Back to results

GM-CSF With Post-Transplant Cyclophosphamide

Primary Purpose

Transplant-Related Hematologic Malignancy

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Sargramostim

Control Arm

About this trial

This is an interventional supportive care trial for Transplant-Related Hematologic Malignancy

Eligibility Criteria

18 Years - 78 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Northside HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

GM-CSF post-transplant

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

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

To estimate incidence of GVHD

How many patients have not relapsed measured by progression-free survival at 12 months following the 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

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

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

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

To estimate the rate of infections

Number of patients achieving platelet engraftment as measured by platelets reaching 20,000 without transfusion for 7 days

To assess time to platelet engraftment

Full Information

NCT ID

NCT04237623

First Posted

January 17, 2020

Last Updated

October 19, 2022

Sponsor

Northside Hospital, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04237623

Brief Title

GM-CSF With Post-Transplant Cyclophosphamide

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Recruiting

Study Start Date

May 18, 2020 (Actual)

Primary Completion Date

May 18, 2024 (Anticipated)

Study Completion Date

May 18, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Northside Hospital, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Transplant-Related Hematologic Malignancy

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

38 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

GM-CSF post-transplant

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Sargramostim

Other Intervention Name(s)

GM-CSF

Intervention Description

250mcg/m2/day IV starting Day +5

Intervention Type

Other

Intervention Name(s)

Control Arm

Other Intervention Name(s)

G-CSF

Intervention Description

Standard G-CSF given to those who decline to receive GM-CSF

Primary Outcome Measure Information:

Title

The number of patients who achieved neutrophil engraftment at 20 days after the initiation of treatment.

Description

Time Frame

3 months after initial treatment

Secondary Outcome Measure Information:

Title

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

Description

To estimate overall survival

Time Frame