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
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- Northside Hospital, Inc.
- Enrollment
- 38
- Locations
- 2
- Primary Endpoint
- The number of patients who achieved neutrophil engraftment at 20 days after the initiation of treatment.
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Supportive Care
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 78 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy 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
Arms & Interventions
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.
Intervention: Control Arm (Other)
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.
Intervention: Sargramostim (Drug)
Outcomes
Primary Outcomes
The number of patients who achieved neutrophil engraftment at 20 days after the initiation of treatment.
Time Frame: 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 Outcomes
- How many patients have not relapsed measured by relapse rates at 12 months following the initiation of treatment.(12 months following initiation of treatment)
- Number of patients achieving platelet engraftment as measured by platelets reaching 20,000 without transfusion for 7 days(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(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(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(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(12 months following initiation of treatment)
- How many patients are still alive measured by overall survival at 12 months following the initiation of treatment.(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(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(12 months following initiation of treatment)