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Study of Lenalidomide to Evaluate Safety, Pharmacokinetics and Effectiveness for Japanese Patients With Symptomatic Anemia Associated With Myelodysplastic Syndromes (MDS) With a Del(5)(q31-33) Abnormality.

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Celgene Corporation
ClinicalTrials.gov Identifier:
NCT00812968
First received: December 17, 2008
Last updated: September 30, 2013
Last verified: September 2013
Results First Received: September 30, 2013  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Myelodysplastic Syndromes
Intervention: Drug: Lenalidomide

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Lenalidomide 10 mg of lenalidomide was administered orally once daily on Days 1 to 21 of every 28-day cycle. Treatment continued until disease progression or relapse following an erythroid improvement was documented, any of the criteria for treatment discontinuation was violated, or lenalidomide became commercially available for the treatment of MDS associated with a del (5q) cytogenetic abnormality, for up to 156 weeks (3 years).

Participant Flow:   Overall Study
    Lenalidomide  
STARTED     11  
COMPLETED     0  
NOT COMPLETED     11  
Relapse after erythroid response                 6  
Disease progression                 1  
No further treatment required                 1  
Study closed due to marketing approval                 3  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Lenalidomide 10 mg of lenalidomide was administered orally once daily on Days 1 to 21 of every 28-day cycle, for up to 156 weeks (3 years).

Baseline Measures
    Lenalidomide  
Number of Participants  
[units: participants]
  11  
Age  
[units: years]
Mean ± Standard Deviation
  71.8  ± 5.95  
Gender  
[units: participants]
 
Female     7  
Male     4  
Region of Enrollment  
[units: participants]
 
Japan     11  
Duration of MDS  
[units: years]
Mean ± Standard Deviation
  2.0  ± 1.44  
International Prognostic Scoring System (IPSS) Score [1]
[units: participants]
 
Low risk     2  
Intermediate-1 risk     9  
Intermediate-2 risk     0  
High risk     0  
French-American-British (FAB) classification of MDS [2]
[units: participants]
 
Refractory anemia (RA)     9  
Refractory anemia with ringed sideroblasts (RARS)     0  
Refractory anemia with excess blasts (RAEB)     2  
Chronic myelomonocytic leukemia (CMML)     0  
RAEB in transformation (RAEB-t)     0  
World Health Organization Classification of MDS [3]
[units: participants]
 
Refractory anemia (RA)     0  
RA with ringed sideroblasts (RARS)     0  
Refractory anemia with excess blasts-1 (RAEB-1)     2  
Refractory anemia with excess blasts-2 (RAEB-2)     0  
Refractory cytopenia multilineage dysplasia (RCMD)     1  
RCMD and ringed sideroblasts (RCMD-RS)     0  
MDS, unclassified (MDS-U)     0  
MDS associated with isolated del(5q)     8  
Bone Marrow Cellularity [4]
[units: participants]
 
Aplastic (0%)     0  
Hypocellular (> 0% to ≤ 30%)     3  
Normocellular (> 30% to ≤ 60%)     5  
Hypercellular (> 60% to ≤ 90%)     2  
Packed (> 90% to ≤ 100%)     0  
Missing     1  
Transfusion Dependency  
[units: participants]
 
Yes     5  
No     6  
[1] The MDS IPSS assesses the severity of MDS based on 3 prognostic factors each assigned a score: the proportion of bone marrow blasts, chromosome changes in the marrow cells (karyotype) and the presence of one or more low blood cell counts (cytopenias). The IPSS score is the sum of the bone marrow blast + karyotype + cytopenia score and ranges from 0 (low risk) to 3.5 (high risk). Prognosis is categorized as Low risk (score = 0), Intermediate-1 (score 0.5 to 1.0), Intermediate-2 (score 1.5 to 2.0) or High risk (score ≥ 2.5).
[2] The French-American-British (FAB) classification of MDS recognizes five subtypes of MDS that are distinguished by the percentage of myeloblasts, presence or absence of ringed sideroblasts (i.e., erythroid precursors with iron deposits surrounding the nucleus), or a monocytosis.
[3] The World Health Organization (WHO) classification recognizes eight subtypes of MDS that are distinguished by the percentage of myeloblasts, presence or absence of ringed sideroblasts (i.e., erythroid precursors with iron deposits surrounding the nucleus), presence of a monocytosis or a deletion 5q.
[4] Bone marrow cellularity is a measure of the percentage of hematopoietic stem cells relative to fat cells in the bone marrow.



  Outcome Measures
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1.  Primary:   Number of Participants With Adverse Events (AE)   [ Time Frame: From the first dose of study drug through 28 days after completion of/discontinuation from the study (maximum time on study was 155 weeks). ]

2.  Secondary:   Maximum Observed Plasma Concentration (Cmax) of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose. ]

3.  Secondary:   Time to Maximum Plasma Concentration (Tmax) of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose. ]

4.  Secondary:   Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUCt) of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose. ]

5.  Secondary:   Area Under the Plasma Concentration-time Curve Over the Dosing Interval (AUCτ) of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, 12 and 24 hours post-dose. ]

6.  Secondary:   Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC∞) of Lenalidomide   [ Time Frame: Day 1 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, 12 and 24 hours post-dose. ]

7.  Secondary:   Terminal Half-life (T1/2) of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose. ]

8.  Secondary:   Apparent Volume of Distribution (VzF) of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose. ]

9.  Secondary:   Apparent Total Plasma Clearance (CL/F) of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose. ]

10.  Secondary:   Apparent Terminal Elimination Rate Constant of Lenalidomide   [ Time Frame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose. ]

11.  Secondary:   Number of Participants With a Erythroid Response   [ Time Frame: Response was assessed every 28 days through Week 156. ]

12.  Secondary:   Time to Erythroid Response   [ Time Frame: From the first dose of study drug through Week 156 ]

13.  Secondary:   Duration of Erythroid Response   [ Time Frame: From the first dose of study drug through Week 156 ]

14.  Secondary:   Change From Baseline in Hemoglobin Concentration   [ Time Frame: Baseline and from Day1 until the maximum observed value (up to 155 weeks) ]

15.  Secondary:   Number of Participants With a Neutrophil Response   [ Time Frame: Response was assessed every 28 days through Week 156 ]

16.  Secondary:   Number of Participants With a Platelet Response   [ Time Frame: Response was assessed every 28 days through Week 156 ]

17.  Secondary:   Number of Participants With a Cytogenetic Response   [ Time Frame: Response was assessed every 12 weeks through Week 156 ]

18.  Secondary:   Change From Baseline in Percentage of Bone Marrow Erythroblasts   [ Time Frame: Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169). ]

19.  Secondary:   Percentage of Bone Marrow Myeloblasts   [ Time Frame: Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169). ]

20.  Secondary:   Percentage of Bone Marrow Promyelocytes   [ Time Frame: Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169). ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Associate Director, Clinical Trials Disclosure
Organization: Celgene Corporation
phone: 1-888-260-1599
e-mail: clinicaltrialdisclosure@celgene.com


No publications provided by Celgene Corporation

Publications automatically indexed to this study:

Responsible Party: Celgene Corporation
ClinicalTrials.gov Identifier: NCT00812968     History of Changes
Other Study ID Numbers: CC-5013-MDS-007
Study First Received: December 17, 2008
Results First Received: September 30, 2013
Last Updated: September 30, 2013
Health Authority: Japan: Pharmaceuticals and Medical Devices Agency
Japan: Ministry of Health, Labor and Welfare