Phase 2
N=52
Phase II Study of Lenalidomide and Eltrombopag in Patients With Symptomatic Anemia
Adult Myelodysplastic Syndrome · Anemia · Chronic Myelomonocytic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT01772420 ↗Enrolled (actual)
52
Serious AEs
25.0%
Results posted
Jul 2023
Primary outcome: Primary: Number of Patients Demonstrating Overall Hematologic Improvement (HI) — 6; 5; 7 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Eltrombopag Olamine (Drug); Laboratory Biomarker Analysis (Other); Lenalidomide (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Albert Einstein College of Medicine
- Primary completion
- Jul 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients Demonstrating Overall Hematologic Improvement (HI) |
6; 5; 7 | — |
| SECONDARY Number of Patients With Hematologic Improvement in Platelet Counts (HI-P) |
0; 3; 6 | — |
| SECONDARY Number of Patients With Hematologic Improvement in Erythrocyte Counts (HI-E) |
6; 3; 4 | — |
| SECONDARY Number of Patients With Hematologic Improvement in Neutrophil Counts (HI-N) |
0; 1; 2 | — |
| SECONDARY Time to Attain Hematologic Improvement (HI) |
12; 8; 8 | — |
| SECONDARY Duration of Hematologic Improvement (HI) |
41; 88; 40 | — |
| SECONDARY Number of Patients With Clinically Significant Bleeding Events |
0; 2; 0 | — |
Summary
This phase II trial studies how well lenalidomide (LEN) and eltrombopag olamine (ELT) work in treating patients with symptomatic anemia in low or intermediate myelodysplastic syndrome (MDS). Lenalidomide may stimulate the immune system in different ways and stop cancer cells from growing. Eltrombopag olamine may increase the number of white blood cells and platelets found in bone marrow or peripheral blood. Giving lenalidomide and eltrombopag olamine may be an effective treatment for myelodysplastic syndrome.
Eligibility Criteria
Inclusion Criteria
- Patient must have a documented diagnosis of myelodysplastic syndrome (MDS) of at least three months duration (MDS duration >= 3 months) according to World Health Organization (WHO) criteria or non-proliferative chronic myelomonocytic leukemia (CMML) (white blood cells [WBC] = = 2 units/month) confirmed for a minimum of 8 weeks before randomization
- Patients must have IPSS score determined by cytogenetic analysis prior to randomization; patients with cytogenetic failure and = = 20% or a serum ferritin >= 100 ng/100 mL or soluble transferring receptor = 500 cells/L (0.5 x 10^9/L)
- Eastern Cooperative Oncology Group (ECOG) performance 0-3
- Subject is able to understand and comply with protocol requirements and instructions
- Patient has signed and dated informed consent
- Prothrombin time (PT/international normalized ratio [INR]) and activated partial thromboplastin time (aPTT) must be within 80 to 120% of the normal range at baseline
Exclusion Criteria
- Pre-existing cardiovascular disease (including congestive heart failure, New York Heart Association [NYHA] grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. atrial fibrillation), or subjects with a corrected QT interval (QTc) > 450 msec
- Patients determined to be at increased risk of arterial or venous thrombosis by the investigator
- Bone marrow fibrosis that leads to a dry tap
- Female subjects who are nursing or pregnant (positive serum or urine beta-human chorionic gonadotropin (beta-hCG) pregnancy test) at screening or pre-dose on day 1
- Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication
- Patients with documented liver cirrhosis
- Patients with splenomegaly with a spleen size > 16 cm
Data sourced from ClinicalTrials.gov (NCT01772420). 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.