Clinical Trial: Lenalidomide or Observation in Treating Patients With Asymptomatic High-Risk Smoldering Multiple Myeloma

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Randomized Phase III Trial of Lenalidomide Versus Observation Alone in Patients With Asymptomatic High-Risk Smoldering Multiple Myeloma

Brief Summary: This randomized phase II/III trial studies how well lenalidomide works and compares it to observation in treating patients with asymptomatic high-risk asymptomatic (smoldering) multiple myeloma. Biological therapies such as lenalidomide, may stimulate the immune system in different ways and stop cancer cells from growing. Sometimes the cancer may not need treatment until it progresses. In this case, observation may be sufficient. It is not yet known whether lenalidomide is effective in treating patients with high-risk smoldering multiple myeloma than observation alone.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To study the risk of grade 3 adverse events that effect vital organ function (such as cardiac, hepatic or thromboembolic) or any grade 4 or higher non-hematologic adverse events among patients receiving lenalidomide as treatment for high-risk asymptomatic, smoldering multiple myeloma. (Phase II) II. To compare progression free survival where failure is defined as death or the development of symptomatic myeloma indicating treatment between patients receiving lenalidomide versus observation alone in high-risk asymptomatic, smoldering multiple myeloma. (Phase III)

SECONDARY OBJECTIVES:

I. To assess the response to therapy of patients treated with lenalidomide as treatment for asymptomatic, smoldering multiple myeloma. (Phase II) II. To determine and compare the response rate, time to progression, 1-year progression-free survival probability, and overall survival between patients randomized to receive lenalidomide or observation in the setting of asymptomatic myeloma. (Phase III) III. To estimate the incidence of adverse events in patients receiving lenalidomide therapy for early-stage multiple myeloma. (Phase III)

TERTIARY OBJECTIVES:

I. To describe the cohort in terms of gene expression profiling (GEP) and cytogenetic risk classification and evaluate baseline immune and magnetic resonance imaging (MRI) parameters. (Phase II) II. To evaluate the impact of therapy within GEP-defined risk groups and GEP as a prognostic marker. (Phase III) III. To study the effects of lenalidomide on laboratory markers of immune function. (Phase III) IV. To study the prognostic value of MRI-detected asymptomatic bone disease on clinical outcome. (Phase III) V. To evaluate the
Sponsor: National Cancer Institute (NCI)

Current Primary Outcome:

  • Incidence of grade 3 or higher toxicities based on Cancer Therapy Evaluation Program-Adverse Event Reporting System expedited reporting (Phase II) [ Time Frame: Up to 6 months ]
  • PFS (Phase III) [ Time Frame: Up to 10 years ]
    PFS in each of the arms will be estimated using the method of Kaplan and Meier. PFS between the two arms will be compared using a stratified log-rank test. Cox proportional hazards model will be used to assess PFS outcome in multiple regression analyses of established prognostic factors.


Original Primary Outcome: Progression-free survival

Current Secondary Outcome:

  • Duration of response [ Time Frame: Up to 10 years ]
    Estimated using the Kaplan-Meier method.
  • Incidence of adverse events graded according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 [ Time Frame: Up to 10 years ]
    Adverse events will be monitored on all patients and summarized by arm. The difference in rates of all grade 3 or higher toxicities will be evaluated for all randomized patients using the Fisher's Exact test.
  • Overall survival (OS) [ Time Frame: Up to 10 years ]
    OS in each of the arms estimated using the method of Kaplan and Meier. OS between the two arms will be compared using stratified log-rank test. Cox proportional hazards model will be used to estimate hazard rate between arms.
  • Response rate (complete and partial response) [ Time Frame: Up to 10 years ]
    Calculated with a 95% confidence interval.


Original Secondary Outcome:

  • Response rate (complete and partial response)
  • Duration of response
  • Adverse events
  • Overall survival
  • Quality of life
  • Gene expression profiling
  • Markers of immune function


Information By: National Cancer Institute (NCI)

Dates:
Date Received: July 23, 2010
Date Started: October 2010
Date Completion:
Last Updated: May 19, 2017
Last Verified: May 2017