Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS

Myelodysplastic Syndrome Clinical Trial

Official title:

Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00520468
• Other study ID #: 2004-0253
• Status: Completed
• Phase: Phase 2
• First received: August 23, 2007
• Last updated: August 1, 2012
• Start date: June 2004
• Est. completion date: June 2009

Study information

• Verified date: August 2012
• Source: M.D. Anderson Cancer Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

Objectives:

Primary: To evaluate the response rate of total cytokine-immunotherapy for low-risk myelodysplastic syndromes (MDS).

Secondary: To evaluate response duration, survival and side effects of the treatment.

Description:

MDS is a disease that produces low blood counts and may cause anemia, infections, and/or bleeding. Erythropoietin and Granulocyte colony-stimulating factor (G-CSF or GCSF) are drugs that stimulate the production of red cells and white cells. Prednisone and cyclosporin are drugs that work against MDS by affecting your immune system.

Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have a complete medical history and physical exam. Routine blood tests (between 4-6 tablespoons) will be performed. You will have a bone marrow aspiration. To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative blood or urine pregnancy test.

If you are found to be eligible to take part in this study, you will receive erythropoietin as an injection under the skin once a week and G-CSF as an injection under the skin 1-2 times a week for as long as you respond well to treatment. You will take prednisone by mouth every day for a month and cyclosporin tablets by mouth every day for 6 months.

During this study, you will need to visit your doctor for a physical exam and measurement of your vital signs. The frequency of doctor visits will vary depending on your physical condition, but will be required at least once every 3 months.

Blood tests (about 2 teaspoons) will be done about every 1-2 weeks during the first 12 weeks of treatment, then every 2 to 4 weeks for the remainder of the study. The blood samples will be collected for routine lab tests. Periodic (every 3 to 6 months) bone marrow samples will also be taken to check cells related to the disease before, during, and after completion of this study.

You will be taken off study if the disease gets worse or intolerable side effects occur.

This is an investigational study. All drugs are FDA approved and commercially available. Up to 60 patients will take part in this study. All will be enrolled at M. D. Anderson.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: June 2009
• Est. primary completion date: June 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Patients with MDS and </= 10% blasts or International Prognostic Scoring System (IPSS) low or intermediate 1. No prior intensive chemotherapy or high-dose ara-C (>/= 1g/m2). Prior cytokines, biologic therapies, targeted therapies, or single agent chemotherapy allowed. Procrit, G-CSF are allowed before therapy. Patients with blasts < 5% must have an indication for therapy, such as transfusion needs, symptomatic anemia or Hb < 11g/dl, platelets < 100 x 10 9/L, or granulocytes < 10 9/L.

2. Performance 0-2 (Eastern Cooperative Oncology Group (ECOG)). Adequate liver function (bilirubin of < 2mg/dl) and renal function (creatinine < 2mg/dl). Adequate cardiac functions (New York Heart Association (NYHA) cardiac III-IV excluded)

3. Signed informed consent

Exclusion Criteria:

1. Nursing and pregnant females are excluded. Women of childbearing potential should practice effective methods of contraception. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.

2. Patients with active and uncontrolled infections.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

Drug:
• Erythropoietin
40,000 units injected under the skin (SQ) weekly
• Cyclosporin A
300 mg (tablets) by mouth daily for 6 months
• G-CSF
300 mcg injected under the skin (SQ) two times per week
• Prednisone
60 mg per day for 7 days, taper over 1 month

Locations

Country	Name	City	State
United States	U.T.M.D. Anderson Cancer Center	Houston	Texas

Sponsors (1)

Lead Sponsor	Collaborator
M.D. Anderson Cancer Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Response	Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.	Response evaluation within first 3 months from start of therapy, then every 3 to 6 months	No

External Links

•   M.D. Anderson Cancer Center's Website