Objective: To test if pembrolizumab, in combination with decitabine, is a possible treatment for people with relapsed or refractory AML. Eligibility: Adults 18 years of age and older with refractory AML or relapsed AML. Design: Participants will be first screened for eligibility. The study is counted in 21-day cycles. The initial phase of the study consists of 8 cycles. Participants may be in the study for up to 2 years if they are responding to the treatment. The first 3 weeks of treatment is usually done in the hospital. The rest may be done as an outpatient. Participants will get pembrolizumab at the beginning of each cycle through an IV. Participants will usually get decitabine by IV on days 8 12 and days 15 19 of every other cycle. Participants will give blood samples. Participants will have bone marrow exams. A needle will be inserted into the hip to extract cells from the bone marrow. Some participants may give a sample of saliva from the inside of their cheek. Some participants may give a small skin sample. The top layer of the skin is removed. Some patients may require leukapheresis before starting treatment. This is a procedure to remove leukemia cells in the blood stream.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Improvement of the anticancer efficacy of PD-1/PD-L1 blockade via combination therapy and PD-L1 regulation.
    Wu M, Huang Q, Xie Y, Wu X, et al · · 2022 · cited 336× · PMID 35279217 · DOI 10.1186/s13045-022-01242-2
  2. Epigenetic regulation in the tumor microenvironment: molecular mechanisms and therapeutic targets.
    Yang J, Xu J, Wang W, Zhang B, et al · · 2023 · cited 251× · PMID 37217462 · DOI 10.1038/s41392-023-01480-x
  3. Recent advances in targeted therapies in acute myeloid leukemia.
    Bhansali RS, Pratz KW, Lai C. · · 2023 · cited 151× · PMID 36966300 · DOI 10.1186/s13045-023-01424-6
  4. Dysregulated haematopoietic stem cell behaviour in myeloid leukaemogenesis.
    Yamashita M, Dellorusso PV, Olson OC, Passegué E. · · 2020 · cited 128× · PMID 32415283 · DOI 10.1038/s41568-020-0260-3
  5. Recent developments in immunotherapy of acute myeloid leukemia.
    Lichtenegger FS, Krupka C, Haubner S, Köhnke T, et al · · 2017 · cited 103× · PMID 28743264 · DOI 10.1186/s13045-017-0505-0
  6. Targeting the Immune Microenvironment in Acute Myeloid Leukemia: A Focus on T Cell Immunity.
    Lamble AJ, Lind EF. · · 2018 · cited 95× · PMID 29951373 · DOI 10.3389/fonc.2018.00213
  7. Advances in targeted therapy for acute myeloid leukaemia.
    Kayser S, Levis MJ. · · 2018 · cited 88× · PMID 29193012 · DOI 10.1111/bjh.15032
  8. Epigenetic regulation in hematopoiesis and its implications in the targeted therapy of hematologic malignancies.
    Zhao A, Zhou H, Yang J, Li M, et al · · 2023 · cited 81× · PMID 36797244 · DOI 10.1038/s41392-023-01342-6

Verify or expand the search:

Other trials of Pembrolizumab

Trials testing the same drug.

Other recruiting trials for Relapsed Acute Myeloid Leukemia

Currently open trials in the same condition.

Other National Heart, Lung, and Blood Institute (NHLBI) trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02996474.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing