Predictive Biomarkers For Pediatric Chronic Graft-Versus-Host Disease

NCT ID: NCT02067832

Last Updated: 2023-12-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

302 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-11-30

Study Completion Date

2025-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Chronic Graft vs Host Disease

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

1. Allogeneic hematopoietic stem cell transplantation for any malignant or non-malignant disease.
2. Age 0-17.99 years at the time of transplantation.
3. Bone marrow, peripheral blood stem cell and umbilical cord blood (including single or double cord blood) as the graft source.
4. Any conditioning regimen with any chemotherapy / radiation therapy combination. Haploidentical donor transplants with post-transplant cyclophosphamide are also allowed.
5. Use of serotherapy is permitted.
6. Any graft-versus-host disease prophylaxis is permitted, including post-HSCT cyclophosphamide.
7. If participant weighs between 0-20 kg, participant must be able to provide 15 ml of whole blood at each time point.
8. 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.
9. Written informed consent from parents.
10. Assent from study participant when appropriate.
11. Participation on other clinical trials is acceptable.

Exclusion Criteria

1. Autologous HSCT.
2. 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.
3. Ex-vivo T-cell depletion of graft source (e.g. CD34 selection).
4. Second (or greater) allogeneic transplants (first allogeneic transplant where a previous autologous transplant was performed is permitted).
5. Syngeneic transplants.
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University of British Columbia

OTHER

Sponsor Role lead

Responsible Party

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Kirk Schultz

Principle Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Geoff Cuvelier, MD

Role: PRINCIPAL_INVESTIGATOR

University of Manitoba

Kirk R Schultz, MD

Role: PRINCIPAL_INVESTIGATOR

University of British Columbia

Locations

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Children's of Alabama

Birmingham, Alabama, United States

Site Status

City of Hope National Medical Center

Duarte, California, United States

Site Status

Loma Linda University Medical Center

Loma Linda, California, United States

Site Status

UCSF Benioff Children's Hospital

San Francisco, California, United States

Site Status

Nemours A. I. DuPont Hospital for Children

Wilmington, Delaware, United States

Site Status

Children's National Medical Center

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

Site Status

Nemours Children's Clinic

Jacksonville, Florida, United States

Site Status

All Children's Hospital

St. Petersburg, Florida, United States

Site Status

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

Chicago, Illinois, United States

Site Status

C S Mott Children's Hospital The University of Michigan

Ann Arbor, Michigan, United States

Site Status

Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status

Blair E. Batson Hospital for Children

Jackson, Mississippi, United States

Site Status

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Morgan Stanley Children's Hospital

New York, New York, United States

Site Status

New York Medical College

Valhalla, New York, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States

Site Status

Utah Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status

ST.Anna Children's Hospital

Vienna, , Austria

Site Status

Alberta Children's Hospital

Calgary, Alberta, Canada

Site Status

University of British Columbia - BC Children's Hospital

Vancouver, British Columbia, Canada

Site Status

University of Manitoba

Winnipeg, Manitoba, Canada

Site Status

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Montreal Children's Hospital

Montreal, Quebec, Canada

Site Status

The Sainte-Justine University Hospital Centre

Montreal, Quebec, Canada

Site Status

Countries

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United States Austria Canada

References

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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.

Reference Type DERIVED
PMID: 36219586 (View on PubMed)

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.

Reference Type DERIVED
PMID: 34534280 (View on PubMed)

Other Identifiers

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TCF-118695

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

H12-02890

Identifier Type: -

Identifier Source: org_study_id