Clinical trial NCT00520468: Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS
Official title: Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelodysplastic Syndrome
Keywords: Myelodysplastic Syndrome
MDS
Erythropoietin
Darbepoetin alfa
Aranesp
Erythropoiesis stimulating protein
G-CSF
Filgrastim
Neupogen
Prednisone
Cyclosporin A
Sandimmune
CYA
Cyclosporine
Lead sponsor: M.D. Anderson Cancer Center
Sponsor class: OTHER
First posted: 2007-08-24
Enrollment: 15
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed 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a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle.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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Response
timeFrame: Response evaluation within first 3 months from start of therapy, then every 3 to 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: U.T.M.D. Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Gautam Borthakur, MBBS
role: PRINCIPAL_INVESTIGATOR
References: