Clinical trial NCT00053001: Thalidomide and Epoetin Alfa in Treating Anemia in Patients With Myelodysplastic Syndrome
Official title: A Phase II Study on the Effectiveness of Thalomid (Thalidomide) Combined With Procrit (Erythropoietin) for the Treatment of Anemia in Patients With Low and Intermediate Risk-1 (IPSS Score Less Than or Equal to 1.5) Myelodysplastic Syndromes
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Anemia
Leukemia
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasms
Keywords: de novo myelodysplastic syndromes
secondary myelodysplastic syndromes
previously treated myelodysplastic syndromes
anemia
atypical chronic myeloid leukemia, BCR-ABL1 negative
myelodysplastic/myeloproliferative neoplasm, unclassifiable
childhood myelodysplastic syndromes
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2003-01-28
Sex: ALL
Minimum age (years): 21
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: SUPPORTIVE_CARE

Brief summary: RATIONALE: Thalidomide may stop or slow the growth of cancer cells. Epoetin alfa may stimulate red blood cell production. Combining thalidomide with epoetin alfa may improve anemia, decrease the need for blood transfusions, and improve the quality of life in patients with myelodysplastic syndrome.

PURPOSE: Phase II trial to study the effectiveness of combining thalidomide with epoetin alfa in treating anemia in patients who have myelodysplastic syndrome.

Detailed description: OBJECTIVES:

* Determine whether the combination of epoetin alfa and thalidomide improves the anemia and/or decreases the need for red cell transfusion in patients with low- or intermediate-risk myelodysplastic syndromes.
* Determine whether this regimen improves the bone marrow morphology and cytogenetics, alters the natural history of the disease, and reduces the frequency of leukemic transformation in these patients.
* Evaluate whether this regimen improves pathophysiologic parameters (e.g., apoptosis, tumor necrosis factor-alpha concentration, microvessel density, vascular endothelial growth factor, and cytotoxic T lymphocytes) in the bone marrow of these patients.
* Determine the safety of this regimen in these patients.

OUTLINE: Patients receive epoetin alfa subcutaneously (SC) once weekly for 8 weeks. After 8 weeks, patients unresponsive to epoetin alfa alone receive oral thalidomide once daily in addition to epoetin alfa SC once weekly for a maximum of 24 weeks in the absence of disease progression or unacceptable toxicity.

PROJECTED ACCRUAL: A total of 30-40 patients will be accrued for this study within 2 years..

Eligibility criteria: DISEASE CHARACTERISTICS:

* Diagnosis of myelodysplastic syndromes

  * Newly diagnosed OR
  * Prior treatment was unsuccessful, including treatment with chemotherapy
* International prognostic scoring system score no greater than 1.5
* Hemoglobin no greater than 10 g/dL (untransfused) AND/OR
* Received at least 3 units of packed red blood cells for symptomatic anemia within the past 6 weeks

PATIENT CHARACTERISTICS:

Age

* Over 21

Performance status

* Karnofsky 70-100%

Life expectancy

* At least 6 months

Hematopoietic

* See Disease Characteristics
* No prior bleeding disorder

Hepatic

* Bilirubin less than 2 mg/dL
* ALT/AST less than 2 times upper limit of normal

Renal

* Creatinine less than 1.5 mg/dL

Cardiovascular

* No prior clinically significant heart disease
* No uncontrolled hypertension
* No recent thromboembolic disease (e.g., deep vein thrombosis)

  * Prior thromboembolic events allowed provided event occurred at least 6 weeks prior to study and patient is on anticoagulants and is clinically stable

Pulmonary

* No unstable pulmonary disease
* No recent pulmonary embolism
* No active pulmonary infection

Neurologic

* No pre-existing peripheral neuropathy greater than grade 2
* No sustained neurologic deficit
* No epilepsy

Other

* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use 2 effective methods (including 1 highly effective method) of contraception for at least 4 weeks before, during, and for at least 4 weeks after study completion
* No active infection
* No concurrent illness that would obscure toxicity or dangerously alter drug metabolism
* No other serious concurrent medical illness
* No uncontrolled diabetes mellitus
* No other malignant disease (except non-melanoma skin cancer or carcinoma in situ of the cervix) unless in complete remission and off therapy for that disease for more than 1 year
* No known hypersensitivity to mammalian cell-derived products or human albumin

PRIOR CONCURRENT THERAPY:

Biologic therapy

* Not specified

Chemotherapy

* See Disease Characteristics

Endocrine therapy

* Not specified

Radiotherapy

* Not specified

Surgery

* Not specified

Other

* At least 4-6 weeks since prior therapy

Primary outcomes:
measure: Clinical response
Secondary outcomes:
Other outcomes:
Locations:
city: Worcester
country: United States
facility: Fallon Clinic at Worcester Medical Center
state: Massachusetts
city: Worcester
country: United States
facility: UMASS Memorial Cancer Center - University Campus
state: Massachusetts
Study officials:
affiliation: Fallon Clinic
name: Laszlo Leb, MD
role: STUDY_CHAIR
References: