Trial Information

A Phase III Randomized, Double-Blind Study of Maintenance Therapy With CC-5013 [NSC # 703813, IND #70116] or Placebo Following Autologous Stem Cell Transplantation for Multiple Myeloma

OBJECTIVES:

Primary

- Compare the efficacy of lenalidomide vs placebo as maintenance therapy after autologous
stem cell transplantation, in terms of prolonging time to disease progression, in
patients with multiple myeloma (arm II closed as of 12/17/09).

Secondary

- Compare the rate of complete response in patients treated with these regimens (arm II
closed as of 12/17/09).

- Compare the progression-free and overall survival of patients treated with these
regimens (arm II closed as of 12/17/09).

- Determine the feasibility of long-term treatment with lenalidomide in these patients.

OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study.

- Peripheral blood stem cell (PBSC) mobilization: Mobilization of autologous peripheral
blood stem cells will be performed according to institutional guidelines. Stem cells
may be collected at any time prior to transplant.

- Autologous PBSC transplantation (PBSCT): Approximately 2-4 weeks after PBSC collection,
patients receive melphalan IV either over 30-60 minutes on day -2 or as a divided dose
over two days on days -3 and -2 or days -2 and -1. Patients undergo autologous PBSCT on
day 0. Patients receive G-CSF SC once daily after day 0 but before day 5 and continuing
until blood counts recover.

- Maintenance therapy*: Approximately 90-100 days after completion of autologous PBSCT,
patients undergo restaging. Patients with disease progression are removed from the
study. Patients with responding or stable disease are stratified according to levels of
β2 microglobulin at baseline (≥ 2.5 mg/dL vs normal), prior thalidomide (yes vs no),
and prior lenalidomide (yes vs no). Patients are randomized to 1 of 2 maintenance
treatment arms (arm II closed as of 12/17/09).

- Arm I: Beginning between day 100-110, patients receive oral lenalidomide once
daily.

- Arm II (closed as of 12/17/09): Beginning between day 100-110, patients receive
oral placebo once daily. Patients who have not yet met the study endpoint receive
oral lenalidomide as in arm I at the discretion of treating physician.

In both arms, treatment continues in the absence of disease progression or unacceptable
toxicity (arm II closed as of 12/17/09) .

NOTE: *The maintenance dose is increased to a maximum dose of 3 pills over 3-6 months.

Treatment assignments were unblinded on 12/17/2009. As of 12/17/2009, no more patients will
be randomized between lenalidomide and placebo.

After completion of study treatment, patients are followed every 3 months for 4 years, every
6 months for 5 years, and then annually for 6 years.

PROJECTED ACCRUAL: A total of 538 will be accrued for this study.