CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome
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ClinicalTrials.gov Identifier: NCT03393611 |
Recruitment Status :
Recruiting
First Posted : January 8, 2018
Last Update Posted : June 17, 2020
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Condition or disease | Intervention/treatment | Phase |
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Myelodysplastic Syndromes Leukemia, Myeloid, Acute Leukemia, Relapsed Adult Acute Myeloid Myelodysplastic Syndromes, Previously Treated | Drug: CPX-351 Drug: Fludarabine Drug: Melphalan Drug: Rabbit Anti-Human T-Lymphocyte Globulin Biological: Haplo-Cord Stem Cell Transplantation | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 10 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Pilot Study of a Novel Sequential Treatment Utilizing CPX-351 as Salvage Chemotherapy Followed by Allogeneic Stem-Cell Transplantation (SCT) Utilizing a Haplo-cord Graft for Patients With Relapsed or Refractory Leukemia or Myelodysplastic Syndrome |
Actual Study Start Date : | November 30, 2012 |
Estimated Primary Completion Date : | November 30, 2020 |
Estimated Study Completion Date : | November 30, 2022 |

Arm | Intervention/treatment |
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Experimental: CPX-351 Salvage Therapy and Transplant
Subjects will receive CPX-351 salvage chemotherapy on Day -21, -19, and -17 as a bridge to allogeneic stem cell transplantation using a Fludarabine/Melphalan/rATG conditioning regimen and a haplo-cord graft.
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Drug: CPX-351
Salvage Chemotherapy: CPX-351 at 120 u/m2 on Days -21, -19, and -17
Other Name: Cytarabine:Daunorubicin Liposome Injection Drug: Fludarabine Fludarabine 150 mg/m2 (30 mg/m2/day x 5 days, Day -7 to Day -3)
Other Name: Fludara Drug: Melphalan Melphalan 140 mg/m2 (Day -2)
Other Name: Alkeran Drug: Rabbit Anti-Human T-Lymphocyte Globulin Rabbit ATG (rATG)-thymoglobulin 4.5 mg/kg (1.5 mg/kg/day x 3 days)
Other Name: Thymoglobulin Biological: Haplo-Cord Stem Cell Transplantation Allogeneic stem cell transplantation using a haploidentical donor and umbilical cord blood unit. |
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 1 year ]Evaluated using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE)
- Neutrophil Engraftment [ Time Frame: 100 days ]Evaluate the time to neutrophil engraftment, defined as the first day in which absolute neutrophil count (ANC) >500/mm3 for three consecutive days
- Overall Survival at Day 100, 6 months, and 1 year [ Time Frame: Day 100, 6 months, and 1 year post-transplant ]Evaluate survival of subjects alive, with or without presence of disease, at the designated time points
- Disease-Free Survival at Day 100, 6 months, and 1 year [ Time Frame: Day 100, 6 months, and 1 year post-transplant ]Evaluate survival of subjects alive without disease at the designated time points
- Non-Relapse Mortality [ Time Frame: Day 100 ]Death that cannot be explained by persistence, relapse, or progression of underlying disease
- Relapse Rate [ Time Frame: Day 100, 6 months, 1 year ]Time to first relapse or progression of underlying disease after initiation of protocol therapy

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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Subject must have refractory or relapsed Acute Myeloid Leukemia (AML) according to previously established criteria:
- Primary induction failure (PIF) after ≥ 2 cycles of chemotherapy
- First relapse
- Relapse refractory to salvage chemotherapy
- Second or subsequent relapse
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Subjects with Myelodysplastic Syndrome (MDS):
(a) Either Refractory Anemia with Excess Blasts I or Refractory Anemia with Excess Blasts II (RAEB I or RAEB II)
- Karnofsky performance status ≥ 70
- Willing to participate as a research subject and sign an informed consent form
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Adequate physical function measured by:
- Cardiac: asymptomatic, or if symptomatic then Left Ventricular Ejection Fraction (LVEF) at rest must be ≥ 45% and must improve with exercise
- Hepatic: ≤3 x upper limit of normal (ULN) alanine aminotransferase (ALT) and ≤ 1.5 total serum bilirubin, unless liver is involved with the disease or there is congenital benign hyperbilirubinemia
- Renal: serum creatinine within normal range, or if serum creatinine is outside the normal range, then calculated creatinine clearance ≥ 60 ml/min
- Pulmonary: asymptomatic, or if symptomatic, diffusing capacity of the lungs for carbon monoxide (DLCO) ≥ 45% of predicted (corrected for hemoglobin)
- If subject has prior malignancy, must be without any evidence of disease of that prior malignancy for at least 2 years (excludes skin cancers that may have been excised within that 2 year period).
Exclusion Criteria:
- Serious active or uncontrolled infection or medical condition
- Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
- Prior daunorubicin therapy with a cumulative dose of more than 368 mg/m2 or equivalent
- Other systemic anticancer therapy or ongoing clinically relevant toxicities from such therapy (at discretion of the investigator)
- History of and/or current evidence of myocardial impairment (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, congestive heart failure), resulting in heart failure by New York Heart Association Class III or IV staging.
- Subjects with Wilson disease or other Copper-related disorders.

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): NCT03393611
Contact: Ashlee Torres, RN | 212-746-7117 | ant9105@med.cornell.edu | |
Contact: Koen van Besien, MD | 646-962-7950 | kov9001@med.cornell.edu |
United States, New York | |
Weill Cornell Medical College | Recruiting |
New York, New York, United States, 10021 | |
Contact: Ashlee Torres, RN 212-746-7117 ant9105@med.cornell.edu | |
Contact: Koen van Besien, MD, PhD 646-962-7950 kov9001@med.cornell.edu | |
Principal Investigator: Koen van Besien, MD, PhD |
Principal Investigator: | Koen van Besien, MD, PhD | Weill Medical College of Cornell University |
Responsible Party: | Weill Medical College of Cornell University |
ClinicalTrials.gov Identifier: | NCT03393611 |
Other Study ID Numbers: |
1107011830 |
First Posted: | January 8, 2018 Key Record Dates |
Last Update Posted: | June 17, 2020 |
Last Verified: | June 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Leukemia Preleukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Myelodysplastic Syndromes Syndrome Disease Pathologic Processes Neoplasms by Histologic Type Neoplasms Bone Marrow Diseases Hematologic Diseases Precancerous Conditions Cytarabine Fludarabine |
Melphalan Daunorubicin Thymoglobulin Antineoplastic Agents Antimetabolites, Antineoplastic Antimetabolites Molecular Mechanisms of Pharmacological Action Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antiviral Agents Anti-Infective Agents Antineoplastic Agents, Alkylating Alkylating Agents Myeloablative Agonists |