Individualized Treatments in Adults With Relapsed/Refractory Cancers

A personalized cancer medicine approach would address therapy resistance, cancer metastasis, and limited options after standard of care is exhausted in advanced cancer participants. This approach may reduce the barriers to approved therapeutic assignment currently limited to a particular cancer type or patient demographic.

Study Overview

• Status: Recruiting
• Conditions: Relapsed Cancer; Refractory Cancer
• Intervention / Treatment: Diagnostic test: Drug Sensitivity Test (DST)

Detailed Description

Treatment itself will not be given as part of this trial. The results of the drug sensitivity test (DST) and genomic screening will be used to inform treating physician about participant-specific drug sensitivity or resistance guiding best therapy choices. The physician will decide the most appropriate treatment for each case, with the option to add one or more personalized (assay-guided) drug(s) from the investigational platform. All participants will need to be consented separately for any subsequent investigational treatment if no standard treatment options are available.

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Diana Azzam, PhD; Phone Number: 305-348-9043; Email: dazzam@fiu.edu
• Study Contact Backup: Name: Jorge Manrique-Succar, MD; Phone Number: (954) 659-5000; Email: manriqj@ccf.org

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33199
      • Recruiting
      • Florida International University
      • Contact: Diana Azzam, PhD; Phone Number: 305-348-9043; Email: dazzam@fiu.edu
      • Principal Investigator: Diana Azzam, PhD
    • Weston, Florida, United States, 33331
      • Recruiting
      • Lerner College of Medicine, Cleveland Clinic Florida
      • Principal Investigator: Jorge Manrique-Succar, MD
      • Sub-Investigator: Elizabeth Stone, MD
      • Sub-Investigator: Chebli Mrad, MD
      • Sub-Investigator: Badih Adada, MD
      • Sub-Investigator: Rafael Arleta-Bulos, MD
      • Sub-Investigator: Diana Saravia, MD
      • Sub-Investigator: Arun Nagarajan, MD
      • Sub-Investigator: Thomas Samuel, MD
      • Sub-Investigator: Chakra Chaulagain, MD
      • Sub-Investigator: Chieh-Lin Fu, MD
      • Sub-Investigator: Amer Hanano, MD
      • Sub-Investigator: Candice Schwartz, MD
      • Sub-Investigator: Mariana Berho, MD
      • Contact: Jillian King; Phone Number: 954-299-5798; Email: kingj19@ccf.org
      • Contact: Kaitlin Bernabe; Phone Number: 56449 954-659-5000; Email: BERNABK@ccf.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participants aged 18 years or older at the time of enrollment on this study of any gender, race, or ethnicity.
• Patients with suspected or confirmed diagnosis of recurrent or refractory cancer.
• Participants who have undergone at least two lines of previous therapy.
• Participants who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers).
• Participants willing to have a blood draw or buccal swab done for the purposes of genetic testing.
• Participants willing to sign informed consent.

Exclusion Criteria:

• Participants who do not have malignant tissue available and accessible.
• Participants for whom the amount of excised malignant tissue is not sufficient for the ex vivo drug testing and/or genetic profiling.
• Participants with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe standard therapy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Drug Sensitivity Testing; The results of the DST and genomic screening will be used to inform treating physician about participant-specific drug sensitivity or resistance guiding best therapy choices. The physician will decide which treatment will be most appropriate for each case. All participants will need to be consented separately for any subsequent investigational treatment if no standard treatment options are available.

Diagnostic test: Drug Sensitivity Test (DST); Refractory cancer tissue will be collected from participants and subjected to single-drug testing while DNA is simultaneously sent for targeted gene sequencing. Drug sensitivity scores from the tests will become available for a final list of therapeutic options ranked best in order of preference together with suggested doses and schedules.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment recommendation feasibility	Primary objective is to determine feasibility of providing treatment recommendations to relapsed and refractory adult cancer patients based on ex vivo drug sensitivity testing (DST). Feasibility will be demonstrated if it is possible to recommend treatment within 4 weeks in at least 22 of 36 participants (62.5%).	Up to 4 weeks post-treatment
Treatment recommendation feasibility	Primary objective is to determine feasibility of providing treatment recommendations to relapsed and refractory adult cancer patients based on genomic profiling. Feasibility will be demonstrated if it is possible to recommend treatment within 4 weeks in at least 22 of 36 participants (62.5%).	Up to 4 weeks post-treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment responsiveness	Treatment responsiveness will be measured by comparing individual outcomes of adult participants with relapsed and refractory cancers treated with DST-guided therapy to non-DST guided (conventional) therapy. To address this goal, the overall response rate will be calculated. A responder to the treatment will be defined as any patient who achieves a best response of "partial response" or "complete response" during the study period. Evaluable patients who demonstrate a complete or partial response confirmed by physician's review will be considered a responder. All other evaluable patients will be considered non- responder. Outcomes will be observed during treatment and follow-up for one year post-treatment or until disease progression, death, or withdrawal, whichever occurs first.	Up tp 1 year post-treatment
Progression-free survival	Progression-free survival will be measured by comparing individual outcomes of participants with relapsed and refractory cancers treated with DST-guided therapy to non-DST guided (conventional) therapy. DFS will be defined as time from start of treatment to even (treatment failure, relapse, second malignancy, death) or last follow-up for those who are event-free. Outcomes will be observed during treatment and follow-up for one year post-treatment or until disease progression, death, or withdrawal, whichever occurs first.	Up tp 1 year post-treatment
Progression-free survival ratio	We will assess changes in progression-free survival from each patient's previous treatment versus their progression-free survival from the treatment assigned during the trial. Assessments will be made both in the DST-guided cohort and the non-DST-guided (conventional) cohort. Analysis will include both the raw ratio as well as the number of incidences of 30% improved PFS on trial versus previous regimen (PFS2/PFS1 > 1.3x).	Up to 1 year post-treatment

Collaborators and Investigators

• Sponsor: Case Comprehensive Cancer Center
• Collaborators: Florida International University; Community Foundation of Broward
• Investigators: Principal Investigator: Jorge Manrique-Succar, MD, Lerner College of Medicine, Cleveland Clinic Florida; Principal Investigator: Diana Azzam, PhD, Robert Stempel College of Public Health and Social Work, Florida International University

Study record dates

Study Major Dates

• Study Start (Actual): November 20, 2023
• Primary Completion (Estimated): November 1, 2025
• Study Completion (Estimated): November 1, 2025

Study Registration Dates

• First Submitted: July 28, 2023
• First Submitted That Met QC Criteria: September 1, 2023
• First Posted (Actual): September 6, 2023

Study Record Updates

• Last Update Posted (Actual): April 23, 2024
• Last Update Submitted That Met QC Criteria: April 22, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Other Study ID Numbers: CASE5Y22

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No