Clinical trial · Interventional
Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)
Phase II Study of Azacytidine Followed by GM-CSF in Patients With Low- or Intermediate-1- Risk Myelodysplastic Syndrome (MDS)
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Slow Accrual
Summary
Brief summary (as posted)
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
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Azacytidine | Drug | Azacitidine | ALIAS |
| GM-CSF | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Azacytidine + GM-CSF
- description
- 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
- interventionNames
- Drug: Azacytidine
- Drug: GM-CSF
Primary outcomes (1)
- measure
- Overall Response Rate (ORR)
- timeFrame
- Baseline up to 2 treatment cycles (8 weeks)
- description
- 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
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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.
References
Publications (0)
Data not yet available