COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

Peptide Vaccination in Combination With Azacitidine for Patients With MDS and AML (AZACTA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02750995
Recruitment Status : Recruiting
First Posted : April 26, 2016
Last Update Posted : September 1, 2017
Technical University of Denmark
Information provided by (Responsible Party):
Inge Høgh Dufva, Herlev Hospital

Brief Summary:
The purpose of this phase I study is to investigate the combination of hypomethylating agents with experimental peptide vaccination against four selected tumor antigens, known to be upregulated in response to hypomethylating agents, in patients with high risk myelodysplastic syndrome and acute myeloid leukemia.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Acute Myeloid Leukemia Biological: NPMW-peptide vaccine Drug: Azacitidine Phase 1

Show Show detailed description

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Peptide Vaccination in Combination With Azacitidine for Patients With Myelodysplastic Syndrome and Acute Myeloid Leukemia - A Phase I Study
Actual Study Start Date : April 2016
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : September 2020

Arm Intervention/treatment
Experimental: Vaccination
Azacitidine + NPMW-peptide vaccine
Biological: NPMW-peptide vaccine
Peptide vaccine against long peptide sequences from NY-ESO-1, PRAME, MAGE-A3, WT-1.

Drug: Azacitidine
Standard therapy. All participants receive azacitidine 6 months prior to inclusion, which continues during the study period.
Other Name: Vidaza

Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Through study completion, up to 24 months. ]
    The safety of combining azacitidine treatment with this peptide vaccine will be judged on basis of the reported adverse events during the study period.

Secondary Outcome Measures :
  1. Immunological evaluation [ Time Frame: weeks: 0, 1, 9, 21. Thereafter months: 12, 18, 24 ]
    Measurements of specific T-cell reactivity against the vaccine components. The immunological response observed before, during and after treatment will be evaluated and compared. Analyses will be performed both on blood and bone marrow samples.

Other Outcome Measures:
  1. Clinical efficacy [ Time Frame: Months: 6, 12, 18, 24 ]
    The clinical efficacy of the treatment will be judged by the objective response rate according to the IWG modified response criteria, transfusion requirements and time to progression and survival.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Participants must have received 6 courses of azacitidine and been evaluated with response to treatment.
  2. Histologically confirmed high-risk MDS or AML (<30% blasts) and a normo- or hypercellular marrow after 6 courses of azacitidine.
  3. Indication for continued treatment with azacitidine.
  4. Age >18 years.
  5. Signed consent form after receiving both written and oral information.
  6. The patients must be willing to follow the scheduled treatment and sampling.

Exclusion Criteria:

  1. Hypocellular bone marrow after 6 courses of azacitidine.
  2. Additional active cancer disease. Participants treated for a second malignancy may be included if the patient is without evidence of disease at least 2 years after completion of treatment.
  3. Participants with a known hypersensitivity to any of the active substances or to any of the excipients.
  4. Participants with secondary MDS or AML
  5. Severe allergies or previous anaphylactic reactions.
  6. Active autoimmune disease, for example autoimmune neutropenia/ thrombocytopenia or hemolytic anemia, systemic lupus erythematosus, Sjögren's syndrome, scleroderma, myasthenia gravis, Goodpasture's syndrome, Addison's disease, Hashimoto's thyroiditis, Grave's disease.
  7. Concomitant treatment with systemic immunosuppressive medications (including prednisone, methotrexate etc.). Participants are allowed to receive up to 10 mg prednisone at the days of azacitidine injection.
  8. Concomitant treatment with other experimental drugs.
  9. Concomitant treatment with other systemic anti-cancer therapy.
  10. Pregnant or breastfeeding females.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02750995

Layout table for location contacts
Contact: Jytte Kock, Nurse +45 38682129
Contact: Staffan Holmberg, MD +45 60744083

Layout table for location information
Dept of Hematology, Herlev Hospital Recruiting
Herlev, Denmark, 2730
Contact: Jytte Kock, Nurse    +45 38682129   
Contact: Staffan Holmberg, MD    +45 60744083   
Principal Investigator: Inge Høgh Dufva, Lector         
Sub-Investigator: Staffan Holmberg, MD         
Sub-Investigator: Anne Ortved Gang, MD         
Sponsors and Collaborators
Inge Høgh Dufva
Technical University of Denmark
Layout table for investigator information
Principal Investigator: Inge Høgh Dufva, Lector Herlev Hospital
Layout table for additonal information
Responsible Party: Inge Høgh Dufva, Clinical Associate Professor, Herlev Hospital Identifier: NCT02750995    
Other Study ID Numbers: AZACTA
First Posted: April 26, 2016    Key Record Dates
Last Update Posted: September 1, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified participant data for primary, secondary and tertiary outcome measurements will be made available within 6 months of study completion.
Keywords provided by Inge Høgh Dufva, Herlev Hospital:
myelodysplastic syndrome
acute myeloid leukemia
peptide vaccine
cancer testis antigen
cancer vaccine
combination therapy
Additional relevant MeSH terms:
Layout table for MeSH terms
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors