Pulmonary Alveolar Proteinosis, AutoimmunePhase 2NCT02835742
Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
A Phase 2 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: Determine the safety and efficacy of GM-CSF inhalation in patients with aPAP. Study Design: multi-center, randomized, double-blind, placebo- controlled, safety/efficacy study. Read more about this study
- Who is it for?
- Ages 16 to 80 years
- Conditions the study is for
- Pulmonary Alveolar Proteinosis, Autoimmune
- What is being tested?
- Sargramostim
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 78 participants
- Where
- 1 siteJapan
- Study dates
- 1 Sep 2016to Jun 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 16 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age over 16 years and below 80 years (as of the date of registration).
- Can provide signed informed consent.
- Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures specified in the protocol (including short-term hospital admission).
- Autoimmune pulmonary alveolar proteinosis diagnosed by both HR-CT and biopsy and/or BAL as well as GM-CSF antibodies in serum positive.
- PaO2 < 70 mmHg after 5 minutes spine position at room air, or PaO2 < 75 mmHg after 5 minutes spine position at room air and with symptom(s) including cough, sputum and exertional dyspnea
Who cannot join
- Diagnosed as secondary or hereditary pulmonary alveolar proteinosis
- WBC of 12,000/mm3 or more
- Fever of 38 degree celsius or more
- Severe edema
- History of malignant disease within recent 5 years (not applied to the treated cases of uterine carcinoma in situ and local basal cell carcinoma)
- Complication of cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc with severe condition.
Show all 19Show fewer
- Complication of respiratory diseases such as pulmonary infectious disease(incl. pulmonary tuberculosis), bronchial asthma, lung fibrosis ,interstitial pneumonitis, or bronchiectasis, in which the evaluations of safety and efficacy of GM-CSF therapy are considered as difficult.
- History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.
- Treatment with other cytokines
- 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, or rituximab within 6 months before the start of the study (this criterion does not apply to patients for whom 6 months or more have elapsed after their last lavage or rituximab)
- Severe liver dysfunction (AST > 100 IU/L and/or ALT > 100 IU/L and/or T-bil >3.0mg/dL)
- Severe renal dysfunction (Ccr < 30 mL/min, calculated by Cockcroft-Gault (CG) formula)
- Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product
- Treatment with oral or intravenous administration or inhalation of corticosteroids.
- Treatment with other inhaled drugs.
- Previously treated with GM-CSF before the start of the study.
- Demonstrate hypersensitivity to GM-CSF agent.
- Other patients judged to be inappropriate for the study by the attending physician (e.g., patients who are unlikely to complete treatment or are uncooperative).
Trial locations and contacts
1 site in 1 country.
About this study
Objective: Determine the safety and efficacy of GM-CSF inhalation in patients with aPAP.
Study Design: multi-center, randomized, double-blind, placebo- controlled, safety/efficacy study.
- Being tested
- Sargramostim, Placebo
- 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
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Group1 | Active comparator | Treatments for Group 1 include GM-CSF inhalation with 250 mcg/day/body of sargramostim (125 mcg BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles. Receives: Drug: Sargramostim |
| Group2 | Placebo comparator | Treatments for Group 2 include placebo inhalation (Placebo BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change value of AaDO2 between baseline and 24 weeks
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2016 (estimated)
- Study start1 Sep 2016
- Primary completionAug 2017
- Study completionJun 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 78 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
- NCT02835742
- Sponsor's protocol ID
- PAGE
- Status last verified
- Feb 2019
- First submitted
- 13 Jul 2016
- Last updated
- 7 Feb 2019
- Results posted
- No
Publications
Tazawa R, Ueda T, Abe M, Tatsumi K, Eda R, Kondoh S, Morimoto K, Tanaka T, Yamaguchi E, Takahashi A, Oda M, Ishii H, Izumi S, Sugiyama H, Nakagawa A, Tomii K, Suzuki M, Konno S, Ohkouchi S, Tode N, Handa T, Hirai T, Inoue Y, Arai T, Asakawa K, Sakagami T, Hashimoto A, Tanaka T, Takada T, Mikami A, Kitamura N, Nakata K. Inhaled GM-CSF for Pulmonary Alveolar Proteinosis. N Engl J Med. 2019 Sep 5;381(10):923-932. doi: 10.1056/NEJMoa1816216.
PubMed 31483963
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
- ClinicalTrials.gov ID
- NCT02835742
- Acronym / sponsor ID
- PAGE
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 78 participants (actual)
- Lead sponsor
- Niigata University Medical & Dental HospitalUniversity, hospital or other
- Study start
- 1 Sep 2016 (actual)
- Primary completion
- Aug 2017 (actual)
- Study completion
- Jun 2018 (actual)
- First posted
- 18 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: Determine the safety and efficacy of GM-CSF inhalation in patients with aPAP.
Study Design: multi-center, randomized, double-blind, placebo- controlled, safety/efficacy study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group1 | Active comparator | Drug: SargramostimTreatments for Group 1 include GM-CSF inhalation with 250 mcg/day/body of sargramostim (125 mcg BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles. |
| Group2 | Placebo comparator | Drug: PlaceboTreatments for Group 2 include placebo inhalation (Placebo BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles. |
Who may qualify
- Ages
- 16 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Age over 16 years and below 80 years (as of the date of registration).
- Can provide signed informed consent.
- Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures specified in the protocol (including short-term hospital admission).
- Autoimmune pulmonary alveolar proteinosis diagnosed by both HR-CT and biopsy and/or BAL as well as GM-CSF antibodies in serum positive.
- PaO2 < 70 mmHg after 5 minutes spine position at room air, or PaO2 < 75 mmHg after 5 minutes spine position at room air and with symptom(s) including cough, sputum and exertional dyspnea
Key exclusion criteria (19 listed)
- Diagnosed as secondary or hereditary pulmonary alveolar proteinosis
- WBC of 12,000/mm3 or more
- Fever of 38 degree celsius or more
- Severe edema
- History of malignant disease within recent 5 years (not applied to the treated cases of uterine carcinoma in situ and local basal cell carcinoma)
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 | Group1 |
| Placebo | Drug | Group2 |
Primary outcome
Change value of AaDO2 between baseline and 24 weeks
Time frame: 24 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 7 Feb 2019.
- Derived from this study: PubMed 31483963Tazawa R, Ueda T, Abe M, Tatsumi K, Eda R, Kondoh S, Morimoto K, Tanaka T, Yamaguchi E, Takahashi A, Oda M, Ishii H, Izumi S, Sugiyama H, Nakagawa A, Tomii K, Suzuki M, Konno S, Ohkouchi S, Tode N, Handa T, Hirai T, Inoue Y, Arai T, Asakawa K, Sakagami T, Hashimoto A, Tanaka T, Takada T, Mikami A, Kitamura N, Nakata K. Inhaled GM-CSF for Pulmonary Alveolar Proteinosis. N Engl J Med. 2019 Sep 5;381(10):923-932. doi: 10.1056/NEJMoa1816216.
Related trials for Pulmonary Alveolar Proteinosis, Autoimmune
Active, not recruitingPhase 3
Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Autoimmune Pulmonary Alveolar Proteinosis
Not yet recruitingPhase 2
A Study of hBMMSC Injection in Patients With aPAP
Autoimmune Pulmonary Alveolar Proteinosis
RecruitingPhase 3
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
Autoimmune Pulmonary Alveolar Proteinosis
Not yet recruitingPhase 2
Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis
Pulmonary Alveolar Proteinosis
Active, not recruitingPhase 2
Study of Human Bone Marrow Mesenchymal Stem Cells in APAP
Autoimmune Pulmonary Alveolar Proteinosis
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 2 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 5 inclusion and 19 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 7 Feb 2019. 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. Some participants in this study may receive a placebo. 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 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?
Change value of AaDO2 between baseline and 24 weeks (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02835742, which also lists 1 linked publication.
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 7 Feb 2019.