NCT00030550

Brief Summary

RATIONALE: Thalidomide may be an effective treatment for anemia caused by myelodysplastic syndrome. PURPOSE: Randomized phase II trial to study the effectiveness of thalidomide in treating anemia in patients who have myelodysplastic syndrome.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2001

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

February 14, 2002

Completed
12 months until next milestone

First Posted

Study publicly available on registry

January 27, 2003

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2003

Completed
Last Updated

January 31, 2013

Status Verified

January 1, 2013

Enrollment Period

1.7 years

First QC Date

February 14, 2002

Last Update Submit

January 30, 2013

Conditions

LeukemiaMyelodysplastic SyndromesMyelodysplastic/Myeloproliferative Neoplasms

Keywords

refractory anemiarefractory anemia with ringed sideroblastsrefractory anemia with excess blastsde novo myelodysplastic syndromeschronic myelomonocytic leukemiapreviously treated myelodysplastic syndromesmyelodysplastic/myeloproliferative neoplasm, unclassifiableatypical chronic myeloid leukemia, BCR-ABL1 negative

Interventions

thalidomideDRUG

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
DISEASE CHARACTERISTICS: * Diagnosis of myelodysplastic syndromes (MDS) of at least 12 weeks duration * Refractory anemia (RA) * RA with ringed sideroblasts * RA with excess blasts * Chronic myelomonocytic * No therapy-related MDS * No myelosclerosis or myelofibrosis occupying more than 30% of marrow space (or assessed as grade 3+ or greater) * No transformation to acute myeloid leukemia * No more than 20% blasts in bone marrow * No more than 5% blasts in peripheral blood * 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) * Transfusion-dependent (received at least 2 units of packed RBCs or whole blood within the past 8 weeks) OR * 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) * No iron deficiency (e.g., absent bone marrow iron store) * If marrow aspirate is not evaluable, transferrin saturation must be at least 20% and ferritin at least 50 ng/mL * No uncorrected B12 or folate deficiency * No other contributing causes of anemia (e.g., autoimmune or hereditary hemolytic disorders or gastrointestinal blood loss) PATIENT CHARACTERISTICS: Age: * 18 and over Performance status: * ECOG 0-2 OR * Zubrod 0-2 Life expectancy: * At least 6 months Hematopoietic: * See Disease Characteristics * Absolute neutrophil count at least 500/mm\^3 Hepatic: * Bilirubin no greater than 2.0 mg/dL * AST and ALT less than 2 times upper limit of normal (ULN) * Hepatitis B surface antigen negative * Hepatitis C negative Renal: * Creatinine no greater than 1.5 times ULN Cardiovascular: * No uncontrolled hypertension * No clinically significant, symptomatic, unstable cardiovascular disease unrelated to MDS Pulmonary: * No clinically significant, symptomatic, unstable pulmonary disease unrelated to MDS Neurologic: * No clinically significant, symptomatic, unstable neurologic disease unrelated to MDS * No history of epilepsy * No sustained neurologic deficit (e.g., stroke) * No grade 2 or greater peripheral neuropathy Other: * Not pregnant or nursing * Negative pregnancy test * 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 * HIV negative * No clinically significant, symptomatic, unstable endocrine, gastrointestinal, or genitourinary disease unrelated to MDS * No other malignancy within the past 5 years except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix * No life-threatening or active infection requiring parenteral antibiotics * No other serious concurrent illness PRIOR CONCURRENT THERAPY: Biologic therapy: * See Disease Characteristics * More than 7 days since prior hematopoietic growth factors (e.g., epoetin alfa, filgrastim (G-CSF), sargramostim (GM-CSF), or interleukin-3) * No prior thalidomide * No prior agents intended to inhibit vascular endothelial growth factor or tumor necrosis factor alfa (e.g., etanercept or infliximab) * No concurrent epoetin alfa Chemotherapy: * No concurrent chemotherapy that may be active against MDS Endocrine therapy: * More than 30 days since prior androgens * No requirement for ongoing therapy with systemic corticosteroids Radiotherapy: * Not specified Surgery: * Not specified Other: * More than 30 days since prior treatment for MDS except RBC transfusion or epoetin alfa * More than 30 days since prior participation in another experimental clinical trial * More than 30 days since prior experimental drugs * No other concurrent investigational agents or treatments

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

PPD Development

Wilmington, North Carolina, 28412, United States

Location

MeSH Terms

Conditions

LeukemiaMyelodysplastic SyndromesMyelodysplastic-Myeloproliferative DiseasesAnemia, RefractoryAnemia, Refractory, with Excess of BlastsLeukemia, Myelomonocytic, ChronicMyeloproliferative DisordersLeukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow DiseasesAnemiaLeukemia, MyeloidChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

PhthalimidesPhthalic AcidsAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsPiperidonesPiperidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsIsoindolesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Study Officials

  • James L. Slack, MD

    Roswell Park Cancer Institute

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

February 14, 2002

First Posted

January 27, 2003

Study Start

September 1, 2001

Primary Completion

June 1, 2003

Last Updated

January 31, 2013

Record last verified: 2013-01

Locations

US(1)