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GM-CSF With Post-Transplant Cyclophosphamide

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ClinicalTrials.gov Identifier: NCT04237623
Recruitment Status : Recruiting
First Posted : January 23, 2020
Last Update Posted : August 31, 2021
Sponsor:
Information provided by (Responsible Party):
Northside Hospital, Inc.

Tracking Information
First Submitted Date  ICMJE January 17, 2020
First Posted Date  ICMJE January 23, 2020
Last Update Posted Date August 31, 2021
Actual Study Start Date  ICMJE May 18, 2020
Estimated Primary Completion Date May 18, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 17, 2020)
The number of patients who achieved neutrophil engraftment at 20 days after the initiation of treatment. [ Time Frame: 3 months after initial treatment ]
The aim of the study is to establish equivalent effectiveness of Sargramostim to a matched control cohort of G-CSF treated patients in time to achieve neutrophil (ANC >500 x3 days) post infusion of HLA-mismatched peripheral blood haploidentical stem cells with post-transplant cyclophosphamide. Patients will be followed for 3 months following the initiation of treatment to see engraftment numbers at 20 days after initial treatment.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 17, 2020)
  • How many patients are still alive measured by overall survival at 12 months following the initiation of treatment. [ Time Frame: 12 months following initiation of treatment ]
    To estimate overall survival
  • How many patients have not relapsed measured by relapse rates at 12 months following the initiation of treatment. [ Time Frame: 12 months following initiation of treatment ]
    To estimate relapse rates
  • How many patients develop graft-versus-host-disease (GVHD) measured by the incidence of GVHD at 12 months following initiation of treatment [ Time Frame: 12 months following initiation of treatment ]
    To estimate incidence of GVHD
  • How many patients have not relapsed measured by progression-free survival at 12 months following the initiation of treatment [ Time Frame: 12 months following initiation of treatment ]
    To estimate non-relapse mortality
  • How many patients died due to infections measured by the incidence and type of infections at 12 months following initiation of treatment [ Time Frame: 12 months following initiation of treatment ]
    To estimate infection-related mortality
  • How many patients died due to a treatment-related adverse events grade 2 or greater as assessed by CTCAE v.4.0 [ Time Frame: 12 months following initiation of treatment ]
    To estimate event-free survival
  • Number of patients to achieve full donor chimerisms at Days 30, 50, 100, and 6 months post-transplant as measured by donor chimerism data [ Time Frame: 12 months following initiation of treatment ]
    To estimate graft failure
  • Number of patients that acquired an infection in the first 100-days post-transplant as measured by the incidence of infections [ Time Frame: 12 months following initiation of treatment ]
    To estimate the rate of infections
  • Number of patients achieving platelet engraftment as measured by platelets reaching 20,000 without transfusion for 7 days [ Time Frame: 12 months following initiation of treatment ]
    To assess time to platelet engraftment
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 GM-CSF With Post-Transplant Cyclophosphamide
Official Title  ICMJE Phase II Trial Evaluating the Efficacy and Safety of Sargramostim Post-Infusion of T-Replete HLA Mismatched Peripheral Blood Haploidentical Hematopoietic Stem Cells and With Post Transplant Cyclophosphamide
Brief Summary Given the increased number of HLA-mismatched haploidentical transplantation with post-transplant cyclophosphamide performed each year and the high risk of infectious complications associated with this type of transplant, the investigators suggest that GM-CSF administration post-infusion of T-replete haploidentical stem cells and post-transplant cyclophosphamide can yield similar count recovery rates to G-CSF with a potential of lowering risk of infectious complications.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Condition  ICMJE Transplant-Related Hematologic Malignancy
Intervention  ICMJE
  • Drug: Sargramostim
    250mcg/m2/day IV starting Day +5
    Other Name: GM-CSF
  • Other: Control Arm
    Standard G-CSF given to those who decline to receive GM-CSF
    Other Name: G-CSF
Study Arms  ICMJE Experimental: GM-CSF post-transplant
Sargramostim (GM-CSF) will start on Day +5 and continue until ANC >1000 x3 days or >1500 x1 day. GM-CSF will be administered not less than 24 hours after the last dose of cyclophosphamide and will be given at a dose of 250mcg/m2/day as an infusion over 2 hours.
Interventions:
  • Drug: Sargramostim
  • Other: Control Arm
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 17, 2020)
38
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 18, 2024
Estimated Primary Completion Date May 18, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Availability of 5/10 to 8/10 matched related donor
  • KPS >/= 70%
  • CML, AML, MDS, ALL, CLL, HD, NHL, MPS/CMML, MM, any other hematologic condition deemed an eligible indication for allogeneic transplant by the treating center

Exclusion Criteria:

  • Poor cardiac, pulmonary, liver, and renal function
  • HIV-positive
  • Patients who have a debilitating medical or psychiatric illness that would preclude them from giving informed consent
  • History of severe or serious allergic reaction to human GM-CSF or yeast-derived products
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 78 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Stacey Brown, BA 404-780-7965 stacey.brown@northside.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04237623
Other Study ID Numbers  ICMJE NSH 1246
Has Data Monitoring Committee Not Provided
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
Plan to Share IPD: No
Responsible Party Northside Hospital, Inc.
Study Sponsor  ICMJE Northside Hospital, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Melhem Solh, MD Northside Hospital
PRS Account Northside Hospital, Inc.
Verification Date August 2021

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