VIRTUUS Children's Study

NCT ID: NCT03719339

Last Updated: 2025-12-12

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

445 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-08-10

Study Completion Date

2026-03-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The objective of the VIRTUUS Children's Study is to adapt identified and validated adult noninvasive diagnostic and prognostic biomarkers for the characterization of allograft status in pediatric recipients of kidney allografts.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Advances in immunosuppressive regimens have significantly improved short-term allograft survival for kidney transplant recipients. Yet, long-term allograft survival remains static. For children with end-stage renal disease (ESRD), improvements in long-term outcomes are greatly needed. Children with ESRD require multiple transplants over a lifetime, incurring repeated surgical and immunological risks with each newly transplanted organ. Allograft injury occurs primarily due to acute cellular rejection (ACR) and/or antibody mediated rejection (AMR) and viral infections, such as BK virus associated nephropathy (BKVN). A major hindrance to promoting long-term allograft survival is the lack of non-invasive diagnostic and prognostic biomarkers to reliably detect early injury in the allograft before clinical manifestations arise.

The incidence of acute rejection (AR) in children in the first year post-transplant is 10-13%\[1\]. The current gold standard for diagnosing AR is core needle biopsy; however, biopsy is highly invasive, incurs risk of bleeding and graft loss, is subject to sampling error and lacks sensitivity and specificity for early injury. Since immune responses are dynamic over time, single biopsies do not adequately capture anti-allograft immunity, but repeat biopsies are impractical in children who often require sedation and hospitalization for biopsies. Other markers, such as serum creatinine, have low sensitivity and specificity for early kidney allograft damage.

The ability to identify sub-clinical kidney allograft injury using minimally invasive, robust, biomarkers with high sensitivity and specificity in pediatric recipients would represent a major advance in pediatric kidney transplant care. In the Clinical Trials in Organ Transplantation (CTOT)-04 study, a NIH-sponsored, multicenter, prospective study of adult kidney allograft recipients, members of the VIRTUUS team were able to diagnose and predict ACR using urinary cell mRNA and metabolite profiles with high sensitivity and specificity\[2, 3\]. In addition, the investigators validated a urinary cell mRNA signature that distinguishes acute rejection (AR) from acute tubular injury (ATI) and ACR from AMR as well as a urinary cell mRNA signature diagnostic and prognostic of BKVN\[3-5\]. The overarching objective of this VIRTUUS proposal is to adapt existing validated adult noninvasive diagnostic and prognostic biomarkers to characterize allograft status in pediatric recipients of kidney allografts. Specifically, the investigators will investigate 1) whether the adult urinary cell 3-gene signature is diagnostic and prognostic of ACR in pediatric recipients of kidney allografts, 2) whether the combined metabolite and the urinary cell 3-gene signature is diagnostic and prognostic of ACR in pediatric recipients of kidney allografts, 3) whether levels of BKV VP-1 mRNA in urinary cells are diagnostic of BKVN, and 4) whether urinary cell levels of plasminogen activator inhibitor -1 (PAI-1) mRNA and serum creatinine levels predict allograft failure.

Investigators propose to validate early immunologic markers that have shown to be prognostic and diagnostic in adult kidney transplant recipients in pediatric kidney transplant recipients. Investigator findings will significantly advance the field of pediatric transplantation by moving toward proactive, tailored immunosuppressive regimens that minimize morbidity and optimize long-term allograft survival.

The Investigator's primary objective is to hypothesize that: (i) the adult urinary cell 3-gene signature will be diagnostic and prognostic of ACR in longitudinally collected urine samples from children with kidney transplants; and (ii) combined metabolite and mRNA biomarkers have greater ability to diagnose ACR than the mRNA or metabolite signature alone and (iii) levels of BKV VP-1 mRNA are diagnostic of BK virus nephropathy (BKVN) and (iv) urinary cell levels of plasminogen activator inhibitor-1 (PAI-1) mRNA and serum creatinine levels predict allograft failure.

Investigators seek to:

1. Determine if the adult urinary cell 3-gene signature is diagnostic and prognostic of ACR in pediatric kidney allograft recipients,
2. Evaluate whether a combined metabolite and the 3-gene urinary mRNA signature is diagnostic and prognostic of ACR and
3. Test the hypothesis that BKV-VP-1 mRNA levels in urinary cells are diagnostic of BKVN, and to test the hypothesis that a two variable prediction model composed of urinary cell level of PAI-1 mRNA and serum creatinine levels, both measured at the time of BKVN biopsy diagnosis, predict future graft failure

Secondary objectives include the following:

1. Create a repository of DNA samples from urine, saliva, discarded blood and tissue, and deceased donor blood and tissue to use for future research studies that will examine genome-wide associations with rejection and viral infections in pediatric kidney transplant recipients and
2. Create a biobank of samples of left-over blood and deceased donor blood to later examine associations between urine and blood proteomics and metabolomics.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Renal Transplant Rejection Pediatric Kidney Disease End Stage Renal Disease Nephropathy BK Virus (Nephropathy) Antibody-mediated Rejection Acute Cellular Graft Rejection

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Males or females between 2 to 18 years at the time of recruitment
* Receiving the first, or additional, incident kidney transplants.
* Have an existing/prevalent transplant with a scheduled kidney allograft biopsy.
* Parental/guardian permission (informed consent) and, if appropriate, child assent.

Exclusion Criteria

• Patient's primary medical team feels the subject's participation is not safe.
Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Brendan Keating, DPhil

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia and Hospital of The University of Pennsylvania

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of California-San Diego, Rady Children's Hospital

La Jolla, California, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

University of California

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

Children's National Medical Center

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

Site Status

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

Chicago, Illinois, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Joan & Sanford I. Weill Medical College of Cornelle University

New York, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

CHOP

Philadelphia, Pennsylvania, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Vancouver Children's Hospital

Vancouver, British Columbia, Canada

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Canada

References

Explore related publications, articles, or registry entries linked to this study.

Ettenger RB, Seifert ME, Blydt-Hansen T, Briscoe DM, Holman J, Weng PL, Srivastava R, Fleming J, Malekzadeh M, Pearl M. Detection of Subclinical Rejection in Pediatric Kidney Transplantation: Current and Future Practices. Pediatr Transplant. 2024 Sep;28(6):e14836. doi: 10.1111/petr.14836.

Reference Type DERIVED
PMID: 39147695 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

1R01HD091185-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

17-013841

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

taVNS for FRNS in Children
NCT05588063 RECRUITING NA
The CDR Implementation Trial
NCT03162354 COMPLETED NA