MedPath

GM-CSF With Post-Transplant Cyclophosphamide

Phase 2
Recruiting
Conditions
Transplant-Related Hematologic Malignancy
Interventions
Other: Control Arm
Registration Number
NCT04237623
Lead Sponsor
Northside Hospital, Inc.
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 available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
38
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
GM-CSF post-transplantControl ArmSargramostim (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.
GM-CSF post-transplantSargramostimSargramostim (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.
Primary Outcome Measures
NameTimeMethod
The number of patients who achieved neutrophil engraftment at 20 days after the initiation of treatment.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.

Secondary Outcome Measures
NameTimeMethod
How many patients have not relapsed measured by relapse rates at 12 months following the initiation of treatment.12 months following initiation of treatment

To estimate relapse rates

How many patients died due to infections measured by the incidence and type of infections at 12 months following initiation of treatment12 months following initiation of treatment

To estimate infection-related mortality

Number of patients achieving platelet engraftment as measured by platelets reaching 20,000 without transfusion for 7 days12 months following initiation of treatment

To assess time to platelet engraftment

How many patients died due to a treatment-related adverse events grade 2 or greater as assessed by CTCAE v.4.012 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 data12 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 infections12 months following initiation of treatment

To estimate the rate of infections

How many patients are still alive measured by overall survival at 12 months following the initiation of treatment.12 months following initiation of treatment

To estimate overall survival

How many patients develop graft-versus-host-disease (GVHD) measured by the incidence of GVHD at 12 months following initiation of treatment12 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 treatment12 months following initiation of treatment

To estimate non-relapse mortality

Trial Locations

Locations (1)

Northside Hospital

🇺🇸

Atlanta, Georgia, United States

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