Matching Patients With Hematologic Malignancy to Adequate Clinical Trials

Study Overview
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Brief Summary
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A molecular profile of a patient with hematologic malignancy, for whom standard-of-care had failed, is identified using next generation sequencing. Patients are assigned to early clinical trials of targeted agent based on the molecular profiling or physician's choice. The improvement of outcomes in the intention-to-treat population is investigated.

Detailed Description
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Most of hematologic malignancy patient becomes incurable with standard treatment during their disease course. Although these patients are recommended to participate in an early phase clinical trials, the response rate have reported be only 4 to 6 percent. Over several decades, a lot of cancer driving mutations has been identified, and targeted agents directed at the mutations are continuously developed and studied in many clinical trials. Most of the clinical trials recruited participants regardless of mutational status of them. Recently, however, participants has been recruited in recent clinical trials according to the presence of specific mutations. The response rate of these recent clinical trials is 12-75%. In this pragmatic clinical trial, cancer driving mutations in hematologic malignancy patients is identified using next generation sequencing, and assign patients to an appropriate clinical trial which is anticipated to exhibit the best response. The improvement of outcome of this biomarker-driven assignment is investigated.

Official Title
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Pragmatic Clinical Trial of Matching Malignant Hematologic Cancer Patients With Adequate Early Phase Clinical Trials by Next-generation Sequencing

Conditions
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Hematologic Malignancies

Intervention / Treatment
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* Other: Matching on the basis of molecular analysis
* Other: Matching on the basis of physician's choice


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: adults with > 18 years old incurable hematologic malignancy patients who failed to respond to standard treatment patients who agreed to this protocol with informed consent Eastern Cooperative Oncology Group performance status ≤ 3 Tolerable major organ function determined by laboratory examination Exclusion Criteria: Expected survival < 3 months patients with poor compliance patients who can not give an informed consent patients who are participating another clinical trials

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Matched<br>A molecular profile is identified using next generation sequencing. A participant in this arm is assigned to early clinical trial studying targeted agent, which is anticipated to have the best response rate. | Other: Matching on the basis of molecular analysis<br> <br> |
| Other: Not matched<br>A participants in this arm is assigned to a clinical trial at physician's choice. | Other: Matching on the basis of physician's choice<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response rate | Response evaluation is based on the criteria suggested in a clinical trial to which a participant is assigned. | one year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Types of identified molecular profile |  | one year | 
| Overall survival |  | one year | 
| Progression-free survival |  | one year | 
| Toxicity profile | according to CTCAE version 4.0 | one year | 
| Frequencies of identified molecular profile |  | one year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Personalized medicine, Next generation sequencing