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CompletedNCT00546897Updated Sep 29, 2014Results posted

Lenalidomide in Older Patients With Acute Myeloid Leukemia Without Chromosome 5q Abnormalities

A Phase 2 interventional study of Lenalidomide in Leukemia, Myeloid, Acute, sponsored by Washington University School of Medicine. Completed at 1 site in United States. Open to participants aged 60 Years and older. Per ClinicalTrials.gov, last updated 2014-09-29.

Sponsored by Washington University School of Medicine · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
48
Allocation
Non-randomized
Ages
60 Years and older
Sex
All
01

Study summary

This study is designed to test the safety and efficacy of lenalidomide in older patients (age > 60 years) with untreated acute myeloid leukemia without chromosomal abnormalities involving 5q.

Read the detailed description

The incidence of AML increases with age, and current treatment options for the older patient population with newly diagnosed AML (AML >= 60) is limited, all with poor outcomes. AML >= 60 patients are more likely to have poor-risk cytogenetics abnormalities, and many have a preceding myelodysplastic syndrome (MDS). Traditional induction chemotherapy approaches in AML with cytarabine and anthracyclines yield remissions in 45-60% of AML >= 60, however the vast majority of these patients relapse with a median survival of about 9 months. These patients are rarely candidates for potentially curative allogeneic stem cell transplantation. Many untreated AML >= 60 patients are not candidates for aggressive therapy, and those who do receive therapy have a significant induction mortality of 10-20%, and significant hematologic toxicity occurs in over 30%, with no change in overall survival compared with supportive care. AML >= 60 patients with favorable risk cytogenetics have a modest improvement in prognosis, for example with a 5 year overall survival of \~20%, compared with 0% in other cytogenetic categories. Thus, all eligible patients with AML >= 60 should be recommended a clinical trial, regardless of whether they would be offered generally ineffective traditional induction chemotherapy. More effective and less toxic therapies are needed for the treatment of AML in this older patient population, indeed the preferred first line therapy in the national cancer center network (NCCN) guidelines for AML is a clinical trial.

In trials of lenalidomide in patients with MDS the dose of lenalidomide has been reduced for myelotoxicity and/or thrombocytopenia. However, current paradigms for the therapy of acute myeloid leukemia are based on using high doses of myelosuppressive chemotherapy and supporting the patient through a 4-5 weeks period of neutropenia/thrombocytopenia in an attempt to eliminate the malignant clone. Based on its efficacy in the related myeloid disorder MDS, and the close relationship between MDS and AML in patients > 60, this trial employs the same paradigm of myelosuppressive therapy using high dose lenalidomide instead of chemotherapy. Importantly, within the MDS trials using low doses of lenalidomide, responses were observed in 3/9 (33%) of patients with excess blasts (RAEB/RAEB-t), which are now classified as evolving into AML or AML. This suggests that the therapeutic effect of lenalidomide occurs in the setting of a large percentage of blasts, such as AML, although the dose and schedule of lenalidomide administration is different. The response of AML >= 60 patients to the proposed high dose lenalidomide regimen is unknown. Following high dose lenalidomide, in those patients that have a response, we propose using a lower dose maintenance strategy similar to the FDA approved dosing for MDS. The maintenance phase will include standard dose reductions for unacceptable toxicities.

02

Conditions studied

  • Leukemia, Myeloid, Acute
03

In context

Leukemia

5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.

This study's enrollment of 48 is above the median of 38 across 4,247 interventional studies indexed under Leukemia.

Browse Leukemia studies →

Lead sponsor

Washington University School of Medicine is the lead sponsor of 1,765 studies on the registry; 271 are open to participants now.

Of its 324 completed or terminated interventional studies of FDA-regulated products, 212 (65%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
60 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • AML, de novo, secondary to prior therapy, or transformed from MDS, as defined by the International Working Group (except acute promyelocytic leukemia (AML M3). Patients must not have abnormalities of chromosome 5q as assessed by routine cytogenetics or FISH. Diagnosis of AML by WHO criteria (≥20% blasts) is determined by CBC, bone marrow assessment, and immunophenotypic analysis performed within 2 weeks of study enrollment.
  • Intermediate or poor-risk cytogenetics as defined by SWOG criteria
  • Age ≥ 60 years at the time of signing the informed consent form.
  • Understand and voluntarily sign an informed consent form.
  • Able to adhere to the study visit schedule and other protocol requirements.
  • No previous treatment for AML, however hydroxyurea, steroids, and leukopheresis are allowed
  • ECOG performance status of ≤ 2 at study entry.
  • Life expectancy > 2 months
  • Adequate organ function as defined by:
  • Serum creatinine ≤ 1.5X institution upper limit of normal (ULN)
  • Total bilirubin ≤ 2.0 mg/dL
  • AST (SGOT) and ALT (SGPT) ≤ 5 x ULN
  • Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 10 - 14 days prior to and again within 24 hours of starting lenalidomide and 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 she starts taking lenalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy. All patients must be counseled at a minimum of every 28 days about pregnancy precautions and risks of fetal exposure.
  • Disease free of prior malignancies for ≥ 5 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.

Exclusion criteria

Exclusion Criteria:

  • Received prior treatment for AML
  • Favorable risk cytogenetic abnormalities as defined by SWOG criteria (http://www.bloodjournal.org/cgi/content/abstract/96/13/4075) that include: inv(16)/t(16;16)/del(16q), t(15;17) with/without secondary aberrations, t(8;21) lacking del(9q) or complex karyotype (16). Prior to enrollment, FISH, molecular studies or routine cytogenetics must be completed to rule out these cytogenetic abnormalities.
  • Known CNS leukemia
  • Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
  • Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
  • Use of any other experimental drug or therapy within 30 days of enrollment.
  • Known hypersensitivity to thalidomide.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
48 participants (actual)

Study arms

  • Experimental
    Cohort 1

    Lenalidomide 50 mg/day oral for 14 days followed by 30 days of rest. Lenalidomide 50 mg/day oral for 21 days (this is Cycle 1 and Cycle 2). If no progressive disease (PD) then lenalidomide 10 mg/day oral for 28 days for 12 cycles.

    Drug: Lenalidomide

  • Experimental
    Cohort 2

    Cycle 1: Oral lenalidomide 50 mg/day x 28 days induction therapy. Treatment will then depend on the response to Cycle 1: if patients obtain a complete remission (CR) they will proceed to low dose lenalidomide therapy, if patients have a non-CR they will receive a second high dose cycle of lenalidomide 50 mg/day x 28 days (Cycle 2) Cycle 2 consists of lenalidomide 50mg/day x 28 days Further treatment will depend on the response to Cycle 2: if patients obtain a CR/partial remission (PR)/stable disease (SD) they will proceed to low dose lenalidomide therapy, if patients have PD they will be removed from the study. Low Dose Cycles: low dose lenalidomide therapy consisting of 10 mg daily for a 28 day cycle.be 1) For patients that achieve a CR, 2 cycles of low dose lenalidomide will be administered, and then patients observed off therapy. For patients with PR/SD, low dose lenalidomide will continue for a total of 6 cycles and then patients will be observed off therapy.

    Drug: Lenalidomide

Interventions

  • DrugLenalidomide

    Also known as: Revlimid, CC-5013

06

What researchers measure

Primary outcomes

  1. Complete Remission Rate (CRm + CRi + CRc)

    CRm = Defined as morphologic leukemia-free state, including \<5% blasts in BM aspirate with marrow spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, no persistent extramedullary disease, ANC \> 1000/uL, platelet count \>100,000/uL. Patient must be independent of transfusions for a minimum of 1 week before each marrow assessment. There is no duration requirement for this designation. CRi = Defined as CR with the exception of neutropenia \<1000/uL or thrombocytopenia \<100,000/ul. Cytogenetic complete remission (CRc): Only patients with an identified cytogenetic abnormality may receive this designation. Defines as a morphologic complete remission plus reversion to a normal karyotype (no clonal abnormalities detected in a minimum of 20 mitotic cells).

    Time frame: After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)

Secondary outcomes

  1. Safety and Tolerability (Removal From Study Due to Adverse Events)

    Toxicity will be scored using CTCAE Version 3.0 for toxicity and adverse event reporting

    Time frame: 4 weeks after last dose of study drug [median duration of therapy was 65 days (range, 3-413 days)]

  2. Response Rate (RR)

    RR = as patients obtaining any response (CRm + CRc +CRi + PR). CRm = Defined as morphologic leukemia-free state, including \<5% blasts in BM aspirate with marrow spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, no persistent extramedullary disease, ANC \> 1000/uL, platelet count \> 100,000/uL. Patient must be independent of transfusions for a minimum of 1 week before each marrow assessment. There is no duration requirement for this designation. CRc = Cytogenetic complete remission (CRc): Only patients with an identified cytogenetic abnormality may receive this designation. Defines as a morphologic complete remission plus reversion to a normal karyotype (no clonal abnormalities detected in a minimum of 20 mitotic cells). Morphologic complete remission with incomplete blood count recovery (CRi): Defined as CR with the exception of neutropenia \<1000/uL or thrombocytopenia \<100,000/ul. Partial remission (PR): Requires

    Time frame: After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)

  3. Morphologic Leukemia Free State

    Morphologic leukemia-free state: Defined as \< 5% blasts on the BM aspirate with spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, and no persistent extramedullary disease.

    Time frame: After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)

  4. Morphologic Complete Remission Rate (CRm)

    CRm = Defined as morphologic leukemia-free state, including \<5% blasts in BM aspirate with marrow spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, no persistent extramedullary disease, ANC \> 1000/uL, platelet count \>100,000/uL. Patient must be independent of transfusions for a minimum of 1 week before each marrow assessment. There is no duration requirement for this designation.

    Time frame: After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)

  5. Cytogenetics CR Rate (CRc)

    Cytogenetic complete remission (CRc): Only patients with an identified cytogenetic abnormality may receive this designation. Defines as a morphologic complete remission plus reversion to a normal karyotype (no clonal abnormalities detected in a minimum of 20 mitotic cells).

    Time frame: After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)

  6. CR With Complete Blood Counts (CRi) Rate

    CRi = Defined as CR with the exception of neutropenia \<1000/uL or thrombocytopenia \<100,000/ul.

    Time frame: After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)

  7. Partial Remission Rate (PR)

    Partial remission (PR): Requires that the criteria for complete remission be met with the following exceptions: decrease of \>50% in the percentage of blasts to 5-25% in the BM aspirate. A value of \< 5% blasts in BM with Auer rods is also considered a partial remission.

    Time frame: After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)

  8. Overall Survival (OS)

    Overall survival: Defined as the date of first dose of study drug to the date of death from any cause.

    Time frame: 2 years

  9. Event Free Survival (EFS)

    Event free survival: Defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause.

    Time frame: 2 years

  10. Progression-free Survival

    Progression-free survival (PFS) denotes the chances of staying free of disease progression for a group of individuals suffering from a cancer after a particular treatment. It is the percentage of individuals in the group whose disease is likely to remain stable (and not show signs of progression) after a specified duration of time. Progression-free survival rates are an indication of how effective a particular treatment is.

    Time frame: 2 years

  11. Relapse Free Survival (RFS) for Complete Responders

    This is determined only for patients achieving a complete remission. Defined as the interval from the date of first documentation of a leukemia free state to date of recurrence or death due to any cause.

    Time frame: 2 years

  12. Duration of CR for Complete Responders

    Duration of remission: Defined as the interval from the date complete remission is documented to the date of recurrence

    Time frame: 2 years

  13. Changes in NK Cell Number and Function

    Peripheral blood mononuclear cells (PBMC) will be viably cryopreserved from patients at baseline (pre-therapy, newly diagnosed AML), during lenalidomide therapy, and posttherapy. Following sample collection, PBMC will be thawed, and flow cytometry will be performed to assess NK cell number (CD56+CD3-), subsets, and phenotype utilizing the Siteman Cancer Center Flow Cytometry / Cell Sorting Core. In addition, NK cell function will be assessed in flow based killing assays using PBMC (containing NK cells) as effectors and NK sensitive cell lines (K562) and/or autologous leukemic blasts as target cells. Thus, analyzing these parameters in patients before, during, and after therapy will provide a comprehensive evaluation of the ability of lenalidomide to modulate NK cells in patients in vivo.

    Time frame: Baseline, during therapy, and posttherapy

  14. Gene Expression Profiles of Bone Marrow and Peripheral Blood

    RNA will be made from total bone marrow cells for labeling and evaluations by RNA profiling. Cellular RNA and corresponding biotinylated cRNA targets will be prepared and hybridized with Affymetrix GeneChip® microarrays within the Multiplexed Gene Analysis SCC Core (Dr. Mark Watson, Director). Microarray data (and eventually corresponding gene sequence data) will be integrated an analyzed with state-of-the-art software packages. The pre- and post-treatment RNA profiling studies will be used as a discovery tool. Patterns of gene expression before and after lenalidomide therapy will be compared within each patient's sample to identify genes with altered expression after lenalidomide therapy. In addition, supervised algorithms will be sued to identify genes that can potentially predict clinical outcome and response to lenalidomide therapy.

    Time frame: Pre and post treatment

  15. Plasma Proteins Via Proteomics

    Proteomic analysis will be performed within the Siteman Cancer Center proteomics core on pre- and post-treatment plasma samples. This pilot proteomic study will identify candidate proteins of interest with altered expression after treatment with lenalidomide. This approach will provide an unbiased method to assess global changes in serum proteins following lenalidomide therapy.

    Time frame: Pre and post treatment

07

Results

Posted Sep 29, 2014

Participant flow

The study opened to participant enrollment on 02/02/2007 and closed to participant enrollment on 03/04/2009.

Participant flow — Overall Study
MilestoneCohort 1Cohort 2
Started1533
Completed1533
Not completed00

Outcome measures

PrimaryComplete Remission Rate (CRm + CRi + CRc)

CRm = Defined as morphologic leukemia-free state, including \<5% blasts in BM aspirate with marrow spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, no persistent extramedullary disease, ANC \> 1000/uL, platelet count \>100,000/uL. Patient must be independent of transfusions for a minimum of 1 week before each marrow assessment. There is no duration requirement for this designation. CRi = Defined as CR with the exception of neutropenia \<1000/uL or thrombocytopenia \<100,000/ul. Cytogenetic complete remission (CRc): Only patients with an identified cytogenetic abnormality may receive this designation. Defines as a morphologic complete remission plus reversion to a normal karyotype (no clonal abnormalities detected in a minimum of 20 mitotic cells).

Time frame:
After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)
Reported as:
Number · participants
Complete Remission Rate (CRm + CRi + CRc)
participantsCohort 1Cohort 2
CRm03
CRi04
CRc13
SecondarySafety and Tolerability (Removal From Study Due to Adverse Events)

Toxicity will be scored using CTCAE Version 3.0 for toxicity and adverse event reporting

Time frame:
4 weeks after last dose of study drug [median duration of therapy was 65 days (range, 3-413 days)]
Reported as:
Number · participants
Safety and Tolerability (Removal From Study Due to Adverse Events)
participantsCohort 1Cohort 2
Safety and Tolerability (Removal From Study Due to Adverse Events)18
SecondaryResponse Rate (RR)

RR = as patients obtaining any response (CRm + CRc +CRi + PR). CRm = Defined as morphologic leukemia-free state, including \<5% blasts in BM aspirate with marrow spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, no persistent extramedullary disease, ANC \> 1000/uL, platelet count \> 100,000/uL. Patient must be independent of transfusions for a minimum of 1 week before each marrow assessment. There is no duration requirement for this designation. CRc = Cytogenetic complete remission (CRc): Only patients with an identified cytogenetic abnormality may receive this designation. Defines as a morphologic complete remission plus reversion to a normal karyotype (no clonal abnormalities detected in a minimum of 20 mitotic cells). Morphologic complete remission with incomplete blood count recovery (CRi): Defined as CR with the exception of neutropenia \<1000/uL or thrombocytopenia \<100,000/ul. Partial remission (PR): Requires

Time frame:
After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)
Reported as:
Number · participants
Response Rate (RR)
participantsCohort 1Cohort 2
CRm03
CRc13
CRi04
PR10
SecondaryMorphologic Leukemia Free State

Morphologic leukemia-free state: Defined as \< 5% blasts on the BM aspirate with spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, and no persistent extramedullary disease.

Time frame:
After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)
Reported as:
Number · participants
Morphologic Leukemia Free State
participantsCohort 1Cohort 2
Morphologic Leukemia Free State110
SecondaryMorphologic Complete Remission Rate (CRm)

CRm = Defined as morphologic leukemia-free state, including \<5% blasts in BM aspirate with marrow spicules and a count of \> 200 nucleated cells and no blasts with Auer rods, no persistent extramedullary disease, ANC \> 1000/uL, platelet count \>100,000/uL. Patient must be independent of transfusions for a minimum of 1 week before each marrow assessment. There is no duration requirement for this designation.

Time frame:
After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)
Reported as:
Number · participants
Morphologic Complete Remission Rate (CRm)
participantsCohort 1Cohort 2
Morphologic Complete Remission Rate (CRm)03
SecondaryCytogenetics CR Rate (CRc)

Cytogenetic complete remission (CRc): Only patients with an identified cytogenetic abnormality may receive this designation. Defines as a morphologic complete remission plus reversion to a normal karyotype (no clonal abnormalities detected in a minimum of 20 mitotic cells).

Time frame:
After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)
Reported as:
Number · participants
Cytogenetics CR Rate (CRc)
participantsCohort 1Cohort 2
Cytogenetics CR Rate (CRc)13
SecondaryCR With Complete Blood Counts (CRi) Rate

CRi = Defined as CR with the exception of neutropenia \<1000/uL or thrombocytopenia \<100,000/ul.

Time frame:
After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)
Reported as:
Number · participants
CR With Complete Blood Counts (CRi) Rate
participantsCohort 1Cohort 2
CR With Complete Blood Counts (CRi) Rate04
SecondaryPartial Remission Rate (PR)

Partial remission (PR): Requires that the criteria for complete remission be met with the following exceptions: decrease of \>50% in the percentage of blasts to 5-25% in the BM aspirate. A value of \< 5% blasts in BM with Auer rods is also considered a partial remission.

Time frame:
After 2 cycles of low dose lenalidomide (approximately Day 113 for Cohort 1 and approximately Day 104 for Cohort 2)
Reported as:
Number · participants
Partial Remission Rate (PR)
participantsCohort 1Cohort 2
Partial Remission Rate (PR)10
SecondaryOverall Survival (OS)

Overall survival: Defined as the date of first dose of study drug to the date of death from any cause.

Time frame:
2 years
Reported as:
Median · months
Overall Survival (OS)
monthsCohort 1Cohort 2
Overall Survival (OS)—4 (3 to 9)
SecondaryEvent Free Survival (EFS)

Event free survival: Defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause.

Time frame:
2 years

No measurements were reported for this outcome.

SecondaryProgression-free Survival

Progression-free survival (PFS) denotes the chances of staying free of disease progression for a group of individuals suffering from a cancer after a particular treatment. It is the percentage of individuals in the group whose disease is likely to remain stable (and not show signs of progression) after a specified duration of time. Progression-free survival rates are an indication of how effective a particular treatment is.

Time frame:
2 years
Reported as:
Median · months
Progression-free Survival
monthsCohort 1Cohort 2
Progression-free Survival—2 (2 to 3)
SecondaryRelapse Free Survival (RFS) for Complete Responders

This is determined only for patients achieving a complete remission. Defined as the interval from the date of first documentation of a leukemia free state to date of recurrence or death due to any cause.

Time frame:
2 years
Reported as:
Median · months
Relapse Free Survival (RFS) for Complete Responders
monthsCohort 1Cohort 2
Relapse Free Survival (RFS) for Complete Responders—10 (2 to NA)
SecondaryDuration of CR for Complete Responders

Duration of remission: Defined as the interval from the date complete remission is documented to the date of recurrence

Time frame:
2 years
Reported as:
Median · months
Duration of CR for Complete Responders
monthsCohort 1Cohort 2
Duration of CR for Complete Responders—10 (1 to 17)
SecondaryChanges in NK Cell Number and Function

Peripheral blood mononuclear cells (PBMC) will be viably cryopreserved from patients at baseline (pre-therapy, newly diagnosed AML), during lenalidomide therapy, and posttherapy. Following sample collection, PBMC will be thawed, and flow cytometry will be performed to assess NK cell number (CD56+CD3-), subsets, and phenotype utilizing the Siteman Cancer Center Flow Cytometry / Cell Sorting Core. In addition, NK cell function will be assessed in flow based killing assays using PBMC (containing NK cells) as effectors and NK sensitive cell lines (K562) and/or autologous leukemic blasts as target cells. Thus, analyzing these parameters in patients before, during, and after therapy will provide a comprehensive evaluation of the ability of lenalidomide to modulate NK cells in patients in vivo.

Time frame:
Baseline, during therapy, and posttherapy

No measurements were reported for this outcome.

SecondaryGene Expression Profiles of Bone Marrow and Peripheral Blood

RNA will be made from total bone marrow cells for labeling and evaluations by RNA profiling. Cellular RNA and corresponding biotinylated cRNA targets will be prepared and hybridized with Affymetrix GeneChip® microarrays within the Multiplexed Gene Analysis SCC Core (Dr. Mark Watson, Director). Microarray data (and eventually corresponding gene sequence data) will be integrated an analyzed with state-of-the-art software packages. The pre- and post-treatment RNA profiling studies will be used as a discovery tool. Patterns of gene expression before and after lenalidomide therapy will be compared within each patient's sample to identify genes with altered expression after lenalidomide therapy. In addition, supervised algorithms will be sued to identify genes that can potentially predict clinical outcome and response to lenalidomide therapy.

Time frame:
Pre and post treatment

No measurements were reported for this outcome.

SecondaryPlasma Proteins Via Proteomics

Proteomic analysis will be performed within the Siteman Cancer Center proteomics core on pre- and post-treatment plasma samples. This pilot proteomic study will identify candidate proteins of interest with altered expression after treatment with lenalidomide. This approach will provide an unbiased method to assess global changes in serum proteins following lenalidomide therapy.

Time frame:
Pre and post treatment

No measurements were reported for this outcome.

Adverse events

Collected over Adverse events were collected from the start of treatment until 30 days following the completion of treatment. Cohort 1 median duration of treatment was 65 days (5-530). Cohort 2 median duration of treatment was 65 days (3-413).. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Cohort 1—10/15 (66.7%)15/15 (100%)
Cohort 2—25/33 (75.8%)33/33 (100%)
Most frequent serious events
Showing 10 of 57
Most frequent serious events
EventCohort 1Cohort 2
Febrile neutropeniaBlood and lymphatic system disorders6/1512/33
PneumoniaInfections and infestations2/159/33
Death due to progressive diseaseGeneral disorders3/156/33
FatigueGeneral disorders0/153/33
SyncopeCardiac disorders0/153/33
AnorexiaMetabolism and nutrition disorders0/153/33
DyspneaRespiratory, thoracic and mediastinal disorders1/150/33
Gouty arthritisMusculoskeletal and connective tissue disorders1/150/33
NauseaGastrointestinal disorders1/151/33
VomitingGastrointestinal disorders1/152/33
Most frequent other events
Showing 10 of 157
Most frequent other events
EventCohort 1Cohort 2
HypocalcemiaInvestigations12/1521/33
PlateletsInvestigations12/1525/33
SGPT (ALT)Investigations12/1514/33
HemoglobinBlood and lymphatic system disorders10/1522/33
Leukocytes (WBC)Investigations9/1515/33
HypoalbuminemiaInvestigations8/1519/33
RashSkin and subcutaneous tissue disorders6/1518/33
DyspneaRespiratory, thoracic and mediastinal disorders8/1513/33
FatigueGeneral disorders8/1517/33
HyponatremiaInvestigations8/1511/33

Baseline characteristics

Age, Continuous
Age, Continuous(years)Cohort 1Cohort 2Total
Median71 (60 to 86)71 (60 to 88)71 (60 to 88)
Sex: Female, Male
Sex: Female, Male(Participants)Cohort 1Cohort 2Total
Female41216
Male112132
Region of Enrollment
Region of Enrollment(participants)Cohort 1Cohort 2Total
United States153348
Eastern Cooperative Oncology Group (ECOG) Performance Status
Eastern Cooperative Oncology Group (ECOG) Performance Status(participants)Cohort 1Cohort 2Total
0101626
141317
2145
Cytogenetic Risk Category
Cytogenetic Risk Category(participants)Cohort 1Cohort 2Total
Intermediate131831
Unfavorable21315
Unknown022
08

Study locations

1 site
  • Washington University School of Medicine
    St. Louis, Missouri 63110, United States
09

References and documents

Publications

  • List A, Kurtin S, Roe DJ, Buresh A, Mahadevan D, Fuchs D, Rimsza L, Heaton R, Knight R, Zeldis JB. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med. 2005 Feb 10;352(6):549-57. doi: 10.1056/NEJMoa041668. PubMed 15703420 ↗
  • List, AF, G Dewald, J Bennett, et al. 2005. Hematologic and Cytogenetic (CTG) Response to Lenalidomide (CC-5013) in Patients with Transfusion-Dependent (TD) Myelodysplastic Syndrome (MDS) and Chromosome 5q31.1 Deletion: Results of the Multicenter MDS-003 Study. In ASCO, Orlando, FL.
  • Bansal D, Vij K, Chang GS, Miller CA, DiPersio JF, Vij R, Heath SE, Westervelt P, Welch JS, Fehniger TA. Lenalidomide results in a durable complete remission in acute myeloid leukemia accompanied by persistence of somatic mutations and a T-cell infiltrate in the bone marrow. Haematologica. 2018 Jun;103(6):e270-e273. doi: 10.3324/haematol.2017.184168. Epub 2018 Mar 22. No abstract available. PubMed 29567774 ↗
  • Fehniger TA, Uy GL, Trinkaus K, Nelson AD, Demland J, Abboud CN, Cashen AF, Stockerl-Goldstein KE, Westervelt P, DiPersio JF, Vij R. A phase 2 study of high-dose lenalidomide as initial therapy for older patients with acute myeloid leukemia. Blood. 2011 Feb 10;117(6):1828-33. doi: 10.1182/blood-2010-07-297143. Epub 2010 Nov 4. PubMed 21051557 ↗
  • Fehniger TA, Byrd JC, Marcucci G, Abboud CN, Kefauver C, Payton JE, Vij R, Blum W. Single-agent lenalidomide induces complete remission of acute myeloid leukemia in patients with isolated trisomy 13. Blood. 2009 Jan 29;113(5):1002-5. doi: 10.1182/blood-2008-04-152678. Epub 2008 Sep 29. PubMed 18824593 ↗
10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 29, 2014, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00546897
Lead sponsor
Washington University School of Medicine
Responsible party
Sponsor
First posted
Oct 19, 2007
Start date
Feb 2007
Primary completion
Jun 2010
Completion
Mar 2012
Results posted
Sep 29, 2014
Last update
Sep 29, 2014

Study contacts

Ravi Vij, M.D.
principal investigator · Washington University School of Medicine

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Sep 2014. You cannot join it, but the record below documents what was studied.

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