Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)

NCT01542684 | Terminated Phase 2 Results

• 1 other identifier: 2011-1123
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

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.

Conditions

Leukemia

Keywords

Leukemia; Myelodysplastic Syndrome; MDS; Azacytidine; 5-Azacytidine; 5-AZA; Vidaza; 5-AZC; Aza-CR; Ladakamycin; NSC-102816; GM-CSF; Sargramostim; Leukine

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR)

  ORR is percentage total participants with overall response (Complete Response (CR) or Partial Response (PR)) within two treatment cycles. Response based on modified International Working Group (IWG) criteria: Complete response - Bone marrow: 5% myeloblasts with normal maturation of all cell lines, Persistent dysplasia noted, Peripheral blood Hgb 11 g/dL, Platelets 100x109/L, Neutrophils 1.0x109/L, Blasts 0%. Partial response: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by 50% over pretreatment but still \> 5% , Cellularity and morphology not relevant; Stable disease - Failure to achieve at least PR, but no evidence of progression for \> 8 weeks; No Response or Failure - Death during treatment or disease progression characterized by worsening of cytopenias, increase in percentage of bone marrow blasts, or progression to a more advanced MDS French-American-British (FAB) classification subtype than pretreatme

  Baseline up to 2 treatment cycles (8 weeks)

Study Arms (1)

Azacytidine + GM-CSF

EXPERIMENTAL

Azacytidine administered intravenously (IV) or subcutaneously (SQ) at starting dose of 40 mg/m\^2, daily for 4 days. GM-CSF administered IV or subcutaneously at 250 mcg/m\^2 one day (the next day) after completion of azacytidine treatment, for 3 consecutive days. Each treatment cycle will last at least 4 weeks

Drug: Azacytidine; Drug: GM-CSF

Interventions

Azacytidine DRUG

Starting dose: 40 mg/m\^2 intravenously (IV) or subcutaneously (SQ) daily for 4 days.

Also known as: 5-Azacytidine, 5-AZA, Vidaza, 5-AZC, AZA-CR, Ladakamycin, NSC-102816

Azacytidine + GM-CSF

GM-CSF DRUG

250 mcg/m\^2 IV or SQ one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.

Also known as: Saragramostim, Leukine

Azacytidine + GM-CSF

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with low- or intermediate-1-risk MDS according to the International Prognostic Scoring System (IPSS) classification
• 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.
• Age \>/= 18 years old.
• Prior therapy with growth factor support, lenalidomide, or other investigational agents is allowed.
• Previously untreated patients are eligible for this study.

You may not qualify if:

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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

UT MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• UT MD Anderson Cancer Center Website

MeSH Terms

Conditions

Leukemia; Myelodysplastic Syndromes

Interventions

Azacitidine; Granulocyte-Macrophage Colony-Stimulating Factor; sargramostim

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: Zeev Estrov, MD/Professor
• Organization: The University of Texas MD Anderson Cancer Center

eharriso@mdanderson.org

713-794-1675

Study Officials

• Zeev Estrov, MD

  UT MD Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2012
• First Posted: March 2, 2012
• Study Start: March 1, 2012
• Primary Completion: April 1, 2013
• Study Completion: April 1, 2013
• Last Updated: April 30, 2014
• Results First Posted: April 30, 2014

Record last verified: 2014-03

Locations

US (1)