NCT00528983

Safety, Pharmacokinetics, and Pharmacodynamics of Oral Azacitidine in Subjects With Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Acute Myelogenous Leukemia

Completed Phase 1 Last updated 8 November 2019

What this trial tests

Phase 1 trial testing Subcutaneous (SC) Azacitidine in Myelodysplastic Syndromes (MDS) in 133 participants. Completed in 5 April 2016.

Timeline

11 September 2007

Primary endpoint
31 July 2013

5 April 2016

Quick facts

Lead sponsor	Celgene
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	133
Start date	11 September 2007
Primary completion	31 July 2013
Estimated completion	5 April 2016
Sites	18 locations across United States

Drugs / interventions tested

• Subcutaneous (SC) Azacitidine — full drug profile →
• Oral Azacitidine
• Oral Azacitidine

Conditions studied

• Myelodysplastic Syndromes (MDS) — all drugs for Myelodysplastic Syndromes (MDS) →
• Chronic Myelomonocytic Leukemia (CMML) — all drugs for Chronic Myelomonocytic Leukemia (CMML) →
• Acute Myelogenous Leukemia (AML) — all drugs for Acute Myelogenous Leukemia (AML) →

Sponsor

Celgene — full company profile →

Who can join

18 and older, any sex, with Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Phase I study of oral azacitidine in myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia.
   Garcia-Manero G, Gore SD, Cogle C, Ward R, et al · · 2011 · cited 191× · PMID 21576646 · DOI 10.1200/jco.2010.34.4226
2. Efficacy and safety of extended dosing schedules of CC-486 (oral azacitidine) in patients with lower-risk myelodysplastic syndromes.
   Garcia-Manero G, Gore SD, Kambhampati S, Scott B, et al · · 2016 · cited 83× · PMID 26442612 · DOI 10.1038/leu.2015.265
3. Demethylating Agents in the Treatment of Cancer.
   Howell PM, Liu Z, Khong HT. · · 2010 · cited 46× · PMID 27713340 · DOI 10.3390/ph3072022
4. Pharmacokinetics and Pharmacodynamics with Extended Dosing of CC-486 in Patients with Hematologic Malignancies.
   Laille E, Shi T, Garcia-Manero G, Cogle CR, et al · · 2015 · cited 45× · PMID 26296092 · DOI 10.1371/journal.pone.0135520
5. Epigenetic treatment of myelodysplastic syndromes and acute myeloid leukemias.
   Leone G, D'Alò F, Zardo G, Voso MT, et al · · 2008 · cited 34× · PMID 18537607 · DOI 10.2174/092986708784534947
6. Epimutational profile of hematologic malignancies as attractive target for new epigenetic therapies.
   Fratta E, Montico B, Rizzo A, Colizzi F, et al · · 2016 · cited 17× · PMID 27329599 · DOI 10.18632/oncotarget.10033
7. Beyond the Edge of Hypomethylating Agents: Novel Combination Strategies for Older Adults with Advanced MDS and AML.
   Kubasch AS, Platzbecker U. · · 2018 · cited 14× · PMID 29795051 · DOI 10.3390/cancers10060158
8. Personalizing initial therapy in acute myeloid leukemia: incorporating novel agents into clinical practice.
   DeStefano CB, Hourigan CS. · · 2018 · cited 8× · PMID 29713444 · DOI 10.1177/2040620718761778