Clinical trial NCT02182986: Biomarkers for Post-Transplant Lymphoproliferative Disorders in Children
Official title: Biomarkers for Post-Transplant Lymphoproliferative Disorders in Children (CTOTC-06)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Heart Transplant
Small Intestine Transplant
Kidney Transplant
Liver Transplant
EBV-Related PTLD
PTLDs
Keywords: Biomarkers
Post-Transplant Lymphoproliferative Disorders (PTLDs)
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Sponsor class: NIH
Collaborators: name: Clinical Trials in Organ Transplantation in Children
First posted: 2014-07-08
Enrollment: 944
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric Candidates for or recipients of heart, liver, heart with liver, kidney, small intestine, or liver with small intestine at participating major pediatric solid organ transplant programs

Brief summary: Solid organ transplantation is an important therapeutic option for children with a variety of end stage diseases. However, the same immunosuppressive medications that are required to prevent the child's immune system from attacking and rejecting the transplanted organ can predispose these individuals to developing a very serious cancer that is linked to Epstein-Barr virus (EBV).

Detailed description: EBV-associated post-transplant lymphoproliferative disease (PTLD) is the most common malignancy in children after transplant. Diagnosis and effective treatment of the EBV-associated cancer is hampered by our inability to determine which children are at risk of developing these cancers and to detect the cancer at an early stage. In this study, we plan to test new "biomarkers" in the blood of children that will tell us very early on if the child is at risk of developing the EBV-associated cancer or if the cancer is present. These studies provide new opportunities for detection, diagnosis, and treatment of children with EBV-associated, post-transplant cancer.

Eligibility criteria: Inclusion Criteria:

* Subject and/or parent or legal guardian must be able to understand and provide informed consent/assent;
* Candidate for or recipient of: heart, liver, heart with liver, small intestine, liver with small intestine, or kidney; and
* Subject enrolled within 3 years of transplant.

Exclusion Criteria:

* Previous diagnosis of PTLD;
* Transplant recipients of lung alone, or in combination with an eligible organ type;
* Pancreas transplantation with the exception of 'en bloc' transplant in combined liver and small intestine multivisceral transplantation;
* Any combination other than listed in inclusion criteria;
* History of any previous solid organ, stem cell, or bone marrow transplantation;
* Inability or unwillingness of the legal guardian and/or the subject to comply with the study protocol.

Primary outcomes:
measure: Incidence of Epstein-Barr Virus (EBV) Positive Post-Transplant Lymphoproliferative Disorders (PTLD)
timeFrame: Receipt of transplanted organ(s) to confirmation of EBV-positive PTLD, up to year 4 post - enrollment
measure: Specified Gain-of-Function Mutations in EBV Latent Membrane Protein 1 (LMP-1)
timeFrame: Receipt of transplanted organ(s) to confirmation of mutations in EBV LMP1 , up to year 4 post - enrollment
measure: Pathogenic Changes in B Cell Clonotype Development
timeFrame: Receipt of transplanted organ(s) to confirmation of changes in B cell clonotype development, up to year 4 post - enrollment
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: Stanford
country: United States
facility: Lucile Packard Children's Hospital Stanford
state: California
city: Washington
country: United States
facility: Medstar Georgetown Transplant Institute
state: District of Columbia
city: Miami
country: United States
facility: University of Miami Health System
state: Florida
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern
state: Texas
Study officials:
affiliation: Stanford University
name: Carlos Esquivel, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Stanford University
name: Daniel Bernstein, M.D.
role: STUDY_CHAIR
References: