Clinical trial NCT00812968: Study of CC-5013 to Evaluate Safety, Pharmacokinetics and Effectiveness for Japanese Patients With Symptomatic Anemia Associated With Myelodysplastic Syndrome With a Del(5)(q31-33) Abnormality.
Official title: A Multicenter, Single-arm Study to Assess the Safety, Pharmacokinetics and Efficacy of Lenalidomide in Japanese Subjects With Low- or Intern=Mediate-1-risk Myelodysplastic Syndromes (MDS) Associated With a Deletion 5 (q31-33) Abnormality and Symptomatic Anemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelodysplastic Syndromes
Keywords: Myelodysplastic Syndromes
Lenalidomide
Lead sponsor: Celgene
Sponsor class: INDUSTRY
First posted: 2008-12-22
Enrollment: 11
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this clinical experience study is to determine whether CC-5013 is safe and effective (to include studying the process by which a drug is absorbed, distributed, metabolized, and eliminated by the body \[pharmacokinetics\]) in Japanese subjects with low- or intermediate-1-risk MDS (IPSS risk categories) associated with a deletion 5(q31-33) abnormality and symptomatic anemia.


Eligibility criteria: Inclusion Criteria:

* Must understand and voluntarily sign an informed consent form.
* Age ≥ 20 years at the time of signing the informed consent form.
* Must be able to adhere to the study visit schedule and other protocol requirements.
* Diagnosis of Myelodysplastic Syndrome (MDS) that meets International Prognostic Scoring System (IPSS) criteria for low- or intermediate-1-risk disease associated with a deletion 5(q31-33) abnormality
* Symptomatic anemia secondary to MDS defined as:Untransfused Hb level \< 10.0 g/dL and a Functional Assessment of Cancer Therapy (FACT)-anemia subscale score of ≤ 74 or Transfusion dependent anemia

Exclusion Criteria:

* Pregnant or lactating females.
* Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
* Prior therapy with lenalidomide.
* Patients with any of the following laboratory abnormalities within 14 days of starting study drug: Absolute Neutrophil Count (ANC) \< 750 cells/μL (0.75 x 10\^9/L) Platelet count \< 50,000/μL (50x10\^9/L) Serum creatinine \> 2.5 mg/dL Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) \> 3.0 x Upper Limit of Normal (ULN)

Primary outcomes:
measure: Number of Participants With Adverse Events (AE)
timeFrame: After the first study dose until 28 days after completion of/discontinuation from the study (maximum time on study was 155 weeks).
Secondary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax) of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.
measure: Time to Maximum Plasma Concentration (Tmax) of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.
measure: Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUCt) of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.
measure: Area Under the Plasma Concentration-time Curve Over the Dosing Interval (AUCτ) of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, 12 and 24 hours post-dose.
measure: Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC∞) of Lenalidomide
timeFrame: Day 1 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, 12 and 24 hours post-dose.
measure: Terminal Half-life (T1/2) of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.
measure: Apparent Volume of Distribution (VzF) of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.
measure: Apparent Total Plasma Clearance (CL/F) of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.
measure: Apparent Terminal Elimination Rate Constant of Lenalidomide
timeFrame: Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.
measure: Number of Participants With a Erythroid Response
timeFrame: Response was assessed every 28 days through Week 156.
measure: Time to Erythroid Response
timeFrame: From the first dose of study drug through Week 156
measure: Duration of Erythroid Response
timeFrame: From the first dose of study drug through Week 156
measure: Change From Baseline in Hemoglobin Concentration
timeFrame: Baseline and from Day1 until the maximum observed value (up to 155 weeks)
measure: Number of Participants With a Neutrophil Response
timeFrame: Response was assessed every 28 days through Week 156
measure: Number of Participants With a Platelet Response
timeFrame: Response was assessed every 28 days through Week 156
measure: Number of Participants With a Cytogenetic Response
timeFrame: Response was assessed every 12 weeks through Week 156
measure: Change From Baseline in Percentage of Bone Marrow Erythroblasts
timeFrame: Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169).
measure: Percentage of Bone Marrow Myeloblasts
timeFrame: Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169).
measure: Percentage of Bone Marrow Promyelocytes
timeFrame: Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169).
Other outcomes:
Locations:
city: Shimono
country: Japan
facility: Celgene Clinical Site
state: Tochigi
city: Shibuya-ku
country: Japan
facility: Celgene Clinical Site
state: Tokyo
city: Hiroshima
country: Japan
facility: Celgene Clinical Site
city: Kyoto
country: Japan
facility: Celgene Clinical Site
city: Osaka
country: Japan
facility: Celgene Clinical Site
city: Shizuoka
country: Japan
facility: Celgene Clinical Site
Study officials:
affiliation: Celgene Corporation
name: Masaaki Takatoku, MD
role: STUDY_DIRECTOR
References:
citation: Matsuda A, Taniwaki M, Jinnai I, Harada H, Watanabe M, Suzuki K, Yanagita S, Suzuki T, Yoshida Y, Kimura A, Tsudo M, Tohyama K, Takatoku M, Ozawa K. Morphologic analysis in myelodysplastic syndromes with del(5q) treated with lenalidomide. A Japanese multiinstitutional study. Leuk Res. 2012 May;36(5):575-80. doi: 10.1016/j.leukres.2011.11.011. Epub 2011 Dec 15.
citation: Harada H, Watanabe M, Suzuki K, Yanagita S, Suzuki T, Yoshida Y, Kimura A, Tsudo M, Matsuda A, Tohyama K, Taniwaki M, Takeshita K, Takatoku M, Ozawa K. Lenalidomide is active in Japanese patients with symptomatic anemia in low- or intermediate-1 risk myelodysplastic syndromes with a deletion 5q abnormality. Int J Hematol. 2009 Oct;90(3):353-360. doi: 10.1007/s12185-009-0400-8. Epub 2009 Aug 25.