Lenalidomide or Observation in Treating Patients With Asymptomatic High-Risk Smoldering Multiple Myeloma
This phase II/III trial is studying lenalidomide to see how well it works compared to observation in treating patients with asymptomatic high-risk 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.
Light Chain Deposition Disease
Smoldering Multiple Myeloma
Other: clinical observation
Other: laboratory biomarker analysis
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Randomized Phase III Trial of Lenalidomide Versus Observation Alone in Patients With Asymptomatic High-Risk Smoldering Multiple Myeloma|
- Grade 3 or higher non-hematologic toxicity based on AdEERS expedited reporting (Phase II) [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]The difference in rates of all Grade 3 or higher toxicities will be evaluated for all randomized patients using the Fisher's Exact Test.
- Progression-free survival (PFS) (Phase III) [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]Cox proportional hazards model will be used to assess PFS outcome in multiple regression analyses of established prognostic factors.
- Mean change of the FACT-FWB+PWB score [ Time Frame: Registration (prior to randomization) to 24 months ] [ Designated as safety issue: No ]
- Response rate (complete and partial response) [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]Calculated with a 95% confidence interval.
- Duration of response [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]Estimated using the Kaplan-Meier method.
- Adverse events [ Time Frame: Up to 10 years ] [ Designated as safety issue: Yes ]
- Overall survival (OS) [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]Estimated using the method of Kaplan and Meier. OS between the two arms will be compared using two-sided log-rank test. Cox proportional hazards model will be used to estimate the hazard rate between arms,
- Difference in FACT-FWB+PWB mean scores between patients that experience disease progression (PD) and patients who do not experience PD [ Time Frame: Up to 48 months ] [ Designated as safety issue: No ]Assessed using mixed effects model.
- Sensitivity for patients that did experience PD during the interval with respect to change in QOL in QOL status since last QOL assessment [ Time Frame: Up to 48 months ] [ Designated as safety issue: No ]
|Study Start Date:||October 2010|
|Estimated Primary Completion Date:||April 2014 (Final data collection date for primary outcome measure)|
Experimental: Arm I (lenalidomide)
Patients receive oral lenalidomide once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Other Names:Other: laboratory biomarker analysis
Active Comparator: Arm II (observation)
Patients receive standard of care and undergo observation in the absence of disease progression.
Other: clinical observation
Other Name: observationOther: laboratory biomarker analysis
Show Detailed Description