The purpose of this study is to determine whether CPI-613 is effective and safe in either patients with refractory or relapsed acute myeloid leukemia (AML) or patients with myelodysplastic syndrome (MDS) who have failed therapy with a hypomethylating agent (such as decitabine \[Vidaza\] and azacitidine \[AZA\]).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
CPI-613 drug product, provided in concentrated form at 50 mg/mL, must be diluted with D5W prior to administration. CPI-613 is to be infused intravenously (IV) via a central venous catheter. The dose of CPI-613 will be either the Maximum Tolerated Dose (MTD) or the highest No-Significant- Adverse-Effects-Dose-Level (NOAEL), as determined from the nearly completed Phase 1 dose-escalation clinical trial in patients with hematologic malignancies (i.e., Cornerstone Study# CL-CPI-613-009 or Wake Forest Study# CCCWFU 29109, under IND 107,800).
Cornerstone Pharmaceuticals, Inc
Cranbury, New Jersey, United States
Overall Survival (OS)
Time frame: Monitored until participants passed away, for an expected average of 6 months.
Overall Remission Rate
Time frame: Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks.
Response Rate
Time frame: Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks.
Duration of Overall Remission
Time frame: Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks.
Progression Free Survival (PFS)
Time frame: Monitored during treatment with CPI-613 and until participants passed away, which will be an expected average of 6 months.
Quality of Life (QOL)
Time frame: Monitored before, during and 1 week after treatment with CPI-613, for an expected average of 20 weeks.
Safety
Safety assessment will be based on clinical signs, vital signs, blood work, adverse events, AEs, etc.
Time frame: Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks.
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