Myelodysplastic SyndromesPhase 1NCT03286114
Augmentation of the Graft vs. Leukemia Effect Via Checkpoint Blockade With Pembrolizumab
A Phase 1 study testing Pembrolizumab in people with Myelodysplastic Syndromes and Acute Myeloid Leukemia, run by University of Michigan Rogel Cancer Center at 1 site in United States.
Trial summary
This is a single arm, open-label, Phase 1b study of pembrolizumab for patients with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), and acute lymphoblastic leukemia (ALL) whose disease has relapsed after receiving allogeneic hematopoetic stem cell transplant. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
- What is being tested?
- Pembrolizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Dec 2017to 28 Apr 2021
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL) or Myelodysplastic Syndrome (MDS) in confirmed relapse
- Confirmation of 'measurable disease'
- Patient may not have received definitive salvage chemotherapy for their post-transplant relapse within the past 21 days.
- Be willing and able to provide written informed consent/assent for the trial
- Be ≥ 18 years of age on day of signing informed consent
- Be willing to provide tissue from bone marrow biopsies
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- Have a performance status of 0, to 1 on the ECOG Performance Scale. Eastern Cooperative Oncology Group Performance Status: an attempt to quantify cancer patients' general well-being and activities of daily life. The score ranges from 0 to 5 where 0 is asymptomatic and 5 is death.
- Demonstrate adequate organ function
- Female subject of childbearing potential should have a negative urine or serum pregnancy within 72 hours prior to receiving the first dose of study medication.
- Female subjects of childbearing potential must be willing to use an adequate method of contraception
- Male subjects of childbearing potential must agree to use an adequate method of contraception
Who cannot join
- Has had relapse prior to primary neutrophil engraftment or ≤21 days post HCT.
- Has received >1 line of chemotherapy or other treatment directed towards post-transplant relapse prior to study entry
- Rapidly progressive relapse requiring urgent chemotherapy as determined by treating physician
- Is currently participating and receiving study therapy of an investigational agent and received study therapy within 2 weeks of the first dose of treatment.
- Has a diagnosis of active GvHD (≥ Grade I)
- Receiving systemic steroid therapy of > 10mg prednisone daily or equivalent*
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- Has received GM-CSF within 14 days of first dose of pembrolizumab
- Has a known history of active TB (Bacillus Tuberculosis)Hypersensitivity to pembrolizumab or any of its excipients
- Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study Day 1 or who has not recovered from adverse events
- Has had prior chemotherapy within 21 days or radiation therapy within 14 days prior to study Day 1 or who has not recovered from adverse events
- Has a known additional (secondary) malignancy that is progressing or requires active treatment
- Has known or suspected active central nervous system (CNS) metastases and/or carcinomatous meningitis
- Has a history of (non-infectious) pneumonitis that required steroids, or current pneumonitis
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
- Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial
- Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent
- Has a known history of Human Immunodeficiency Virus (HIV)
- Has known active Hepatitis B or Hepatitis C
- Has received a live vaccine within 30 days of planned start of study therapy
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Michigan Cancer Center
Ann Arbor, Michigan, 48109
About this study
Official title: Augmentation of the Graft vs. Leukemia Effect Via Checkpoint Blockade With Pembrolizumab for Relapse of Primary Malignancy After Allogeneic Hematopoietic Stem Cell Transplant: A Feasibility Study
This is a single arm, open-label, Phase 1b study of pembrolizumab for patients with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), and acute lymphoblastic leukemia (ALL) whose disease has relapsed after receiving allogeneic hematopoetic stem cell transplant.
- Being tested
- Pembrolizumab
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PembrolizumabDrug
Also called KEYTRUDA
200mg IV every 21 days
| Study group | Role | What they receive |
|---|---|---|
| Pembrolizumab | Experimental | Not described Receives: Drug: Pembrolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Number of Patients That Demonstrate Clinical Benefit From Treatment
This study will assess if the study drug is promising for further study. The study drug will be considered promising if at least 4 patients receive a clinical benefit or if any complete response is seen. Clinical benefit is defined as either stable disease, partial remission or complete remission to treatment.
Complete remission (CR) will be defined as achieving a morphologic leukemia free state by achieving all of the following criteria: bone marrow myeloblasts < 5% by morphologic assessment; AND absence of circulating blasts with phenotypic or morphologic features of leukemia (e.g. Auer rods) AND no evidence of extramedulary disease.
Partial remission (PR) will be defined as a ≥ 50% reduction in bone marrow blast percentage to 5-25% or marrow blasts < 5% with persistent Auer rods, flow cytometric or cytogenetic disease.
SD will be defined as ≤ 5% increase in blasts or decreased blast percentage in the bone marrow that does not meet the criteria for PR.
Measured: Day 77
The Number of Patients That Respond to Treatment
This study will assess the number of patients that respond to treatment by overall response rate (ORR). ORR is defined as the number of patients will complete remission and partial remission.
Complete remission (CR) will be defined as achieving a morphologic leukemia free state by achieving all of the following criteria: bone marrow myeloblasts < 5% by morphologic assessment; AND absence of circulating blasts with phenotypic or morphologic features of leukemia (e.g. Auer rods) AND no evidence of extramedulary disease.
Partial remission (PR) will be defined as a ≥ 50% reduction in bone marrow blast percentage to 5-25% or marrow blasts < 5% with persistent Auer rods, flow cytometric or cytogenetic disease.
Measured: Day 77
Graft Versus Host Disease (GvHD) or Other Significant Immune Mediated Toxicities
The number of patients that experience Graft Versus Host Disease (GvHD) or other significant immune mediated toxicities
Measured: 30 Days Post Treatment
Secondary outcomes (2)
Overall Survival
The number of patients alive at 1 year
Measured: 1 Year
Event- Free Survival
The number of patients alive at 1 year without disease
Measured: 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2017
- Study start21 Dec 2017
- Primary completion22 Oct 2020
- Study completion28 Apr 2021
Why it stopped: Lack of patient population
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by University of Michigan Rogel Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Michigan Rogel Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Responsible party
- Sponsor
- Investigators
- John Magenau, M.D., Principal investigator, University of Michigan Rogel Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03286114
- Sponsor's protocol ID
- UMCC 2017.056
- Other IDs
- HUM00129255
- Status last verified
- Apr 2025
- First submitted
- 8 Sep 2017
- Last updated
- 11 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Magenau JM, Frame DG, Riwes M, Maciejewski J, Anand S, Pawarode A, Perry AM, Geer M, Braun T, Ghosh M, Reddy P. PD-1 inhibition for relapse after allogeneic transplantation in acute myeloid leukemia and myelodysplastic syndrome. Blood Adv. 2025 Aug 12;9(15):3878-3886. doi: 10.1182/bloodadvances.2024015200.
PubMed 40198769
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Augmentation of the Graft vs. Leukemia Effect Via Checkpoint Blockade With Pembrolizumab for Relapse of Primary Malignancy After Allogeneic Hematopoietic Stem Cell Transplant: A Feasibility Study
- ClinicalTrials.gov ID
- NCT03286114
- Acronym / sponsor ID
- UMCC 2017.056
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of patient population
- Enrolment
- 16 participants (actual)
- Lead sponsor
- University of Michigan Rogel Cancer CenterUniversity, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Study start
- 21 Dec 2017 (actual)
- Primary completion
- 22 Oct 2020 (actual)
- Study completion
- 28 Apr 2021 (actual)
- First posted
- 18 Sep 2017 (actual)
- Results
- Posted 11 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single arm, open-label, Phase 1b study of pembrolizumab for patients with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), and acute lymphoblastic leukemia (ALL) whose disease has relapsed after receiving allogeneic hematopoetic stem cell transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pembrolizumab | Experimental | Drug: Pembrolizumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL) or Myelodysplastic Syndrome (MDS) in confirmed relapse
- Confirmation of 'measurable disease'
- Patient may not have received definitive salvage chemotherapy for their post-transplant relapse within the past 21 days.
- Be willing and able to provide written informed consent/assent for the trial
- Be ≥ 18 years of age on day of signing informed consent
Key exclusion criteria (20 listed)
- Has had relapse prior to primary neutrophil engraftment or ≤21 days post HCT.
- Has received >1 line of chemotherapy or other treatment directed towards post-transplant relapse prior to study entry
- Rapidly progressive relapse requiring urgent chemotherapy as determined by treating physician
- Is currently participating and receiving study therapy of an investigational agent and received study therapy within 2 weeks of the first dose of treatment.
- Has a diagnosis of active GvHD (≥ Grade I)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PembrolizumabAlso: KEYTRUDA200mg IV every 21 days | Drug | Pembrolizumab |
Primary outcome
The Number of Patients That Demonstrate Clinical Benefit From Treatment
Time frame: Day 77
This study will assess if the study drug is promising for further study. The study drug will be considered promising if at least 4 patients receive a clinical benefit or if any complete response is seen. Clinical benefit is defined as either stable disease, partial remission or complete remission to treatment. Complete remission (CR) will be defined as achieving a morphologic leukemia free state by achieving all of the following criteria: bone marrow myeloblasts < 5% by morphologic assessment; AND absence of circulating blasts with phenotypic or morphologic features of leukemia (e.g. Auer rods) AND no evidence of extramedulary disease. Partial remission (PR) will be defined as a ≥ 50% reduction in bone marrow blast percentage to 5-25% or marrow blasts < 5% with persistent Auer rods, flow cytometric or cytogenetic disease. SD will be defined as ≤ 5% increase in blasts or decreased blast percentage in the bone marrow that does not meet the criteria for PR.
The Number of Patients That Respond to Treatment
Time frame: Day 77
This study will assess the number of patients that respond to treatment by overall response rate (ORR). ORR is defined as the number of patients will complete remission and partial remission. Complete remission (CR) will be defined as achieving a morphologic leukemia free state by achieving all of the following criteria: bone marrow myeloblasts < 5% by morphologic assessment; AND absence of circulating blasts with phenotypic or morphologic features of leukemia (e.g. Auer rods) AND no evidence of extramedulary disease. Partial remission (PR) will be defined as a ≥ 50% reduction in bone marrow blast percentage to 5-25% or marrow blasts < 5% with persistent Auer rods, flow cytometric or cytogenetic disease.
Graft Versus Host Disease (GvHD) or Other Significant Immune Mediated Toxicities
Time frame: 30 Days Post Treatment
The number of patients that experience Graft Versus Host Disease (GvHD) or other significant immune mediated toxicities
2 secondary outcomes
Overall Survival
Time frame: 1 Year
Event- Free Survival
Time frame: 1 Year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40198769Magenau JM, Frame DG, Riwes M, Maciejewski J, Anand S, Pawarode A, Perry AM, Geer M, Braun T, Ghosh M, Reddy P. PD-1 inhibition for relapse after allogeneic transplantation in acute myeloid leukemia and myelodysplastic syndrome. Blood Adv. 2025 Aug 12;9(15):3878-3886. doi: 10.1182/bloodadvances.2024015200.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Magenau, M.D. principal investigator
University of Michigan Rogel Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Pembrolizumab in people with Myelodysplastic Syndromes and Acute Myeloid Leukemia, run by University of Michigan Rogel Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Number of Patients That Demonstrate Clinical Benefit From Treatment (time frame: Day 77). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03286114, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 May 2025.