Ignite Creation Date:
2025-12-25 @ 4:17 AM
Ignite Modification Date:
2025-12-26 @ 3:17 AM
Study NCT ID:
NCT01772420
Status:
COMPLETED
Last Update Posted:
2023-07-27
First Post:
2013-01-15
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Phase II Study of Lenalidomide and Eltrombopag in Patients With Symptomatic Anemia
Sponsor:
Albert Einstein College of Medicine
Organization:
Study Overview
Official Title:
Phase II Study of Lenalidomide and Eltrombopag in Patients With Symptomatic Anemia in Low or Intermediate I Myelodysplastic Syndrome (MDS)
Status:
COMPLETED
Status Verified Date:
2023-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Detailed Description:
PRIMARY OBJECTIVES (not Outcome Measures):
I. To evaluate the rate of hematologic improvement of the eltrombopag (eltrombopag olamine)/lenalidomide combination (as per Modified International Working Group \[IWG\] criteria).
II. To evaluate the safety and tolerability of the combination.
SECONDARY OBJECTIVES (not Outcome Measures):
I. To compare the time to hematologic improvement. II. To evaluate the duration of hematologic improvement III. To evaluate the effect of combination treatment on platelet counts, platelet transfusions and bleeding events.
IV. To evaluate the frequency of bone marrow response (complete response \[CR\] + partial response \[PR\]) and cytogenetic response.
V. To evaluate the relationship between mutations in bone marrow stem cells and response.
VI. To evaluate the relationship between various stem and progenitor alterations and response.
OUTLINE: Patients are initially assigned to 1 of 2 treatment arms.
ARM A: Patients with platelet counts \>= 50,000 receive lenalidomide orally (PO) daily or every other day (QOD) on days 1-21. If platelet counts fall below 50,000, patients discontinue lenalidomide and receive eltrombopag olamine PO daily or QOD until platelet count is maintained above 50,000 for 2 weeks. Patients then resume lenalidomide PO daily or QOD. If platelets fall below 50,000 again, patients receive eltrombopag olamine as before. When platelet counts are maintained above 50,000 for 2 weeks, patients resume lenalidomide concurrently with eltrombopag for all subsequent courses.
ARM B: Patients with platelet counts \< 50,000 receive eltrombopag olamine PO daily or QOD on days 1-28 until platelet counts is maintained above 50,000 for 2 weeks. Patients then receive treatment as in Arm A.
In both arms, treatment repeats every 28 days for 4 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3 months for 2 years, every 6 months for 3 years, and then every 12 months for 5 years.
Eligible patients with a diagnosis of MDS or non-proliferative chronic myelomonocytic leukemia (CMML) (WBC ≤ 12,000/mL) of at least 3-month duration according to WHO criteria and International Prognostic Scoring System categories of low or intermediate-1-risk disease. Patients either had symptomatic anemia untransfused with hemoglobin ≤ 10 g/dL in the 8 weeks before starting the study or had RBC transfusion dependence (i.e., ≥ 2 units/mo) confirmed 8 weeks before starting the study and/or PLTs \<50,000 k/uL with hemoglobin \>10.0 g/dL. Patients must not have received prior therapy with LEN (for \> 2 months) nor ELT
I. To evaluate the rate of hematologic improvement of the eltrombopag (eltrombopag olamine)/lenalidomide combination (as per Modified International Working Group \[IWG\] criteria).
II. To evaluate the safety and tolerability of the combination.
SECONDARY OBJECTIVES (not Outcome Measures):
I. To compare the time to hematologic improvement. II. To evaluate the duration of hematologic improvement III. To evaluate the effect of combination treatment on platelet counts, platelet transfusions and bleeding events.
IV. To evaluate the frequency of bone marrow response (complete response \[CR\] + partial response \[PR\]) and cytogenetic response.
V. To evaluate the relationship between mutations in bone marrow stem cells and response.
VI. To evaluate the relationship between various stem and progenitor alterations and response.
OUTLINE: Patients are initially assigned to 1 of 2 treatment arms.
ARM A: Patients with platelet counts \>= 50,000 receive lenalidomide orally (PO) daily or every other day (QOD) on days 1-21. If platelet counts fall below 50,000, patients discontinue lenalidomide and receive eltrombopag olamine PO daily or QOD until platelet count is maintained above 50,000 for 2 weeks. Patients then resume lenalidomide PO daily or QOD. If platelets fall below 50,000 again, patients receive eltrombopag olamine as before. When platelet counts are maintained above 50,000 for 2 weeks, patients resume lenalidomide concurrently with eltrombopag for all subsequent courses.
ARM B: Patients with platelet counts \< 50,000 receive eltrombopag olamine PO daily or QOD on days 1-28 until platelet counts is maintained above 50,000 for 2 weeks. Patients then receive treatment as in Arm A.
In both arms, treatment repeats every 28 days for 4 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3 months for 2 years, every 6 months for 3 years, and then every 12 months for 5 years.
Eligible patients with a diagnosis of MDS or non-proliferative chronic myelomonocytic leukemia (CMML) (WBC ≤ 12,000/mL) of at least 3-month duration according to WHO criteria and International Prognostic Scoring System categories of low or intermediate-1-risk disease. Patients either had symptomatic anemia untransfused with hemoglobin ≤ 10 g/dL in the 8 weeks before starting the study or had RBC transfusion dependence (i.e., ≥ 2 units/mo) confirmed 8 weeks before starting the study and/or PLTs \<50,000 k/uL with hemoglobin \>10.0 g/dL. Patients must not have received prior therapy with LEN (for \> 2 months) nor ELT
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| NCI-2013-01219 | REGISTRY | CTRP (Clinical Trial Reporting Program) | View | |
| P30CA013330 | NIH | None | https://reporter.nih.gov/quic… | View |
| 115479 | OTHER | GlaxoSmithKline Tracking Number | View | |
| RV--MDS-PI-0645 | OTHER | Celgene Tracking Number | View |