Precision Medicine for Patients With Identified Actionable Mutations
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|ClinicalTrials.gov Identifier: NCT04111107|
Recruitment Status : Recruiting
First Posted : October 1, 2019
Last Update Posted : November 9, 2020
The goal of the current pragmatic trial is to evaluate the impact of a simple method of selecting a treatment approach for identified mutations on participants' progression free survival (PFS). The study also intends to collect information on barriers that investigators encounter when prescribing treatment options using the Next Generation Sequencing (NGS) reports. Additionally, patients' quality of life will be measured before, after, and during treatment.
Patients will be followed until death for monitoring survival study endpoints.
|Condition or disease||Intervention/treatment||Phase|
|Solid Tumor Lymphoma Multiple Myeloma Malignant Neoplasm Mutation Abnormality||Drug: Investigational Agent Other: Supportive Care Regimens Diagnostic Test: Next Gen Sequencing Report||Phase 2|
• To estimate the progression-free ratio, as defined by the progression-free survival time on study treatment divided by the progression-free survival time on the last treatment received by patient, for an identified actionable mutation, who will be treated with an off-label treatment off label therapy based on a simplified selection methodology using the Next Generation Sequencing results.
- To estimate patient response rate on off-label treatments for actionable mutations based on Next Generation Sequencing results.
- To estimate overall survival (OS) for patients treated with off-label treatments for actionable mutations based on Next Generation Sequencing results.
- To describe the safety of using off-label or other experimental treatments for patients with actionable mutations based on Next Generation Sequencing results.
- To describe health related quality of life in patients undergoing off-label treatment targeting genetic mutations, as measured by the PROMIS-29 Overall Health-Related Quality of Life, Including 4-Item Anxiety Subscale.
- Using the Satisfaction with Medical Decision Scale, to describe patient satisfaction with decision to pursue off-label treatment.
- To identify types of actionable mutations with available targeted treatment occurring in cancer patients.
- To characterize the historical treatment regimens for these patients relative to the targetable mutation.
- To describe patient clinical and demographic characteristics of those with actionable mutations based on Next Generation Sequencing results.
- To identify barriers to treatment based on Next Generation Sequencing results.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||337 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Precision Medicine for Patients With Identified Actionable Mutations at Wake Forest Baptist Comprehensive Cancer Center (WFBCCC): A Pragmatic Trial-|
|Actual Study Start Date :||April 22, 2020|
|Estimated Primary Completion Date :||April 2021|
|Estimated Study Completion Date :||April 2023|
Experimental: Drug Administration Based on Next Gen Sequencing Report
Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity.
Drug: Investigational Agent
Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label.
Other: Supportive Care Regimens
Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion.
Diagnostic Test: Next Gen Sequencing Report
1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
- Progression Free Survival [ Time Frame: From the start of treatment to the time of progression, death, or date of last contact, assessed up to 2 years ]Estimation of progression-free ratio defined as the duration of time from start of treatment to the time of progression divided by the duration of time from the last treatment received pre-trial to the time of progression on that treatment. The median progression-free ratio will be estimated with the range and a two-sided Wilcoxon Signed Rank test will be calculated to see if the progression free survival ratio is different from 1.0. This trial is powered to detect differences in the progression-free ratio for those with actionable mutations identified by NGS results and then treated with a targeted therapy. A hypothesized PFS ratio larger than 1.3 would suggest that the targeted therapy is doing better than the previous treatment received (not targeted), and we assume a null hypothesis PFS ratio of 1.0 (no difference).
- Overall Survival [ Time Frame: From the start of treatment to date of death or date of last contact, up to 2 years ]Overall survival will be displayed using Kaplan-Meier curves with median survival times and 95% confidence intervals.
- Incidence of Adverse Events [ Time Frame: Up to 30 days after treatment ends ]Adverse events will be summarized in incidence tables by type and grade severity for all patients and presented by type of treatment received.
- Response Rate [ Time Frame: Up to 30 days after treatment ends ]Response rate will be estimated for all patients with corresponding 95% confidence intervals.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04111107
|Contact: Study Coordinatorfirstname.lastname@example.org|
|United States, North Carolina|
|Wake Forest Baptist Comprehensive Cancer Center||Recruiting|
|Winston-Salem, North Carolina, United States, 27157|
|Contact: Study Coordinator 336-716-5440 email@example.com|
|Principal Investigator: Stefan Grant, MD|
|Principal Investigator:||Stefan Grant, MD||Wake Forest University Health Sciences|