Kidney DiseasesPhase 2NCT03063242
Sargramostim for Myeloid Dendritic Cell Deficiency
A Phase 2 study testing Sargramostim and Blood samples in people with Kidney Diseases and Kidney Transplant, run by University of Florida at 1 site in United States.
Trial summary
The study will determine whether administration of sargramostim will improve myeloid dendritic cell deficiency in various study groups, including healthy subjects and patients with chronic kidney disease, including those with kidney transplants. Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Kidney Diseases, Kidney Transplant
- What is being tested?
- Sargramostim and Blood samples
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Feb 2017to 17 Sep 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Age >18 years < 80 years
- Absence of acute or chronic medical condition and taking no prescription medications (Project I)
- Stable native or transplant kidney function (Project II/III)
Who cannot join
- Age < 18 or > 80 years
- History of non-adherence to prescribed medications (Projects II and III)
- Active drug or heavy alcohol use (defined as > 4 drinks/day)
- Pregnancy or breast feeding
- Active infection (bacterial or viral) or clinically significant infections within the past three months (e.g. those requiring hospitalization, or as judged by the PI, except for CMV viremia in Project III)
- Active malignancy (with the exception of excised non-metastatic basal cell carcinoma or squamous cell carcinoma of the skin, or adequately treated pre-invasive cervical cancer in situ)
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- Unstable cardiovascular status (angina, arrhythmias, congestive heart failure (CHF) etc…)
- History of liver disease (as defined by a diagnosis of uncompensated cirrhosis)
- History of lung disease (including moderate-severe Chronic Obstructive Pulmonary Disease (COPD), interstitial lung disease, or asthma)
- Known hypersensitivity to yeast-derived products
- Hemoglobin < 10 g/dL and hematocrit < 30%.
- Abnormal white blood cell count (WBC) count at baseline (< 3 or > 12 x 103 cells/mm3, except Project III)
- Treatment with WBC growth factors (G-CSF or GM-CSF) or immunosuppressive medications (tacrolimus, cyclosporine, mycophenolate, azathioprine, corticosteroids, chlorambucil, cyclophosphamide) within 4 weeks of study (erythropoiesis-stimulating agents will be allowed for Project II and immunosuppression for Project III)
- Treatment with lithium within 4 weeks of study
- History of arterial or venous thrombosis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Sargramostim to Reverse Myeloid Dendritic Cell Deficiency
The study will determine whether administration of sargramostim will improve myeloid dendritic cell deficiency in various study groups, including healthy subjects and patients with chronic kidney disease, including those with kidney transplants.
Read the detailed description
The overall objective of this project is to study the ability of sargramostim to enhance mDC level and function, including subsequent stimulation of T cell responses, in various human subjects with demonstrated myeloid dendritic cell (mDC) and T cell deficiency.
Single center nonrandomized trial with an interrupted time series design involving measures on blood samples from three separate populations before and after administration of sargramostim.
The objective is to determine the safety and dose response of sargramostim administration in healthy participants and in patients with chronic kidney disease (CKD) and kidney transplants.
Additionally to determine whether reversal of mDC/T cell deficiency by sargramostim results in augmented T cell responses in these three groups.
- Conditions
- Kidney Diseases, Kidney Transplant
- Being tested
- Sargramostim, Blood samples
- Keywords
- Sargramostim
- Medical subject headings
- Kidney Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SargramostimDrug
Also called Leukine®, GM-CSF
Study participants (n=5 per project) will receive subcutaneous injection of sargramostim (6 ug/kg) daily until maximal mDC levels are achieved, as determined by a dose response curve.
Blood samplesBiological
Blood samples will be drawn at baseline and during each subsequent visit
| Study group | Role | What they receive |
|---|---|---|
| Project I: Healthy participants | Experimental | 5 healthy participants will be used to optimize the dosage and timing of sargramostim administration with regard to the primary and secondary outcomes. Blood samples will be drawn and analyzed for mDC levels. Receives: Drug: Sargramostim; Biological: Blood samples |
| Project II: Patients with CKD stage IV/V | Experimental | 5 Patients with CKD stage IV/V who are cytomegalovirus (CMV) seropositive with mean blood mDC levels <1.0x104/mL will receive sargramostim treatment once all 5 healthy participants have completed treatment and the data have been analyzed to guide subsequent dosing. Blood samples will be drawn and analyzed for mDC levels. Receives: Drug: Sargramostim; Biological: Blood samples |
| Project III: kidney transplant patients | Experimental | 5 Kidney transplant recipients who are CMV seropositive with neutropenia (defined as absolute neutrophil count <1.0 x103/mm3) and/or CMV viremia will receive sargramostim treatment once all 5 Project I participants have completed treatment and the data have been analyzed to guide subsequent dosing. Blood samples will be drawn and analyzed for mDC levels. Receives: Drug: Sargramostim; Biological: Blood samples |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in peripheral blood mDC levels
mDC levels to >2.0 x104 mDCs/mL, with the target level defined as levels at or above upper quartile values in healthy controls
Measured: Baseline to 2 weeks
Secondary outcomes (2)
Proportion of patients with adverse events during the intervention.
Measured: Baseline to 2 weeks
Increase in T cell levels, mDC Interleukin (IL)-12 production, and interferon-gamma (IFN-y) production in QuantiFERON-CMV and QuantiFERON-Monitor assays after the intervention.
Measured: Baseline to 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2017
- Study start23 Feb 2017
- Primary completion17 Sep 2018
- Study completion17 Sep 2018
Why it stopped: Insufficient resources necessary for completion.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Kidney DiseasesStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Project I will be used to optimize the dosage and timing of sargramostim administration with regard to the primary and secondary outcomes. Thus, Project II and III treatment will only begin once all 5 Project I participants have completed treatment and the data have been analyzed to guide subsequent dosing
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Karl Womer, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03063242
- Sponsor's protocol ID
- IRB201600815
- Other IDs
- OCR16461
- Status last verified
- Dec 2018
- First submitted
- 21 Feb 2017
- Last updated
- 31 Dec 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sargramostim to Reverse Myeloid Dendritic Cell Deficiency
- ClinicalTrials.gov ID
- NCT03063242
- Acronym / sponsor ID
- IRB201600815
- Conditions
- Kidney Diseases, Kidney Transplant
- Keywords
- Sargramostim
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Insufficient resources necessary for completion.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Study start
- 23 Feb 2017 (actual)
- Primary completion
- 17 Sep 2018 (actual)
- Study completion
- 17 Sep 2018 (actual)
- First posted
- 24 Feb 2017 (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.
The study will determine whether administration of sargramostim will improve myeloid dendritic cell deficiency in various study groups, including healthy subjects and patients with chronic kidney disease, including those with kidney transplants.
Detailed description
The overall objective of this project is to study the ability of sargramostim to enhance mDC level and function, including subsequent stimulation of T cell responses, in various human subjects with demonstrated myeloid dendritic cell (mDC) and T cell deficiency.
Single center nonrandomized trial with an interrupted time series design involving measures on blood samples from three separate populations before and after administration of sargramostim.
The objective is to determine the safety and dose response of sargramostim administration in healthy participants and in patients with chronic kidney disease (CKD) and kidney transplants.
Additionally to determine whether reversal of mDC/T cell deficiency by sargramostim results in augmented T cell responses in these three groups.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Project I will be used to optimize the dosage and timing of sargramostim administration with regard to the primary and secondary outcomes. Thus, Project II and III treatment will only begin once all 5 Project I participants have completed treatment and the data have been analyzed to guide subsequent dosing
| Arm | Type | Receives |
|---|---|---|
| Project I: Healthy participants | Experimental | Drug: Sargramostim, Biological: Blood samples5 healthy participants will be used to optimize the dosage and timing of sargramostim administration with regard to the primary and secondary outcomes. Blood samples will be drawn and analyzed for mDC levels. |
| Project II: Patients with CKD stage IV/V | Experimental | Drug: Sargramostim, Biological: Blood samples5 Patients with CKD stage IV/V who are cytomegalovirus (CMV) seropositive with mean blood mDC levels <1.0x104/mL will receive sargramostim treatment once all 5 healthy participants have completed treatment and the data have been analyzed to guide subsequent dosing. Blood samples will be drawn and analyzed for mDC levels. |
| Project III: kidney transplant patients | Experimental | Drug: Sargramostim, Biological: Blood samples5 Kidney transplant recipients who are CMV seropositive with neutropenia (defined as absolute neutrophil count <1.0 x103/mm3) and/or CMV viremia will receive sargramostim treatment once all 5 Project I participants have completed treatment and the data have been analyzed to guide subsequent dosing. Blood samples will be drawn and analyzed for mDC levels. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Age >18 years < 80 years
- Absence of acute or chronic medical condition and taking no prescription medications (Project I)
- Stable native or transplant kidney function (Project II/III)
Key exclusion criteria (15 listed)
- Age < 18 or > 80 years
- History of non-adherence to prescribed medications (Projects II and III)
- Active drug or heavy alcohol use (defined as > 4 drinks/day)
- Pregnancy or breast feeding
- Active infection (bacterial or viral) or clinically significant infections within the past three months (e.g. those requiring hospitalization, or as judged by the PI, except for CMV viremia in Project III)
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: Leukine®, GM-CSFStudy participants (n=5 per project) will receive subcutaneous injection of sargramostim (6 ug/kg) daily until maximal mDC levels are achieved, as determined by a dose response curve. | Drug | Project I: Healthy participants, Project II: Patients with CKD stage IV/V, Project III: kidney transplant patients |
| Blood samplesBlood samples will be drawn at baseline and during each subsequent visit | Biological | Project I: Healthy participants, Project II: Patients with CKD stage IV/V, Project III: kidney transplant patients |
Primary outcome
Change in peripheral blood mDC levels
Time frame: Baseline to 2 weeks
mDC levels to >2.0 x104 mDCs/mL, with the target level defined as levels at or above upper quartile values in healthy controls
2 secondary outcomes
Proportion of patients with adverse events during the intervention.
Time frame: Baseline to 2 weeks
Increase in T cell levels, mDC Interleukin (IL)-12 production, and interferon-gamma (IFN-y) production in QuantiFERON-CMV and QuantiFERON-Monitor assays after the intervention.
Time frame: Baseline to 2 weeks
Results and safety
The study was stopped early (reason given: Insufficient resources necessary for completion.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2018.
- 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. This study is not recruiting at the moment.
Karl Womer, MD principal investigator
University of Florida
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 and Blood samples in people with Kidney Diseases and Kidney Transplant, run by University of Florida 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 15 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: stopped early, as of the sponsor's last update on 31 Dec 2018. 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?
The study was stopped early (reason given: Insufficient resources necessary for completion.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in peripheral blood mDC levels (time frame: Baseline to 2 weeks). 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 31 Dec 2018.