Thalidomide in Treating Patients With Myelodysplastic Syndrome

NCT00015990 | Completed Phase 2

• 4 other identifiers: NCI-2012-01856N998BU10CA025224CDR0000068580
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

Phase II trial to study the effectiveness of thalidomide in treating patients who have myelodysplastic syndrome. Thalidomide may improve the immune system's ability to fight myelodysplastic syndrome

Conditions

Chronic Myelomonocytic Leukemia; de Novo Myelodysplastic Syndromes; Previously Treated Myelodysplastic Syndromes; Refractory Anemia; Refractory Anemia With Excess Blasts; Refractory Anemia With Excess Blasts in Transformation; Refractory Anemia With Ringed Sideroblasts; Secondary Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (2)

• Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al

  Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.

  Up to 3 months

• Incidence and severity of toxicities, graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0

  Up to 5 years

Secondary Outcomes (4)

• Survival time

  Time from registration to death due to any cause, assessed up to 5 years

• Time to disease progression

  Time from registration to documentation of disease progression, assessed up to 5 years

• Duration of response measured using criteria reported by Cheson et al

  Date at which the patient's objective status is first noted to be either a CHR or PR to the date progression is documented, assessed up to 5 years

• Time to treatment failure

  Time from the date of registration to the date at which the patient is removed from treatment due to progression, toxicity, or refusal, assessed up to 5 years

Study Arms (1)

Treatment (thalidomide)

EXPERIMENTAL

Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.

Drug: thalidomide; Other: laboratory biomarker analysis

Interventions

thalidomide DRUG

Given orally

Also known as: Kevadon, Synovir, THAL, Thalomid

Treatment (thalidomide)

laboratory biomarker analysis OTHER

Correlative studies

Treatment (thalidomide)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Pre-transfusion hemoglobin =\< 10 g/dL
• Pre-transfusion platelet count =\< 50,000/μL
• Absolute neutrophil count \< 1000/μL
• Total bilirubin ≤ 1.5 x UNL
• Alkaline phosphatase ≤ 3 x UNL
• AST ≤ 3 x UNL
• Creatinine ≤ 1.5 x UNL
• A diagnosis of MDS as demonstrated in the bone marrow; any subtypes are eligible including:
• Refractory anemia (cytopenia)
• Refractory anemia with ringed sideroblasts
• Chronic myelomonocytic leukemia
• Refractory anemia with excess blasts
• Refractory anemia with excess blasts in transformation
• Unclassified MDS
• Patients with refractory anemia with excess blasts in transformation who are not candidates for (or who decline) induction chemotherapy are eligible; those patients who were candidates for (and accepted) induction chemotherapy should have failed at least 1 chemotherapy regimen prior to entry

You may not qualify if:

• Any of the following as this regimen may be harmful to a developing fetus or nursing child:
• Pregnant women
• Nursing women
• Women of childbearing potential or their sexual partners who are unwilling to employ 2 adequate methods of contraception (condoms, diaphragm, birth control pills, injections, intrauterine device \[IUD\], surgical sterilization, subcutaneous implants, or abstinence, etc.)
• Peripheral neuropathy (by history or clinical exam)
• Concomitant therapy ≤ 30 days for myelodysplastic syndrome with any specific agent including chemotherapy, corticosteroids and/or growth factors (i.e. erythropoietin, G-CSF, GM-CSF, thrombopoietic agent); patients on chronic low-dose corticosteriods (\< 20 mg/d) for reasons other than MDS are allowed
• Uncontrolled infections

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

North Central Cancer Treatment Group

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Leukemia, Myelomonocytic, Chronic; Anemia, Refractory; Anemia, Refractory, with Excess of Blasts

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Anemia; Myelodysplastic Syndromes

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Alvaro Moreno Aspitia

  North Central Cancer Treatment Group

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2001
• First Posted: February 27, 2004
• Study Start: April 1, 2001
• Primary Completion: February 1, 2007
• Last Updated: January 24, 2013

Record last verified: 2013-01

Locations

US (1)