Pharmacogenomic Testing in Pediatric Hematology/Oncology Patients

NCT06744712 | Not Yet Recruiting DMC

• 3 other identifiers: IRB00104368LCI-PED-NDPO-PGx-001NCI-2025-00567
• Study Type: interventional
• Target: 130
• Locations: 1 country
• Sites: 1

Brief Summary

Pharmacogenomic (PGx) testing involves analyzing variants of genes associated with drug metabolism, transport and medication targets. PGx testing uses an individual's genetic factors, such as single nucleotide polymorphisms (SNPs), to personalize therapy or dose a selection of medications. PGx testing has traditionally been used to test single genes, but there are now platforms allowing a panel of genes to be tested at once. To date there has not been a comprehensive screening of pediatric oncology patients to determine the prevalence of genetic variants that may affect anticancer therapy and supportive care medications. This study would allow us to summarize the frequency of clinically relevant gene-drug interactions and actionable genetic polymorphisms in pediatric oncology patients.

Conditions

Pediatric Cancer

Keywords

Pharmacogenomic; Anti-Cancer Therapy

Outcome Measures

Primary Outcomes (2)

• Gene-drug interaction

  A gene-drug interaction occurs when an individual carrying a variant form of a gene is administered a drug that serves as a substrate for the enzyme or transporter encoded by that gene. Clinically relevant gene-drug interactions are defined in the CPIC (Clinical Pharmacogenetics Implementation Consortium) guidelines or the FDA Table of Pharmacogenomic Associations. Gene-drug interaction is a binary variable (interaction present or interaction absent). Each clinically relevant gene-drug interaction from the pharmacogenomic testing will be reported.

  3, 6, 9, 12 months after enrollment

• Genotype

  Genes involved in toxicity and efficacy of pediatric anticancer therapy and supportive care medications, as listed in CPIC or FDA Table of Pharmacogenomic Association guidelines, are tested for genetic polymorphisms using the OneOme RightMed Comprehensive Test. Each gene's genotype is classified as a binary variable (actionable or not actionable).

  Baseline

Secondary Outcomes (2)

• Anticancer treatment modification based on PGx test result

  3, 6, 9, 12 months after enrollment

• Modification in supportive medication based on PGx test result

  3, 6, 9, 12 months after enrollment

Study Arms (1)

Pharmacogenomic (PGx) Testing

EXPERIMENTAL

Other: Pharmacogenomic Testing

Interventions

Pharmacogenomic Testing OTHER

Pharmacogenomic testing will be completed by the vendor. The vendor is certified under CLIA-88 and accredited by the College of American Pathologists as qualified to perform high-complexity testing. Genomic DNA extracted from the swab will be analyzed by PCR using Thermo Fisher TaqMan® and/or LGC Biosearch BHQ® probe-based methods to interrogate the variant locations listed in (See Appendix II). Twenty-seven genes are evaluated and reported.

Pharmacogenomic (PGx) Testing

Eligibility Criteria

• Age: Up to 26 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Written informed consent and HIPAA authorization for release of personal health information, and assent when applicable, from the participant, parent or legal guardian.
• Age ≤ 26 years at the time of consent.
• Newly diagnosed with a malignancy and planning to undergo anti-cancer therapy; or bone marrow transplant candidate with a non-malignant diagnosis who has not yet undergone myeloablative conditioning regimen.

You may not qualify if:

• Anti-cancer therapy has already been initiated. Note: Enrollment after initiation of intrathecal chemotherapy will be allowed.
• Previously received bone marrow transplant or planning to receive as part of initial upfront therapy for a malignant condition.
• Prior history of tissue or organ transplant.

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead
• Atrium Health Levine Cancer Institute: collaborator
• OneOme, LLC: collaborator

Study Sites (1)

Levine Childrens Hospital Pediatric Cancer and Blood Disorders

Charlotte, North Carolina, 28203, United States

Location

MeSH Terms

Conditions

Neoplasms

Interventions

Pharmacogenomic Testing

Intervention Hierarchy (Ancestors)

Genetic Testing; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Genetic Techniques; Genetic Services; Health Services; Health Care Facilities Workforce and Services; Diagnostic Services; Preventive Health Services

Study Officials

• Erin Trovillion, MD

  Wake Forest University Health Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Meg Lattanze

CONTACT

980-349-6887; megan.lattanze@advocatehealth.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 27, 2024
• First Posted: December 20, 2024
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): July 1, 2028
• Last Updated: March 25, 2026

Record last verified: 2026-03

Locations

US (1)