NCT01390311

Brief Summary

This phase I trial studies the effects and safety of adding azacitidine (5-AzaC) to the standard of care (Soc) for patients with relapsed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after being treated with donor stem cell transplant. SoC includes giving an infusion of the donor's white blood cells (donor lymphocyte infusion or DLI) to boost the anticancer effects of the transplant. Giving 5-AzaC after DLI may alter the function of T-cells resulting in reduced incidence of graft versus host disease (GVHD) while maintaining the anticancer effects.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2012

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 23, 2011

Completed
18 days until next milestone

First Posted

Study publicly available on registry

July 11, 2011

Completed
9 months until next milestone

Study Start

First participant enrolled

April 1, 2012

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2014

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2015

Completed
Last Updated

April 27, 2015

Status Verified

April 1, 2015

Enrollment Period

1.9 years

First QC Date

June 23, 2011

Last Update Submit

April 24, 2015

Conditions

Outcome Measures

Primary Outcomes (1)

  • MTD and DLT of azacitidine when given after DLI

    MTD is defined as the maximum dose level immediately below the dose level at which 2 patients of a cohort (3 to 6 patients) experience dose-limiting toxicity during the first cycle.

    40 days (after DLI)

Secondary Outcomes (4)

  • Rate of Grade II-IV and III-IV acute GVHD based on Glucksberg criteria

    100 days (after DLI)

  • Rates of CR, CRi, PR, and overall response (CR+CRi+PR = overall response)

    1 year (after DLI)

  • Overall survival

    100 days (after DLI)

  • Effects of increasing dose of azacitidine on frequency and absolute number of (resting T-cells) rTregs and (T-cells)Tregs

    64 days (Baseline, 7 days, 14 days, 21 days, and ~60 days after first dose of azacitidine

Study Arms (3)

Control Cohort

ACTIVE COMPARATOR

1. Pre-DLI Salvage Chemotherapy (at the discretion of the treating physician) 2. DLI will be administered 2 +/- 1 weeks after pre-DLI chemotherapy. The minimum CD3+ cell counts in DLI product should be 1 x 107 cells/kg for sibling and \~1 x 106/kg for match unrelated donors based on recipient weight 3. No Azacitidine will be given

Drug: Pre-DLI Salvage ChemotherapyBiological: Donor Leukocyte Infusion (DLI)

Cohort 1 (Starting Dose)

EXPERIMENTAL

1. Pre-DLI Salvage Chemotherapy (at the discretion of the treating physician) 2. DLI will be administered 2 +/- 1 weeks after pre-DLI chemotherapy. The minimum CD3+ cell counts in DLI product should be 1 x 107 cells/kg for sibling and \~1 x 106/kg for match unrelated donors based on recipient weight 3. Azacitidine 45 mg/m2 IV on Days 4, 6, 8, and 10 post-DLI.

Drug: AzacitidineDrug: Pre-DLI Salvage ChemotherapyBiological: Donor Leukocyte Infusion (DLI)

Cohort 2

EXPERIMENTAL

1. Pre-DLI Salvage Chemotherapy (at the discretion of the treating physician) 2. DLI will be administered 2 +/- 1 weeks after pre-DLI chemotherapy. The minimum CD3+ cell counts in DLI product should be 1 x 107 cells/kg for sibling and \~1 x 106/kg for match unrelated donors based on recipient weight 3. Azacitidine 75 mg/m2 IV on Days 4, 6, 8, and 10 post-DLI.

Drug: AzacitidineDrug: Pre-DLI Salvage ChemotherapyBiological: Donor Leukocyte Infusion (DLI)

Interventions

Also known as: 5-AC, 5-azacytidine, azacytidine, Vidaza
Cohort 1 (Starting Dose)Cohort 2

At the discretion of the treating physician

Cohort 1 (Starting Dose)Cohort 2Control Cohort
Cohort 1 (Starting Dose)Cohort 2Control Cohort

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Must have a diagnosis of AML/MDS based on 2008 World Health Organization (WHO) classification of myeloid malignancies
  • Must have laboratory, histologic, or cytogenetic evidence of disease relapse after allogeneic hematopoietic stem cell transplant (HSCT) and require salvage therapy followed by DLI
  • Must have original donor
  • Must have life expectancy \>= 2 months
  • Must be ≥ 18 years old. Azacitidine is not approved by the FDA for use in children
  • Must have an Eastern Cooperative Oncology Group (ECOG) performance status =\< 3
  • Must have laboratory results indicating:
  • Total bilirubin \< 2.0 mg/dL
  • Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =\< 3 X the upper limit of institutional normal
  • Serum creatinine =\< 2.0 mg/dL
  • Patient must have ability to understand and willingness to provide written informed consent prior to participation in the study and any related procedures being performed
  • The effects of 5-AzaC on the developing human fetus at the recommended therapeutic dose are unknown; for this reason and because category D agents as well as other therapeutic agents used in this trial are known to be teratogenic, women of childbearing age must have a negative serum pregnancy test (Beta \[B\]-human chorionic gonadotropin) within 72 hours prior to initiating therapy and be willing to not become pregnant by using effective contraception while undergoing treatment and for at least 3 months afterwards; azacitidine is a pregnancy category D drug and could be harmful to or cause loss of a fetus; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
  • Men must be willing not to father a new child while receiving therapy; they must use an effective barrier method of contraception during the study and for 3 months following the last dose
  • Both men and women and members of all races and ethnic groups are eligible for this trial
  • Must be the original donor for the allogeneic bone marrow transplant patient
  • +3 more criteria

You may not qualify if:

  • Must not have Grade III-IV GVHD
  • Must not have an advanced malignant hepatic tumor
  • Must not receive anti-thymocyte globulin, campath (alemtuzumab) or daclizumab within 4 weeks of DLI
  • Must not receive any other forms of chemotherapy after cell infusion during the treatment protocol
  • Must not be receiving any other investigational agents within 14 days of first dose of study drug
  • Must not have uncontrolled intercurrent illness including ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or psychiatric illness/social situations that would limit compliance with study requirements
  • Must not be pregnant or breastfeeding; pregnant women are excluded from this study because azacitidine is a Category D agent with the potential for teratogenic or abortifacient effects; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with azacitidine, breastfeeding should be discontinued if the mother is treated with azacitidine; these potential risks may also apply to other agents used in this study
  • Must not have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to azacitidine or other agents used in the study
  • Must not have a known or suspected hypersensitivity to azacitidine or mannitol.
  • Must not be human immunodeficiency virus (HIV)-positive and on combination antiretroviral therapy; these patients are ineligible because of the potential for pharmacokinetic interactions with azacitidine; in addition, these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy; appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated
  • Must not have any underlying conditions which would contra-indicate apheresis
  • Must not be pregnant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteMyelodysplastic Syndromes

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Diseases

Intervention Hierarchy (Ancestors)

Aza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Peter Westervelt, M.D., Ph.D.

    Washington University School of Medicine

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 23, 2011

First Posted

July 11, 2011

Study Start

April 1, 2012

Primary Completion

March 1, 2014

Study Completion

April 1, 2015

Last Updated

April 27, 2015

Record last verified: 2015-04

Locations