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LeukemiaPhase 2NCT00030550

Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome

A Phase 2 study testing thalidomide in people with Leukemia and Myelodysplastic Syndromes, run by Roswell Park Cancer Institute at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
thalidomide
How many people
Not given
Where
1 siteUnited States
Study dates
Sep 2001to Jun 2003
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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

Trial locations and contacts

1 site in 1 country.

United States1 site
  • PPD Development

    Wilmington, North Carolina, 28412

Other trials in United States

About this study

Official title: 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

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.

Read the detailed description

OBJECTIVES:

  • Determine the efficacy of thalidomide for the treatment of anemia in patients with myelodysplastic syndromes.
  • Determine whether this drug reduces the frequency of leukemia transformation and decreases bone marrow blast percentage in these patients.
  • Determine the effect of this drug on neutrophil and platelet production and the number of episodes of febrile neutropenia in these patients.
  • Determine the safety of this drug in these patients.

OUTLINE: 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.

  • Arm I: Patients receive oral thalidomide once daily on weeks 1-24.
  • 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.

Patients are followed at 4 weeks.

PROJECTED ACCRUAL: A total of 220 patients (110 per treatment arm) will be accrued for this study.

Being tested
thalidomide
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
Medical subject headings
Leukemia, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Anemia, Refractory, Anemia, Refractory, with Excess of Blasts, Leukemia, Myelomonocytic, Chronic, Myeloproliferative Disorders, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Jan 2003 (estimated)
  2. Study startSep 2001
  3. Primary completionJun 2003

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Masking
Double blind
IDs, dates and publications
ClinicalTrials.gov ID
NCT00030550
Sponsor's protocol ID
DS 01-16
Other IDs
RPCI-DS-0116, CELGENE-T-MDS-001, NCI-G01-2044
Status last verified
Jan 2013
First submitted
14 Feb 2002
Last updated
31 Jan 2013
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing thalidomide in people with Leukemia and Myelodysplastic Syndromes, run by Roswell Park Cancer Institute at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2013.

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