Study Stopped
Insufficient resources necessary for completion.
Sargramostim for Myeloid Dendritic Cell Deficiency
Sargramostim to Reverse Myeloid Dendritic Cell Deficiency
2 other identifiers
interventional
4
1 country
1
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Feb 2017
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 21, 2017
CompletedStudy Start
First participant enrolled
February 23, 2017
CompletedFirst Posted
Study publicly available on registry
February 24, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 17, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
September 17, 2018
CompletedDecember 31, 2018
December 1, 2018
1.6 years
February 21, 2017
December 27, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Baseline to 2 weeks
Secondary Outcomes (2)
Proportion of patients with adverse events during the intervention.
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.
Baseline to 2 weeks
Study Arms (3)
Project I: Healthy participants
EXPERIMENTAL5 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
EXPERIMENTAL5 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
EXPERIMENTAL5 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.
Interventions
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 samples will be drawn at baseline and during each subsequent visit
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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)
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University of Florida
Gainesville, Florida, 32610, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Karl Womer, MD
University of Florida
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 21, 2017
First Posted
February 24, 2017
Study Start
February 23, 2017
Primary Completion
September 17, 2018
Study Completion
September 17, 2018
Last Updated
December 31, 2018
Record last verified: 2018-12
Data Sharing
- IPD Sharing
- Will not share