Clinical trial NCT01542684: Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)
Official title: Phase II Study of Azacytidine Followed by GM-CSF in Patients With Low- or Intermediate-1- Risk Myelodysplastic Syndrome (MDS)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Leukemia
Keywords: Leukemia
Myelodysplastic Syndrome
MDS
Azacytidine
5-Azacytidine
5-AZA
Vidaza
5-AZC
Aza-CR
Ladakamycin
NSC-102816
GM-CSF
Sargramostim
Leukine
Lead sponsor: M.D. Anderson Cancer Center
Sponsor class: OTHER
First posted: 2012-03-02
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this clinical research study is to learn if the combination of azacitidine and GM-CSF can help to control MDS. The safety of these drugs will also be studied.

Azacitidine is designed to block certain proteins that stop the function of tumor-fighting genes. By blocking the "bad" proteins, the tumor-fighting genes may be able to work better.

Granulocyte-macrophage colony-stimulating factor (GM-CSF) is designed to help produce white blood cells. This may help to fight infections.

Detailed description: Study Drug Administration:

If you are found to be eligible to take part in this study, on Days 1-4 of every cycle, you will receive azacitidine by vein over 15-30 minutes.

You may receive drugs to help prevent nausea and vomiting before you receive your dose of azacitidine.

On Days 5-7 of every cycle, you will receive GM-CSF by vein over about 15 minutes or by injection.

Each study cycle will be about 4-6 weeks, depending on the study doctor's decision.

Study Visits:

One (1) time each week during every cycle, blood (about 2-3 teaspoons) will be drawn for routine tests.

At any time, if your doctor thinks it is needed, you will have a bone marrow aspirate to check the status of the disease.

Length of Study:

You may continue taking the study drugs for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

Your follow-up visits will be per standard of care for the disease.

This is an investigational study. Both azacitidine and GM-CSF are FDA approved and commercially available for the treatment of MDS. The study drug combination to treat MDS is considered investigational.

Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.

Eligibility criteria: Inclusion Criteria:

1. Patients with low- or intermediate-1-risk MDS according to the International Prognostic Scoring System (IPSS) classification
2. Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UT MD Anderson Cancer Center.
3. Age \>/= 18 years old.
4. Prior therapy with growth factor support, lenalidomide, or other investigational agents is allowed.
5. Previously untreated patients are eligible for this study.

Exclusion Criteria:

1. Any previous adverse reaction (\>/= Common Terminology Criteria for Adverse Events (CTCAE) grade 2) to GM-CSF.
2. Prior treatment with azacytidine or decitabine.
3. Unresolved diarrhea \>/= CTCAE grade 2.

Primary outcomes:
measure: Overall Response Rate (ORR)
timeFrame: Baseline up to 2 treatment cycles (8 weeks)
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: UT MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: UT MD Anderson Cancer Center
name: Zeev Estrov, MD
role: PRINCIPAL_INVESTIGATOR
References: