Ex Vivo Drug Sensitivity Testing and Mutation Profiling

Sponsor
Florida International University (Other)
Overall Status
Active, not recruiting
CT.gov ID
NCT03860376
Collaborator
Nicklaus Children's Hospital f/k/a Miami Children's Hospital (Other)
25
1
45.8
0.5

Study Details

Study Description

Brief Summary

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.

Detailed Description

PRIMARY OBJECTIVE: The primary objective of the study is to determine feasibility of providing pediatric cancer patients with access to personalized treatment options and clinical management recommendations based on ex vivo drug sensitivity testing (DST) and genomic profiling.

SECONDARY OBJECTIVE: The secondary objective of the study is to compare individual outcomes (response and disease-free survival) in patients with pediatric cancers treated with DST-guided therapy as compared to non-DST guided (conventional) therapy.

EXPLORATORY OBJECTIVE: To explore associations between genetic abnormalities in malignancies and ex vivo drug response.

Study Design

Study Type:
Observational
Actual Enrollment :
25 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Personalized Ex Vivo Drug Screening and Genomics Profiling to Guide Individualized Treatments for Children With Relapsed or Refractory Solid Tumors and Leukemias
Actual Study Start Date :
Feb 21, 2019
Anticipated Primary Completion Date :
Dec 15, 2022
Anticipated Study Completion Date :
Dec 15, 2022

Arms and Interventions

Arm Intervention/Treatment
Chemorefractory or relapsed patients

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.

Outcome Measures

Primary Outcome Measures

  1. Percentage of patients that receive DST-guided treatmens [Upto 4 years]

    This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a combination drug regimen within 4 weeks in at least 10 out of 16 patients (62.5%). With that outcome, we would be 90% confidence that the true feasibility rate is at least 43% (90% CI: 0.425, 0.824).

Secondary Outcome Measures

  1. Assessing response to DST-guided therapy. [Upto 4 years]

    Will be measured by comparing patients treated with DST-guided therapy versus non-DST guided conventional therapy.

  2. Assessing Disease Free Survival (DFS). [Upto 4 years]

    DFS will be measured from start of treatment to event (event defined as treatment failure, relapse, second malignancy, or death) or last follow-up for patients who are event free.

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Day to 21 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Nicklaus Children's Hospital Miami Florida United States 33155

Sponsors and Collaborators

  • Florida International University
  • Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Investigators

  • Principal Investigator: Diana Azzam, PhD, Florida International University
  • Principal Investigator: Daria Salyakina, PhD, Nicklaus Children's Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Diana Azzam, PhD, Assistant Professor, Florida International University
ClinicalTrials.gov Identifier:
NCT03860376
Other Study ID Numbers:
  • 1186919
  • 8LA05
First Posted:
Mar 1, 2019
Last Update Posted:
Aug 22, 2022
Last Verified:
Aug 1, 2022
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Product Manufactured in and Exported from the U.S.:
No
Keywords provided by Diana Azzam, PhD, Assistant Professor, Florida International University
Additional relevant MeSH terms:

Study Results

No Results Posted as of Aug 22, 2022