A Phase 2a Open-Label Clinical Trial Evaluating Efficacy & Safety of CPI-613 in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML), and in Patients With Myelodysplastic Syndrome (MDS) Who Failed Hypomethylating Agents
A new therapy for AML is necessary because, although there are several treatment options for
patients with AML, these treatments are very toxic and not available to all AML patients or
only useful for acute promyelocytic leukemia (APL). Also, there is essentially no treatment
for patients with refractory or relapsed AML outside of bone marrow transplant.
Accordingly, there is a great medical need for a safe and effective therapy for AML,
especially refractory and relapsed AML. Also, although hypomethylating agents have been
found to be effective against MDS, these agents are toxic. Furthermore, after relapsing from
a hypomethylating agent, there is no treatment for this disease.
A nearly completed clinical trial of CPI-613 (Cornerstone Study# CL-CPI-613-009 or Wake
Forest Study# CCCWFU 29109, under IND# 107,800) shows that CPI-613 is well tolerated at
doses as high as 3,000 mg/m2. Results from this nearly completed trial also suggest that
CPI- 613 may be effective against refractory and relapsed AML, as well as against MDS that
is relapsed from a hypomethylating agent. Therefore, CPI-613 may be a suitable treatment
option for refractory/relapsed AML and MDS relapsed from a hypomethylating agent. The
promising preliminary efficacy data from Study# CL-CPI-613-009 (Wake Forest Study# CCCWFU
29109, under IND# 107,800) is the basis on which Cornerstone is conducting the current Phase
2a trial to further assess the efficacy of CPI-613 against these diseases.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall Survival (OS)
Monitored until participants passed away, for an expected average of 6 months.
King C Lee, Ph.D.
Cornerstone Pharmaceuticals, Inc.
United States: Food and Drug Administration
|Cornerstone Pharmaceuticals, Inc||Cranbury, New Jersey 08512|