A Study of Lenalidomide Versus Placebo in Subjects With Transfusion Dependent Anemia in Low Risk Myelodysplastic Syndrome (MDS) Without Del 5Q - Full Text View

Purpose

The purpose of this study is to investigate whether lenalidomide would reduce the number of red blood cell transfusions needed by anemic (RBC transfusion-dependent) subjects with low or intermediate-1 risk MDS without a deletion 5q chromosome abnormality. The study will also investigate the safety of lenalidomide use in these subjects. Two-thirds of the subjects will receive lenalidomide and one-third of the subjects will receive placebo (does not contain lenalidomide).

Condition	Intervention	Phase
Anemia	Drug: Lenalidomide Other: Placebo	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	A Phase 3, Multicenter, Placebo-Controlled Study to Compare the Efficacy and Safety of Lenalidomide vs. Placebo in Subjects With Transfusion Dependent Anemia Due to Low or Intermediate Risk MDS and Unresponsive to ESA Therapies

Resource links provided by NLM:

MedlinePlus related topics: Anemia Blood Transfusion and Donation Myelodysplastic Syndromes

Drug Information available for: Lenalidomide

U.S. FDA Resources

Further study details as provided by Celgene Corporation:

Primary Outcome Measures:

Proportion of subjects that become transfusion independent. Proportion of subjects with an erythroid differentiation gene expression signature that become transfusion independent. [ Time Frame: Up to 4 years for each subject (likely to be 6 months to 2 years) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Evaluate the safety of lenalidomide versus placebo. [ Time Frame: up to 6 years from study start through follow-up ] [ Designated as safety issue: Yes ]
Evaluate the impact of lenalidomide therapy on health-related quality of life (HRQOL) and use of healthcare resources. [ Time Frame: up to 6 years from study start through follow-up ] [ Designated as safety issue: No ]

Estimated Enrollment:	228
Study Start Date:	November 2009
Estimated Study Completion Date:	December 2018
Estimated Primary Completion Date:	April 2016 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Arm #1 - Lenalidomide 10 mg lenalidomide once daily (administered as one 10-mg lenalidomide capsule + 2 placebo capsules)	Drug: Lenalidomide One 10 mg Lenalidomide capsule + 2 placebo capsules or (3 placebo capsules) once daily for subjects with a creatinine clearance ≥ 60 mL/min. Alternatively-one 5 mg Lenalidomide capsule + 2 placebo capsules (or 3 placebo capsules) once daily for subjects with a creatinine clearance between 40 and 60 mL/min. Subjects may take study drug for at least 168 days unless there are intolerable side effects or disease progresses. Subjects may continue study drug beyond 168 days if they have an erythroid response (increase in their hemoglobin levels and fewer transfusions administered than before starting study drug) Other Name: Revlimid
Placebo Comparator: Arm #2 - placebo Three placebo capsules once daily. Subjects will be randomized using a 2:1 ratio in a double-blind manner to receive oral lenalidomide 10 mg once daily or placebo once daily. Subjects will receive oral lenalidomide 10 mg once daily (one 10 mg lenalidomide capsules + 2 placebo capsule) or matching placebo once daily (3 placebo capsules).	Other: Placebo 3 placebo capsules once daily. Subjects may take study drug for at least 168 days unless there are intolerable side effects or disease progresses. Subjects may continue study drug beyond 168 days if they have an erythroid response (increase in their hemoglobin levels and fewer transfusions administered than before starting study drug)

Arms

Assigned Interventions

Experimental: Arm #1 - Lenalidomide

10 mg lenalidomide once daily (administered as one 10-mg lenalidomide capsule + 2 placebo capsules)

Drug: Lenalidomide

One 10 mg Lenalidomide capsule + 2 placebo capsules or (3 placebo capsules) once daily for subjects with a creatinine clearance ≥ 60 mL/min.

Alternatively-one 5 mg Lenalidomide capsule + 2 placebo capsules (or 3 placebo capsules) once daily for subjects with a creatinine clearance between 40 and 60 mL/min.

Subjects may take study drug for at least 168 days unless there are intolerable side effects or disease progresses. Subjects may continue study drug beyond 168 days if they have an erythroid response (increase in their hemoglobin levels and fewer transfusions administered than before starting study drug)

Other Name: Revlimid

Placebo Comparator: Arm #2 - placebo

Three placebo capsules once daily. Subjects will be randomized using a 2:1 ratio in a double-blind manner to receive oral lenalidomide 10 mg once daily or placebo once daily. Subjects will receive oral lenalidomide 10 mg once daily (one 10 mg lenalidomide capsules + 2 placebo capsule) or matching placebo once daily (3 placebo capsules).

Other: Placebo

3 placebo capsules once daily. Subjects may take study drug for at least 168 days unless there are intolerable side effects or disease progresses. Subjects may continue study drug beyond 168 days if they have an erythroid response (increase in their hemoglobin levels and fewer transfusions administered than before starting study drug)

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

18 years or older
Diagnosis of low or intermediate-1 risk MDS with any chromosome karyotype except del 5q[31]
Anemia that requires red blood cell transfusions
Resistant to erythropoiesis stimulating agents (ESAs) or blood erythropoietin level > 500 mU/mL
ECOG Performance Status ≤ 2
Must agree to follow pregnancy precautions as required by the protocol.
Must agree to receive counseling related to teratogenic and other risks of lenalidomide
Must agree not to donate blood or semen
Must be willing to consent to two or more bone marrow aspirate procedures to be completed during study

Exclusion Criteria:

Subjects previously receiving immunomodulating or immunosuppressive agents, or epigenetic or DNA modulation agents
Allergic reaction to thalidomide
Renal insufficiency (CrC1<40 mL/min by Cockroft-Gault method)
Prior history of cancer, other than MDS, unless the subject has been free of the disease for ≥ 5 years. (Basal cell carcinoma of the skin, Carcinoma in situ of the cervix, or stage T1a or T1b prostate cancer is allowed)
Absolute neutrophil count < 500/uL
Platelets < 50,000/uL
AST or ALT > 3X upper limit of normal
Uncontrolled hyperthyroidism or hypothyroidism
Significant neuropathy
Prior stem cell transplantation
Anemia due to reasons other than MDS
History of deep venous thrombosis (DVT) or pulmonary embolus (PE) within past 3 years
Significant active cardiac disease within the past 6 months
Known HIV infection; known Hepatitis C infection or active Hepatitis B infection

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01029262

Contacts

Contact: Anne McClain, RN, BSN	913-266-0336	amcclain@celgene.com
Contact: Joanna Sobierska	011-41-327298414	jsobierska@celgene.com

Show 98 Study Locations

Sponsors and Collaborators

Celgene Corporation

Investigators

Study Director:

Bouchra Benettaib, MD

Celgene Corporation

More Information

No publications provided

Responsible Party:	Celgene Corporation
ClinicalTrials.gov Identifier:	NCT01029262 History of Changes
Other Study ID Numbers:	CC-5013-MDS-005
Study First Received:	December 8, 2009
Last Updated:	March 11, 2013
Health Authority:	United States: Food and Drug Administration Australia: Department of Health and Ageing Therapeutic Goods Administration Austria: Federal Office for Safety in Health Care Belgium: Federal Agency for Medicinal Products and Health Products Canada: Health Canada Czech Republic: State Institute for Drug Control France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Germany: Federal Institute for Drugs and Medical Devices Israel: Ministry of Health Japan: Pharmaceuticals and Medical Devices Agency Italy: The Italian Medicines Agency Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Portugal: National Pharmacy and Medicines Institute Spain: Spanish Agency of Medicines Turkey: Ministry of Health United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Celgene Corporation:

Myelodysplastic Syndromes
MDS
transfusion dependent anemia
Erythropoiesis stimulating agents
non-del 5q

Additional relevant MeSH terms:

Anemia
Myelodysplastic Syndromes
Preleukemia
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Neoplasms
Lenalidomide
Thalidomide
Hematinics
Antineoplastic Agents
Therapeutic Uses

Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Hematologic Agents

ClinicalTrials.gov processed this record on May 21, 2013

A Study of Lenalidomide Versus Placebo in Subjects With Transfusion Dependent Anemia in Low Risk Myelodysplastic Syndrome (MDS) Without Del 5Q (MDS-005)