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NCT04111107 · REGISTRY CONDITION SEARCH

Precision Medicine for Patients With Identified Actionable Mutations

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE2
Status at capture
TERMINATED
Registry last update
2024-07-03

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Progression Free Survival Ratio

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outcome · POSTED

Overall Survival

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outcome · POSTED

Number of Participants With Adverse Events

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outcome · POSTED

Response Rate

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
Inclusion Criteria: * Cancer patients at Wake Forest Baptist Comprehensive Cancer Center and its satellites who have next generation DNA sequencing results on their tumor biopsy or surgically resected tissue and/or blood samples and/or consent to the Wake Forest Comprehensive Cancer Center Precision Oncology Registry to use their information for research. * Actionable mutation (defined as a mutation or gene amplification for which an off-label therapy is identified on the patient's NGS report for which NCCN guidelines do not recommend a specific treatment in the particular disease or for which there is no documentation in the patient's medical record of clinical data demonstrating lack of activity with the targeting of the specific mutation or amplification in the patient's specific disease) uncovered by the genomic sequencing of a tumor or those that have undergone liquid biopsy assay of their tumor genomic, performed by Wake Forest or another and who are medically able to receive targeted therapy based on those results. * Sequencing on a sample collected within 3 months prior to registration is strongly encouraged but must have been performed within the 12 months prior to registration. * Patients must have progressed through at least two lines of treatment, or are not candidates for or unwilling to receive any standard therapies. Patients who have received treatment on the present protocol who have progression of disease may be recruited to the trial for treatment using another targeted therapy provided that they fulfill the other criteria for participation in the trial. If a patient discontinues treatment on this protocol they can be considered for further participation in this trial provided they meet all of the eligibility criteria and are eligible for re-registration and re-consent. * Eastern Cooperative Oncology Group (ECOG) of less than or equal to 3 * Life expectancy of greater than 6 weeks * The effects of the drugs used for cancer treatment on the developing human fetus are unknown. For this reason and because of the possibility that the agents are teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Exclusion Criteria: * Patients who have had or will receive chemotherapy or radiotherapy to major bone marrow bearing sites within 2 weeks prior to receiving treatment on the study * Patients who have not recovered from toxicity of prior treatment if such toxicity will preclude treatment with the proposed targeted agent. * Patients may not be receiving any other investigational agents. * Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. * Pregnant women are excluded from this study because of the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the targeted agent, breastfeeding should be discontinued if the mother is treated with the targeted agent.
healthy Volunteers
false
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    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.
    intervention Names
    1. Drug: Investigational Agent
    2. Other: Supportive Care Regimens
    3. Diagnostic Test: Next Gen Sequencing Report
    label
    Drug Administration Based on Next Gen Sequencing Report
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Drug Administration Based on Next Gen Sequencing Report
    description
    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.
    name
    Investigational Agent
    type
    DRUG
  2. arm Group Labels
    1. Drug Administration Based on Next Gen Sequencing Report
    description
    Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion.
    name
    Supportive Care Regimens
    type
    OTHER
  3. arm Group Labels
    1. Drug Administration Based on Next Gen Sequencing Report
    description
    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.
    name
    Next Gen Sequencing Report
    type
    DIAGNOSTIC_TEST
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
45
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    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).
    measure
    Progression Free Survival Ratio
    time Frame
    From the start of treatment to the time of progression, death, or date of last contact, assessed up to 2 years
secondary Outcomes
  1. description
    Overall survival will be displayed using Kaplan-Meier curves with median survival times and 95% confidence intervals.
    measure
    Overall Survival
    time Frame
    From the start of treatment to date of death or date of last contact, up to 2 years
  2. description
    Adverse events will be summarized in incidence tables by type for all patients who received at least one cycle of treatment.
    measure
    Number of Participants With Adverse Events
    time Frame
    Up to 30 days after treatment ends, up to 33 months
  3. description
    Response rate will be estimated for all patients with corresponding 95% confidence intervals. Complete response is the disappearance of all target lesions and normalization of tumor marker level. Any pathological lymph nodes must have reduction in short axis to less than 10 mm. Partial response is at least 30% decrease in the sum of diameters of target lesions. Progressive disease is greater than 20% increase and a minimum 5 mm increase over the nadir. Stable disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
    measure
    Response Rate
    time Frame
    Up to 30 days after treatment ends, up to 33 months
Full study description
brief 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.
Source references
Source notices and limitations
    Discovery and provenance
    1. release id
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      edge id
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      payload
      clinical indication matching status
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      id
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      nct id
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      occurrences
      1. context exact
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        exact text
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        start
        0
      offset unit
      unicode_code_points
      query cond
      Plasma Cell Neoplasms
      query disease state id
      ds-plasma-cell-neoplasms
      source file
      /Users/arunverma/Documents/ChatGPT/Cancer Statistics Website/research-data/2026-09-10/clinicaltrials-condition/round-0000/raw/cond-plasma-cell-neoplasms-000.json
      source file sha256
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      source json pointer
      /studies/497/protocolSection/identificationModule/nctId
      source kind
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      source record id
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      source string basis
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      source string sha256
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    Preserved source evidence · Not medical advice