Lenalidomide Safety/Efficacy in Myelodysplastic Syndromes (MDS) Associated With a Deletion (Del)(5q) Cytogenetic Abnormality

NCT00065156 | Completed Phase 2 Results

• 1 other identifier: CC-5013-MDS-003
• Study Type: interventional
• Target: 148
• Locations: 2 countries
• Sites: 32

Brief Summary

This study is a multicenter, single-arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion-dependent subjects with low- or intermediate-1-risk Myelodysplastic Syndromes (MDS) associated with a del (5q31-33) cytogenetic abnormality. Screening procedures will take place within 28 days of the first day of lenalidomide treatment. Subjects will receive lenalidomide in 28-day cycles for up to 6 cycles, or until bone marrow disease progression or progression/relapse following erythroid hematologic improvement is documented. Study visits will occur every cycle (every 28 days) and laboratory monitoring to assess hematological parameters will occur every 14 days. Safety and efficacy assessments to be performed during the study are outlined in the Schedule of Study Assessments.

Conditions

Myelodysplastic Syndromes

Keywords

MDS; CC-5013; Revlimid; Celgene

Outcome Measures

Primary Outcomes (1)

• Participants Who Achieved Red Blood Cell (RBC) -Transfusion Independence

  Number of participants who achieved RBC-transfusion independence, which was defined as the absence of an intravenous infusion of any RBC transfusion during any consecutive "rolling" 56 days during the treatment period (eg, Days 1 to 56, Days 2 to 57, Days 3 to 58, etc), and accompanied by at least a 1 g/dL increase from screening/baseline in hemoglobin.

  Up to 2 years

Secondary Outcomes (11)

• Participants With Adverse Experiences

  Up to 2 Years

• Participants With a >= 50% Decrease From Baseline in Red Blood Cell (RBC) Transfusion Requirements Over Any Consecutive 56 Days During Study

  Baseline (Day -54 to Day 0), During study (Day 1 up to 2 years)

• Time to Transfusion Independence

  up to 2 years

• Participants Who Relapsed or Maintained Their Transfusion Independence After Achieving Transfusion Independence During the Study

  up to 2 years

• Kaplan Meier Estimate for Duration of Transfusion Independence Response

  up to 2 years

Study Arms (1)

Lenalidomide

EXPERIMENTAL

Drug: lenalidomide

Interventions

lenalidomide DRUG

10 mg orally once daily for 21 days out of a 28-day cycle (syncopated); subsequently amended (Amendment 1, dated 27 August 2003) to employ a continuous dosage regimen in which 10 mg was taken once daily for 28 day cycles (continuous). Subjects who initially began a syncopated regimen and who did not experience a dose-limiting adverse event were allowed to switch to the continuous regimen.

Also known as: CC-5013

Lenalidomide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must understand and voluntarily sign an informed consent form
• Age 18 years or older at the time of signing the informed consent
• Must be able to adhere to the study visit schedule and other protocol requirements.
• Diagnosis of low or intermediate-1-risk International Prognostic Scoring System (IPSS) Myelodysplastic Syndromes (MDS) without an abnormality of chromosome 5 involving a deletion between bands q31 and q33.
• Red blood cell (RBC) transfusion-dependent anemia defined as having received greater than or equal to 2 units of RBCs within 8 weeks of the first day of study drug treatment.
• Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1 or 2.
• Women of childbearing potential (WCBP) must have a negative serum or urine pregnancy test within 7 days of starting study drug.
• Sexually active WCBP must agree to use adequate contraceptive methods (oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) while on study drug.
• WCBP must agree to have pregnancy tests every 4 weeks while on study drug.

You may not qualify if:

• Pregnant or lactating females
• Prior therapy with lenalidomide.
• An abnormality of chromosome 5 involving a deletion between bands q31 and q33.
• Lab Abnormality: Absolute neutrophil count (ANC) \<500 cell/mm\^3 (0.5\*10\^9/L)
• Lab Abnormality: Platelet count \<50,000/mm\^3 (50\*10\^9/L)
• Lab Abnormality: Serum creatinine \>2.5 mg/dL (221 mmol/L)
• Lab Abnormality: Serum total bilirubin \>2.0 mg/dL (34 mmol/L)
• Prior greater than or equal to grade 3 National Cancer Institute (NCI) Common Toxicity Criteria (CTC) allergic reaction/hypersensitivity to thalidomide.
• Clinically significant anemia due to factors such as iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis or gastrointestinal bleeding
• If a marrow aspirate is not evaluable for storage iron, transferrin saturation must be \> 20% and serum ferritin not less than 50 ng/mL
• Use of hematopoietic growth factors within 7 days of the first day of study drug treatment.
• Prior greater than or equal to grade 3 NCI CTC rash or any desquamation (blistering) while taking thalidomide.
• Chronic use (\>2 weeks) of greater than physiologic doses of a corticosteroid agent (dose equivalent to \>10 mg/day of prednisone) within 28 days of the first day of study drug treatment.
• Use of experimental or standard drugs (i.e. chemotherapeutic, immunosuppressive, and cytoprotective agents) for the treatment of MDS within 28 days of the first day of study drug treatment.
• 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 greater than or equal to 3 years.

Sponsors & Collaborators

• Celgene: lead

Study Sites (32)

Arizona Cancer Center

Scottsdale, Arizona, 85258, United States

Location

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

Arizona Cancer Center

Tucson, Arizona, 85724-5024, United States

Location

Desert Hematology & Oncology Medical Group

Rancho Mirage, California, 92270, United States

Location

Stanford University Medical Center

Stanford, California, 94305-5750, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Cancer & Blood Disease Center

Lecanto, Florida, 34461, United States

Location

University of Miami Sylvester Comp Cancer Center

Miami, Florida, 33136, United States

Location

H. Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, 33612-9497, United States

Location

Northwest Georgia Oncology - Wellstar Cancer Research

Marietta, Georgia, 30060, United States

Location

Rush-Presbyterian- St. Luke's Medical Center

Chicago, Illinois, 60612, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637-1470, United States

Location

Midwest Cancer Research Group

Skokie, Illinois, 60077, United States

Location

Johns Hopkins Oncology Center

Baltimore, Maryland, 21287-8963, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115-6084, United States

Location

Wayne State University School of Medicine

Detroit, Michigan, 48201-2097, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198-7680, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

New York Hospital-Cornell

New York, New York, 10021-0034, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10021-6007, United States

Location

Mt. Sinai Medical Center

New York, New York, 10029, United States

Location

University of Rochester- James P. Wilmot Cancer Center

Rochester, New York, 14642, United States

Location

Wake Forest University School of Medicine

Winston-Salem, North Carolina, 27157-1082, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Oregon Health & Science University

Portland, Oregon, 97201, United States

Location

Kaiser Permanente Northwest Region

Portland, Oregon, 97227, United States

Location

Western Pennsylvania Cancer Institute

Pittsburgh, Pennsylvania, 15224, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Swedish Cancer Institute

Seattle, Washington, 98104, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109-4417, United States

Location

St. Johannes Hospital

Duisburg, Germany

Location

Related Publications (5)

• Sekeres MA, Maciejewski JP, Giagounidis AA, Wride K, Knight R, Raza A, List AF. Relationship of treatment-related cytopenias and response to lenalidomide in patients with lower-risk myelodysplastic syndromes. J Clin Oncol. 2008 Dec 20;26(36):5943-9. doi: 10.1200/JCO.2007.15.5770. Epub 2008 Nov 17.

  PMID: 19018091 BACKGROUND

• Prebet T, Cluzeau T, Park S, Sekeres MA, Germing U, Ades L, Platzbecker U, Gotze K, Vey N, Oliva E, Sugrue MM, Bally C, Kelaidi C, Al Ali N, Fenaux P, Gore SD, Komrokji R. Outcome of patients treated for myelodysplastic syndromes with 5q deletion after failure of lenalidomide therapy. Oncotarget. 2017 Jun 14;8(47):81926-81935. doi: 10.18632/oncotarget.18477. eCollection 2017 Oct 10.

  PMID: 29137233 BACKGROUND

• List A, Dewald G, Bennett J, Giagounidis A, Raza A, Feldman E, Powell B, Greenberg P, Thomas D, Stone R, Reeder C, Wride K, Patin J, Schmidt M, Zeldis J, Knight R; Myelodysplastic Syndrome-003 Study Investigators. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006 Oct 5;355(14):1456-65. doi: 10.1056/NEJMoa061292.

  PMID: 17021321 RESULT

• A.F. List, et.al. Results of the MDS-002 and -003 international phase II studies evaluating lenalidomide (CC-5013; Revlimid) in the treatment of transfusion-dependent (TD) patients with myelodysplastic syndrome (MDS). European Hematology Association 10th Conference June 2-5, 2005 Abstract #0772

  RESULT

• 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.

  PMID: 28651604 DERIVED

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Lenalidomide

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Associate Director, Clinical Trials Disclosure
• Organization: Celgene Corporation

clinicaltrialdisclosure@celgene.com

1-888-260-1599

Study Officials

• Alan F List, MD

  H. Lee Moffitt Cancer Center and Research Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 17, 2003
• First Posted: July 18, 2003
• Study Start: June 1, 2003
• Primary Completion: August 1, 2008
• Study Completion: August 1, 2008
• Last Updated: November 19, 2019
• Results First Posted: June 9, 2010

Record last verified: 2019-11

Locations

DE (1); US (31)