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AzTrials

Acute Myelogenous LeukemiaPhase 2NCT02458235

Donor Lymphocyte Infusion With Azacitidine to Prevent Hematologic Malignancy Relapse After Stem Cell Transplantation

A Phase 2 study testing azacitidine and donor lymphocyte infusion in people with Acute Myelogenous Leukemia and Acute Lymphoid Leukemia, run by University of California, San Francisco at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The goal of this study is to determine whether post-transplant consolidation with azacitidine combined with donor lymphocyte infusion (DLI) is a safe and effective approach for the prevention of relapse in pediatric and young adult patients with hematologic malignancies who have undergone hematopoietic stem cell… Read more about this study

Who is it for?
Ages Up to 29 years
What is being tested?
azacitidine and donor lymphocyte infusion
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
1 siteUnited States
Study dates
2 Jun 2015to 15 Mar 2019
Phase
Phase 2
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 Up to 29 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients age 0 - 29.9 years undergoing allogeneic peripheral blood stem cell transplant
  • Patients with acute myelogenous leukemia (AML) or acute lymphoblastic leukemia (ALL)
  • Patients with juvenile myelomonocytic leukemia (JMML)
  • Patients with myelodysplastic syndrome (MDS)

Who cannot join

  • Patients who have had a prior transplant.
  • Patients with Fanconi anemia or other cancer-predisposition syndromes
  • Patients with expected survival <12 weeks
  • Lansky score <60%

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of California San Francisco

    San Francisco, California, 94143

Other trials in United States

About this study

Official title: A Phase II Study of Risk-adapted Donor Lymphocyte Infusion and Azacitidine for the Prevention of Hematologic Malignancy Relapse Following Allogeneic Stem Cell Transplantation

The goal of this study is to determine whether post-transplant consolidation with azacitidine combined with donor lymphocyte infusion (DLI) is a safe and effective approach for the prevention of relapse in pediatric and young adult patients with hematologic malignancies who have undergone hematopoietic stem cell transplantation (HSCT).

Read the detailed description

This is a phase II single-arm trial of azacitidine (IV or SC) in combination with escalating donor lymphocyte infusion (DLI). Patients will be enrolled on the study by day +28 +/- 7 post-transplant, prior to withdrawal of immunosuppression or administration of donor lymphocyte infusion (DLI). They will have donor chimerism and minimal residual disease (MRD) testing from peripheral blood (PB) and bone marrow (BM) on day +28 ± 7. Patients will be stratified according to risk categories (low, standard and high), defined by GVHD status, mixed versus full donor chimerism, and positive versus negative MRD results. Depending on risk assessment, immunosuppression will be tapered according to standard or fast schedules, and patients (with the exception of low-risk ALL patients) will receive one cycle of low-dose azacitidine (40mg/m2 IV/SC daily x 4 days). After tapering immunosuppression, chimerism will be repeated and patients will receive up to 6 additional cycles of low-dose azacitidine, depending on risk assessment. For patients who meet criteria for high risk of relapse, azacitidine will be combined with escalating doses of DLI for a maximum of 7 cycles in total. Risk and safety assessments, including routine laboratory parameters, donor chimerism, minimal residual disease, and GHVD activity will be assessed following each cycle. Chimerism and minimal residual disease testing will be repeated every cycle by peripheral blood (PB), and bone marrow (BM) will be tested every other cycle. Patients will be followed by laboratory monitoring and physician evaluation prior to each cycle, and will be followed for two years post-transplant to study toxicity and GVHD outcomes.

Being tested
azacitidine, donor lymphocyte infusion
Keywords
pediatric leukemia myelodysplastic syndrome azacitidine
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelomonocytic, Juvenile, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • azacitidineDrug

    Also called Vidaza®, Ladakamycin

    40mg/m2 IV/SC daily x 4 days, maximum of 7 cycles at 6 weekly intervals

  • donor lymphocyte infusionBiological

    For patients with cells available for DLI who are in the high risk group and do not have graft-versus-host disease (GVHD), DLI will be adminstered on day 5 of each cycle.

Study groups in NCT02458235
Study groupRoleWhat they receive
Azacitidine/donor lymphocyte infusionExperimentalPatients will be stratified according to risk categories (low, standard and high), defined by GVHD status, mixed versus full donor chimerism, and positive versus negative Minimal Residual Disease (MRD) results. Patients will receive up to 7 cycles of low-dose azacitidine (40mg/m2 IV/SC daily x 4 days) at 6 weekly intervals, except for low risk ALL patients who may not receive treatment after withdrawal of immunosuppression. Standard risk patients will receive an additional 6 cycles of azacitidine alone. High risk patients will receive an additional 6 cycles of azacitidine plus escalating DLI.
Receives: Drug: azacitidine; Biological: donor lymphocyte infusion

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Relapse Rate

    Relapse rate will be estimated using a percentage of participants who relapsed. It is assumed that the rate of relapse in pediatric acute leukemia post-transplant would be 40%, azacitidine +/- Donor Lymphocyte Infusion (DLI) would reduce the 2-year relapse rate by approximately 40% to a rate of 25%.

    Measured: Up to 2 years

  2. Frequency of System Specific Grade 3 or Higher Treatment-related Adverse Events

    Frequency of system specific adverse events of interest include renal, hepatic, cardiac, pulmonary, or neurologic toxicities. Toxicities will be graded using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.

    Measured: Up to 2 years

  3. Proportion of Participants With Acute and Chronic Graft Versus Host Disease (GVHD)

    Proportion of participants with Grade 3-4 acute GVHD and moderate to severe chronic GVHD will be reported.

    Measured: Up to 2 years

  4. Proportion of Participants With Serious Infection

    The proportion of participants will be reported for Grade 3-4 invasive fungal infection or disease caused by viral infections

    Measured: Up to 2 years

  5. Proportion of Participants With Severe Hematologic Toxicity Including Graft Failure

    The proportion of participants will be reported for Grade 4 severe hematologic toxicities including graft failure

    Measured: Up to 2 years

  6. Number of Participants Whom Had >2 Dose Reductions for Any Reason

    The number of participants whom had greater than 2 dose reductions for any reason.

    Measured: Up to 2 years

Secondary outcomes (2)

  1. Median Relapse-free Survival

    Release-free survival rate is defined as the median length of time after beginning treatment that the participant survives without progression or relapse, reported in months

    Measured: Up to 2 years

  2. Median Time to Relapse

    Time to relapse is defined as the length of time after beginning treatment until the participant has experienced a relapse in disease, measured in months.

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Jun 2015 (estimated)
  2. Study start2 Jun 2015
  3. Primary completion15 Mar 2019
  4. Study completion15 Mar 2019

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
17 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02458235
Sponsor's protocol ID
140813
Other IDs
NCI-2015-02240
Status last verified
Oct 2020
First submitted
5 Jan 2015
Last updated
12 Oct 2020
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Justin T. Wahlstrom, Biljana N. Horn, Carol Fraser-Browne, Rebecca Hoeweler, Ying Lu, Alexis Melton, Jennifer Willert, Christopher C. Dvorak; Azacitidine Administration Following Hematopoietic Stem Cell Transplantation Is Safe and Feasible in Children with Acute Leukemia. Blood 2016; 128 (22): 4805. https://doi.org/10.1182/blood.V128.22.4805.4805

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing azacitidine and donor lymphocyte infusion in people with Acute Myelogenous Leukemia and Acute Lymphoid Leukemia, run by University of California, San Francisco 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 4 inclusion and 4 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: finished, as of the sponsor's last update on 12 Oct 2020. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 12 Oct 2020). 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?

Relapse Rate (time frame: Up to 2 years). The study lists 6 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 NCT02458235, 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 12 Oct 2020.

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