Lenalidomide +/− Epoetin Alfa for Myelodysplastic Syndrome

Recruiting at 577 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: National Cancer Institute (NCI)

Must be taking: Lenalidomide

Must not be taking: Cytotoxic agents

Disqualifiers: Pregnancy, Uncontrolled hypertension, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 7

Duration: 16 weeks

247 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that participants stop all non-transfusion therapy for myelodysplastic syndrome (MDS) for 28 days before starting the study treatment. This includes all types of growth factors.

Is the combination of Lenalidomide and Epoetin Alfa safe for treating myelodysplastic syndrome?

Lenalidomide is generally considered safe for treating certain types of myelodysplastic syndrome, but it can cause side effects like low blood cell counts, which are usually managed by adjusting the dose. There is a risk of blood clots when used with erythropoiesis-stimulating agents like Epoetin Alfa, so monitoring is important. ^{1 2 3 4 5}

What makes the drug Lenalidomide unique for treating myelodysplastic syndrome?

Lenalidomide is unique because it is an oral drug that not only helps improve anemia in patients with myelodysplastic syndrome, especially those with a specific chromosome abnormality (5q deletion), but also has anti-cancer and anti-inflammatory properties. It can be used alone or with epoetin alfa to enhance the body's response to anemia treatment. ^{2 5 6 7 8}

What data supports the effectiveness of the drug Lenalidomide combined with Epoetin Alfa for treating myelodysplastic syndrome?

Research shows that Lenalidomide can improve hemoglobin levels and reduce the need for blood transfusions in patients with certain types of myelodysplastic syndrome (MDS), especially those with a specific chromosome abnormality (5q deletion). Combining it with Epoetin Alfa, which helps stimulate red blood cell production, may enhance these benefits for patients with anemia due to MDS. ^{2 7 8 9 10}

Who Is on the Research Team?

Amit K Verma

Principal Investigator

ECOG-ACRIN Cancer Research Group

Are You a Good Fit for This Trial?

Adults diagnosed with low- or intermediate-1-risk myelodysplastic syndrome (MDS) and anemia, who have not responded to erythropoietin treatment or are unlikely to respond. Participants must have adequate organ function, no severe allergies to thalidomide, no recent malignancies other than MDS, and not be pregnant. They should also not have had any MDS treatments in the last 28 days.

Inclusion Criteria

I haven't received any non-transfusion treatments for MDS in the last 28 days.

I have been diagnosed with MDS or CMML for at least 3 months.

I have been diagnosed with MDS or CMML with low white blood cell count for 3 months or more.

Exclusion Criteria

Women pregnant or breastfeeding

Serious medical condition, unstable medical co-morbidity, or psychiatric illness preventing informed consent or placing subject at unacceptable risk

I have been treated with lenalidomide before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive lenalidomide with or without epoetin alfa for 4 cycles of 28 days each

16 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

2 visits (in-person)

Extension

Participants who achieve a major erythroid response may continue treatment beyond 4 cycles

Variable

What Are the Treatments Tested in This Trial?

Interventions

• Epoetin Alfa
• Lenalidomide

Trial Overview The trial is testing whether lenalidomide works better alone or combined with epoetin alfa for treating anemia in patients with MDS. Lenalidomide may inhibit tumor growth by cutting off blood supply to cancer cells while epoetin alfa could boost immune cell numbers.

How Is the Trial Designed?

Group I: Arm B (lenalidomide, epoetin alfa) Experimental Treatment 4 Interventions

Patients receive lenalidomide PO QD on days 1-21 and epoetin alfa SC once weekly. Patients undergo bone marrow biopsy at screening and during follow-up. Patients undergo blood specimen collection on study.

Group II: Arm A (lenalidomide) Active Control 3 Interventions

Patients receive lenalidomide PO QD on days 1-21. Patients undergo bone marrow biopsy at screening and during follow-up. Patients undergo blood specimen collection on study.

Lenalidomide is already approved in European Union, United States for the following indications:

Approved in European Union as Revlimid for:

• Multiple myeloma
• Myelodysplastic syndromes
• Mantle cell lymphoma
• Follicular lymphoma
• Marginal zone lymphoma

Approved in United States as Revlimid for:

• Multiple myeloma
• Myelodysplastic syndromes
• Mantle cell lymphoma
• Follicular lymphoma
• Marginal zone lymphoma

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Published Research Related to This Trial

In a phase III trial with 195 patients suffering from lower-risk, non-del(5q) myelodysplastic syndromes (MDS), the combination of lenalidomide (LEN) and epoetin (EPO) alfa resulted in a significantly higher major erythroid response (28.3%) compared to LEN alone (11.5%).

The combination treatment not only increased the response rate but also provided a longer duration of response, with a median major erythroid response duration of 23.8 months for the LEN-EPO group compared to 13 months for LEN alone.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lenalidomide-Epoetin Alfa Versus Lenalidomide Monotherapy in Myelodysplastic Syndromes Refractory to Recombinant Erythropoietin. List, AF., Sun, Z., Verma, A., et al.[2022]

Lenalidomide has become a crucial treatment option for multiple myeloma and del-5q myelodysplastic syndromes, and it has been tested for various other cancers, showcasing its broad therapeutic potential.

The drug received FDA approval for use in combination with dexamethasone for previously treated multiple myeloma, highlighting its efficacy and safety in this specific patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lenalidomide: a novel anticancer drug with multiple modalities. Galustian, C., Dalgleish, A.[2019]

Lenalidomide is an effective treatment for transfusion-dependent anemia in lower-risk myelodysplastic syndromes (MDS), particularly in patients with the deletion 5q chromosomal abnormality, but it also shows clinical activity in those without this specific abnormality.

The use of lenalidomide in MDS exemplifies reciprocal translational research, where laboratory findings informed clinical applications, enhancing our understanding of both the drug's mechanisms of action and the biology of MDS.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lenalidomide in myelodysplastic syndromes: an erythropoiesis-stimulating agent or more? Komrokji, RS., Lancet, JE., List, AF.[2021]

Lenalidomide (REVLIMID®) is an effective treatment for multiple myeloma and transfusion-dependent anemia in myelodysplastic syndromes, showing better activity and less neurotoxicity compared to its predecessor, thalidomide.

This drug has multiple beneficial properties, including anti-inflammatory and antiangiogenic effects, and may play a significant role in treating various cancers beyond its current approved uses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The potential of immunomodulatory drugs in the treatment of solid tumors. Dalgleish, A., Galustian, C.[2018]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Lenalidomide-Epoetin Alfa Versus Lenalidomide Monotherapy in Myelodysplastic Syndromes Refractory to Recombinant Erythropoietin. [2022]

2. England pubmed.ncbi.nlm.nih.gov

A decade of progress in myelodysplastic syndrome with chromosome 5q deletion. [2020]

3. England pubmed.ncbi.nlm.nih.gov

Lenalidomide: a novel anticancer drug with multiple modalities. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Lenalidomide in myelodysplastic syndromes: an erythropoiesis-stimulating agent or more? [2021]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Lenalidomide: a review of its use in patients with transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndrome associated with 5q chromosome deletion. [2021]

6. Italy pubmed.ncbi.nlm.nih.gov

Treatment with lenalidomide does not appear to increase the risk of progression in lower risk myelodysplastic syndromes with 5q deletion. A comparative analysis by the Groupe Francophone des Myelodysplasies. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Lenalidomide as a novel treatment of acute myeloid leukemia. [2023]

8. New Zealand pubmed.ncbi.nlm.nih.gov

Venous thromboembolism in myelodysplastic syndrome patients receiving lenalidomide: results from postmarketing surveillance and data mining techniques. [2022]

9. England pubmed.ncbi.nlm.nih.gov

The evolving role of lenalidomide in the treatment of hematologic malignancies. [2019]

10. England pubmed.ncbi.nlm.nih.gov

The potential of immunomodulatory drugs in the treatment of solid tumors. [2018]