Pharmacogenomic Testing for Pediatric Cancer

Overseen By Meg Lattanze

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Wake Forest University Health Sciences

Disqualifiers: Prior cancer therapy, Organ transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how pharmacogenomic testing (genetic testing for drug response) can personalize cancer treatment for children and young adults. By analyzing specific genes, researchers aim to understand how different genetic profiles affect the effectiveness and safety of cancer medications. The goal is to identify gene-drug interactions that could tailor treatments to each patient's unique genetic makeup. This trial includes individuals 26 years or younger who have just been diagnosed with cancer and are about to start treatment, or who are candidates for a bone marrow transplant without prior conditioning. As an unphased trial, this study offers a unique opportunity to contribute to groundbreaking research that could revolutionize personalized cancer treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the trial coordinators or your doctor.

What prior data suggests that pharmacogenomic testing is safe for pediatric cancer patients?

Research has shown that pharmacogenomic (PGx) testing is safe and can enhance drug safety for children. In one study involving children with cancer, PGx testing tailored treatments to their individual genetic profiles. This approach predicts how well a child might respond to certain medications, potentially reducing the risk of harmful side effects.

Other studies have found that PGx testing can identify genetic differences affecting how children process cancer treatments. For instance, one review noted that PGx testing could help avoid harmful side effects from chemotherapy.

Overall, PGx testing is generally well-tolerated and can make cancer treatments safer for children by customizing medications to their unique genetic makeup. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about pharmacogenomic testing for pediatric cancer because it offers a personalized approach to treatment. Unlike standard treatments like chemotherapy and radiation, which can affect healthy cells as well as cancer cells, pharmacogenomic testing uses a patient’s genetic information to tailor therapies specifically to their tumor's unique characteristics. This precision means potentially fewer side effects and more effective targeting of cancer cells. By understanding how a child's genes affect drug response, doctors can choose the most effective medications right from the start, which could lead to better outcomes and improved quality of life for young patients.

What evidence suggests that pharmacogenomic testing is effective for pediatric cancer?

Research has shown that genetic testing, such as the Pharmacogenomic (PGx) Testing studied in this trial, can make medications safer and more effective for children with cancer. This testing identifies gene differences that affect how a child processes medicine. One study found that 16% of children with cancer could benefit from this testing by avoiding harmful side effects or improving medication efficacy. Genetic testing customizes treatments for each child, ensuring they receive the right medicine at the right dose. This approach aims to enhance the safety and effectiveness of cancer treatment for children. ^{1 3 4 6 7}

Who Is on the Research Team?

Erin Trovillion, MD

Principal Investigator

Wake Forest University Health Sciences

Are You a Good Fit for This Trial?

This trial is for children with cancer. It's looking to see how often certain genetic variations occur in these young patients, which might affect their response to cancer treatment and supportive care medications.

Inclusion Criteria

Written informed consent and HIPAA authorization for release of personal health information, and assent when applicable, from the participant, parent or legal guardian

I am 26 years old or younger.

I am newly diagnosed with cancer and will start treatment soon, or I need a bone marrow transplant but haven't started the prep treatment.

Exclusion Criteria

I have had or am planning to have a bone marrow transplant for cancer treatment.

I have had an organ or tissue transplant in the past.

I have started treatment for my cancer.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Enrollment and PGx Testing

Participants undergo buccal swab collection for PGx testing at the time of enrollment

Baseline

1 visit (in-person)

Monitoring and Dose Modification

Dose modifications to anticancer therapy and supportive care medications based on PGx results are collected every three months

12 months

Quarterly reviews

Follow-up

Participants are monitored for safety and effectiveness after PGx testing and treatment modifications

3 months

What Are the Treatments Tested in This Trial?

Interventions

• Pharmacogenomic Testing

Trial Overview The study tests a group of genes at once (pharmacogenomic testing) to find out if there are any genetic factors that could help doctors choose the best medication or dosage for each child based on their unique DNA.

How Is the Trial Designed?

Group I: Pharmacogenomic (PGx) Testing Experimental Treatment 1 Intervention

Pharmacogenomic Testing is already approved in United States, Canada, European Union for the following indications:

Approved in United States as Pharmacogenomic Testing for:

• Guiding medication selection and dosing based on genetic variations

Approved in Canada as Pharmacogenetic Testing for:

• Informing drug dosing and monitoring for efficacy and safety in children and pregnant women

Approved in European Union as Pharmacogenomic Testing for:

• Personalized medicine through genetic-guided drug therapy

Who Is Running the Clinical Trial?

Wake Forest University Health Sciences

Lead Sponsor

Trials

1,432

Recruited

2,506,000+

Atrium Health Levine Cancer Institute

Collaborator

Trials

30

Recruited

4,700+

OneOme, LLC

Industry Sponsor

Trials

3

Recruited

1,100+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9544158/

Pharmacogenomic testing in paediatrics - PubMed Central - NIH Implementing PGx testing in paediatric patients can enhance drug safety, helping to improve drug efficacy or reduce the risk of toxicity.

2. sciencedirect.com sciencedirect.com/science/article/pii/S1040842825001088

Pharmacogenomics in pediatric oncology patients with ... This review provides an overview of pharmacogenomic studies in relation to chemotherapy-induced toxicity in children with solid tumors.

3. frontiersin.org frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1568320/full

Clinical and cost-effectiveness of pharmacogenomic ... This systematic review and meta-analysis evaluates the effectiveness of pharmacogenomic testing for ACT in childhood cancer.

4. ascpt.onlinelibrary.wiley.com ascpt.onlinelibrary.wiley.com/doi/full/10.1002/cpt.3685

Implementing Pre‐Emptive Pharmacogenetics: Impact of Early ... In a pediatric oncology cohort of 1,151 subjects, 16% (n = 184) could have benefited from pre-emptive pharmacogenetic screening, and 227 first- ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06744712?term=cancer%20pharmacogenomic&viewType=Table&rank=1

Pharmacogenomic Testing in Pediatric Hematology ... Pharmacogenomic (PGx) testing involves analyzing variants of genes associated with drug metabolism, transport and medication targets. PGx testing uses an ...

6. cancer.org cancer.org/cancer/managing-cancer/making-treatment-decisions/pharmacogenomic-testing.html

Pharmacogenomics in Cancer Treatment Pharmacogenomic or PGx testing looks at specific genes in a person's DNA to help predict how well a treatment might work for them.

7. nature.com nature.com/articles/s41397-024-00326-1

A review of real-world evidence on preemptive ... - Nature The study findings indicate that PGx testing has yielded significant outcomes in the Vanderbilt PREDICT program and related initiatives. Among ...