Clinical trial NCT04237623: 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
Overall status: RECRUITING
Phase: PHASE2
Conditions: Transplant-Related Hematologic Malignancy
Lead sponsor: Northside Hospital, Inc.
Sponsor class: OTHER
First posted: 2020-01-23
Enrollment: 38
Sex: ALL
Minimum age (years): 18
Maximum age (years): 78
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

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.


Eligibility criteria: 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

Primary outcomes:
measure: The number of patients who achieved neutrophil engraftment at 20 days after the initiation of treatment.
timeFrame: 3 months after initial treatment
Secondary outcomes:
measure: How many patients are still alive measured by overall survival at 12 months following the initiation of treatment.
timeFrame: 12 months following initiation of treatment
measure: How many patients have not relapsed measured by relapse rates at 12 months following the initiation of treatment.
timeFrame: 12 months following initiation of treatment
measure: How many patients develop graft-versus-host-disease (GVHD) measured by the incidence of GVHD at 12 months following initiation of treatment
timeFrame: 12 months following initiation of treatment
measure: How many patients have not relapsed measured by progression-free survival at 12 months following the initiation of treatment
timeFrame: 12 months following initiation of treatment
measure: How many patients died due to infections measured by the incidence and type of infections at 12 months following initiation of treatment
timeFrame: 12 months following initiation of treatment
measure: How many patients died due to a treatment-related adverse events grade 2 or greater as assessed by CTCAE v.4.0
timeFrame: 12 months following initiation of treatment
measure: Number of patients to achieve full donor chimerisms at Days 30, 50, 100, and 6 months post-transplant as measured by donor chimerism data
timeFrame: 12 months following initiation of treatment
measure: Number of patients that acquired an infection in the first 100-days post-transplant as measured by the incidence of infections
timeFrame: 12 months following initiation of treatment
measure: Number of patients achieving platelet engraftment as measured by platelets reaching 20,000 without transfusion for 7 days
timeFrame: 12 months following initiation of treatment
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Northside Hospital
state: Georgia
Study officials:
affiliation: Northside Hospital
name: Melhem Solh, MD
role: PRINCIPAL_INVESTIGATOR
References: