Testing drug sensitivity and genetic profiling for pediatric cancer patients
Official title: Advancing Personalized Treatment in Pediatric Oncology Through Functional Precision Medicine
Run by Florida International University · NCT05857969 · Data updated 2026-11-12
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 |
| Ages | 1 Day to 21 Years |
| Sex | All |
| Enrollment | 65 (estimated) |
| Sponsor | Florida International University Academic / other |
| Locations | 1 site (Miami, Florida) |
| Status | Recruiting |
| Started | February 22, 2023 |
| Main results expected | February 22, 2028 |
| Study ends | December 31, 2028 |
| Registry record updated | August 26, 2026 |
| Trial ID | NCT05857969 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include pediatric patients aged 21 years or younger with recurrent or refractory cancers who have available tumor tissue for testing.
✕Probably not a fit
Patients with newly diagnosed tumors or those with high cure rates using standard therapies may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
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.
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 gone: Other studies utilizing similar approaches of drug sensitivity testing and genomic profiling have shown promise, indicating potential for success in this novel application.
Who can join: the full rules
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 it's running
-
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
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
HA-1 TCR T cell therapy for leukemia that returned or didn't respond after a donor stem cell transplant
This Phase 1 study will test whether giving specially engineered donor T cells called HA-1 TCR T cells is safe and what dose works for people whose acute leukemia came back or didn't respond after a donor stem cell transplant.
-
FH-FOLR1 CAR T-cell therapy for children with relapsed or refractory AML
This trial will test FH-FOLR1 CAR T cells in children aged six and under whose FOLR1-positive acute myeloid leukemia has returned or not responded to treatment.
-
Finding the best dose of imetelstat with chemotherapy for children with certain blood cancers
This study is testing the best dose of a new drug called imetelstat combined with chemotherapy to see if it can help children with tough blood cancers like leukemia and myelodysplastic syndrome.
-
Using edetate calcium disodium or succimer to treat acute myeloid leukemia or myelodysplastic syndrome during chemotherapy
This study is testing whether two drugs, edetate calcium disodium and succimer, can safely help people with acute myeloid leukemia or myelodysplastic syndrome feel better during chemotherapy.
-
Combination chemotherapy for relapsed or refractory acute lymphoblastic leukemia and related conditions
This study is testing a new combination of chemotherapy drugs to see if it can help people with relapsed or hard-to-treat acute lymphoblastic leukemia and related blood cancers feel better and live longer.
-
Combining cladribine, idarubicin, cytarabine, and venetoclax for treating certain blood cancers
This study is testing a new combination of four medications to see if it helps people with certain types of blood cancers feel better and live longer.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.