Dendritic Cell DeficiencyPhase 1NCT01871467
Sargramostim for Myeloid Dendritic Cell Deficiency
A Phase 1 study testing Sargramostim in people with Dendritic Cell Deficiency, run by Johns Hopkins University at 1 site in United States.
Trial summary
Previous studies have demonstrated a deficiency of blood dendritic cells in patients with kidney disease that is associated with the development of viral infections after kidney transplantation. Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Dendritic Cell Deficiency
- What is being tested?
- Sargramostim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- May 2013to Jun 2014
- Phase
- Phase 1
- 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 with diagnosis of end stage renal disease and currently undergoing outpatient hemodialysis (HD) at one of the Johns Hopkins University-affiliated HD units
Who cannot join
- Age<18or>80years
- History of non-adherence to prescribed HD treatment
- 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)
- 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 etc...)
- History of liver disease (as defined by a diagnosis of uncompensated cirrhosis) • History of lung disease (including moderate-severe chronic obstructive pulmonary disease, interstitial lung disease, or asthma)
- Known hypersensitivity to yeast-derived products
- Hemoglobin < 10 g/dL and hematocrit < 30%.
- Abnormal white blood cell (WBC) count at baseline (< 3 or > 12 x 10 cells/mm )
- 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)
- Treatment with lithium within 4 weeks of study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Sargramostim for Myeloid Dendritic Cell Deficiency - Phase I
Previous studies have demonstrated a deficiency of blood dendritic cells in patients with kidney disease that is associated with the development of viral infections after kidney transplantation. We plan to test the ability of sargramostim to increase blood dendritic cell levels in patients with kidney disease in the hopes of developing new therapies to prevent viral infections after kidney transplantation.
- Conditions
- Dendritic Cell Deficiency
- Being tested
- Sargramostim
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SargramostimDrug
| Study group | Role | What they receive |
|---|---|---|
| Sargramostim administration | Experimental | Subjects will receive sargramostim. Receives: Drug: Sargramostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Target blood myeloid dendritic cell level of > 2.0 x 10<4>/mL
Measured: 2-4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2013 (estimated)
- Study startMay 2013
- Primary completionJun 2014
- Study completionJun 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dendritic Cell DeficiencyStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe 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 1
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Karl L Womer, MD, Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01871467
- Sponsor's protocol ID
- KW-5001-1
- Status last verified
- Aug 2017
- First submitted
- 4 Jun 2013
- Last updated
- 22 Aug 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sargramostim for Myeloid Dendritic Cell Deficiency - Phase I
- ClinicalTrials.gov ID
- NCT01871467
- Acronym / sponsor ID
- KW-5001-1
- Conditions
- Dendritic Cell Deficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Study start
- May 2013
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 6 Jun 2013 (estimated)
- 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.
Previous studies have demonstrated a deficiency of blood dendritic cells in patients with kidney disease that is associated with the development of viral infections after kidney transplantation. We plan to test the ability of sargramostim to increase blood dendritic cell levels in patients with kidney disease in the hopes of developing new therapies to prevent viral infections after kidney transplantation.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sargramostim administration | Experimental | Drug: SargramostimSubjects will receive sargramostim. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Age >18 years < 80 years with diagnosis of end stage renal disease and currently undergoing outpatient hemodialysis (HD) at one of the Johns Hopkins University-affiliated HD units
Key exclusion criteria (13 listed)
- Age<18or>80years
- History of non-adherence to prescribed HD treatment
- 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)
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 |
|---|---|---|
| Sargramostim | Drug | Sargramostim administration |
Primary outcome
Target blood myeloid dendritic cell level of > 2.0 x 10<4>/mL
Time frame: 2-4 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2017.
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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 L Womer, MD principal investigator
Johns Hopkins University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Sargramostim in people with Dendritic Cell Deficiency, run by Johns Hopkins University 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 1 inclusion and 13 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: finished, as of the sponsor's last update on 22 Aug 2017. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Target blood myeloid dendritic cell level of > 2.0 x 10<4>/mL (time frame: 2-4 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 22 Aug 2017.