Phase 3
N=239
A Study of Lenalidomide Versus Placebo in Subjects With Transfusion Dependent Anemia in Lower Risk Myelodysplastic Syndrome (MDS) Without Del 5q
Anemia
Bottom Line
View on ClinicalTrials.gov: NCT01029262 ↗Enrolled (actual)
239
Serious AEs
32.6%
Results posted
Jun 2015
Primary outcome: Primary: Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC) — 2.5; 26.9 percentage of participants — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Lenalidomide (Drug); Placebo (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Celgene
- Primary completion
- Mar 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC) |
2.5; 26.9 | <0.001 sig |
| PRIMARY Percentage of Participants With a Erythroid Gene Signature Who Achieved RBC Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC) |
0.0; 7.1 | 1.000 |
| SECONDARY Percentage of Participants Who Achieved RBC Transfusion Independence With a Duration of ≥ 24 Weeks (168 Days) as Determined by the Sponsor |
0.0; 17.5 | < 0.001 sig |
| SECONDARY Kaplan Meier Estimates of Duration of 56-day RBC Transfusion Independence Response as Determined by the Sponsor |
NA; 30.9 | 0.639 |
| SECONDARY Percentage of Participants Who Achieved an Erythroid Response Based on the Modified International Working Group (IWG) 2006 Criteria |
30.4; 38.8 | 0.252 |
| SECONDARY Time to 56-Day RBC-Transfusion-Independent (TI) Response as Determined by the Sponsor |
0.3; 10.1 | — |
| SECONDARY Kaplan Meier Estimates for Progression to Acute Myeloid Leukemia (AML) |
NA; NA | 0.864 |
| SECONDARY Kaplan Meier Estimate for Overall Survival (OS) |
3.0; 3.8 | 0.980 |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAE) |
74; 160; 42; 144; 3; 40 | — |
| SECONDARY Compliance Rates Using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) From Baseline to Week 48 |
88.6; 90; 78.5; 83.8; 80.6; 85.8 | 0.823 |
| SECONDARY Mean Change From Baseline in the EORTC QLQ-C30 Fatigue Domain at Week 12 and 24 |
0.6; 2.4; 7.6; -1.5 | 0.323 |
| SECONDARY Mean Change From Baseline in the EORTC QLQ-C30 Dyspnea Domain at Week 12 and 24 |
0.6; 2.2; 4.3; 1.2 | 0.760 |
| SECONDARY Mean Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain at Week 12 and 24 |
-1.4; -2.1; -5.7; -0.4 | 0.424 |
| SECONDARY Mean Change From Baseline in the EORTC QLQ-C30 Global Health Status/Quality of Life (QOL) Domain at Week 12 and 24 |
-2.1; -1.4; -4.1; -2.4 | 0.746 |
| SECONDARY Mean Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain at Week 12 and 24 |
1.2; -1.4; -7.1; 0.8 | 0.265 |
| SECONDARY Mean Change From Baseline in Fatigue Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 |
-0.464; 3.497; 7.376; 0.196 | 0.2909 |
| SECONDARY Mean Change From Baseline in the Dyspnea Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 |
1.696; 3.374; 5.998; -0.206 | 0.6957 |
| SECONDARY Mean Change From Baseline in the Physical Functioning Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 |
0.732; -2.919; -5.451; -1.484 | 0.3975 |
| SECONDARY Mean Change From Baseline in the Global Health Status/QoL Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 |
-1.201; -2.690; -4.502; -2.441 | 0.6408 |
| SECONDARY Mean Change From Baseline in the Emotional Functioning Domain Associated With the EORTC QLQ-C30 Scale at Weeks 12 and 24 |
1.458; -1.876; -6.746; -1.129 | 0.2848 |
| SECONDARY Percentage of Participants With a Clinically Meaningful Improvement in QOL (EORTC QLQ-C-30 Scale) From Baseline in Fatigue Domain at Weeks 12 and 24 |
30.4; 39.3; 29.8; 38.6 | 0.042 sig |
| SECONDARY Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Dyspnea Domain at Weeks 12 and 24 |
19.6; 21.3; 12.8; 20.5 | 0.825 |
| SECONDARY Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline Within the Physical Functioning Domain at Weeks 12 and 24 |
26.8; 16.4; 12.8; 24.1 | 0.119 |
| SECONDARY Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Global Health Status/QOL Domain at Weeks 12 and 24 |
19.6; 22.1; 14.9; 26.5 | 0.792 |
| SECONDARY Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Emotional Functioning Domain at Weeks 12 and 24 |
25.0; 20.5; 17.0; 21.7 | 0.476 |
| SECONDARY Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Related to Adverse Events Per Person Year |
0.47; 0.77 | — |
| SECONDARY Healthcare Resource Utilization (HRU): Duration of Hospitalizations Due to Adverse Events |
9.0; 11.0 | — |
| SECONDARY Healthcare Resource Utilization (HRU): Number of Days of Hospitalization Due to Adverse Events Per Person-Years |
6.37; 8.92 | — |
Summary
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.
Eligibility Criteria
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) 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
Data sourced from ClinicalTrials.gov (NCT01029262). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.