Ignite Creation Date:
2025-12-24 @ 4:46 PM
Ignite Modification Date:
2026-02-27 @ 12:49 AM
Study NCT ID:
NCT00030550
Status:
COMPLETED
Last Update Posted:
2013-01-31
First Post:
2002-02-14
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D054437', 'term': 'Myelodysplastic-Myeloproliferative Diseases'}, {'id': 'D000753', 'term': 'Anemia, Refractory'}, {'id': 'D000754', 'term': 'Anemia, Refractory, with Excess of Blasts'}, {'id': 'D015477', 'term': 'Leukemia, Myelomonocytic, Chronic'}, {'id': 'D009196', 'term': 'Myeloproliferative Disorders'}, {'id': 'D054438', 'term': 'Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative'}], 'ancestors': [{'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D013792', 'term': 'Thalidomide'}], 'ancestors': [{'id': 'D010797', 'term': 'Phthalimides'}, {'id': 'D010795', 'term': 'Phthalic Acids'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D010881', 'term': 'Piperidones'}, {'id': 'D010880', 'term': 'Piperidines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D054833', 'term': 'Isoindoles'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE'}, 'primaryPurpose': 'TREATMENT'}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2001-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-01', 'lastUpdateSubmitDate': '2013-01-30', 'studyFirstSubmitDate': '2002-02-14', 'studyFirstSubmitQcDate': '2003-01-26', 'lastUpdatePostDateStruct': {'date': '2013-01-31', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2003-01-27', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2003-06', 'type': 'ACTUAL'}}, 'conditionsModule': {'keywords': ['refractory anemia', 'refractory anemia with ringed sideroblasts', 'refractory anemia with excess blasts', 'de novo myelodysplastic syndromes', 'chronic myelomonocytic leukemia', 'previously treated myelodysplastic syndromes', 'myelodysplastic/myeloproliferative neoplasm, unclassifiable', 'atypical chronic myeloid leukemia, BCR-ABL1 negative'], 'conditions': ['Leukemia', 'Myelodysplastic Syndromes', 'Myelodysplastic/Myeloproliferative Neoplasms']}, 'descriptionModule': {'briefSummary': 'RATIONALE: Thalidomide may be an effective treatment for anemia caused by myelodysplastic syndrome.\n\nPURPOSE: Randomized phase II trial to study the effectiveness of thalidomide in treating anemia in patients who have myelodysplastic syndrome.', 'detailedDescription': 'OBJECTIVES:\n\n* Determine the efficacy of thalidomide for the treatment of anemia in patients with myelodysplastic syndromes.\n* Determine whether this drug reduces the frequency of leukemia transformation and decreases bone marrow blast percentage in these patients.\n* Determine the effect of this drug on neutrophil and platelet production and the number of episodes of febrile neutropenia in these patients.\n* Determine the safety of this drug in these patients.\n\nOUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified according to International Prognostic Scoring System score (low and intermediate-1 vs intermediate-2 and high) and transfusion dependence (yes vs no). Patients are randomized to one of two treatment arms.\n\n* Arm I: Patients receive oral thalidomide once daily on weeks 1-24.\n* Arm II: Patients receive oral placebo once daily on weeks 1-24. In both arms, patients who have not progressed to leukemia after 24 weeks of therapy may receive open-label thalidomide for an additional 24 weeks in the absence of disease progression or unacceptable toxicity.\n\nPatients are followed at 4 weeks.\n\nPROJECTED ACCRUAL: A total of 220 patients (110 per treatment arm) will be accrued for this study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'DISEASE CHARACTERISTICS:\n\n* Diagnosis of myelodysplastic syndromes (MDS) of at least 12 weeks duration\n\n * Refractory anemia (RA)\n * RA with ringed sideroblasts\n * RA with excess blasts\n * Chronic myelomonocytic\n* No therapy-related MDS\n* No myelosclerosis or myelofibrosis occupying more than 30% of marrow space (or assessed as grade 3+ or greater)\n* No transformation to acute myeloid leukemia\n* No more than 20% blasts in bone marrow\n* No more than 5% blasts in peripheral blood\n* Patients with an erythropoietin level 100 mU/mL or less must have failed epoetin alfa treatment (i.e., at least 30,000 units of epoetin alfa weekly for at least 6 weeks)\n* Transfusion-dependent (received at least 2 units of packed RBCs or whole blood within the past 8 weeks) OR\n* Transfusion-independent (no packed RBC or whole blood transfusions within the past 8 weeks with 2 hemoglobin levels (at least 7 days apart) less than 11 g/dL)\n* No iron deficiency (e.g., absent bone marrow iron store)\n\n * If marrow aspirate is not evaluable, transferrin saturation must be at least 20% and ferritin at least 50 ng/mL\n* No uncorrected B12 or folate deficiency\n* No other contributing causes of anemia (e.g., autoimmune or hereditary hemolytic disorders or gastrointestinal blood loss)\n\nPATIENT CHARACTERISTICS:\n\nAge:\n\n* 18 and over\n\nPerformance status:\n\n* ECOG 0-2 OR\n* Zubrod 0-2\n\nLife expectancy:\n\n* At least 6 months\n\nHematopoietic:\n\n* See Disease Characteristics\n* Absolute neutrophil count at least 500/mm\\^3\n\nHepatic:\n\n* Bilirubin no greater than 2.0 mg/dL\n* AST and ALT less than 2 times upper limit of normal (ULN)\n* Hepatitis B surface antigen negative\n* Hepatitis C negative\n\nRenal:\n\n* Creatinine no greater than 1.5 times ULN\n\nCardiovascular:\n\n* No uncontrolled hypertension\n* No clinically significant, symptomatic, unstable cardiovascular disease unrelated to MDS\n\nPulmonary:\n\n* No clinically significant, symptomatic, unstable pulmonary disease unrelated to MDS\n\nNeurologic:\n\n* No clinically significant, symptomatic, unstable neurologic disease unrelated to MDS\n* No history of epilepsy\n* No sustained neurologic deficit (e.g., stroke)\n* No grade 2 or greater peripheral neuropathy\n\nOther:\n\n* Not pregnant or nursing\n* Negative pregnancy test\n* Fertile patients must use at least 1 highly effective and 1 additional effective method of contraception for 4 weeks prior to, during, and for 4 weeks after study participation\n* HIV negative\n* No clinically significant, symptomatic, unstable endocrine, gastrointestinal, or genitourinary disease unrelated to MDS\n* No other malignancy within the past 5 years except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix\n* No life-threatening or active infection requiring parenteral antibiotics\n* No other serious concurrent illness\n\nPRIOR CONCURRENT THERAPY:\n\nBiologic therapy:\n\n* See Disease Characteristics\n* More than 7 days since prior hematopoietic growth factors (e.g., epoetin alfa, filgrastim (G-CSF), sargramostim (GM-CSF), or interleukin-3)\n* No prior thalidomide\n* No prior agents intended to inhibit vascular endothelial growth factor or tumor necrosis factor alfa (e.g., etanercept or infliximab)\n* No concurrent epoetin alfa\n\nChemotherapy:\n\n* No concurrent chemotherapy that may be active against MDS\n\nEndocrine therapy:\n\n* More than 30 days since prior androgens\n* No requirement for ongoing therapy with systemic corticosteroids\n\nRadiotherapy:\n\n* Not specified\n\nSurgery:\n\n* Not specified\n\nOther:\n\n* More than 30 days since prior treatment for MDS except RBC transfusion or epoetin alfa\n* More than 30 days since prior participation in another experimental clinical trial\n* More than 30 days since prior experimental drugs\n* No other concurrent investigational agents or treatments'}, 'identificationModule': {'nctId': 'NCT00030550', 'briefTitle': 'Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome', 'organization': {'class': 'OTHER', 'fullName': 'Roswell Park Cancer Institute'}, 'officialTitle': 'A Randomized, Multi-Center, Double-Blind, Placebo-Controlled Trial Assessing The Safety And Efficacy Of Thalidomide (THALOMID) For The Treatment Of Anemia In Red Blood Cell Transfusion-Dependent Patients With Myelodysplastic Syndromes', 'orgStudyIdInfo': {'id': 'DS 01-16'}, 'secondaryIdInfos': [{'id': 'RPCI-DS-0116'}, {'id': 'CELGENE-T-MDS-001'}, {'id': 'NCI-G01-2044'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'thalidomide', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'locations': [{'zip': '28412', 'city': 'Wilmington', 'state': 'North Carolina', 'country': 'United States', 'facility': 'PPD Development', 'geoPoint': {'lat': 34.23556, 'lon': -77.94604}}], 'overallOfficials': [{'name': 'James L. Slack, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Roswell Park Cancer Institute'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Roswell Park Cancer Institute', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Cancer Institute (NCI)', 'class': 'NIH'}]}}}