A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes
Status:
Completed
Trial end date:
2020-12-01
Target enrollment:
Participant gender:
Summary
This is a Phase 2, two-stage study of the safety and efficacy of pracinostat in combination
with azacitidine in patients with IPSS-R high and very high risk MDS who are previously
untreated with HMAs. Enrollment in this study will be limited to high/very high risk MDS
because this group represents the highest unmet need, with median survival of less than 18
months.
Stage 1a will be conducted as an open-label single arm in up to 40 subjects to assess if this
regimen with a lower pracinostat dose results in a discontinuation rate that meets a
predefined threshold and in efficacy that justifies expansion of enrollment into Stage 1b.
A discontinuation rate of approximately ≤10% in Stage 1a, a rate comparable to that observed
with azacitidine alone in study MEI-003, supports expansion into Stage 1b.
Stage 1b will be conducted as expansion of stage 1a. Approximately 20 additional subjects
will be enrolled. Study drugs should be continued until disease progression or intolerable
toxicity. It is important to note that the median time to achieving a response with
azacitidine alone is 4 to 5 months. Furthermore, the median time to achieving a CR in study
MEI-003 was 4 cycles. Therefore, early (<6 months) discontinuation of trial therapy for 'no
response' should be avoided. The Medical Monitor should be contacted prior to discontinuing a
subject from the study to discuss the rationale for discontinuation.