Efficacy and Safety Study of CC-5013 Monotherapy in Subjects With Myelodysplastic Syndromes
- Registration Number
- NCT00064974
- Lead Sponsor
- Celgene Corporation
- Brief Summary
This study is a multi-center, single-arm, open-label study of oral CC-5013 monotherapy administered at a dose of 10 mg daily on Days 1-21 every 28 days (28-day cycles) to red blood cell (RBC) transfusion-dependent subjects with low- or intermediate-1-risk MDS who do not have a del (5q31-33) cytogenetic abnormality. Screening procedures will take place within 28 days of first day of study drug treatment. Subjects will receive study drug (CC-5013) in 28-day cycles for up to 6 cycles, or until bone marrow disease progression or progression/relapse following erythroid hematologic improvement (Appendix I) 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 215
- Must understand and voluntarily sign an informed consent form.
- Age ≥ 18 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 low - or intermediate-1-risk IPSS (Appendix III) 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 ≥ to 2 units of RBCs within 8 weeks of the first day of study drug treatment.
- Eastern Cooperative Oncology Group (ECOG) (Appendix IV) 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.
- 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 cells/mm3 (0.5 x 109/L)
- Lab Abnormality: Platelet count <50,000/mm3 (50 x 109/L)
- Lab Abnormality: Serum creatinine >2.5 mg/dL (221 mmol/L)
- Lab Abnormality: Serum glutamic oxaloacetic transaminase/Aspartate transaminase (SGOT/AST) or Serum glutamic pyruvic transaminase/Alanine transaminase (SGPT/ALT) >3.0 x upper limit of normal (ULN)
- Lab Abnormality: Serum total bilirubin >2.0 mg/dL (34 mmol/L)
- Prior ≥ grade 3 National Cancer Institute (NCI) Common Toxicity Criteria (CTC) (Appendix VI) allergic reaction/hypersensitivity to thalidomide.
- Prior ≥ grade 3 NCI CTC (Appendix VI) rash or any desquamation (blistering) while taking 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.
- 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.
- Use of any other experimental therapy within 28 days of the first day of study drug treatment.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description CC-5013 CC-5013 CC-5013 10 mg (two 5 mg capsules) daily on days 1-28 every 28 days (28 day cycles)
- Primary Outcome Measures
Name Time Method RBC Transfusion Independence
- Secondary Outcome Measures
Name Time Method ≥ 50% decrease in RBC transfusion requirement Platelet Response Platelet Response
Neutrophil Response Neutrophil Response
Bone marrow Response Bone marrow Response
Duration of Response Duration of Response
Hemoglobin concentration Change of hemoglobin concentration from baseline
Number of Participants with Adverse Event Number of Participants with Adverse Event
Trial Locations
- Locations (116)
Alta Bates Cancer Center
🇺🇸Berkeley, California, United States
Midwest Cancer Research Group
🇺🇸Skokie, Illinois, United States
The Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
Desert Hematology Oncology Medical Group, Inc.
🇺🇸Rancho Mirage, California, United States
Florida Cancer Specialists
🇺🇸Fort Myers, Florida, United States
Northwest Georgia Oncology - Wellstar Cancer Research
🇺🇸Marietta, Georgia, United States
Rush Presbyterian-St. Luke's Medical Center
🇺🇸Chicago, Illinois, United States
University of Chicago Medical Center
🇺🇸Chicago, Illinois, United States
Johns Hopkins Oncology Center
🇺🇸Baltimore, Maryland, United States
Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
Western Pennsylvania Cancer Institute
🇺🇸Pittsburgh, Pennsylvania, United States
Swedish Cancer Institute
🇺🇸Seattle, Washington, United States
Fred Hutchinson Cancer Research Center
🇺🇸Seattle, Washington, United States
Royal Adelaide Hospital - SA Pathology Haematology
🇦🇺Adelaide, South Australia, Australia
Indiana University Medical Center
🇺🇸Indianapolis, Indiana, United States
Kaiser Permanente Northwest Region
🇺🇸Portland, Oregon, United States
Wayne State University School of Medicine
🇺🇸Detroit, Michigan, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Odense University Hospital
🇩🇰Odense, Denmark
Ospedale Guglielmo da Saliceto - hematooncology
🇮🇹Piacenza, Italy
Border Medical Oncology
🇦🇺Wodonga, Australia
Cancer & Blood Disease Center
🇺🇸Lecanto, Florida, United States
St. Vincents Comprehensive Cancer Center
🇺🇸New York, New York, United States
University of Miami- Sylvester Comp Cancer Center
🇺🇸Miami, Florida, United States
Arizona Cancer Center
🇺🇸Tucson, Arizona, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Stanford University Medical Center
🇺🇸Stanford, California, United States
H. Lee Moffitt Cancer Center and Research Institute
🇺🇸Tampa, Florida, United States
St. Luke's Oncology and Hematology Associates
🇺🇸Duluth, Minnesota, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Roswell Park Cancer Institute
🇺🇸Buffalo, New York, United States
New York Hospital- Cornell
🇺🇸New York, New York, United States
Wake Forest University School of Medicine
🇺🇸Winston Salem, North Carolina, United States
Mt. Sinai Medical Center
🇺🇸New York, New York, United States
Winthrop University Hospital
🇺🇸Mineola, New York, United States
Memorial Sloan-Kettering Cancer Center
🇺🇸New York, New York, United States
University of Rochester-James P. Wilmot Cancer Center
🇺🇸Rochester, New York, United States
Drexel University College of Medicine
🇺🇸Philadelphia, Pennsylvania, United States
MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Royal Prince Alfred Hospital - Institute of Haematology
🇦🇺Camperdown, Australia
Princess Alexandra Hospital - Haematology
🇦🇺Brisbane, Australia
Frankston Hospital-peninsula Health - Oncology Day Unit
🇦🇺Frankston, Australia
The Alfred Hospital - malignant haematology & stem cell transplantation
🇦🇺Melbourne, Australia
Wollongong Hospital - Haematology
🇦🇺Wollongong, Australia
Peter McCallum Cancer Institute - Directorate of Cancer Medecine
🇦🇺East Melbourne, Australia
Calvary Mater Newcastle - Haematology
🇦🇺Waratah, Australia
Aalborg Sygemus - Haematology
🇩🇰Aalborg, Denmark
UZ Gent - Hematology
🇧🇪Gent, Belgium
Charles university Hospital - Internal Medicine
🇨🇿Prague, Czech Republic
University Hospital Leuven - Hematology
🇧🇪Leuven, Belgium
Cliniques Universitaires ULC de Mont-Godinne - Hematology
🇧🇪Yvoir, Belgium
Fakultní nemocnice Hradec Králové - Hematology
🇨🇿Hradec Kralove, Czech Republic
Aarhus University Hospital
🇩🇰Aarhus, Denmark
Vejle Hospital - Hematology
🇩🇰Vejle, Denmark
Centre Hospitalier de la côte basque - Hematologie
🇫🇷Bayonne, France
CHU Angers - Service des maladies du sang
🇫🇷Angers, France
Centre Hospitalier Départemental Vendée - Onco-hematologie
🇫🇷La Roche sur Yon, France
Institut Paoli Calmette - Hematology 1
🇫🇷Marseille, France
CHRU de Lille - Service des maladies du sang
🇫🇷Lille, France
CHU Hôtel-Dieu - Hematologie
🇫🇷Nantes, France
Hôpital Saint Louis - Immuno-hematologie
🇫🇷Paris, France
CHU Saint Antoine - Service des maladies du sang
🇫🇷Paris, France
Centre Hospitalier Lyon sud - Hematologie
🇫🇷Pierre-Benite, France
CHRU Hôpital Purpan - Hematologie
🇫🇷Toulouse, France
CHRU - Hôpital du Haut Lévêque - Centre François Magendie
🇫🇷Pessac, France
Hôpital Bretonneau - Hématologie & Thérapie cellulaire
🇫🇷Tours, France
Universitätsklinikum Essen, Klinik für Hämatologie
🇩🇪Essen, Germany
Universitätsklinikum Leipzig - Medizinische Klinik und Poliklinik II
🇩🇪Leipzig, Germany
CHU Nancy - Hematologie
🇫🇷Vandoeuvre-les-Nancy, France
Universitätsklinikum Jena - Klinik fur Innere Medizin II-Hamatologie/Onkologie
🇩🇪Jena, Germany
Universitätsklinikum Ulm - Klinik fur Innere Medizin III
🇩🇪Ulm, Germany
Universitätsklinikum Heidelberg - Medizinische Klinik und Poliklinik V
🇩🇪Heidelberg, Germany
Universitätsklinikum Tübingen - Medizinische Klinik und Poliklinik - Abteilung II
🇩🇪Tübingen, Germany
Universitätsklinikum Münster - Medizinische Klinik und Poliklinik A
🇩🇪Münster, Germany
Universitätsklinikum Würzburg - Medizinische Klinik und Poliklinik II
🇩🇪Würzburg, Germany
University of Athens - Alexandra Hospital; Clinical Therapeutics
🇬🇷Athens, Greece
Università degli Studi di Bologna - Policlinico S. Orsola - Hematology
🇮🇹Bologna, Italy
AO Universitaria San Martino - hematooncology
🇮🇹Genova, Italy
Fondazione "G. Pascale" - Hematology
🇮🇹Napoli, Italy
Universita degli Studi di Padova - Clinical & Experimental Medicine
🇮🇹Padova, Italy
Unità di Ematologia Arcispedale S. Maria Nuova - Haematology
🇮🇹Reggio Emilia, Italy
Ospedale San Luigi AO Luigi Gonzaga - Hematology
🇮🇹Orbassano, Italy
Policlinico Umberto I, Università "La Sapienza" di Roma - Hematology
🇮🇹Roma, Italy
Erasmus Medical Center - Hematology
🇳🇱Rotterdam, Netherlands
University Medical Center - Hematology
🇳🇱Utrecht, Netherlands
VUMC - Hematology
🇳🇱Amsterdam, Netherlands
A.O.U. San Giovanni Battista - Hematology
🇮🇹Torino, Italy
Medical Sciences - Hematology & BMT
🇷🇺Moscow, Russian Federation
Moscow State Medical Institution Municipal Clinical Hospital n.a. S.P. Botkin - Hematology
🇷🇺Moscow, Russian Federation
Russian Research Institute of Hematology and Blood Transfusion - Hematology
🇷🇺St. Petersburg, Russian Federation
Hospital Germans Trias i Pujol - Hematology
🇪🇸Badalona, Spain
State Higher Educational Institution St. Petersburg State Medical University - Onco-hematology
🇷🇺St. Petersburg, Russian Federation
Hospital Clinic i Provincial de Barcelona - Hematology
🇪🇸Barcelona, Spain
Hospital de Donostia - Hematology
🇪🇸Guipúzcoa, Spain
Hospital de La Princesa - Hematology
🇪🇸Madrid, Spain
Hospital 12 de Octubre - Hematology
🇪🇸Madrid, Spain
Hospital de Salamanca - Hematology
🇪🇸Salamanca, Spain
Hospital Universitario Marqués de Valdecilla - Hematology
🇪🇸Santander, Spain
Karolinska University Hospital Huddinge - Center of hematology
🇸🇪Stockholm, Sweden
Sahlgrenska Hospital, University of Goteborg - Hematology
🇸🇪Goteborg, Sweden
Inselspital, Institut für Medizinische Onkologie
🇨🇭Bern, Switzerland
Overlakare Medocomcentrum - Hematology
🇸🇪Uppsala, Sweden
Karolinska University Hospital Solna- medicine
🇸🇪Stockholm, Sweden
Hôpitaux Universitaire de Genève - Oncologie
🇨🇭Genève, Switzerland
Royal Bournemouth Hospital - Haematology
🇬🇧Bournemouth, United Kingdom
Klinik und Poliklinik für Onkologie - UniversitätsSpital Zürich
🇨🇭Zurich, Switzerland
St Bartholomew's Hospital - Medical Oncology
🇬🇧London, United Kingdom
St James's University Hospital - Haematology
🇬🇧Leeds, United Kingdom
Hospital La Fe - Hematology
🇪🇸Valencia, Spain
King's College Hospital - Haematology Clinical Trials
🇬🇧London, United Kingdom
Freeman Hospital - Northern Centre for Cancer Care
🇬🇧Newcastle Upon Tyne, United Kingdom
Nottingham City Hospital - Centre for Clinical Haematology
🇬🇧Nottingham, United Kingdom
Derriford Hospital - Haematology
🇬🇧Plymouth, United Kingdom
Royal Wolverhampton hospitals trust - Research and development
🇬🇧Wolverhampton, United Kingdom
Royal Marsden NHS Foundation Trust - Haematology
🇬🇧Surrey, United Kingdom
Royal hallamshire Hospital - Haematology
🇬🇧Sheffield, United Kingdom