Clinical trial NCT04447651: Patient Response to Immunotherapy Using Spliceosome Mutational Markers (PRISMM)
Official title: A Remote-Directed "Virtual" Clinical Trial in Metastatic Solid Tumors to Determine Feasibility of Evaluating Patient Response to Immunotherapy Using Spliceosome Mutational Markers (PRISMM)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Metastatic Solid Tumor
SF3B1 Gene Mutation
Spliceosome Mutation
U2AF1 Gene Mutation
SRSF2 Gene Mutation
Keywords: sequencing
metastatic solid tumor
spliceosome mutation
immune checkpoint inhibition
immunotherapy
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sponsor class: OTHER
Collaborators: name: Vanderbilt University
name: Bristol-Myers Squibb
name: Avon Breast Health Access Fund
First posted: 2020-06-25
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Metastatic solid tumor patients with a SF3B1,U2AF1 or SRSF2 mutation.

Brief summary: This study is being done to see if patients with metastatic solid tumors (hematologic malignancies and lymphoma excluded) who have a specific genetic mutation in patients' tumor (the SF3B1, U2AF1 or SRSF2 mutation), are more likely to respond to immunotherapy agents that are now commercially available.

Detailed description: This is a non-therapeutic study, meaning that while Johns Hopkins is providing a treatment recommendation based on participants' genetic information, participants and participants' oncologist will decide ultimately what to do, and participants' oncologist will monitor participants' day to day care while on therapy. Participation involves allowing the investigators access to participants' sequencing report and medical records, providing a blood sample (about 8 tablespoons) at baseline and possibly again after three months, and answering questionnaires. If participants join the study, a panel of experts (Johns Hopkins Molecular Tumor Board) will review participants' genetic information participants entered and make a determination regarding treatment recommendation. Blood will be collected at baseline irrespective of what treatment is recommended. If immunotherapy is recommended, the investigators may collect blood again at 3 months. Participants and participants' oncologist will ultimately decide if participants proceed with the recommend treatment or not; the recommendation made from Johns Hopkins is not binding in any way. Participants' oncologist will continue with usual care according to standard practices while participants are on therapy. The investigators will get health information and conduct questionnaires with participants and participants' oncologist to assess how participants are doing on therapy. The main risks are the discomforts of the blood draw (which are expected to be minor and not last), boredom from completing questionnaires and the risk that information may become known to people outside of the study. Participants may or may not benefit directly from being in the study and there is no payment for participation.

Eligibility criteria: Inclusion Criteria:

* Performance status eligible for immune checkpoint blockade as determined by local physician
* Able to demonstrate histologically proven locally advanced or metastatic solid tumors (hematologic malignancies and lymphoma excluded)
* genomic testing demonstrating a spliceosome mutation (SF3B1, U2AF1 or SRSF2)

Exclusion Criteria:

* Local physician determines has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
* Local physician determines the patient has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate

Primary outcomes:
measure: Feasibility of study as assessed by completion of study accrual within study time frame
timeFrame: 2 years
measure: Feasibility of study as assessed by physician responses
timeFrame: 1 year
measure: Feasibility of case review as assessed by time to issuance of recommendations
timeFrame: 4 weeks from consent
Secondary outcomes:
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
Study officials:
affiliation: Johns Hopkins University
name: Cesar Santa-Maria, MD
role: PRINCIPAL_INVESTIGATOR
References: