Testing drug sensitivity and genetic profiling for pediatric cancer patients
Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments
This study is testing a new way to find the best cancer treatments for children by looking at how their tumor cells respond to different drugs and comparing those results to standard treatments.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 65 (estimated) |
| Ages | 1 Day to 21 Years |
| Sex | All |
| Sponsor | Florida International University Academic / other |
| Locations | 1 site (Miami, Florida) |
| Trial ID | NCT05857969 on ClinicalTrials.gov |
What this trial studies
This study focuses on providing personalized treatment options for pediatric cancer patients by utilizing Functional Precision Medicine (FPM), which combines ex vivo drug sensitivity testing and genomic profiling. The study aims to enroll children with recurrent or refractory cancers who have available tumor tissue for testing. By exposing isolated tumor cells to clinically approved drugs, the study seeks to determine the most effective individualized therapies. Additionally, it will compare patient outcomes between those treated with FPM-guided therapy and conventional therapy, while exploring the relationship between tumor characteristics and drug response across different ethnicities.
Who should consider this trial
Good fit: Ideal candidates include pediatric patients aged 21 years or younger with recurrent or refractory cancers who have available tumor tissue for testing.
Not a fit: Patients with newly diagnosed tumors or those with high cure rates using standard therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could lead to more effective and personalized treatment options for children with recurrent or refractory cancers.
How similar studies have performed: Other studies utilizing similar approaches of drug sensitivity testing and genomic profiling have shown promise, indicating potential for success in this novel application.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients aged 21 years or younger at the time of enrollment on this study of any gender, race or ethnicity. Subjects with suspected or confirmed diagnosis of recurrent or refractory cancer Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers) Subjects willing to have a blood draw or buccal swab done for the purposes of genetic testing Subjects or their parents or legal guardians willing to sign informed consent Subjects aged 7 to 17 willing to sign assent Exclusion Criteria: * Subjects who do not have malignant tissue available and accessible The amount of excised malignant tissue is not sufficient for the ex vivo drug testing and/or genetic profiling. Patients with newly diagnosed tumors and tumors that have high (\>90%) cure rate with safe standard therapy.
Where this trial is running
Miami, Florida
- Nicklaus Children's Hospital — Miami, Florida, United States (Recruiting)
Study contacts
- Principal investigator: Diana Azzam — Florida International University
- Study coordinator: Diana Azzam, PhD
- Email: fpmlab@fiu.edu
- Phone: 305-348-9043
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.