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RecruitingInterventionalPhase 2

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

NCT ID: NCT04237623Sponsor: Northside Hospital, Inc.Last updated: 2026-04-16

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.

Arms & interventions

  • DrugSargramostim

    250mcg/m2/day IV starting Day +5

  • OtherControl Arm

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

Outcome measures

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.

    Time frame: 3 months after initial treatment

Secondary

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

Eligibility criteria

Sex: AllAge: 18 Years to 78 YearsHealthy volunteers: No
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 locations (1)

Northside Hospital

Atlanta, Georgia, 30342

Recruiting
Stacey Brown, BA · Contact
Caitlin Guzowski, MBA, MHA · Contact
H. Kent Holland, MD · Sub Investigator
Asad Bashey, MD · Sub Investigator
Lawrence E Morris, MD · Sub Investigator
Scott Solomon, MD · Sub Investigator
Melhem Solh, MD · Principal Investigator