Clinical trial · Observational
Ex Vivo Drug Sensitivity Testing and Mutation Profiling
Personalized Ex Vivo Drug Screening and Genomics Profiling to Guide Individualized Treatments for Children With Relapsed or Refractory Solid Tumors and Leukemias
NCT03860376CI-TRIAL-00066332completedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This study is a prospective, non-randomized feasibility study. Freshly isolated tumor cells from patients will be screened using state-of-the-art viability assay designed for ex vivo high-throughput drug sensitivity testing (DST). In addition, genetic information will be obtained from cancer and normal (germline) tissue and correlated with drug response. This study will provide the platform for informing treating physician about individualized treatment options. The main outcome of this study will be the proportions of the patients whose treatment was guided by the personalized medicine approach.
Conditions
Conditions (14)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Recurrent Childhood Acute Lymphoblastic Leukemia | Childhood Acute Lymphoblastic Leukemia | CURATED_BROADER | 0.78 |
| Recurrent Childhood Acute Myeloid Leukemia | Childhood Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| Recurrent Childhood Brainstem Glioma | Childhood Brain Stem Glioma | CURATED_BROADER | 0.78 |
| Recurrent Childhood Brain Tumor | Childhood Brain Neoplasm | CURATED_BROADER | 0.78 |
| Recurrent Childhood Ependymoma | Childhood Ependymoma | CURATED_BROADER | 0.78 |
| Recurrent Childhood Gliosarcoma | Childhood Gliosarcoma | CURATED_BROADER | 0.78 |
| Recurrent Childhood Large Cell Lymphoma | — |
Interventions
Interventions (0)
Data not yet available
No intervention recorded.
Design
Arms and outcomes
Arms (1)
- label
- Chemorefractory or relapsed patients
- description
- We intend to enroll chemorefractory or relapsed pediatric patients with all types of cancers where tumor tissue would be available for ex vivo drug screening and genomic profiling. The results of the drug sensitivity assay and genetic screening will be used to inform treating physician about patient-specific drug sensitivity or resistance guiding best therapy choices.
Primary outcomes (1)
- measure
- Percentage of patients that receive DST-guided treatmens
- timeFrame
- Up to 4 years
- description
- This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a monotherapy or combination drug regimen based on functional and/or genomics data within 4 weeks in at least 16 out of 25 patients (64%). To achieve at least 90% power, the null hypothesis will be rejected when at least 16 out of 25 patients receive treatment recommendations through functional and/or genomics data within 4 weeks on the study. With that outcome, we would have 95% confidence that the true feasibility rate is at least 30% (95% CI: 0.425, 1).
Secondary outcomes (3)
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Day
- Maximum age
- 21 Years
Show eligibility criteria text
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.
References
Publications (2)
- RESULTAcanda De La Rocha AM, Fader M, Coats ER, Espinal PS, Berrios V, Saghira C, Sotto I, Shakya R, Janvier M, Khatib Z, Abdella H, Bittle M, Andrade-Feraud CM, Guilarte TR, McCafferty-Fernandez J, Salyakina D, Azzam DJ. Clinical Utility of Functional Precision Medicine in the Management of Recurrent/Relapsed Childhood Rhabdomyosarcoma. JCO Precis Oncol. 2021 Oct 27;5:PO.20.00438. doi: 10.1200/PO.20.00438. eCollection 2021. No abstract available. PMID 34738048
- DERIVEDAcanda De La Rocha AM, Berlow NE, Fader M, Coats ER, Saghira C, Espinal PS, Galano J, Khatib Z, Abdella H, Maher OM, Vorontsova Y, Andrade-Feraud CM, Daccache A, Jacome A, Reis V, Holcomb B, Ghurani Y, Rimblas L, Guilarte TR, Hu N, Salyakina D, Azzam DJ. Feasibility of functional precision medicine for guiding treatment of relapsed or refractory pediatric cancers. Nat Med. 2024 Apr;30(4):990-1000. doi: 10.1038/s41591-024-02848-4. Epub 2024 Apr 11. PMID 38605166