NCT04111107

Precision Medicine for Patients With Identified Actionable Mutations

Precision Medicine for Patients With Identified Actionable Mutations at Wake Forest Baptist Comprehensive Cancer Center (WFBCCC): A Pragmatic Trial-

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: Wake Forest University Health Sciences · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

Primary Objective: • 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. Secondary Objectives: * 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. Exploratory Objectives: * 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.

Conditions

• Solid Tumor
• Lymphoma
• Multiple Myeloma
• Malignant Neoplasm
• Mutation Abnormality

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2020-04-22
• Primary completion: 2022-12-09
• Completion: 2023-02-09
• First posted: 2019-10-01
• Last updated: 2024-07-03
• Results posted: 2024-07-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study