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Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality

Primary Purpose

Myelodysplastic Syndromes

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Lenalidomide 5 mg
Lenalidomide 10 mg
Placebo
Sponsored by
Celgene Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring MDS, transfusion dependent, anaemia, cytogenetic abnormality 5q-, erythroid response, leukaemia, CC-5013, Celgene, revlimid, lenalidomide

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Must understand and voluntarily sign an informed consent form Age 18 years at the time of signing the informed consent form Documented diagnosis of myelodysplastic syndromes (MDS) that meets International Prognostic Scoring System (IPSS) criteria for low to intermediate-1-risk disease and has an associated del 5q(31) cytogenetic abnormality Red blood cell (RBC) transfusion dependent anaemia defined as not having any 56 days without a RBC transfusion within at least the immediate 112 days Must be able to adhere to the study visit schedule and other protocol requirements Women of childbearing potential must have a negative pregnancy test prior to inclusion Exclusion Criteria: Pregnant or lactating females Prior therapy with lenalidomide Proliferative (white blood cell (WBC)= 12,000/mL) chronic myelomonocytic leukemia (CMML) Prior >= grade-2 (using the National Cancer Institute (NCI)'s Common Terminology Criteria for AEs (CTCAE) (v 3.0)) allergic reaction to thalidomide Prior desquamating (blistering) rash while taking thalidomide Prior history of malignancy other than MDS (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been free of disease for >3 years Use of cytotoxic chemotherapeutic agents or experimental agents (agents that are not commercially available) for the treatment of MDS within 28 days Less than 6 months since prior allogeneic bone marrow transplantation Less than 3 months since prior autologous bone marrow or stem cell transplantation Less than 28 days since prior myelosuppressive anticancer biologic therapy Recombinant human erythropoietin (rHuEPO) therapy received within 28 days Known human immunodeficiency virus (HIV-1) positivity Any serious medical condition or psychiatric illness that will prevent the subject from signing the informed consent form or will place the subject at unacceptable risk if he or she participates in the study

Sites / Locations

  • AZ St-Jan Brugge AV
  • UZ Gent
  • UZ Gasthuisberg
  • CHU Mont Godine
  • Institut Paoli-Calmettes
  • CHU d'Angers Service des Maladies du Sang
  • Hopital Avicenne
  • CHRU Lille Service des Maladies du Sang
  • CHU Nantes Hematologie et Medicine interne
  • CHU Archet 1Hematologie Clinique
  • Hôpital Cochin Hematologie Clinique
  • Centre Jean Bernhard Service Onco-Hematologie
  • Centre Henri Becquerel Service d'Hematologie Clinique
  • CHU Purpan, Place du Dr Baylac, Pavillon des Médecines
  • CHU Purpan, Place du Dr. Baylac, Pavillion des Medecines
  • CHU Nancy Hematologie et Medecine interne
  • Universitaetsklinikum Carl Gustav Carus
  • St Johannes Hospital
  • Universitaetsklinikum Freiburg
  • Hannover Medical School
  • Tel Aviv Sourasky Medical Center
  • Ospedale Niguarda Ca Granda
  • University of Pavia Division of Hematology
  • University of Medical Centre
  • Hematologie Erasmus MC
  • Hospital Universitario de Salamanca
  • Hospital Universitario La Fe
  • SU/Sahlgrenska Section of Hematology & Coagulation
  • Department of Medicine University Hospital
  • Korolinska Institutet Department of Hematology
  • University Hospital of Wales, Dept of Haematology
  • Leed General Infirmary
  • The Royal Bournemouth Hospital
  • Ninewells Hospital and Medical School
  • Kings College Hospital, Denmark Hill
  • Central Manchester and Manchester Children's University Hospitals NHS Trust
  • Nottingham City Hospital
  • John Radcliffe Hospital and the Weatherall Institute of Molecular Medicine

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Experimental

Experimental

Arm Label

Placebo

Lenalidomide 5 mg

Lenalidomide 10 mg

Arm Description

Placebo matching to active study arms.

Lenalidomide 5 mg daily 28/28 days

Lenalidomide 10 mg daily 21/28 days

Outcomes

Primary Outcome Measures

Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for >= 26 Weeks (182 Days)
The count of study participants who had no RBC transfusions for 26 consecutive weeks or more during the double-blind period.

Secondary Outcome Measures

Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for 56 Days
Count of study participants who had no RBC transfusions during any 56 or more consecutive study days during the double-blind period.
Duration of Red Blood Cell (RBC) Transfusion Independence for Participants Who Became RBC Transfusion Independent for at Least 182 Days
Mean number of weeks that participants who achieved RBC transfusion independence for at least 182 days were able to maintain RBC transfusion independence. Both double-blind and open-label periods are included.
Maximum Change From Baseline in Hemoglobin During the Double-blind Period for Participants Who Became Red Blood Cell (RBC) Transfusion Independent for at Least 182 Days
For participants who became RBC transfusion independent for at least 182 days during the double-blind study period, the mean maximum change from baseline in hemoglobin is summarized.
Participants' Response in Platelet Counts as Defined by the International MDS Working Group (IWG 2000) During Double-blind Period
The International MDS Working Group (IWG) defines a major platelet response for participants with a pre-treatment platelet count of <100,000/mm^3 as an absolute increase of ≥30,000/mm^3 whereas a minor response is defined as a ≥50% increase in platelet count with a net increase greater than 10,000/mm^3 but less than 30,000/mm^3.
Participants' Response in Absolute Neutrophil Counts as Defined by the International MDS Working Group (IWG 2000) During Double-blind Period
A major neutrophil response is defined by the International MDS Working Group (IWG) criteria as at least a 100% increase, or an absolute increase of ≥500/mm^3 for participants with absolute neutrophil counts (ANC) of less than 1,500/mm^3 before therapy, whichever is greater. A minor response for such participants is defined as an ANC increase of at least 100%, but absolute increase <500/mm^3.
Participants' Response Based on Bone Marrow Samples by the International MDS Working Group (IWG 2000) During Double-blind Period
The IWG criteria for bone marrow improvement: a complete remission is bone marrow sampling showing less than 5% myeloblasts with normal maturation of all cell lines, with no evidence for dysplasia. A partial remission is ≥ 50% decrease in blasts over pre-treatment. Bone marrow progression is a ≥ 50% increase in blasts that exceed the top range of the pretreatment percentile range: a) <5% blasts b) 5-10% blasts c) 10-20% blasts d) 20-30% blasts. For example, a participant with <5% blasts pretreatment with an on study blast increase of 50% which is now >5% showed bone marrow progression.
Participants Showing Cytogenetic Response by the International MDS Working Group (IWG 2000) During Double-blind Period as Evaluated by Central Review
The IWG criteria for evaluating cytogenetic response require a minimum of 20 baseline and post-baseline analyzable metaphases using conventional cytogenetic techniques. A major cytogenetic response is defined as no detectable cytogenetic abnormality if preexisting abnormality was present whereas a minor response requires ≥50% reduction in abnormal metaphases. Progression could be concluded based on as few as 3 metaphases if there were additional abnormalities. The best response is represented.
Participants Who Progressed to Acute Myeloid Leukemia (AML) During the Study
Number of participants who progressed to acute myeloid leukemia during the study, summarized at three different timepoints: first 16 weeks of the double-blind study, week 52 of the double-blind study, and up to 36 months which includes the double-blind and open-label periods of the study. The counts are cumulative by timeframe.
Kaplan Meier Estimates of Overall Survival by Randomized Group
Kaplan Meier estimate for median length of survival for study participants as they were randomized at the start of the study.
Participant Count of Deaths During Double-blind and Open-label by Randomized Group
Count of participant deaths throughout the entire study and reported by the original treatment assignment.
Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia (FACT-An) Endpoints at Week 12
The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire (Yellen, 1997) was used to assess health-related quality of life (HRQoL). In addition to general HRQoL, the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning. The overall score range for the FACT-An is 0-188. Higher scores indicate better HRQoL.
Change From Baseline in the Trial Outcome Index-Anemia (TOI-An) Endpoints at Week 12
The Trial Outcome Index-Anemia (TOI-An) composed of the physical and functional subscales of the FACT-G along with the Anemia subscale was used to assess health-related quality of life (HRQoL). The overall score range for the TOI-An is 0-136. Higher scores indicate better HRQoL.
Change From Baseline in the Trial Outcome Index-Fatigue (TOI-F) Endpoints at Week 12
The Trial Outcome Index-Fatigue(TOI-F) composed of the physical and functional subscales of the FACT-G along with the fatigue items from the Anemia subscale was used to assess health-related quality of life (HRQoL). The overall score range for the TOI-F is 0-108. Higher scores indicate better HRQoL.
Summary of Participants Who Had Adverse Events (AE) During the Double-blind Period
Counts of study participants who had adverse events (AEs) during the double-blind period by MedDRA System Organ Class (SOC) and preferred term. A participant with multiple occurrences of an adverse event within a category is counted only once in that category. Adverse events were evaluated by the investigator. The National Cancer Institute (NCI)'s Common Terminology Criteria for AEs (CTCAE) was used to grade AE severity. Severity grade 3= severe and undesirable AE. Severity grade 4= life-threatening or disabling AE.

Full Information

First Posted
September 10, 2005
Last Updated
April 12, 2011
Sponsor
Celgene Corporation
Collaborators
ICON Clinical Research
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1. Study Identification

Unique Protocol Identification Number
NCT00179621
Brief Title
Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality
Official Title
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Arm Study of the Efficacy and Safety of 2 Doses of Lenalidomide Versus Placebo in Red Blood Cell (RBC) Transfusion-Dependent Subjects With Low- or Intermediate-1-Risk Myelodysplastic Syndromes Associated With a Deletion (Del) 5q[31] Cytogenetic Abnormality
Study Type
Interventional

2. Study Status

Record Verification Date
April 2011
Overall Recruitment Status
Completed
Study Start Date
July 2005 (undefined)
Primary Completion Date
June 2008 (Actual)
Study Completion Date
June 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Celgene Corporation
Collaborators
ICON Clinical Research

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study was to compare 2 doses (10 mg and 5 mg) of lenalidomide to that of placebo in subjects with red blood cell (RBC) transfusion-dependent low- or intermediate-1-risk IPSS MDS associated with a deletion (del) 5q[31] cytogenetic abnormality. Study participants were randomized to one of the two treatment groups or to placebo and took the study drug for 16 weeks. At this timepoint, participants were evaluated for erythroid response. If participants did not achieve at least a minor erythroid response, they were discontinued from the Double-Blind phase and entered into the Open-Label phase. All erythroid responders at Week 16 were to continue in the Double-Blind phase for up to 52 weeks. For participants that were still responding at the end of Double-Blind phase, they could then rollover into the Open-Label phase for an additional two years. Participants could remain on study for up to a total of 3 years. All participants who discontinued from the study were followed every 4 months for overall survival and progression to acute myeloid leukemia (AML).
Detailed Description
MDS-004 was a multicenter, randomized, double-blind, placebo-controlled, 3-arm study of 2 doses of lenalidomide versus placebo administered to RBC transfusion-dependent adults with low- or intermediate-1 risk MDS associated with a del 5q[31] cytogentetic abnormality. Potential participants that had a del 5q[31] cytogenetic abnormality plus other additional cytogenetic abnormalities were also eligible for enrollment. Transfusion-dependent anemia was defined as documentation that a participant with anemia due to MDS did not have any consecutive 56 days (8 weeks) that were RBC transfusion free during at least the 112 days (16 weeks) prior to Day 1 of the Pre-Randomization Phase. This study was conducted in three phases: a Pre-Randomization Phase a Double-Blind Treatment Phase an Open-Label Extension Phase Potentially protocol-eligible participants entered the Pre-Randomization Phase and were evaluated for the inclusion and exclusion criteria for the Double-Blind Treatment Phase. The Pre-Randomization Phase was not to last for more than 56 days (8 weeks). When the participant's baseline RBC transfusion requirement was calculated, and it had been determined that all eligibility criteria had been met, the participant could be randomized for treatment in the Double-Blind Treatment Phase at the time of their next RBC transfusion. This RBC transfusion had to occur within 56 days of the participant's last previous RBC transfusion. Participants meeting eligibility criteria were randomized (1:1:1 ratio) to receive either lenalidomide 10 mg/day on days 1-21, lenalidomide 5 mg/day on days 1-28, or placebo on days 1-28; all on a 28-day cycle. Randomization was performed using a validated interactive voice response system. Participants were stratified according to karyotype (IPSS karyotype score: 0 vs > 0; i.e., isolated del 5q[31] vs del 5q[31] plus ≥ 1 additional cytogenetic abnormality). A complete blood count (CBC), serum or plasma ferritin, and EPO levels were measured to determine baseline levels. Participants who achieved at least a minor erythroid response (i.e. 50% decrease in transfusion requirements) by week 16 could continue treatment in the Double-Blind phase for up to 52 weeks, unless there was evidence of erythroid relapse, disease progression, or unacceptable toxicity. Those who did not achieve at least a minor erythroid response by week 16 were discontinued from the Double-Blind phase for lack of therapeutic efficacy, and unblinded and were potentially eligible for Open-Label treatment. All participants who completed the double-blind treatment phase (the first 52 weeks of the trial) without disease progression or erythroid relapse were unblinded and entered the Open-Label extension phase at their current lenalidomide dose. Participants in the placebo or lenalidomide 5 mg arms who failed to achieve at least a minor erythroid response by week 16 or who had an erythroid relapse could cross over to lenalidomide 5 mg or 10 mg, respectively, in the Open-Label Extension phase. Lenalidomide treatment could be continued in the Open-label Extension phase for up to 3 years (156 weeks) of total study participation. Participants with disease progression at any time and participants in the lenalidomide 10 mg group who did not achieve at least a minor erythroid response by week 16 were withdrawn from the study and were ineligible for Open-Label treatment. Serial measurements for efficacy and safety were performed every 28 days. In addition, CBCs were monitored weekly for the first 8 weeks, every 2 weeks for the next 8 weeks, and every 4 weeks thereafter. Bone marrow aspirate (BMA) and standard cytogenetic studies were performed at baseline, weeks 12, week 24, and every 24 weeks thereafter and when clinically indicated for assessment of disease progression. BMAs were submitted for central pathology review and sent to a central cytogenetics laboratory for processing and review. All participants were followed for overall survival (OS) and progression to acute myeloid leukemia (AML). Lenalidomide or placebo dosing was reduced for dose-limiting toxicities according to the following dose reduction schedule: Lenalidomide 5 mg (starting dose) dose level -1 (5 mg every other day) dose level -2 (5 mg twice a week) dose level -3 (5 mg weekly) Lenalidomide 10 mg (starting dose) dose level -1 (5 mg daily) dose level -2 (5 mg every other day) dose level -3 (5 mg twice a week) Participants who could not tolerate dose level -3 discontinued treatment. For grade 4 neutropenia, lenalidomide was required per protocol to be interrupted and resumed at a decreased dose level when the absolute neutrophil count (ANC) recovered to ≥ 500/μL. For grade 4 thrombocytopenia, lenalidomide was interrupted and then resumed at a decreased dose level when the platelet count recovered to: between ≥ 25,000/μL and < 50,000/μL on at least 2 occasions for ≥ 7 days; or ≥ 50,000 at any time, respectively. Prophylactic and therapeutic use of granulocyte colony-stimulating factors (G-CSF) or granulocyte macrophage colony-stimulating factors (GM-CSF) was allowed.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes
Keywords
MDS, transfusion dependent, anaemia, cytogenetic abnormality 5q-, erythroid response, leukaemia, CC-5013, Celgene, revlimid, lenalidomide

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
205 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo matching to active study arms.
Arm Title
Lenalidomide 5 mg
Arm Type
Experimental
Arm Description
Lenalidomide 5 mg daily 28/28 days
Arm Title
Lenalidomide 10 mg
Arm Type
Experimental
Arm Description
Lenalidomide 10 mg daily 21/28 days
Intervention Type
Drug
Intervention Name(s)
Lenalidomide 5 mg
Other Intervention Name(s)
Revlimid
Intervention Description
Lenalidomide 5 mg daily 28/28 days
Intervention Type
Drug
Intervention Name(s)
Lenalidomide 10 mg
Other Intervention Name(s)
Revlimid
Intervention Description
Lenalidomide 10 mg daily 21/28 days
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo, matching to active study drug arms
Primary Outcome Measure Information:
Title
Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for >= 26 Weeks (182 Days)
Description
The count of study participants who had no RBC transfusions for 26 consecutive weeks or more during the double-blind period.
Time Frame
Up to 52 weeks
Secondary Outcome Measure Information:
Title
Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for 56 Days
Description
Count of study participants who had no RBC transfusions during any 56 or more consecutive study days during the double-blind period.
Time Frame
Up to 52 weeks
Title
Duration of Red Blood Cell (RBC) Transfusion Independence for Participants Who Became RBC Transfusion Independent for at Least 182 Days
Description
Mean number of weeks that participants who achieved RBC transfusion independence for at least 182 days were able to maintain RBC transfusion independence. Both double-blind and open-label periods are included.
Time Frame
up to 3 years
Title
Maximum Change From Baseline in Hemoglobin During the Double-blind Period for Participants Who Became Red Blood Cell (RBC) Transfusion Independent for at Least 182 Days
Description
For participants who became RBC transfusion independent for at least 182 days during the double-blind study period, the mean maximum change from baseline in hemoglobin is summarized.
Time Frame
Baseline, up to 52 weeks
Title
Participants' Response in Platelet Counts as Defined by the International MDS Working Group (IWG 2000) During Double-blind Period
Description
The International MDS Working Group (IWG) defines a major platelet response for participants with a pre-treatment platelet count of <100,000/mm^3 as an absolute increase of ≥30,000/mm^3 whereas a minor response is defined as a ≥50% increase in platelet count with a net increase greater than 10,000/mm^3 but less than 30,000/mm^3.
Time Frame
up to 52 weeks
Title
Participants' Response in Absolute Neutrophil Counts as Defined by the International MDS Working Group (IWG 2000) During Double-blind Period
Description
A major neutrophil response is defined by the International MDS Working Group (IWG) criteria as at least a 100% increase, or an absolute increase of ≥500/mm^3 for participants with absolute neutrophil counts (ANC) of less than 1,500/mm^3 before therapy, whichever is greater. A minor response for such participants is defined as an ANC increase of at least 100%, but absolute increase <500/mm^3.
Time Frame
up to week 52
Title
Participants' Response Based on Bone Marrow Samples by the International MDS Working Group (IWG 2000) During Double-blind Period
Description
The IWG criteria for bone marrow improvement: a complete remission is bone marrow sampling showing less than 5% myeloblasts with normal maturation of all cell lines, with no evidence for dysplasia. A partial remission is ≥ 50% decrease in blasts over pre-treatment. Bone marrow progression is a ≥ 50% increase in blasts that exceed the top range of the pretreatment percentile range: a) <5% blasts b) 5-10% blasts c) 10-20% blasts d) 20-30% blasts. For example, a participant with <5% blasts pretreatment with an on study blast increase of 50% which is now >5% showed bone marrow progression.
Time Frame
up to 52 weeks
Title
Participants Showing Cytogenetic Response by the International MDS Working Group (IWG 2000) During Double-blind Period as Evaluated by Central Review
Description
The IWG criteria for evaluating cytogenetic response require a minimum of 20 baseline and post-baseline analyzable metaphases using conventional cytogenetic techniques. A major cytogenetic response is defined as no detectable cytogenetic abnormality if preexisting abnormality was present whereas a minor response requires ≥50% reduction in abnormal metaphases. Progression could be concluded based on as few as 3 metaphases if there were additional abnormalities. The best response is represented.
Time Frame
up to 52 weeks
Title
Participants Who Progressed to Acute Myeloid Leukemia (AML) During the Study
Description
Number of participants who progressed to acute myeloid leukemia during the study, summarized at three different timepoints: first 16 weeks of the double-blind study, week 52 of the double-blind study, and up to 36 months which includes the double-blind and open-label periods of the study. The counts are cumulative by timeframe.
Time Frame
up to 3 years
Title
Kaplan Meier Estimates of Overall Survival by Randomized Group
Description
Kaplan Meier estimate for median length of survival for study participants as they were randomized at the start of the study.
Time Frame
up to 3 years
Title
Participant Count of Deaths During Double-blind and Open-label by Randomized Group
Description
Count of participant deaths throughout the entire study and reported by the original treatment assignment.
Time Frame
up to 3 years
Title
Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia (FACT-An) Endpoints at Week 12
Description
The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire (Yellen, 1997) was used to assess health-related quality of life (HRQoL). In addition to general HRQoL, the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning. The overall score range for the FACT-An is 0-188. Higher scores indicate better HRQoL.
Time Frame
Baseline, Week 12
Title
Change From Baseline in the Trial Outcome Index-Anemia (TOI-An) Endpoints at Week 12
Description
The Trial Outcome Index-Anemia (TOI-An) composed of the physical and functional subscales of the FACT-G along with the Anemia subscale was used to assess health-related quality of life (HRQoL). The overall score range for the TOI-An is 0-136. Higher scores indicate better HRQoL.
Time Frame
Baseline, Week 12
Title
Change From Baseline in the Trial Outcome Index-Fatigue (TOI-F) Endpoints at Week 12
Description
The Trial Outcome Index-Fatigue(TOI-F) composed of the physical and functional subscales of the FACT-G along with the fatigue items from the Anemia subscale was used to assess health-related quality of life (HRQoL). The overall score range for the TOI-F is 0-108. Higher scores indicate better HRQoL.
Time Frame
Baseline, Week 12
Title
Summary of Participants Who Had Adverse Events (AE) During the Double-blind Period
Description
Counts of study participants who had adverse events (AEs) during the double-blind period by MedDRA System Organ Class (SOC) and preferred term. A participant with multiple occurrences of an adverse event within a category is counted only once in that category. Adverse events were evaluated by the investigator. The National Cancer Institute (NCI)'s Common Terminology Criteria for AEs (CTCAE) was used to grade AE severity. Severity grade 3= severe and undesirable AE. Severity grade 4= life-threatening or disabling AE.
Time Frame
up to week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Must understand and voluntarily sign an informed consent form Age 18 years at the time of signing the informed consent form Documented diagnosis of myelodysplastic syndromes (MDS) that meets International Prognostic Scoring System (IPSS) criteria for low to intermediate-1-risk disease and has an associated del 5q(31) cytogenetic abnormality Red blood cell (RBC) transfusion dependent anaemia defined as not having any 56 days without a RBC transfusion within at least the immediate 112 days Must be able to adhere to the study visit schedule and other protocol requirements Women of childbearing potential must have a negative pregnancy test prior to inclusion Exclusion Criteria: Pregnant or lactating females Prior therapy with lenalidomide Proliferative (white blood cell (WBC)= 12,000/mL) chronic myelomonocytic leukemia (CMML) Prior >= grade-2 (using the National Cancer Institute (NCI)'s Common Terminology Criteria for AEs (CTCAE) (v 3.0)) allergic reaction to thalidomide Prior desquamating (blistering) rash while taking thalidomide Prior history of malignancy other than MDS (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been free of disease for >3 years Use of cytotoxic chemotherapeutic agents or experimental agents (agents that are not commercially available) for the treatment of MDS within 28 days Less than 6 months since prior allogeneic bone marrow transplantation Less than 3 months since prior autologous bone marrow or stem cell transplantation Less than 28 days since prior myelosuppressive anticancer biologic therapy Recombinant human erythropoietin (rHuEPO) therapy received within 28 days Known human immunodeficiency virus (HIV-1) positivity Any serious medical condition or psychiatric illness that will prevent the subject from signing the informed consent form or will place the subject at unacceptable risk if he or she participates in the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jay Backstrom, MD
Organizational Affiliation
Celgene Corporation
Official's Role
Study Director
Facility Information:
Facility Name
AZ St-Jan Brugge AV
City
Brugge
ZIP/Postal Code
8000
Country
Belgium
Facility Name
UZ Gent
City
Gent
ZIP/Postal Code
900
Country
Belgium
Facility Name
UZ Gasthuisberg
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
CHU Mont Godine
City
Yvoir
ZIP/Postal Code
5530
Country
Belgium
Facility Name
Institut Paoli-Calmettes
City
Marseille
State/Province
Cedex 9
ZIP/Postal Code
B.P.156 - 13272
Country
France
Facility Name
CHU d'Angers Service des Maladies du Sang
City
Angers
ZIP/Postal Code
49933
Country
France
Facility Name
Hopital Avicenne
City
Bobigny Cedex
Country
France
Facility Name
CHRU Lille Service des Maladies du Sang
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
CHU Nantes Hematologie et Medicine interne
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
CHU Archet 1Hematologie Clinique
City
Nice
ZIP/Postal Code
06202
Country
France
Facility Name
Hôpital Cochin Hematologie Clinique
City
Paris
ZIP/Postal Code
75014
Country
France
Facility Name
Centre Jean Bernhard Service Onco-Hematologie
City
Poitiers
ZIP/Postal Code
86021
Country
France
Facility Name
Centre Henri Becquerel Service d'Hematologie Clinique
City
Rouen
ZIP/Postal Code
76038
Country
France
Facility Name
CHU Purpan, Place du Dr Baylac, Pavillon des Médecines
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
CHU Purpan, Place du Dr. Baylac, Pavillion des Medecines
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
CHU Nancy Hematologie et Medecine interne
City
Vandoeuvre
ZIP/Postal Code
54511
Country
France
Facility Name
Universitaetsklinikum Carl Gustav Carus
City
Dresden
ZIP/Postal Code
01307
Country
Germany
Facility Name
St Johannes Hospital
City
Duisburg
ZIP/Postal Code
47166
Country
Germany
Facility Name
Universitaetsklinikum Freiburg
City
Freiburg
ZIP/Postal Code
79106
Country
Germany
Facility Name
Hannover Medical School
City
Hannover
ZIP/Postal Code
D-30625
Country
Germany
Facility Name
Tel Aviv Sourasky Medical Center
City
Tel Aviv
ZIP/Postal Code
64239
Country
Israel
Facility Name
Ospedale Niguarda Ca Granda
City
Milano
ZIP/Postal Code
20162
Country
Italy
Facility Name
University of Pavia Division of Hematology
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
University of Medical Centre
City
Nijmegen
ZIP/Postal Code
6526 GA
Country
Netherlands
Facility Name
Hematologie Erasmus MC
City
Rotterdam
ZIP/Postal Code
3000CA
Country
Netherlands
Facility Name
Hospital Universitario de Salamanca
City
Salamanca
ZIP/Postal Code
37007
Country
Spain
Facility Name
Hospital Universitario La Fe
City
Valencia
ZIP/Postal Code
46009
Country
Spain
Facility Name
SU/Sahlgrenska Section of Hematology & Coagulation
City
Goteborg
ZIP/Postal Code
SE 413 45
Country
Sweden
Facility Name
Department of Medicine University Hospital
City
Lund
ZIP/Postal Code
S-221 85
Country
Sweden
Facility Name
Korolinska Institutet Department of Hematology
City
Stockholm
ZIP/Postal Code
14186
Country
Sweden
Facility Name
University Hospital of Wales, Dept of Haematology
City
Cardiff
State/Province
Wales
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Facility Name
Leed General Infirmary
City
Leeds
State/Province
West Yorkshire
ZIP/Postal Code
LS1 3 EX
Country
United Kingdom
Facility Name
The Royal Bournemouth Hospital
City
Bournemouth
ZIP/Postal Code
BH7 7DW
Country
United Kingdom
Facility Name
Ninewells Hospital and Medical School
City
Dundee
ZIP/Postal Code
DD1 9SY
Country
United Kingdom
Facility Name
Kings College Hospital, Denmark Hill
City
London
ZIP/Postal Code
SE 5 9RS
Country
United Kingdom
Facility Name
Central Manchester and Manchester Children's University Hospitals NHS Trust
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
Nottingham City Hospital
City
Nottingham
ZIP/Postal Code
NG5 1PB
Country
United Kingdom
Facility Name
John Radcliffe Hospital and the Weatherall Institute of Molecular Medicine
City
Oxford
ZIP/Postal Code
OX3 9DS
Country
United Kingdom

12. IPD Sharing Statement

Citations:
Citation
Fenaux, Pierre, Giagounidis, Aristotle, Selleslag, Dominik, Beyne-Rauzy, Odile, Mufti, Ghulam J, Mittelman, Moshe, Muus, Petra, te Boekhorst, Peter, Sanz, Guillermo, del Canizo, Consuelo, Guerci-Bresler, Agnes, Schlegelberger, Brigitte, Aul, Carlo, Kreipe, Hans, Goehring, Gudrun, Knight, Robert, Francis, John, Fu, Tommy, Hellstrom-Lindberg, Eva. RBC Transfusion Independence and Safety Profile of Lenalidomide 5 or 10 mg in Pts with Low- or Int-1-Risk MDS with Del5q: Results From a Randomized Phase III Trial (MDS-004). ASH Annual Meeting Abstracts 2009 114: 944.
Results Reference
result
Citation
Fenaux, P., Giagounidis, A., Selleslag, D. L., Beyne-Rauzy, O., Mittelman, M., Muus, P., Knight, R. D., Fu, T., Hellstrom-Lindberg, E., The MDS-004 Len del(5q) Study Group. Safety of lenalidomide (LEN) from a randomized phase III trial (MDS-004) in low-/int-1-risk myelodysplastic syndromes (MDS) with a del(5q) abnormality. J Clin Oncol (Meeting Abstracts) 2010 28: 6598
Results Reference
result
Citation
Fenaux, P., Giagounidis, A., Selleslag, D., Knight, R., Fu, T., Hellström-Lindberg, E. Effect of baseline EPO and prior erythropoiesis stimulating agents on RBC transfusion independence in Low-/Int-1-Risk MDS with del 5q treated with lenalidomide: A randomized phase 3 study (MDS-004). Haematologica 2010; 95[suppl.2]:125, abs. 0311.
Results Reference
result
Citation
Brandenburg, N., Fu, T., Revicki, D., Knight, R., Muus, P., Fenaux, P. Impact of lenalidomide on health-related quality of life in patients with RBC transfusion-dependent low- or int-1-risk myelodysplastic syndromes with del 5q: a randomized Phase 3 study (MDS-004). Haematologica 2010; 95[suppl.2]:127, abs. 0316
Results Reference
result
Citation
Fenaux, P., Giagounidis, A., Beyne-Rauzy, O., Mufti, G., Mittelman, M., Muus, P., te Boekhorst, P., Sanz, G., Cazzola, M., Backstrom, J., Fu, T., Hellström-Lindberg, E. Prognostic Factors of Long-Term Outcomes In Low- or Int-1-Risk MDS with del5q Treated with Lenalidomide (LEN): Results From a Randomized Phase 3 Trial (MDS-004). Blood ASH Annual Meeting Abstracts 2010 116:21 abs. 4027.
Results Reference
result
Citation
Brandenburg, N., Yu, R., Revicki, D. Reliability and Validity of the FACT-AN In Patients with Low or Int-1-Risk Myelodysplastic Syndromes with Deletion 5q. Blood ASH Annual Meeting Abstracts 2010 116:21 abs. 3827.
Results Reference
result
PubMed Identifier
28651604
Citation
Fenaux P, Giagounidis A, Selleslag D, Beyne-Rauzy O, Mittelman M, Muus P, Nimer SD, Hellstrom-Lindberg E, Powell BL, Guerci-Bresler A, Sekeres MA, Deeg HJ, Del Canizo C, Greenberg PL, Shammo JM, Skikne B, Yu X, List AF. Clinical characteristics and outcomes according to age in lenalidomide-treated patients with RBC transfusion-dependent lower-risk MDS and del(5q). J Hematol Oncol. 2017 Jun 26;10(1):131. doi: 10.1186/s13045-017-0491-2.
Results Reference
derived
PubMed Identifier
24682512
Citation
Saft L, Karimi M, Ghaderi M, Matolcsy A, Mufti GJ, Kulasekararaj A, Gohring G, Giagounidis A, Selleslag D, Muus P, Sanz G, Mittelman M, Bowen D, Porwit A, Fu T, Backstrom J, Fenaux P, MacBeth KJ, Hellstrom-Lindberg E. p53 protein expression independently predicts outcome in patients with lower-risk myelodysplastic syndromes with del(5q). Haematologica. 2014 Jun;99(6):1041-9. doi: 10.3324/haematol.2013.098103. Epub 2014 Mar 28.
Results Reference
derived
PubMed Identifier
21753188
Citation
Fenaux P, Giagounidis A, Selleslag D, Beyne-Rauzy O, Mufti G, Mittelman M, Muus P, Te Boekhorst P, Sanz G, Del Canizo C, Guerci-Bresler A, Nilsson L, Platzbecker U, Lubbert M, Quesnel B, Cazzola M, Ganser A, Bowen D, Schlegelberger B, Aul C, Knight R, Francis J, Fu T, Hellstrom-Lindberg E; MDS-004 Lenalidomide del5q Study Group. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. Blood. 2011 Oct 6;118(14):3765-76. doi: 10.1182/blood-2011-01-330126. Epub 2011 Jul 13.
Results Reference
derived
PubMed Identifier
21109690
Citation
Gohring G, Giagounidis A, Busche G, Hofmann W, Kreipe HH, Fenaux P, Hellstrom-Lindberg E, Schlegelberger B. Cytogenetic follow-up by karyotyping and fluorescence in situ hybridization: implications for monitoring patients with myelodysplastic syndrome and deletion 5q treated with lenalidomide. Haematologica. 2011 Feb;96(2):319-22. doi: 10.3324/haematol.2010.026658. Epub 2010 Nov 25.
Results Reference
derived

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Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality

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