Predictive Biomarkers For Pediatric Chronic Graft-Versus-Host Disease

NCT02067832 | Unknown

• 2 other identifiers: H12-02890TCF-118695
• Study Type: observational
• Target: 302
• Locations: 3 countries
• Sites: 25

Brief Summary

Chronic graft-versus-host disease (cGVHD) can be hard to diagnose, difficult to manage and contributes significantly to morbidity and mortality in hematopoietic stem cell transplantation patients. The research will look into identifying and validating cGVHD biological indicators (=bio-markers) which will be evaluated whether they can predict a future development of the disease. The study hypothesis is that a number of previously reported cGVHD bio-markers, known to be present at the time of cGVHD diagnosis, will also be present at earlier time points, before cGVHD develops. Following validation, the bio-markers will be beneficial for finding those patients who are in higher risk to develop cGVHD. By identifying the higher-risk group, which is more likely to develop cGVHD, a pre-emptive therapy might be applied in order to prevent or reduce the prevalence of the disease.

Conditions

Chronic Graft vs Host Disease

Outcome Measures

Primary Outcomes (1)

• Identification of predictive bio-markers for pediatric chronic Graft-Versus-Host Disease (cGVHD) in Hematopoietic Stem Cell Transplant (HSCT) recipients

  The study will try to determine the prevalence (or levels) of high-probability predictive plasma and cellular cGVHD bio-markers in pediatric patients undergoing allogeneic HSCT from blood samples

  Just before transplant to 12 months post transplant or until diagnosis of cGVHD if precede the 12 months

Secondary Outcomes (1)

• Validation of "predictive" cGVHD bio-markers

  Measure will be assessed following the submission of all samples. During the last year of the study (Oct. 2016 - Sept. 2017)

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Recipients and donors of allogeneic hematopoietic stem cell transplantation

You may qualify if:

• Allogeneic hematopoietic stem cell transplantation for any malignant or non-malignant disease.
• Age 0-17.99 years at the time of transplantation.
• Bone marrow, peripheral blood stem cell and umbilical cord blood (including single or double cord blood) as the graft source.
• Any conditioning regimen with any chemotherapy / radiation therapy combination. Haploidentical donor transplants with post-transplant cyclophosphamide are also allowed.
• Use of serotherapy is permitted.
• Any graft-versus-host disease prophylaxis is permitted, including post-HSCT cyclophosphamide.
• If participant weighs between 0-20 kg, participant must be able to provide 15 ml of whole blood at each time point.
• If participant weighs over 20 kg, participant must be able to provide 1ml/kg of whole blood, up to a maximum of 23 mL for the pre-conditioning sample and 32 mL for samples at day +100, 6-months, 12-months, +/- the cGVHD sample.
• Written informed consent from parents.
• Assent from study participant when appropriate.
• Participation on other clinical trials is acceptable.

You may not qualify if:

• Autologous HSCT.
• Patients referred to a Bone Marrow Transplant (BMT) center from a non-BMT center, where it is anticipated (at the discretion of the center PI) that adequate follow up according to the rules of this protocol can not be met, including the requirement for a reassessment by the BMT center at the time of cGVHD diagnosis.
• Ex-vivo T-cell depletion of graft source (e.g. CD34 selection).
• Second (or greater) allogeneic transplants (first allogeneic transplant where a previous autologous transplant was performed is permitted).
• Syngeneic transplants.

Sponsors & Collaborators

• University of British Columbia: lead

Study Sites (25)

Children's of Alabama

Birmingham, Alabama, 35233, United States

Location

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

Loma Linda University Medical Center

Loma Linda, California, 92354, United States

Location

UCSF Benioff Children's Hospital

San Francisco, California, 94143, United States

Location

Nemours A. I. DuPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Nemours Children's Clinic

Jacksonville, Florida, 32207, United States

Location

All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611-2605, United States

Location

C S Mott Children's Hospital The University of Michigan

Ann Arbor, Michigan, 48109-1274, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Blair E. Batson Hospital for Children

Jackson, Mississippi, 39216, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198-7140, United States

Location

Morgan Stanley Children's Hospital

New York, New York, 10032, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States

Location

Utah Primary Children's Medical Center

Salt Lake City, Utah, 84132, United States

Location

ST.Anna Children's Hospital

Vienna, Austria

Location

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

Location

University of British Columbia - BC Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

University of Manitoba

Winnipeg, Manitoba, R3T 2N2, Canada

Location

Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Montreal Children's Hospital

Montreal, Quebec, H3H 1P3, Canada

Location

The Sainte-Justine University Hospital Centre

Montreal, Quebec, H3T 1C5, Canada

Location

Related Publications (2)

• Cuvelier GDE, Ng B, Abdossamadi S, Nemecek ER, Melton A, Kitko CL, Lewis VA, Schechter T, Jacobsohn DA, Harris AC, Pulsipher MA, Bittencourt H, Choi SW, Caywood EH, Kasow KA, Bhatia M, Oshrine BR, Chaudhury S, Coulter D, Chewning JH, Joyce M, Savasan S, Pawlowska AB, Megason GC, Mitchell D, Cheerva AC, Lawitschka A, Ostroumov E, Schultz KR. A diagnostic classifier for pediatric chronic graft-versus-host disease: results of the ABLE/PBMTC 1202 study. Blood Adv. 2023 Jul 25;7(14):3612-3623. doi: 10.1182/bloodadvances.2022007715.

  PMID: 36219586 DERIVED

• Subburaj D, Ng B, Kariminia A, Abdossamadi S, Lauener M, Nemecek ER, Rozmus J, Kharbanda S, Kitko CL, Lewis VA, Schechter-Finklestein T, Jacobsohn DA, Harris AC, Pulsipher MA, Bittencourt H, Choi SW, Caywood EH, Kasow KA, Bhatia M, Oshrine BR, Coulter D, Chewning JH, Joyce M, Pawlowska AB, Megason GC, Lawitschka A, Ostroumov E, Klein Geltink R, Cuvelier GDE, Schultz KR. Metabolomic identification of alpha-ketoglutaric acid elevation in pediatric chronic graft-versus-host disease. Blood. 2022 Jan 13;139(2):287-299. doi: 10.1182/blood.2021013244.

  PMID: 34534280 DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

From recipient: whole blood From donor: bone marrow or apheresis product

Study Officials

• Geoff Cuvelier, MD

  University of Manitoba

  PRINCIPAL INVESTIGATOR

• Kirk R Schultz, MD

  University of British Columbia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: January 20, 2014
• First Posted: February 20, 2014
• Study Start: November 1, 2013
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2025
• Last Updated: December 5, 2023

Record last verified: 2023-12

Locations

CA (6); US (18); AU (1)