NCT00843882|Active Not RecruitingPhase 3Results

Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia

Randomized Phase III Trial Comparing the Frequency of Major Erythroid Response (MER) to Treatment With Lenalidomide (Revlimid®)) Alone and in Combination With Epoetin Alfa (Procrit®)) in Subjects With Low- or Intermediate-1 Risk MDS and Symptomatic Anemia

National Cancer Institute (NCI)ClinicalTrials.gov

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8 other identifiers

NCI-2009-01173CDR0000634119ECOG-E290509-0095E2905U10CA021115U10CA180820U24CA196172

Study Type

interventional

Target

247

Locations

1 country

Sites

508

Timeline

RegisteredFeb 2009

StartedJan 2009

CompletionOct 2026

Brief Summary

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

247

participants targeted

Target at P50-P75 for phase_3

Timeline

5mo left

Started Jan 2009

Longer than P75 for phase_3

Geographic Reach

1 country

508 active sites

Status

active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

17.7 years study duration

Study Progress98%

Jan 2009Oct 2026

Study Start

First participant enrolled

January 29, 2009

Completed

14 days until next milestone

First Submitted

Initial submission to the registry

February 12, 2009

Completed

1 day until next milestone

First Posted

Study publicly available on registry

February 13, 2009

Completed

10.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 8, 2019

Completed

2 years until next milestone

Results Posted

Study results publicly available

July 8, 2021

Completed

5.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 2, 2026

Expected

Last Updated

May 1, 2026

Status Verified

April 1, 2026

Enrollment Period

10.4 years

First QC Date

February 12, 2009

Results QC Date

May 12, 2021

Last Update Submit

April 30, 2026

Conditions

Anemia Chronic Myelomonocytic Leukemia De Novo Myelodysplastic Syndrome Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (1)

Proportion of Patients With Major Erythroid Response (MER)
Major erythroid response is defined as transfusion-independence for ≥ 8 consecutive weeks for patients who were red blood cell transfusion-dependent at baseline AND a ≥ 1 g/dL hemoglobin rise compared to mean pre-transfusion baseline value; or a \> 2 g/dL rise in hemoglobin without transfusion for non-transfusion dependent patients.
Assessed after completion of 16 weeks of treatment

Secondary Outcomes (9)

Time to Major Erythroid Response (MER)
Assessed every cycle during treatment and then 3 and 6 months after last protocol treatment
Duration of Major Erythroid Response (MER)
Assessed every cycle during treatment and then 3 and 6 months after last protocol treatment
Proportion of Patients With Major Erythroid Response (MER) to Salvage Combination Therapy
Assessed after completion of 16 weeks of treatment
Proportion of Patients With Minor Erythroid Response
Assessed every cycle during treatment and after completion of 16 weeks of treatment
Proportion of Patients With Major Erythroid Response (MER) Among Those With Chromosome 5q31.1 Deletion
Assessed after completion of 16 weeks of treatment
+4 more secondary outcomes

Other Outcomes (3)

The Association Between CD45 Isoform Profile and ex Vivo Augmentation of STAT5 Phosphorylation by Lenalidomide
Assessed at baseline and after completion of 16 weeks of treatment
RNA and Protein Expression Level of Cdc25C, PP2A and Their Phosphatase Substrates, Cdc2phospho-Tyr15 and Cdc25Cphospho-Ser216
Assessed at baseline and after completion of 16 weeks of treatment
Proportion of Patients With Cryptic Chromosome 5q31.1 Deletions
Assessed at baseline and after completion of 16 weeks of treatment

Study Arms (2)

Arm A (lenalidomide)

ACTIVE COMPARATOR

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.

Procedure: Bone Marrow BiopsyOther: Laboratory Biomarker AnalysisDrug: Lenalidomide

Arm B (lenalidomide, epoetin alfa)

EXPERIMENTAL

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.

Procedure: Bone Marrow BiopsyBiological: Epoetin AlfaOther: Laboratory Biomarker AnalysisDrug: Lenalidomide

Interventions

Bone Marrow BiopsyPROCEDURE

Undergo bone marrow biopsy

Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow

Arm A (lenalidomide)Arm B (lenalidomide, epoetin alfa)

Epoetin AlfaBIOLOGICAL

Given SC

Also known as: EPO, Epoetin alfa-epbx, Epogen, Eprex, Procrit, Retacrit

Arm B (lenalidomide, epoetin alfa)

LenalidomideDRUG

Given PO

Also known as: CC 5013, CC-5013, CC5013, CDC 501, Revlimid

Arm A (lenalidomide)Arm B (lenalidomide, epoetin alfa)

Laboratory Biomarker AnalysisOTHER

Correlative studies

Arm A (lenalidomide)Arm B (lenalidomide, epoetin alfa)

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Age \>= 18 years
NOTE: Results of the bone marrow biopsy and aspirate as well as cytogenetics are mandatory to register subjects onto study, which are indispensable to determine International Prognostic Scoring System (IPSS) category needed for eligibility; please note that it is not necessary to wait for the week 16, week 32, or week bone marrow and cytogenetic results prior to starting the next cycle unless deemed necessary by the treating physician; one example of this exception can include if the subject shows signs of progression, such as increased peripheral blood blast percentage; at that juncture, the treating physician may prefer to await the results prior to starting a new cycle; if a cycle is started, and based on the bone marrow results it is felt by the treating physician that the subject should not continue on treatment, please be sure to note this information on the case report forms at end of treatment
Patient must have documented diagnosis of MDS lasting at least three months (MDS duration \>= 3 months) according to World Health Organization (WHO) criteria or non-proliferative chronic myelomonocytic leukemia (CMML) (white blood cell \[WBC\] \< 12,000/mcL)
Patient must have International Prognostic Scoring System (IPSS) categories of low- or intermediate-1-risk disease; patients must have IPSS score determined by cytogenetic analysis prior to randomization; patients must have cytogenetic analysis done (to calculate IPSS); if the current bone marrow biopsy is a dry tap, patients with cytogenetic failure and \< 10% marrow blasts will be eligible; subjects with cytogenetic failure must have previous cytogenetic results (fluorescence in situ hybridization \[FISH\] is not a substitute) within the last 6 months post last type of MDS treatment (in this case, not referring to growth factors as type of MDS treatment)
Must have symptomatic anemia untransfused with hemoglobin \< 9.5 g/dL =\< 8 weeks prior to randomization or with red blood cells (RBC) transfusion dependence (i.e., \>= 2 units/month) confirmed for =\< 8 weeks before randomization
NOTE: For non-transfusion dependent patients (i.e., receiving \< 2 units/4 weeks x 8 weeks pre-study) who receive periodic transfusions, the mean 8 week pre-transfusion hemoglobin should be used to determine protocol eligibility and response reference
For non-transfusion dependent patients, a minimum of 2 pre-transfusion or un-transfused hemoglobin values are required
Applies only for patients without the deletion 5q 31.1; patients must have failed treatment with an erythropoietic growth factor, or have a low probability of response to rhu-erythropoietin; patients with low probability of response to rhu-erythropoietin or prior erythropoietin failures are defined as follows:
Prior erythropoietin failure-requires a minimum trial of \>= 40,000 units epoetin alfa/week x 8 weeks or equivalent dose of darbepoetin alfa for 8 weeks with failure to achieve transfusion independence in dependent patients or a failure to achieve a \>= 2 g rise in hemoglobin sustained for \>= 4 weeks in non-transfusion dependent patients
Low erythropoietin response profile-rhu-erythropoietin and epoetin alfa-naïve patients receiving \>= 2U packed (p)RBC/month for a minimum of 8 weeks, and serum erythropoietin \> 500 mU/mL in the 8 weeks prior to randomization for a hemoglobin \< 9.5 g/dL
Patients must be off all non-transfusion therapy for MDS for 28 days prior to initiation of study treatment, including all types of growth factors; patients may receive hydrocortisone prophylactically to prevent transfusion reactions
Patients must have a serum erythropoietin level documented before randomization and =\< 56 days before day 1 of study treatment; NOTE: hemoglobin must be \< 9.5 g/dL at time that serum erythropoietin is drawn
Effective contraception must be used by patients participating in lenalidomide therapy, and all patients must agree to counseling by a trained counselor every 28 days about pregnancy precautions and risks of fetal exposure; females of childbearing potential (FCBP) must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control: one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before starting lenalidomide, during lenalidomide therapy, during dose interruptions, and for at least 28 days following discontinuation of lenalidomide therapy; females of childbearing potential should be referred to a qualified provider of contraceptive methods, if needed; males receiving lenalidomide must agree to use a latex condom during any sexual contact with females of childbearing potential even if they have undergone a successful vasectomy
Platelet count \>= 50,000/mcL (50 x 10\^9/L) without platelet transfusion (within 56 days prior to randomization)
Absolute neutrophil count (ANC) \>= 500 cells/mcL (0.5 x 10\^9/L); hence ANC must be \>= 500/mcL without myeloid growth factor support (within 56 days prior to randomization)
+5 more criteria

You may not qualify if:

Patients must not have documented iron deficiency; all patients must have documented marrow iron stores; if marrow iron stain is not available, the transferrin saturation must be \> 20% or a serum ferritin \> 100 ng/mL
Women must not be pregnant or breastfeeding; females of childbearing potential must have a negative serum or urine pregnancy test with a sensitivity of at least 25 mIU/mL within 10-14 days and again within 24 hours prior to starting cycle 1 of lenalidomide; a female of childbearing potential (FCBP) is any woman, regardless of sexual orientation or whether they have undergone tubal ligation, who meets the following criteria: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months; FCBP must also agree to ongoing pregnancy testing)
Patients must not have prior therapy with lenalidomide
Patients must not have a diagnosis of uncontrolled seizure or uncontrolled hypertension
Patients must not have proliferative (WBC \>= 12,000/mcL) chronic myelomonocytic leukemia (CMML); WBC must be \< 12,000/mcL
Patients must not have MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
Prior thalidomide therapy is allowed, however, patients must not have prior \>= grade-3 allergic reactions to thalidomide
Patients must not have prior history of desquamating rash from thalidomide at time of study entry
Patients must not have clinically significant anemia resulting from iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis, or gastrointestinal bleeding
Patients must not have used cytotoxic chemotherapeutic agents or experimental agents (agents that are not commercially available) for the treatment of MDS within 8 weeks of randomization
Patients must not have prior history of malignancy other than MDS (except basal cell or squamous skin cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been confirmed free of disease for \>= 3 years
Patients must not have any serious medical condition or any other unstable medical co-morbidity, or psychiatric illness that will prevent the subject from signing the informed consent form or will place the subject at unacceptable risk if he/she participates in the study
Patients must not have a history of thrombo-embolic events within 3 years prior to study randomization
Patients must not have known human immunodeficiency virus (HIV)-1 seropositivity because HIV can be an alternate cause of anemia.
Patients must not have a known allergic reaction to epoetin alfa (Procrit) or human serum albumin
+1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

National Cancer Institute (NCI)lead

Study Sites (508)

University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States

Location

Alaska Breast Care and Surgery LLC