Pulmonary Alveolar Proteinosis, AutoimmunePhase 1NCT02840708
SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study
A Phase 1 study testing Sargramostim in people with Pulmonary Alveolar Proteinosis, Autoimmune, run by Niigata University Medical & Dental Hospital at 1 site in Japan.
Trial summary
Objective: Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation. Study Design: Pharmacokinetic open study Read more about this study
- Who is it for?
- Ages 20 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Pulmonary Alveolar Proteinosis, Autoimmune
- What is being tested?
- Sargramostim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteJapan
- Study dates
- May 2016to 1 Sep 2017
- 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 20 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Subjects judged to be appropriate for the study by the attending physician
- can provide signed informed consent.
- aPAP patient must meet the following
- aPAP patient aged over 20 and below 80 years old (as of the date of registration)
- aPAP severity is mild or moderate. (not severe)
- Healthy volunteer must meet the following
- Japanese healthy male subject aged over 20 and below 45 years old (as of the date of registration).
- BMI (Body mass index) is between 18 and 25.
Who cannot join
- WBC of 12,000/mcl or more
- Fever of 38 degree celsius or more
- History of malignant disease within recent 5 years (not applied to the treated cases of local basal cell carcinoma)
- Complication of severe cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc.
- Complication of respiratory diseases such as pulmonary infectious disease(incl. pulmonary tuberculosis), interstitial pneumonitis, lung fibrosis or bronchiectasis, in which the evaluations of safety of GM-CSF therapy are considered to be difficult.
- History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.
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- liver dysfunction
- renal dysfunction
- Previous experience of severe and unexplained adverse events during aerosol delivery of any kind of medicinal product
- Positive for HIV-Ab, HBs-Ag, HCV-Ab, STS or TPHA
- allergic to GM-CSF.
- addicted to illegal drugs
- Participation to other clinical trials within 12 weeks before registration.
- smoking within 5 years
- cannot follow the procedure defined in this protocol
- aPAP patient must exclude the following
- Pregnant or possibly pregnant women, lactating women, and women who desire to become pregnant during the study period
- Patients who have been treated with whole-lung lavage, repeated segmental-lung lavage, GM-CSF or rituximab within 6 months before the start of the study.
- taking other inhalation.
- Healthy volunteer must exclude the following
- taking any medicines (incl. OTC).
Trial locations and contacts
1 site in 1 country.
About this study
Objective: Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation.
Study Design: Pharmacokinetic open study
- Being tested
- Sargramostim
- Medical subject headings
- Pulmonary Alveolar Proteinosis, Acquired
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 |
|---|---|---|
| 125mcg | Active comparator | SK-1401 125mcg single inhalation Receives: Drug: Sargramostim |
| 250mcg | Active comparator | SK-1401 250mcg single inhalation Receives: Drug: Sargramostim |
| 500mcg | Active comparator | SK-1401 500mcg single inhalation Receives: Drug: Sargramostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum level of Sargramostim
Measured: 0.5,1,2,4,8,12 and 24 hours after inhalation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2016 (estimated)
- Study startMay 2016
- Primary completionDec 2016
- Study completion1 Sep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Alveolar Proteinosis, AutoimmuneStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Niigata University Medical & Dental Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02840708
- Sponsor's protocol ID
- NTU
- Status last verified
- Dec 2017
- First submitted
- 13 Jul 2016
- Last updated
- 18 Dec 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study
- ClinicalTrials.gov ID
- NCT02840708
- Acronym / sponsor ID
- NTU
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Niigata University Medical & Dental HospitalUniversity, hospital or other
- Study start
- May 2016
- Primary completion
- Dec 2016 (actual)
- Study completion
- 1 Sep 2017 (actual)
- First posted
- 21 Jul 2016 (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.
Objective: Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation.
Study Design: Pharmacokinetic open study
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 125mcg | Active comparator | Drug: SargramostimSK-1401 125mcg single inhalation |
| 250mcg | Active comparator | Drug: SargramostimSK-1401 250mcg single inhalation |
| 500mcg | Active comparator | Drug: SargramostimSK-1401 500mcg single inhalation |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Subjects judged to be appropriate for the study by the attending physician
- can provide signed informed consent.
- aPAP patient must meet the following
- aPAP patient aged over 20 and below 80 years old (as of the date of registration)
- aPAP severity is mild or moderate. (not severe)
Key exclusion criteria (21 listed)
- WBC of 12,000/mcl or more
- Fever of 38 degree celsius or more
- History of malignant disease within recent 5 years (not applied to the treated cases of local basal cell carcinoma)
- Complication of severe cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc.
- Complication of respiratory diseases such as pulmonary infectious disease(incl. pulmonary tuberculosis), interstitial pneumonitis, lung fibrosis or bronchiectasis, in which the evaluations of safety of GM-CSF therapy are considered to be difficult.
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 | 125mcg, 250mcg, 500mcg |
Primary outcome
Serum level of Sargramostim
Time frame: 0.5,1,2,4,8,12 and 24 hours after inhalation
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 18 Dec 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.
The record lists no contact. Use the official record to reach the sponsor.
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 Pulmonary Alveolar Proteinosis, Autoimmune, run by Niigata University Medical & Dental Hospital at 1 site in Japan.
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 8 inclusion and 21 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 18 Dec 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 Japan. 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?
Serum level of Sargramostim (time frame: 0.5,1,2,4,8,12 and 24 hours after inhalation). 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 18 Dec 2017.