Stanford, California 94305

  • Dysmyelopoietic Syndromes

Purpose:

This is a single-center, single arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion dependent adult subjects with Diamond-Blackfan Anemia (DBA). Primary Objective: To evaluate the erythroid response rate as measured by rate of red blood cell transfusion independence (MDS IWG 2000 Criteria will be applied) Secondary Objective: 1)To evaluate the tolerability and safety profile of lenalidomide in patients with DBA and other inherited marrow failure syndromes 2) To correlate response to lenalidomide with biologic surrogates of DBA including ribosomal protein mutation status, ex vivo erythroid colony growth, and microarray gene expression


Criteria:

Inclusion Criteria:1. Understand and voluntarily sign an informed consent form. 2. Diagnosis of DBA. 3.. Age >=18 years at the time of signing the informed consent form. 4. Able to adhere to the study visit schedule and other protocol requirements. 5. Red blood cell transfusion-dependent with a requirement of at least one unit of RBCs per month for the 2 months prior to study enrollment (e.g. 2 units/8 weeks) 6. If applicable, ongoing therapy with a stable or decreasing dose of prednisone <= 60 mg/d or corticosteroid equivalent, for which there has been no treatment-related improvement in RBC transfusion requirements for at least 2 months prior to study entry 7. ECOG performance status of <= 2 at study entry. 8. Laboratory test results within these ranges: - Absolute neutrophil count >= 1500/mm>= - Platelet count >= 100,000/mm>= - Serum creatinine <= 2.0 mg/dL - Direct bilirubin <= 1.5 mg/dL - AST (SGOT) and ALT (SGPT) <= 2.5 x ULN - Disease free of prior malignancies for >= 5 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in-situ" of the cervix or breast. 9. Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 10 - 14 days prior to and again within 24 hours of prescribing lenalidomide (prescriptions must be filled within 7 days) and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking lenalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy. See Appendix A: Risks of Fetal Exposure, Pregnancy Testing Guidelines and Acceptable Birth Control Methods. 10. Able to take aspirin (81 - >=25 mg) daily as prophylactic anticoagulation (patients intolerant to ASA may use warfarin or low molecular weight heparin) Exclusion Criteria:1. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form. 2. Pregnant or breast feeding females. (Lactating females must agree not to breast feed while taking lenalidomide). 3. Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study. 4. Use of any other experimental drug or therapy (excluding steroids) specifically used for DBA within 28 days of baseline including metoclopramide, leucine, danazol, or other hormonal therapy. 5. Clinically significant anemia due to factors such as iron, B12, folate deficiencies, autoimmune or hereditary hemolysis, or gastrointestinal bleeding. 6. Known hypersensitivity to thalidomide. 7. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs. 8. Any prior use of lenalidomide. 9. Concurrent use of other anti-cancer agents or treatments. 10. Known positive for HIV or infectious hepatitis, type A, B or C.


NCT ID:

NCT01034592


Primary Contact:

Principal Investigator
Jason Robert Gotlib
Stanford University

Andrea Linder
Phone: (650) 725-4047
Email: andrea.linder@stanford.edu


Backup Contact:

N/A


Location Contact:

Stanford, California 94305
United States

Andrea Linder
Phone: 650-725-4047
Email: andrea.linder@stanford.edu

Site Status: Recruiting


Data Source: ClinicalTrials.gov

Date Processed: September 27, 2021

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