Peptide Vaccination in Combination With Azacitidine for Patients With MDS and AML

NCT02750995 | Completed Phase 1 DMC

• 1 other identifier: AZACTA
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Myelodysplastic Syndrome; Acute Myeloid Leukemia

Keywords

myelodysplastic syndrome; acute myeloid leukemia; azacitidine; peptide vaccine; cancer testis antigen; cancer vaccine; immunotherapy; combination therapy; NY-ESO-1; PRAME; MAGE-A3; WT-1; vaccine

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

  The safety of combining azacitidine treatment with this peptide vaccine will be judged on basis of the reported adverse events during the study period.

  Through study completion, up to 24 months.

Secondary Outcomes (1)

• Immunological evaluation

  weeks: 0, 1, 9, 21. Thereafter months: 12, 18, 24

Other Outcomes (1)

• Clinical efficacy

  Months: 6, 12, 18, 24

Study Arms (1)

Vaccination

EXPERIMENTAL

Azacitidine + NPMW-peptide vaccine

Biological: NPMW-peptide vaccine; Drug: Azacitidine

Interventions

NPMW-peptide vaccine BIOLOGICAL

Peptide vaccine against long peptide sequences from NY-ESO-1, PRAME, MAGE-A3, WT-1.

Vaccination

Azacitidine DRUG

Standard therapy. All participants receive azacitidine 6 months prior to inclusion, which continues during the study period.

Also known as: Vidaza

Vaccination

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Hypocellular bone marrow after 6 courses of azacitidine.
• 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.
• Participants with a known hypersensitivity to any of the active substances or to any of the excipients.
• Participants with secondary MDS or AML
• Severe allergies or previous anaphylactic reactions.
• 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.
• 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.
• Concomitant treatment with other experimental drugs.
• Concomitant treatment with other systemic anti-cancer therapy.
• Pregnant or breastfeeding females.

Sponsors & Collaborators

• Daniel El Fassi: lead
• Technical University of Denmark: collaborator

Study Sites (1)

Dept of Hematology, Herlev Hospital

Herlev, 2730, Denmark

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Leukemia, Myeloid, Acute

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Daniel El Fassi, MD PhD

  Herlev Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Associate Professor

Study Record Dates

• First Submitted: April 18, 2016
• First Posted: April 26, 2016
• Study Start: April 1, 2016
• Primary Completion: January 2, 2018
• Study Completion: June 1, 2019
• Last Updated: November 25, 2020

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will share

Locations

DK (1)