MRD Testing Before and After Hematopoietic Cell Transplantation for Pediatric Acute Myeloid Leukemia
The Role of Minimal Residual Disease Testing Before and After Hematopoietic Cell Transplantation for Pediatric Acute Myeloid Leukemia
1 other identifier
observational
150
2 countries
38
Brief Summary
This is a non-therapeutic study. Pediatric AML patients undergoing HCT with a myeloablative preparative regimen may be enrolled. Subjects can be enrolled 10-40 days prior to HCT. Three samples for MRD (measured by WT1 PCR and flow cytometry) will be collected from peripheral blood and bone marrow: 1) pre-HCT (\<3 weeks prior to starting the preparative regimen), 2) day 42 +/- 14 days post HCT (early post-engraftment), and 3) day 100 (+/-20 days) post HCT. For two years after transplant, the subject's follow-up data will be collected using the Research Level Forms in the CIBMTR Forms Net internet data entry system. The main objective is to determine whether there is any association between level of pre-transplant and post-transplant bone marrow MRD using WT1 and flow cytometry with 2-year event-free-survival, and to estimate the strength of that association in terms of the predictive accuracy of MRD. The investigators hypothesize that measurable MRD at either time point will be associated with decreased 2-year event-free survival.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Oct 2011
Longer than P75 for all trials
38 active sites
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
First Submitted
Initial submission to the registry
June 28, 2011
CompletedFirst Posted
Study publicly available on registry
June 30, 2011
CompletedStudy Start
First participant enrolled
October 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2017
CompletedSeptember 7, 2017
September 1, 2017
5.6 years
June 28, 2011
September 5, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Two-year Event Free Survival (EFS)
Event-free survival is defined as the time from HCT to relapse, death, initiation of post-HCT therapy to treat AML relapse, loss to follow up or end of study whichever comes first.
2 years post-HCT
Secondary Outcomes (8)
Two-year overall survival (OS)
2 years post-HCT
Disease relapse at 2 years
2 years post-HCT
Occurrence of acute grade II-IV and grade III-IV GVHD by 200 days post-HCT
200 days post-HCT
Occurrence of chronic GVHD at 2 years post-HCT
2 years post-HCT
Time to neutrophil engraftment
42 days post-HCT
- +3 more secondary outcomes
Eligibility Criteria
Participating institutions (transplant centers)
You may qualify if:
- Subject or legal guardian to understand and voluntarily sign an informed consent.
- Age 0-21 at time of transplant.
- Karnofsky score ≥ 70% (age ≥ 16 years old), or Lansky score ≥ 70% (age\<16 years old).
- Patients with adequate physical function as measured by:
- Cardiac: Left ventricular ejection fraction at rest must be \> 40%, or shortening fraction \> 26%
- Hepatic: Bilirubin ≤ 2.5 mg/dL; and ALT, AST and Alkaline Phosphatase≤ 5 x ULN
- Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) \> 70 mL/min/1.73 m2.
- Pulmonary: DLCO, FEV1, FVC (diffusion capacity) \> 50% of predicted (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation \> 92% in room air.
- Acute myelogenous leukemia (AML) at the following stages:
- High risk first complete remission (CR1), defined as:
- Having preceding myelodysplasia (MDS) -or-
- Diagnostic high risk karyotypes: del (5q) -5, -7, abn (3q), t (6;9), abnormalities of 12, t (9:22), complex karyotype (≥3 abnormalities), the presence of a high FLT3 ITD-AR (\> 0.4) -or-
- Having \>15% bone marrow blasts after 1st cycle and/or \>5% after 2nd cycle before achieving CR -and-
- \<5% blasts in the bone marrow, with peripheral ANC\>500
- Intermediate risk first complete remission (CR1), defined as:
- +20 more criteria
You may not qualify if:
- Women who are pregnant (positive HCG) or breastfeeding.
- Evidence of HIV infection or HIV positive serology.
- Positive viral load (PCR) for Hepatitis B or C (negative serology, surface antigen, and core antibody may substitute for PCR).
- Current uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms).
- Autologous transplant \< 12 months prior to enrollment.
- Prior allogeneic hematopoietic stem cell transplant.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (38)
The Children's Hospital of Alabama, University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Loma Linda University
Loma Linda, California, 92354, United States
University of California San Francisco
San Francisco, California, 94143, United States
The Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20910, United States
Miami Children's Hospital
Miami, Florida, 33155, United States
All Children's Hospital
St. Petersburg, Florida, 33701, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Riley Hospital for Children/Indiana University
Indianapolis, Indiana, 46202, United States
University of Louisville
Louisville, Kentucky, 40202, United States
Johns Hopkins
Baltimore, Maryland, 21287, United States
Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
Washington University, St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Roswell Park Cancer Institute
Buffalo, New York, 14263, United States
Mount Sinai School of Medicine
New York, New York, 10029, United States
Columbia University - The Morgan Stanley Children's Hospital of New York
New York, New York, 10032, United States
New York Medical College
Valhalla, New York, 10595, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
University Hospitals of Cleveland Case Medical Ctr
Cleveland, Ohio, 44106, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
Oregon Health & Sciences University - Doerbecher Children's
Portland, Oregon, 97239, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Methodist Children's Hospital of South Texas/Texas Institute of Medicine and Surgery
San Antonio, Texas, 78229, United States
University of Utah - Primary Children's Medical Center
Salt Lake City, Utah, 84108, United States
Virginia Commonwealth University
Richmond, Virginia, 23219, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
Children's & Women's Health Centre of British Columbia
Vancouver, British Columbia, V6T 1Z3, Canada
The Montreal Children's Hospital
Montreal, Quebec, H3H 1P3, Canada
Hopital Ste. Justine
Montreal, Quebec, H3T 1C5, Canada
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
David A. Jacobsohn, MD, ScM
Children's National Research Institute
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 28, 2011
First Posted
June 30, 2011
Study Start
October 1, 2011
Primary Completion
May 1, 2017
Study Completion
May 1, 2017
Last Updated
September 7, 2017
Record last verified: 2017-09