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CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03393611
Recruitment Status : Recruiting
First Posted : January 8, 2018
Last Update Posted : June 17, 2020
Sponsor:
Information provided by (Responsible Party):
Weill Medical College of Cornell University

Study Description
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Brief Summary:
This pilot study is designed to evaluate outcomes with the combination of CPX-351 salvage therapy and haplo-cord graft stem cell transplantation for subjects with relapsed or refractory AML or myelodysplastic syndrome.

Condition or disease Intervention/treatment Phase
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 Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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.
 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.


Outcome Measures
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Primary Outcome Measures :
  1. 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)

  2. 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

  3. 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

  4. 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


Secondary Outcome Measures :
  1. Non-Relapse Mortality [ Time Frame: Day 100 ]
    Death that cannot be explained by persistence, relapse, or progression of underlying disease

  2. Relapse Rate [ Time Frame: Day 100, 6 months, 1 year ]
    Time to first relapse or progression of underlying disease after initiation of protocol therapy


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subject must have refractory or relapsed Acute Myeloid Leukemia (AML) according to previously established criteria:

    1. Primary induction failure (PIF) after ≥ 2 cycles of chemotherapy
    2. First relapse
    3. Relapse refractory to salvage chemotherapy
    4. Second or subsequent relapse

  2. 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)

  3. Karnofsky performance status ≥ 70
  4. Willing to participate as a research subject and sign an informed consent form

  5. Adequate physical function measured by:

    1. Cardiac: asymptomatic, or if symptomatic then Left Ventricular Ejection Fraction (LVEF) at rest must be ≥ 45% and must improve with exercise
    2. 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
    3. Renal: serum creatinine within normal range, or if serum creatinine is outside the normal range, then calculated creatinine clearance ≥ 60 ml/min
    4. Pulmonary: asymptomatic, or if symptomatic, diffusing capacity of the lungs for carbon monoxide (DLCO) ≥ 45% of predicted (corrected for hemoglobin)
  6. 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:

  1. Serious active or uncontrolled infection or medical condition
  2. Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
  3. Prior daunorubicin therapy with a cumulative dose of more than 368 mg/m2 or equivalent
  4. Other systemic anticancer therapy or ongoing clinically relevant toxicities from such therapy (at discretion of the investigator)
  5. 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.
  6. Subjects with Wilson disease or other Copper-related disorders.
Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Ashlee Torres, RN 212-746-7117 ant9105@med.cornell.edu
Contact: Koen van Besien, MD 646-962-7950 kov9001@med.cornell.edu

Locations
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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         
Sponsors and Collaborators
Weill Medical College of Cornell University
Investigators
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Principal Investigator: Koen van Besien, MD, PhD Weill Medical College of Cornell University
More Information
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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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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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