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Genetic Analysis in Determining Individualized Treatment for Patients with Recurrent or Refractory Blood Cancers

Trial Status: Active

This trial uses genetic analysis to determine a personalized treatment plan for patients with blood cancers that have come back or do not respond to treatment. Personalized cancer therapy is the practice of making decisions about what kind of treatment patients should receive based on the genetic makeup of their tumors. Genes carry the instructions to make proteins, and certain gene changes can cause cells to evade normal growth controls and become cancer. Genes in cancer cells have changed from those in normal cells. Researchers believe that abnormal genes in tumors may affect how individuals respond to cancer treatments. Studying the information collected from medical records about the tests and treatments patients have received may help doctors describe whether or not patients respond better when they receive treatment based on the genetic makeup of their tumor.

Inclusion Criteria

  • Patients with incurable malignancies with >= 50% 2-year cancer-associated mortality. Diseases include, but are not limited to:
  • B-cell neoplasms: * Diffuse large B-cell lymphoma (DLBCL) * High grade B-cell lymphoma (HGBL) * Gray zone lymphoma (GZL) * Double hit lymphoma (DHL) * B-cell lymphoma unclassifiable (BCLU) * Mantle cell lymphoma (MCL) * Follicular lymphoma (FL) * Marginal zone lymphoma (MZL) * Small lymphocytic lymphoma (SLL) / * Chronic lymphocytic leukemia (CLL) * Acute lymphoid B-cell leukemia (B-ALL) * Hairy cell leukemia (HCL) * Lymphoplasmacytic lymphoma (LPL) * Waldenstrom’s macroglobulinemia (WM) * Multiple myeloma (MM) /plasmacytoma * Monoclonal immunoglobulin deposition disease * Intravascular large B-cell lymphoma * T-cell/histiocyte-rich large B-cell lymphoma * Primary central nervous system (CNS) lymphoma (PCNSL) * Epstein-Barr virus (EBV) + DLBCL, not otherwise specified (NOS) * HHV8+ primary effusion lymphoma (PEL) * Primary bone lymphoma (PBL) * Burkitt lymphoma (BL) * Plasmablastic lymphoma (PL) * Post-transplant lymphoproliferative disorders (PTLD) * Human immunodeficiency virus (HIV) associated lymphoproliferative disorders
  • T-cell/natural killer (NK) cell neoplasms: * Cutaneous T-cell lymphoma (CTCL) * Anaplastic large cell lymphoma (ALCL) Alk+/Alk- * Enteropathy associated T-cell lymphoma (EATL) * Angioimmunoblastic T-cell lymphoma (AITL) * Mycosis fungoides (MF)/Sezary syndrome (SS) * Gamma/ delta T-cell lymphoma (G/D TL) * NK-cell lymphoma * Hepatosplenic T-cell lymphoma (HTCL) * T-cell prolymphocytic leukemia (T-PLL) * Aggressive NK cell leukemia/lymphoma * Adult T-cell leukemia/lymphoma (T-ALL) * Monomorphic epitheliotropic intestinal T-cell lymphoma * Breast-implant associated anaplastic large T-cell lymphoma * Extranodal NK/T cell lymphoma (nasal type) * T-cell large granular lymphocytic leukemia (T-LGL) * Hepatosplenic T-cell lymphoma * Subcutaneous panniculitis like T-cell lymphoma * Peripheral T-cell lymphoma (PTCL) * Primary cutaneous acral CD8+T-cell lymphoma * Primary cutaneous CD8+ aggressive epidermotropic T-cell * Nodal peripheral T-cell lymphoma with T follicular helper (TFH) phenotype * T-cell prolymphocytic T-cell leukemia/lymphoma.
  • Histiocytic/dendritic cell neoplasms: * Langerhans cell histiocytosis (LCH) * Erdheim-Chester disease (ECD) * Indeterminate dendritic cell tumor * Fibroblastic reticular cell tumor.
  • Hodgkin lymphoma (HL): * Classical HL * Nodular lymphocyte-predominant HL
  • Patients with a rare tumor histology (i.e., fewer than 6 cases per 100,000 per year) with no approved therapies.
  • Relapsed or refractory to available systemic therapy regimens. For disease histologies where effective, Food and Drug Administration (FDA)-approved, targeted treatment already exists, disease progression is required.
  • Patients with incurable malignancies, irrespective of 2-year mortality, who, in the opinion of the investigator, have no treatment option expected to yield significant clinical benefit.
  • Patients must have at least one of the following for a diagnosis/disease status: * Advanced symptomatic disease * Medically unfit for standard therapy * Disease where no conventional therapy leads to a survival benefit > 3 months in the respective cohort and line of therapy for which the patient is otherwise eligible * Actionable biologically informed targets determined by certified genomic profiling, immunophenotyping or other clinically validated techniques.
  • Ability to understand and the willingness to sign a written informed consent.
  • Patients must have measurable disease for malignancies: defined as at least one lesion that can be accurately measured in at least one dimension with spiral computed tomography (CT) scan, positron emission tomography (PET)-CT, magnetic resonance imaging (MRI), or calipers by clinical exam. Or presence of hematologic abnormalities with or without bone marrow involvement.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
  • New York Heart Association (NYHA) functional classification I-II.
  • Adequate organ function that reasonably allows for safe administration of therapy.
  • At the time of treatment, patients should be off other anti-tumor agents for at least 5 half-lives of the agent or 2 weeks from the last day of treatment, whichever is shorter, so long as there is recovery from clinically significant side effects from previous therapy to less than or equal grade 1.
  • Able to swallow and/or retain oral medication, if needed.
  • Patients must have evaluable tissue/blood with adequate tumor content/purity for testing as specified by the molecular profiling lab. This will be obtained during the standard of care tumor diagnosis and tumor staging evaluation.
  • Female patients of childbearing potential must agree to use at least one form of contraception during the study and for at least one month after treatment discontinuation. For the purposes of this study, child- bearing potential is defined as: all female patients that were not in post- menopause for at least one year or are surgically sterile. Male patients must use a form of barrier pregnancy prevention approved by the investigator /treating physician during the study and for at least one month after treatment discontinuation.

Exclusion Criteria

  • Severe or uncontrolled medical disorder that would, in the investigator’s opinion, confound study analyses of treatment response or preclude the patient from safely receiving treatment (i.e. substance abuse or psychiatric illness/social situations that would limit compliance with study requirements).
  • Pregnancy, breast-feeding women or any patient with childbearing potential not using adequate pregnancy prevention.
  • Inadequate end organ function that would preclude safe administration of anti-neoplastic therapy; including hepatic dysfunction (liver function tests [LFTs] > 5 x normal limit, total bilirubin > 3 and creatinine [Cr] > 3 x normal limit or glomerular filtration rate [GFR] < 20 cc/min, or symptomatic heart failure [ejection fraction (EF) < 20%]), except when organ function impairment is a consequence of underlying malignancy and there is a reasonable expectation for improvement following initiation of appropriate therapy.
  • Uncontrolled infections or sepsis. Patients with chronic viral infections (including HIV, hepatitis B virus [HBV]/hepatitis C virus [HCV]) that are controlled with appropriate concurrent therapy are allowed to participate in the study, provided ongoing compliance with antiviral therapy can be reasonably expected throughout the duration of the study. Patients with acute infections must start appropriate anti-microbial therapy and demonstrate stabilization of infection prior to study initiation.
  • Patients with acute infections must start appropriate anti-microbial therapy and demonstrate stabilization of infection prior to study initiation.


San Diego
University of California San Diego
Status: ACTIVE
Contact: Natalie Galanina
Phone: 858-534-0196


I. Assess overall response rates to molecularly targeted matched treatment and physician’s choice of unmatched standard-of-care treatment.


I. Determine the incidence of grade 3-5 adverse events in all groups according to Common Terminology Criteria for Adverse Events (CTCAE) version (v) 4.03.

II. Determine the overall response rate (ORR) defined as partial response (PR) or complete response (CR) according to disease specific National Comprehensive Cancer Network (NCCN) response criteria.

III. Determine the progression free survival (PFS) defined as time from first dose to disease progression or death whichever occurs first.

IV. Determine the overall survival (OS) defined as time from first dose to death due to any cause.


I. Assess the ability to identify actionable genomic alterations.

II. Assess the ability to procure molecularly targeted matched treatment based on genomic analysis (through insurance coverage/compassionate use mechanism or patient assistance programs).

III. Assess the ability to deliver matched therapy in a timely fashion.


Patients undergo blood and tumor sample collection for genomic profiling. Based on the results, patients receive either molecularly matched targeted therapy or physician's choice of traditional unmatched therapy.

After completion of study, patients are followed every 3 months.

Trial Phase Phase NA

Trial Type Treatment

Lead Organization
University of California San Diego

Principal Investigator
Natalie Galanina

  • Primary ID 181350
  • Secondary IDs NCI-2019-00959
  • ID NCT03955276