Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidi… (NCT02103478) | Clinical Trial Compass
CompletedPhase 1/2
Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)
United States, Canada130 participantsStarted 2014-10-28
Plain-language summary
This first-in-human, 3-stage, open-label study evaluated the safety and pharmacokinetics of ASTX727, as well as determined the dose for later stages.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* International Prognostic Scoring System (IPSS) low, intermediate -1, intermediate-2, or high risk MDS (including chronic myelomonocytic leukemia; CMML) in Dose Escalation and Dose Confirmation-Randomization; only intermediate-2, or high risk MDS in Dose Confirmation-Open Label
* Eastern Cooperative Oncology Group (ECOG) 0 to 2
* No major surgery within 2 weeks of starting study treatment
* No cytotoxic chemotherapy within 2 weeks of starting study treatment
* Able to swallow pills
Exclusion Criteria:
* Previous treatment with 2 or more courses of decitabine (all stages) or azacitidine (Dose Confirmation stage only)
* Treatment with investigational therapy within 2 weeks of study treatment
* Uncontrolled medical disease(s) or active, uncontrolled infection
* Diagnosed with acute myeloid leukemia (AML)
* Active uncontrolled gastric or duodenal ulcer
* Known history of HIV or hepatitis C or B
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Mean Decitabine Area Under the Concentration Versus Time Curve (AUC0-t) on Day 5 by Cohort in Phase 1
Timeframe: Day 5
2
Mean Decitabine Area Under the Plasma Concentration Versus Time Curve Ratio (5-day AUC0-t) in Phase 2
Timeframe: Pre-dose to Day 5
3
Number of Participants With Dose-limiting Toxicity in Phase 1
Timeframe: Up to Day 28 in Course 1 (28 days per course)
4
Mean Maximum %LINE Demethylation in Phase 2
Timeframe: Pre-dose to Day 28 in Course 2 (28 days per course)
5
Number of Participants With Overall Response in Phase 2