GM-CSF With Post-Transplant Cyclophosphamide

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

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.

Study Overview

• Status: Recruiting
• Conditions: Transplant-Related Hematologic Malignancy
• Intervention / Treatment: Drug: Sargramostim; Other: Control Arm

• Study Type: Interventional
• Enrollment (Estimated): 38
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Stacey Brown, BA; Phone Number: 404-780-7965; Email: stacey.brown@northside.com

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Recruiting
      • Northside Hospital
      • Sub-Investigator: Scott Solomon, MD
      • Contact: Caitlin Guzowski, MBA, MHA; Phone Number: 404-851-8523; Email: caitlin.guzowski@northside.com
      • Principal Investigator: Melhem Solh, MD
      • Sub-Investigator: H. Kent Holland, MD
      • Sub-Investigator: Asad Bashey, MD
      • Sub-Investigator: Lawrence E Morris, MD
      • Contact: Stacey Brown, BA; Phone Number: 404-780-7965; Email: stacey.brown@northside.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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.	Drug: Sargramostim; 250mcg/m2/day IV starting Day +5; Other Names: GM-CSF; Other: Control Arm; Standard G-CSF given to those who decline to receive GM-CSF; Other Names: G-CSF

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
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
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
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
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
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

Collaborators and Investigators

• Sponsor: Northside Hospital, Inc.
• Investigators: Principal Investigator: Melhem Solh, MD, Northside Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 18, 2020
• Primary Completion (Estimated): September 18, 2026
• Study Completion (Estimated): September 18, 2026

Study Registration Dates

• First Submitted: January 17, 2020
• First Submitted That Met QC Criteria: January 17, 2020
• First Posted (Actual): January 23, 2020

Study Record Updates

• Last Update Posted (Actual): April 16, 2026
• Last Update Submitted That Met QC Criteria: April 15, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Hematologic Neoplasms; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Carbohydrates; Intercellular Signaling Peptides and Proteins; Glycoproteins; Glycoconjugates; Colony-Stimulating Factors; Hematopoietic Cell Growth Factors; Cytokines; Granulocyte Colony-Stimulating Factor; sargramostim; Granulocyte-Macrophage Colony-Stimulating Factor
• Other Study ID Numbers: NSH 1246

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No