CPX-351 in Patients Treated for Higher-risk Myelodysplastic Syndromes Experiencing Hypomethylating Agent Failure.
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|ClinicalTrials.gov Identifier: NCT04109690|
Recruitment Status : Recruiting
First Posted : September 30, 2019
Last Update Posted : September 11, 2020
|Condition or disease||Intervention/treatment||Phase|
|MDS||Drug: CPX-351||Phase 1 Phase 2|
Proposed is a two-phase study. The Phase I portion will confirm the tolerability and safety of CPX-351 chemotherapy. Patients who meet eligibility criteria will receive dose level 1 of CPX-351 (44mg/m2 of daunorubicin and 100mg/m2 of cytarabine) on 2 days (day 1 and day 5) of the cycle.
If less than 2 dose limiting toxicity (DLT) are observed in the first cohort of 6, we will increase level of exposure to Dose Level 2 by giving (44mg/m2 of daunorubicin and 100mg/m2 of cytarabine) on days 1, 3 and 5 of
- If less than 2 DLTs are observed Dose Level 2 will become the Recommended Phase II dose (RP2D)
- If 2 or more DLTs are observed Dose Level 1 will become the RP2D If 2 or more DLTs are observed in the first cohort of 6 patients, another 6 patients will be enrolled at dose level -1 (29mg/m2 of daunorubicin and 65mg/m2 of cytarabine) on day 1 and 5.
- If less than 2 DLTs are observed Dose Level -1 will become the RP2D
- If 2 or more patients experience a DLT at dose -1 the study will be stopped Patients failing to achieve a response after cycle 1 will be offered a second cycle of induction with for dose level 1 and 2 (44mg/m2 of daunorubicin and 100mg/m2 of cytarabine) on day 1 and 5 of induction 2. And for dose level-1, (29mg/m2 of daunorubicin and 65mg/m2 of cytarabine) on day 1 and 5 of induction 2.
Phase II: Once the RP2D is confirmed we will enroll 12 patients. If 3 or more responses are observed an additional 12 patients will be enrolled for a total of 24. If 7 out of 24 evaluable patients achieve response, an additional 24 patients will be enrolled for a total of 48 patients.
• If less than 3 responses are observed in the first 12 patients, the study will be terminated.
The outcomes presented in this protocol are associated with the Phase II of the study.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||48 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I-II Study of a Liposomal Formulation of Cytarabine and Daunorubicin (CPX-351) in Patients Treated for Higher-risk Myelodysplastic Syndromes Experiencing Hypomethylating Agent Failure.|
|Actual Study Start Date :||December 30, 2019|
|Estimated Primary Completion Date :||October 31, 2023|
|Estimated Study Completion Date :||April 30, 2024|
Phase I will evaluate the safety and tolerability of CPX-351 (44mg/m2 of daunorubicin and 100mg/m2 of cytarabine) administered on 2 days (day 1 and day 5) to determine the Phase II dose. Phase II will evaluate the efficacy of the RP2D.
Patients in both Phase I and II can receive a maximum of two induction and six consolidation cycles on an inpatient or outpatient basis per local hospital standard of care.
- Overall Response Rate [ Time Frame: Up to 24 months ]The Phase II primary objective is to determine the overall response rate (CR/CRi) of CPX-351 in MDS patients experiencing hypomethylation (HMA) failure.
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): NCT04109690
|Contact: Tara McPartland, MSW, MPH||(203) firstname.lastname@example.org|
|United States, Connecticut|
|Yale University; Smilow Cancer Center||Recruiting|
|New Haven, Connecticut, United States, 06519|
|Contact: Laura Leary email@example.com|
|Principal Investigator: Thomas Prebet, MD, PHD|
|Principal Investigator:||Thomas Prebet, MD, PHD||Yale University|