Clinical trial · Interventional
Safety, Pharmacokinetics, and Pharmacodynamics of Oral Azacitidine in Subjects With Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Acute Myelogenous Leukemia
A Phase 1, Open-label, Dose-Escalation Trial to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Oral Azacitidine in Subjects With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML) or Acute Myelogenous Leukemia (AML).
NCT00528983CI-TRIAL-00041533completedPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The purpose of this study is to determine whether a tablet form of azacitidine that taken by mouth is safe. This Phase I study will also look at different doses and different treatment schedules in order to better understand the effects (positive and negative) of oral azacitidine on the body and on the disease MDS, AML and CMML.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myelogenous Leukemia (AML) | Acute Myeloid Leukemia | ALIAS | 0.85 |
| Chronic Myelomonocytic Leukemia (CMML) | Chronic Myelomonocytic Leukemia | ALIAS | 0.90 |
| Myelodysplastic Syndromes (MDS) | Myelodysplastic Syndrome | ALIAS | 0.85 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Oral Azacitidine | Drug | Azacitidine | ALIAS |
| Subcutaneous (SC) Azacitidine | Drug | Azacitidine | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Subcutaneous (SC) Azacitidine and Oral Azacitidine
- description
- Cycle 1 subjects receive SC Azacitidine for first 7 days of 28 day cycle. For Cycle 2 and beyond subjects receive Oral Azacitidine (experimental) for first 7 days of 28 day cycle.
- interventionNames
- Drug: Subcutaneous (SC) Azacitidine
- Drug: Oral Azacitidine
- type
- EXPERIMENTAL
- label
- Oral Azacitidine
- description
- Subjects receive Oral Azacitidine (experimental) QD or BID for the first 14 or 21 days of 28 day cycle.
- interventionNames
- Drug: Oral Azacitidine
Primary outcomes (3)
- measure
- Safety evaluation as measured by monitoring AEs, dose limiting toxicities, scheduled lab assessments, vital sign measurements, ECGs, & physical exams for study duration. Adverse changes in physical signs/symptoms will be graded according to CTC AE V.3.0.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * 18 years or older. * Diagnosis of low or Int-1 risk MDS * Low platelet count, and/or low hemoglobin, and/or RBC transfusion-dependent and/or platelet transfusion-dependent * ECOG Performance status 0-2 * Standard safety inclusion for serum creatinine, AST, ALT, bilirubin. * Serum bicarbonate greater than or equal to 20 mEq/L. * Use of acceptable birth control. * Signed, written informed consent. Exclusion Criteria: * Diagnosis of acute PML. * Previous or concurrent malignancy. * Prior treatment with azacitidine or other demethylating agents. * Treatment with any anticancer therapy or investigational drugs within 21 days. * Hypersensitivity to azacitidine or mannitol. * Presence of GI disease. * Active, uncontrolled infection. * Known Human Immunodeficiency Virus (HIV) or Hepatitis C, or known active viral Hepatitis B. * Breastfeeding or Pregnant females; * Presence of serious illness, medical condition, or other medical history which would be likely to interfere with a subject's participation in the study or with the interpretation of the results. * Current congestive heart failure (NY Heart Association Class III-IV), unstable angina or angina requiring surgical or medical intervention within 6 months, myocardial infarct within 6 months, or uncontrolled cardiac arrhythmia.
References
Publications (3)
- BACKGROUNDGarcia-Manero G, Gore SD, Cogle C, Ward R, Shi T, Macbeth KJ, Laille E, Giordano H, Sakoian S, Jabbour E, Kantarjian H, Skikne B. Phase I study of oral azacitidine in myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia. J Clin Oncol. 2011 Jun 20;29(18):2521-7. doi: 10.1200/JCO.2010.34.4226. Epub 2011 May 16. PMID 21576646
- BACKGROUNDGarcia-Manero G, Gore SD, Kambhampati S, Scott B, Tefferi A, Cogle CR, Edenfield WJ, Hetzer J, Kumar K, Laille E, Shi T, MacBeth KJ, Skikne B. Efficacy and safety of extended dosing schedules of CC-486 (oral azacitidine) in patients with lower-risk myelodysplastic syndromes. Leukemia. 2016 Apr;30(4):889-96. doi: 10.1038/leu.2015.265. Epub 2015 Oct 7. PMID 26442612
- DERIVEDLaille E, Shi T, Garcia-Manero G, Cogle CR, Gore SD, Hetzer J, Kumar K, Skikne B, MacBeth KJ. Pharmacokinetics and Pharmacodynamics with Extended Dosing of CC-486 in Patients with Hematologic Malignancies. PLoS One. 2015 Aug 21;10(8):e0135520. doi: 10.1371/journal.pone.0135520. eCollection 2015. PMID 26296092