GM-CSF With Post-Transplant Cyclophosphamide

Transplant-Related Hematologic Malignancy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04237623
• Other study ID #: NSH 1246
• Status: Recruiting
• Phase: Phase 2
• Start date: May 18, 2020
• Est. completion date: September 18, 2025

Study information

• Verified date: April 2024
• Source: Northside Hospital, Inc.
• Contact: Stacey Brown, BA
• Phone: 404-780-7965
• Email: stacey.brown[@]northside.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 38
• Est. completion date: September 18, 2025
• Est. primary completion date: September 18, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 78 Years

Eligibility

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

Related Conditions & MeSH terms

• Hematologic Neoplasms
• Transplant-Related Hematologic Malignancy

Intervention

Drug:
• Sargramostim
250mcg/m2/day IV starting Day +5

Other:
• Control Arm
Standard G-CSF given to those who decline to receive GM-CSF

Locations

Country	Name	City	State
United States	Northside Hospital	Atlanta	Georgia

Sponsors (1)

Lead Sponsor	Collaborator
Northside Hospital, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The number of patients who achieved neutrophil engraftment at 20 days after the initiation of 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.	3 months after initial treatment
Secondary	How many patients are still alive measured by overall survival at 12 months following the initiation of treatment.	To estimate overall survival	12 months following initiation of treatment
Secondary	How many patients have not relapsed measured by relapse rates at 12 months following the initiation of treatment.	To estimate relapse rates	12 months following initiation of treatment
Secondary	How many patients develop graft-versus-host-disease (GVHD) measured by the incidence of GVHD at 12 months following initiation of treatment	To estimate incidence of GVHD	12 months following initiation of treatment
Secondary	How many patients have not relapsed measured by progression-free survival at 12 months following the initiation of treatment	To estimate non-relapse mortality	12 months following initiation of treatment
Secondary	How many patients died due to infections measured by the incidence and type of infections at 12 months following initiation of treatment	To estimate infection-related mortality	12 months following initiation of treatment
Secondary	How many patients died due to a treatment-related adverse events grade 2 or greater as assessed by CTCAE v.4.0	To estimate event-free survival	12 months following initiation of treatment
Secondary	Number of patients to achieve full donor chimerisms at Days 30, 50, 100, and 6 months post-transplant as measured by donor chimerism data	To estimate graft failure	12 months following initiation of treatment
Secondary	Number of patients that acquired an infection in the first 100-days post-transplant as measured by the incidence of infections	To estimate the rate of infections	12 months following initiation of treatment
Secondary	Number of patients achieving platelet engraftment as measured by platelets reaching 20,000 without transfusion for 7 days	To assess time to platelet engraftment	12 months following initiation of treatment