NCT00843882

Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia

Randomized Phase III Trial Comparing the Frequency of Major Erythroid Response (MER) to Treatment With Lenalidomide (Revlimid®)) Alone and in Combination With Epoetin Alfa (Procrit®)) in Subjects With Low- or Intermediate-1 Risk MDS and Symptomatic Anemia

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 247 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This randomized phase III trial studies lenalidomide to see how well it works with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia. Lenalidomide may stop the growth of myelodysplastic syndrome by blocking blood flow to the cells. Colony stimulating factors, such as epoetin alfa, may increase the number of immune cells found in bone marrow or peripheral blood. It is not yet known whether lenalidomide is more effective with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia.

Detailed description

PRIMARY OBJECTIVE: I. To compare the rate of major erythroid response (MER) between lenalidomide monotherapy and combined treatment of lenalidomide and epoetin alfa in erythropoietin non-responsive low-/intermediate-1 (Int-1)-risk myelodysplastic syndrome (MDS) patients or erythropoietin treatment naïve patients with low probability of erythropoietin benefit. SECONDARY OBJECTIVES: I. To compare the time to MER by treatment assignment. II. To evaluate the duration of MER by treatment assignment. III. To estimate the frequency of MER to salvage combination therapy in patients who fail to experience a MER with lenalidomide monotherapy. IV. To evaluate and compare the frequency of minor erythroid response by treatment assignment. V. To investigate the mechanism and target of lenalidomide action in patients with chromosome 5q31.1 deletion. VI. To evaluate the frequency of cytogenetic response and progression, and the relation between cytogenetic pattern and erythroid response. VII. To evaluate the frequency of bone marrow response (complete response \[CR\] + partial response \[PR\]). VIII. To evaluate the relationship between erythroid response and laboratory correlates outlined below: VIIIa. Pretreatment and on study endogenous erythropoietin level (Arm A). VIIIb. To evaluate the effect of CD45 isoform profile on lenalidomide enhancement of erythropoietin-induced STAT5 phosphorylation in CD71\^Hi erythroid precursors and the relationship to erythroid response. VIIIc. To characterize molecular targets relevant to lenalidomide cytotoxicity in del5q31.1 cells. VIIId. To evaluate the frequency of cryptic chromosome 5q31.1 deletions in patients with non-del5q31.1 MDS by array-based genomic scan, and to determine the relationship to hematologic response. OUTLINE: Patients are randomized to 1 of 2 treatment arms. Patients with del 5q31.1 karyotype are assigned to Arm A. ARM A: Patients receive lenalidomide orally (PO) once daily (QD) on days 1-21. Patients undergo bone marrow biopsy at screening and during follow-up. Patients undergo blood specimen collection on study. ARM B: Patients receive lenalidomide PO QD on days 1-21 and epoetin alfa subcutaneously (SC) once weekly. Patients undergo bone marrow biopsy at screening and during follow-up. Patients undergo blood specimen collection on study. In both arms, treatment repeats every 28 days for 4 cycles. Patients who achieve a major erythroid response (MER) may continue treatment beyond 4 cycles in the absence of disease progression, disease conversion to acute myeloid leukemia, or unacceptable toxicity. Patients in Arm A who fail to achieve MER or who achieve MER but relapse after 16 weeks of treatment with lenalidomide may crossover and receive treatment in Arm B. After completion of study treatment, patients are followed up for 6 months.

Conditions

• Anemia
• Chronic Myelomonocytic Leukemia
• De Novo Myelodysplastic Syndrome
• Myelodysplastic Syndrome

Interventions

Type	Name	Description
PROCEDURE	Bone Marrow Biopsy	Undergo bone marrow biopsy
BIOLOGICAL	Epoetin Alfa	Given SC
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Lenalidomide	Given PO

Timeline

• Start date: 2009-01-29
• Primary completion: 2019-07-08
• Completion: 2026-10-02
• First posted: 2009-02-13
• Last updated: 2026-04-13
• Results posted: 2021-07-08

Locations

508 sites across 1 country: United States