Clinical Trials

PRIMARY OBJECTIVES:

     I. Study the risk of grade 3-4 non-hematologic adverse events in patients with asymptomatic high-risk smoldering multiple myeloma treated with lenalidomide. (Phase II) II. Compare the progression-free survival (PFS) of patients with asymptomatic high-risk smoldering multiple myeloma treated with lenalidomide versus observation alone. (Phase III)

     SECONDARY OBJECTIVES:

     I. Assess the response of patients treated with lenalidomide. (Phase II) II. Determine and compare the response rate, time to progression, 1-year PFS probability, and the overall survival of patients treated with lenalidomide versus observation alone. (Phase III) III. Estimate the incidence of adverse events of these regimens in these patients. (Phase III)

     TERTIARY OBJECTIVES:

     I. Describe the cohort in terms of GEP and cytogenetic risk classification and evaluate baseline immune and MRI parameters. (Phase II) II. Evaluate the impact of therapy within GEP-defined risk groups and GEP as a prognostic marker. (Phase III) III. Study the effects of lenalidomide on laboratory markers of immune function. (Phase III) IV. Study the prognostic value of MRI-detected asymptomatic bone disease on clinical outcome. (Phase III V. Evaluate the prognostic effect of baseline high-risk cytogenetic abnormalities on clinical outcome. (Phase III)

     QUATERNARY OBJECTIVES:

     I. To compare QOL change between treatment and observation arms based on the functional (FWB) and physical (PWB) well-being components of the FACT-General (G) patient-reported outcome (PRO) measure from registration (prior to initiation of treatment) up to cycle 24.

     II. To examine the impact of differential treatment response (PFS), if observed, on QOL based on the FACT FWB+PWB up to cycle 48.

     III. To obtain prospective data on Myeloma specific QOL attributes, utilizing and evaluating the Multiple Myeloma Subscale (MMS).

     OUTLINE: This is a multicenter study.

     PHASE II: Patients receive lenalidomide orally (PO) once daily (QD) on days 1-21. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.

     PHASE III: Patients are randomized to 1 of 2 treatment arms.

     ARM I: Patients receive lenalidomide PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

     ARM II: Patients receive standard of care and undergo observation in the absence of disease progression.

     Peripheral blood, bone marrow aspirate, and bone marrow core biopsy samples are collected for gene expression profiling, immune function, and cytogenetic abnormalities. Patients complete the Functional Assessment of Cancer Therapy (FACT) Physical Well-Being (PWB), and FACT Functional Well-Being (FWB) questionnaires at baseline and periodically during study.

     After completion of study therapy, patients are followed up periodically for 10 years.