Safety and Effectiveness Study of CPI-613 to Treat Refractory or Relapsed Leukemia and Myelodysplastic Syndrome
|ClinicalTrials.gov Identifier: NCT01520805|
Recruitment Status : Withdrawn
First Posted : January 30, 2012
Last Update Posted : December 29, 2016
|Condition or disease||Intervention/treatment||Phase|
|Acute Myeloid Leukemia (AML) Myelodysplastic Syndrome (MDS)||Drug: CPI-613||Phase 2|
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 (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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|
|Study Start Date :||January 2016|
|Estimated Primary Completion Date :||December 2018|
|Estimated Study Completion Date :||December 2018|
CPI-613 will be intravenously infused over 2 hours, given twice weekly for 3 weeks followed by a week of rest.
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).
- 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 [ Time Frame: Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks. ]Safety assessment will be based on clinical signs, vital signs, blood work, adverse events, AEs, etc.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01520805
|United States, New Jersey|
|Cornerstone Pharmaceuticals, Inc|
|Cranbury, New Jersey, United States, 08512|
|Study Director:||King C Lee, Ph.D.||Rafael Pharmaceuticals Inc.|