CPX-351 Therapy for MDS After Hypomethylating Agent Failure
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|ClinicalTrials.gov Identifier: NCT03957876|
Recruitment Status : Recruiting
First Posted : May 21, 2019
Last Update Posted : July 19, 2019
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndrome (MDS)||Drug: CPX-351||Phase 2|
This study will evaluate efficacy of treatment with CPX-351in participants with MDS while using a new stratification tool to predict outcomes of patients following HMA failure in order to gain a better understanding and insight into identifying new opportunities for drug approvals in this setting.
CPX-351is an investigational (experimental) drug for the indication of myelodysplastic syndrome that works by delivering two chemotherapy medications (daunorubicin and cytarabine) together which are then concentrated into the bone marrow (the part of the body that makes blood cells). CPX-351 is experimental because it is not approved by the Food and Drug Administration (FDA) for the indication of myelodysplastic syndrome. This drug is approved by theFDA for the indication of acute myeloid leukemia. One or more of the Investigators conducting this study serve as consultants for the company that makes products used in this study. These financial interests are within permissible limits established by the local institutional Conflict of Interest Policy.
Prior to beginning treatment, all eligible participants will be grouped into low risk versus high risk based on a stratification tool used to determine their disease. Group 1 will be low risk participants and group 2 will be high risk participants. All study participants will get the same study drug, CPX-351. Participants will receive the CPX-351 on days 1, 3 and 5 of the first 28 day cycle. After participants finish the first round of treatment and based on their response they may be eligible for another 4 cycles of treatment. The participant's doctor will inform them if this is an available option when the time comes. Once participant have finished treatment, their doctor will continue observe for side effects and follow their condition for 1 year.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study of CPX-351 as a Novel Therapeutic Approach for Patients With Myelodysplastic Syndromes (MDS) After Hypomethylating Agent Failure|
|Actual Study Start Date :||July 15, 2019|
|Estimated Primary Completion Date :||February 2022|
|Estimated Study Completion Date :||February 2023|
Experimental: intravenous CPX-351 with potential maintenance therapy
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
Other Name: (daunorubicin and cytarabine)
- Efficacy of CPX-351 as measured by overall response rate (ORR) [ Time Frame: day 28 +/- 7 days of induction ]Efficacy of CPX as measured by ORR as defined by IWG 2006 criteria for MDS participants at end of induction.
- Time to response (TTR) associated with CPX-351 [ Time Frame: day 28 +/- 7 days of induction ]TTR associated with CPX-351 in participant with MDS at the end of induction. TTR defined by the time between starting the treatment and the time of achieving best response.
- Duration of response (DOR) in participants achieving a response [ Time Frame: At the end of cycle 1 (each cycle is 28 days), up to 1 year after end of treatment ]DOR in participants achieving a response defined by the time between first response (day C1 D28 +/-7 days from induction) and the day of loss of response
- Event-free survival (EFS) [ Time Frame: up to 1 year after end of treatment ]EFS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.
- Overall survival (OS) [ Time Frame: up to 1 year after end of treatment ]OS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03957876
|Contact: Aziz Nazha, MD||866-223-8100||CancerCenterResearch@ccf.org|
|United States, Ohio|
|Cleveland Clinic, Case Comprehensive Cancer Center||Recruiting|
|Cleveland, Ohio, United States, 44195|
|Contact: Aziz Nazha, MD 866-223-8100|
|Principal Investigator:||Aziz Nazha, MD||The Cleveland Clinic|