NCT00553202|CompletedPhase 2ResultsDMC

Donor Stem Cell Transplant in Treating Young Patients With Acute Myeloid Leukemia With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory or Relapsed Acute Myelogenous Leukemia

Killer Immunoglobulin-like Receptor (KIR) Incompatible Unrelated Donor Hematopoietic Cell Transplantation (SCT) for AML With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory and Relapsed Acute Myelogenous Leukemia (AML) in Children: A Children's Oncology Group (COG) Study

Children's Oncology Group ClinicalTrials.gov

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3 other identifiers

AAML05P1COG-AAML05P1NCI-2009-00321

Study Type

interventional

Target

158

Locations

2 countries

Sites

Timeline

RegisteredNov 2007

StartedJan 2008

CompletionMar 2020

Brief Summary

RATIONALE: Giving chemotherapy before a donor stem cell transplant using stem cells that closely match the patient's stem cells, helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving antithymocyte globulin before transplant and cyclosporine, tacrolimus, and methotrexate before and after transplant may stop this from happening. PURPOSE: Natural Killer (NK) cells from the donor's bone marrow may be important in fighting leukemia. Bone marrow donors can be selected based on the type of NK cells they have, specifically the killer immunoglobulin receptor (KIR) type. This study provides information on KIR type from potential donors, which can be used in selecting the bone marrow donor. This phase II trial of unrelated donor stem cell transplant in patients with high risk AML (monosomy 7, -5/5q-, high FLT3-ITD AR, or refractory or relapsed AML) in which KIR typing of the patients and potential donors will be available to the treating transplant physician at the time of donor selection.

Trial Health

On Track

Trial Health Score

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

Enrollment

158

participants targeted

Target at P75+ for phase_2 leukemia

Timeline

Completed

Started Jan 2008

Longer than P75 for phase_2 leukemia

Geographic Reach

2 countries

52 active sites

Status

completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

12.3 years study duration

First Submitted

Initial submission to the registry

November 2, 2007

Completed

3 days until next milestone

First Posted

Study publicly available on registry

November 5, 2007

Completed

2 months until next milestone

Study Start

First participant enrolled

January 1, 2008

Completed

8.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2016

Completed

9 months until next milestone

Results Posted

Study results publicly available

February 24, 2017

Completed

3.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2020

Completed

Last Updated

April 16, 2020

Status Verified

July 1, 2016

Enrollment Period

8.4 years

First QC Date

November 2, 2007

Results QC Date

October 31, 2016

Last Update Submit

April 7, 2020

Conditions

Leukemia

Keywords

recurrent childhood acute myeloid leukemiachildhood myelodysplastic syndromes

Outcome Measures

Primary Outcomes (2)

Overall Survival (OS)
OS - Time from HSCT until death
At 5 years from HSCT date
Cumulative Incidence of NK Cell Reconstitution
Cumulative incidence of successful reconstitution to donor level is calculated.
At 5 years from HSCT date

Other Outcomes (3)

Disease-free Survival
From the date of SCT to the date of relapse, the date of death, or the date of last follow-up, whichever occurs first
Acute and Chronic Graft-versus-host Disease
Up to 5 years
Time to the Donor-specific NK-cell Receptor Expression
Up to 42 days after SCT

Study Arms (1)

Treatment (chemotherapy and allogeneic SCT)

EXPERIMENTAL

Patients receive busulfan IV every 6 hours on days -9 to -6, high-dose cyclophosphamide IV over 1 hour on days -5 to -2, anti-thymocyte globulin IV once or twice daily over 4 hours on days -3 to -1, and methylprednisolone IV on days -3 to -1. Patients undergo allogeneic hematopoietic stem cell transplantation (SCT) or allogeneic bone marrow transplantation (BMT) on day 0. Patients receive cyclosporine or tacrolimus IV or orally beginning on day -2 and continuing until day 50, followed by a taper until week 24. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Blood samples will be collected periodically from both patients and donors for studies of natural killer cells in support of the pharmacological study objectives

Biological: anti-thymocyte globulinDrug: busulfanDrug: cyclophosphamideDrug: cyclosporineDrug: methotrexateDrug: methylprednisoloneDrug: tacrolimusOther: laboratory biomarker analysisOther: pharmacological studyProcedure: allogeneic bone marrow transplantationProcedure: allogeneic hematopoietic stem cell transplantation