Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.

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 : January 4, 2019
Sponsor:
Information provided by (Responsible Party):
Weill Medical College of Cornell University

Tracking Information
First Submitted Date  ICMJE December 7, 2017
First Posted Date  ICMJE January 8, 2018
Last Update Posted Date January 4, 2019
Actual Study Start Date  ICMJE November 30, 2012
Estimated Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 2, 2018)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03393611 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 2, 2018)
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Myelodysplastic Syndromes
  • Leukemia, Myeloid, Acute
  • Leukemia, Relapsed Adult Acute Myeloid
  • Myelodysplastic Syndromes, Previously Treated
Intervention  ICMJE
  • 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.
Study Arms  ICMJE 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.
Interventions:
  • Drug: CPX-351
  • Drug: Fludarabine
  • Drug: Melphalan
  • Drug: Rabbit Anti-Human T-Lymphocyte Globulin
  • Biological: Haplo-Cord Stem Cell Transplantation
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 2, 2018)
10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 30, 2022
Estimated Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Ashlee Torres, RN 212-746-7117 ant9105@med.cornell.edu
Contact: Usama Gergis, MD 646-962-7950 usg2001@med.cornell.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03393611
Other Study ID Numbers  ICMJE 1107011830
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Weill Medical College of Cornell University
Study Sponsor  ICMJE Weill Medical College of Cornell University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Usama Gergis, MD Weill Medical College of Cornell University
PRS Account Weill Medical College of Cornell University
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP