Solid TumorPhase 2NCT04111107

Precision Medicine for Patients With Identified Actionable Mutations

A Phase 2 study testing Investigational Agent, Supportive Care Regimens and Next Gen Sequencing Report in people with Solid Tumor and Lymphoma, run by Wake Forest University Health Sciences at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Solid Tumor, Lymphoma, Multiple Myeloma
What is being tested?: Investigational Agent, Supportive Care Regimens and Next Gen Sequencing Report
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 45 participants
Where: 1 siteUnited States
Study dates: 22 Apr 2020to 9 Feb 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

United States1 site

Wake Forest Baptist Comprehensive Cancer Center
Winston-Salem, North Carolina, 27157

Other trials in United States

About this study

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

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.

Read the 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
Being tested: Investigational Agent, Supportive Care Regimens, Next Gen Sequencing Report
Medical subject headings: Lymphoma, Multiple Myeloma, Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Investigational AgentDrug
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.
Supportive Care RegimensOther
Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion.
Next Gen Sequencing ReportDiagnostic test
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.

Study groups in NCT04111107
Study group	Role	What they receive
Drug Administration Based on Next Gen Sequencing Report	Experimental	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. Receives: Drug: Investigational Agent; Other: Supportive Care Regimens; Diagnostic Test: Next Gen Sequencing Report

What is being measured

The primary outcome is the main question the study is designed to answer.

Progression Free Survival Ratio
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).
Measured: From the start of treatment to the time of progression, death, or date of last contact, assessed up to 2 years

Secondary outcomes (3)

Overall Survival
Overall survival will be displayed using Kaplan-Meier curves with median survival times and 95% confidence intervals.
Measured: From the start of treatment to date of death or date of last contact, up to 2 years
Number of Participants With Adverse Events
Adverse events will be summarized in incidence tables by type for all patients who received at least one cycle of treatment.
Measured: Up to 30 days after treatment ends, up to 33 months
Response Rate
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.
Measured: Up to 30 days after treatment ends, up to 33 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Apr 20209 Feb 2023

First posted1 Oct 2019
Study start22 Apr 2020
Primary completion9 Dec 2022
Study completion9 Feb 2023

Why it stopped: PI left the institution and site decided not to continue the study

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Solid TumorStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 45 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04111107
Sponsor's protocol ID: IRB00061185
Other IDs: WFBCCC 04519, P30CA012197, NCI-2019-06802
Status last verified: Jun 2024
First submitted: 27 Sep 2019
Last updated: 3 Jul 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Enrolling by invitationObservational study
Collection of Pharmacokinetic Samples From People With Unanticipated Response, Significant Toxicity or Concern of Future Toxicity
Cancer, Solid Tumors
Active, not recruitingPhase 1
Research Study Utilizing Expanded Multi-antigen Specific Lymphocytes for the Treatment of Solid Tumors
Solid Tumors
RecruitingPhase 3
Long-term Safety and Efficacy Extension Study for Participants With Advanced Tumors Who Are Currently on Treatment or in Follow-up in a Pembrolizumab (MK-3475) Study (MK-3475-587/KEYNOTE-587)
Solid Tumors, Hematologic Malignancies
RecruitingObservational study
Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors
Malignant Solid Tumors, Other Neoplasms Solid Tumors
RecruitingPhase 1/2
Study of STK-012 Alone and With Other Treatments in Patients With Advanced Lung Cancer and Other Cancers
Advanced Solid Tumor, Non Small Cell Lung Cancer

All Solid Tumor trials

Study at a glance

From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Precision Medicine for Patients With Identified Actionable Mutations at Wake Forest Baptist Comprehensive Cancer Center (WFBCCC): A Pragmatic Trial-
ClinicalTrials.gov ID: NCT04111107
Acronym / sponsor ID: IRB00061185
Conditions: Solid Tumor, Lymphoma, Multiple Myeloma, Malignant Neoplasm, Mutation Abnormality
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: PI left the institution and site decided not to continue the study
Enrolment: 45 participants (actual)
Lead sponsor: Wake Forest University Health SciencesUniversity, hospital or other
Collaborators: National Cancer Institute (NCI)
Study start: 22 Apr 2020 (actual)
Primary completion: 9 Dec 2022 (actual)
Study completion: 9 Feb 2023 (actual)
First posted: 1 Oct 2019 (actual)
Results: Posted 3 Jul 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Drug Administration Based on Next Gen Sequencing Report	Experimental	Drug: Investigational Agent, Other: Supportive Care Regimens, Diagnostic Test: Next Gen Sequencing ReportInvestigators 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.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

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

Key exclusion criteria (5 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Investigational AgentTreatment 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.	Drug	Drug Administration Based on Next Gen Sequencing Report
Supportive Care RegimensSupportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion.	Other	Drug Administration Based on Next Gen Sequencing Report
Next Gen Sequencing Report1) 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.	Diagnostic test	Drug Administration Based on Next Gen Sequencing Report

Primary outcome

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
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).

3 secondary outcomes

Overall Survival
Time frame: From the start of treatment to date of death or date of last contact, up to 2 years
Number of Participants With Adverse Events
Time frame: Up to 30 days after treatment ends, up to 33 months
Response Rate
Time frame: Up to 30 days after treatment ends, up to 33 months

Results and safety

Results posted to the registry on 3 Jul 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Drug Administration Based on Next Gen Sequencing Report	45	34

Primary outcome: 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. Median (Full Range). Unit: ratio

Measure	Drug Administration Based on Next Gen Sequencing Report n = 18
Value	0.73 (0.26 to 6.67)

Drug Administration Based on Next Gen Sequencing Report: p = 0.966, Wilcoxon signed rank (2 sided)

Population analysed: For various reasons, 27 patients were not evaluable for the primary outcome. Five patients had no prior treatment but did have an actionable mutation to qualify them for inclusion in the study. Another ten patients were enrolled but never started treatment under this protocol. Nine patients started treatment but died prior to testing for best response and for progression. Two patients died with stable disease and one patient is alive with stable disease and no sign of progression .

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Drug Administration Based on Next Gen Sequencing Report	18 of 34 (52.9%)	34 of 34 (100%)	32 of 34

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Solid Tumor

Enrolling by invitationObservational study
Collection of Pharmacokinetic Samples From People With Unanticipated Response, Significant Toxicity or Concern of Future Toxicity
Cancer, Solid Tumors
Active, not recruitingPhase 1
Research Study Utilizing Expanded Multi-antigen Specific Lymphocytes for the Treatment of Solid Tumors
Solid Tumors
RecruitingPhase 3
Long-term Safety and Efficacy Extension Study for Participants With Advanced Tumors Who Are Currently on Treatment or in Follow-up in a Pembrolizumab (MK-3475) Study (MK-3475-587/KEYNOTE-587)
Solid Tumors, Hematologic Malignancies
RecruitingObservational study
Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors
Malignant Solid Tumors, Other Neoplasms Solid Tumors
RecruitingPhase 1/2
Study of STK-012 Alone and With Other Treatments in Patients With Advanced Lung Cancer and Other Cancers
Advanced Solid Tumor, Non Small Cell Lung Cancer

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Stefan Grant, MD principal investigator

Wake Forest University Health Sciences

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 3 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome 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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04111107.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jul 2024; AzTrials last refreshed its copy on 30 Oct 2026.