Predictive Biomarkers For Pediatric Chronic Graft-Versus-Host Disease

Active, not recruiting

• Conditions: Chronic Graft vs Host Disease

• Registration Number: NCT02067832
• Lead Sponsor: University of British Columbia

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-11
• Study First Submitted: 2014-01-20
• Study First Submitted QC: 2014-02-17
• Study First Posted: 2014-02-20
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-02
• Last Update Posted: 2023-12-05
• Primary Completion: 2024-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 302

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.

Read More

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.

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Identification of predictive bio-markers for pediatric chronic Graft-Versus-Host Disease (cGVHD) in Hematopoietic Stem Cell Transplant (HSCT) recipients	Just before transplant to 12 months post transplant or until diagnosis of cGVHD if precede the 12 months	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

Secondary Outcome Measures

Name	Time	Method
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)	To determine and validate whether "predictive" cGVHD bio-markers present before the onset of cGVHD are able to predict a subset of pediatric patients at greatest risk for the development of cGVHD in the future

Trial Locations

Locations (25)

Loma Linda University Medical Center; 🇺🇸 Loma Linda, California, United States

Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

UCSF Benioff Children's Hospital; 🇺🇸 San Francisco, California, United States

Vanderbilt-Ingram Cancer Center; 🇺🇸 Nashville, Tennessee, United States

Utah Primary Children's Medical Center; 🇺🇸 Salt Lake City, Utah, United States

Children's of Alabama; 🇺🇸 Birmingham, Alabama, United States

Ann and Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

All Children's Hospital; 🇺🇸 Saint Petersburg, Florida, United States

Blair E. Batson Hospital for Children; 🇺🇸 Jackson, Mississippi, United States

Nemours Children's Clinic; 🇺🇸 Jacksonville, Florida, United States

C S Mott Children's Hospital The University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Children's Hospital of Michigan; 🇺🇸 Detroit, Michigan, United States

City of Hope National Medical Center; 🇺🇸 Duarte, California, United States

Nemours A. I. DuPont Hospital for Children; 🇺🇸 Wilmington, Delaware, United States

Children's National Medical Center; 🇺🇸 Washington, District of Columbia, United States

Morgan Stanley Children's Hospital; 🇺🇸 New York, New York, United States

ST.Anna Children's Hospital; 🇦🇹 Vienna, Austria

Montreal Children's Hospital; 🇨🇦 Montreal, Quebec, Canada

The Sainte-Justine University Hospital Centre; 🇨🇦 Montreal, Quebec, Canada

New York Medical College; 🇺🇸 Valhalla, New York, United States

University of Manitoba; 🇨🇦 Winnipeg, Manitoba, Canada

University of Nebraska Medical Center; 🇺🇸 Omaha, Nebraska, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

Alberta Children's Hospital; 🇨🇦 Calgary, Alberta, Canada

University of British Columbia - BC Children's Hospital; 🇨🇦 Vancouver, British Columbia, Canada