Transplant-Related Hematologic MalignancyPhase 2NCT04237623
GM-CSF With Post-Transplant Cyclophosphamide
A Phase 2 study testing Sargramostim in people with Transplant-Related Hematologic Malignancy, run by Northside Hospital, Inc. at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 78 years
- Conditions the study is for
- Transplant-Related Hematologic Malignancy
- What is being tested?
- Sargramostim
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- 18 May 2020to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 78 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Northside Hospital
Atlanta, Georgia, 30342
Stacey Brown, BA, 404-780-7965, stacey.brown@northside.com
Caitlin Guzowski, MBA, MHA, 404-851-8523, caitlin.guzowski@northside.com
About this study
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
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.
- Being tested
- Sargramostim, Control Arm
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SargramostimDrug
Also called GM-CSF
250mcg/m2/day IV starting Day +5
Control ArmOther
Also called G-CSF
Standard G-CSF given to those who decline to receive GM-CSF
| Study group | Role | What they receive |
|---|---|---|
| GM-CSF post-transplant | Experimental | 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. Receives: Drug: Sargramostim; Other: Control Arm |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 months after initial treatment
Secondary outcomes (9)
How many patients are still alive measured by overall survival at 12 months following the initiation of treatment.
To estimate overall survival
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 12 months following initiation of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2020
- Study start18 May 2020
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transplant-Related Hematologic MalignancyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Northside Hospital, Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Melhem Solh, MD, Principal investigator, Northside Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04237623
- Sponsor's protocol ID
- NSH 1246
- Status last verified
- Oct 2026
- First submitted
- 17 Jan 2020
- Last updated
- 14 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04237623
- Acronym / sponsor ID
- NSH 1246
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Northside Hospital, Inc.University, hospital or other
- Study start
- 18 May 2020 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 23 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GM-CSF post-transplant | Experimental | Drug: Sargramostim, Other: Control ArmSargramostim (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. |
Who may qualify
- Ages
- 18 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SargramostimAlso: GM-CSF250mcg/m2/day IV starting Day +5 | Drug | GM-CSF post-transplant |
| Control ArmAlso: G-CSFStandard G-CSF given to those who decline to receive GM-CSF | Other | GM-CSF post-transplant |
Primary outcome
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.
9 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Melhem Solh, MD principal investigator
Northside Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sargramostim in people with Transplant-Related Hematologic Malignancy, run by Northside Hospital, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Stacey Brown, BA (404-780-7965, stacey.brown@northside.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number of patients who achieved neutrophil engraftment at 20 days after the initiation of treatment. (time frame: 3 months after initial treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.