A Study of Lenalidomide Versus Placebo in Subjects With Transfusion Dependent Anemia in Lower Risk Myelodysplastic Syndrome (MDS) Without Del 5q (MDS-005)

Celgene

Status and phase: Completed; Phase 3

Conditions: Anemia

Treatments: Other: Placebo; Drug: Lenalidomide

Study type: Interventional

Funder types: Industry

Identifiers

NCT01029262

CC-5013-MDS-005

Details and patient eligibility

About

The purpose of this study is to investigate whether lenalidomide would reduce the number of red blood cell transfusions (RBC) needed in anemic (RBC transfusion-dependent) participants with low or intermediate-1 risk MDS without a deletion 5q chromosome abnormality. The study also investigated the safety of lenalidomide use in these participants. Two-thirds of the participants received oral lenalidomide and one-third of the participants received oral placebo.

Enrollment: 239 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 18 years or older
• Diagnosis of low or intermediate-1 risk Myelodysplastic (MDS) with any chromosome karyotype except del 5q[31]
• Anemia that requires red blood cell transfusions
• Resistant to erythropoiesis stimulating agents (ESAs) or blood erythropoietin level > 500 mU/mL
• Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) ≤ 2
• Must agree to follow pregnancy precautions as required by the protocol.
• Must agree to receive counseling related to teratogenic and other risks of lenalidomide
• Must agree not to donate blood or semen
• Must be willing to consent to two or more bone marrow aspirate procedures to be completed during study

Exclusion criteria

• Subjects previously receiving immunomodulating or immunosuppressive agents, or epigenetic or deoxyribonucleic acid (DNA) modulation agents
• Allergic reaction to thalidomide
• Renal insufficiency creatinine clearance (CrC1)<40 mL/min by Cockcroft-Gault method)
• Prior history of cancer, other than MDS, unless the subject has been free of the disease for ≥ 5 years. (Basal cell carcinoma of the skin, carcinoma in situ of the cervix, or stage Tumor (T) 1a or T1b prostate cancer is allowed)
• Absolute neutrophil count (ANC) < 500/uL
• Platelets < 50,000/uL
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3X upper limit of normal
• Uncontrolled hyperthyroidism or hypothyroidism
• Significant neuropathy
• Prior stem cell transplantation
• Anemia due to reasons other than MDS
• History of deep venous thrombosis (DVT) or pulmonary embolus (PE) within past 3 years
• Significant active cardiac disease within the past 6 months
• Known Human Immunodeficiency Virus (HIV) infection; known Hepatitis C infection or active Hepatitis B infection

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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239 participants in 2 patient groups, including a placebo group

Arm #1 - Lenalidomide plus placebo

Experimental group

Description:

Lenalidomide 10 mg by mouth (PO) daily plus 2 placebo capsules for participants with a creatinine clearance ≥ 60 mL/min for at least 168 days until disease progression, intolerable side effects or withdrawal of consent. Lenalidomide 5 mg PO daily plus 2 placebo capsules for participants with a creatinine clearance ≥ 40 and \< 60 mL/min.

Treatment:

Drug: Lenalidomide

Arm #2 - placebo

Placebo Comparator group

Description:

Three placebo capsules once daily for at least 168 days until disease progression occurred, intolerable side effects or withdrawal of consent.

Treatment:

Other: Placebo