- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02835742
Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
February 6, 2019 updated by: Niigata University Medical & Dental Hospital
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 Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
78
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Niigata, Japan
- Niigata University Med & Dental Hospital
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
16 years to 80 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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.
- 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).
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: Group1
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.
|
|
Placebo Comparator: Group2
Treatments for Group 2 include placebo inhalation (Placebo BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change value of AaDO2 between baseline and 24 weeks
Time Frame: 24 weeks
|
24 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
September 1, 2016
Primary Completion (Actual)
August 1, 2017
Study Completion (Actual)
June 1, 2018
Study Registration Dates
First Submitted
July 13, 2016
First Submitted That Met QC Criteria
July 15, 2016
First Posted (Estimate)
July 18, 2016
Study Record Updates
Last Update Posted (Actual)
February 7, 2019
Last Update Submitted That Met QC Criteria
February 6, 2019
Last Verified
February 1, 2019
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- PAGE
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Pulmonary Alveolar Proteinosis, Autoimmune
-
Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd...The First Affiliated Hospital of Guangzhou Medical UniversityNot yet recruitingAutoimmune Pulmonary Alveolar Proteinosis
-
Children's Hospital Medical Center, CincinnatiNational Heart, Lung, and Blood Institute (NHLBI); Rare Diseases Clinical Research...CompletedAutoimmune Pulmonary Alveolar ProteinosisUnited States
-
Children's Hospital Medical Center, CincinnatiCompletedAutoimmune Pulmonary Alveolar ProteinosisUnited States
-
Savara Inc.CompletedAutoimmune Pulmonary Alveolar ProteinosisNetherlands, Denmark, France, Germany, Greece, Israel, Italy, Russian Federation, Turkey, United Kingdom
-
Savara Inc.Active, not recruitingAutoimmune Pulmonary Alveolar ProteinosisUnited States, Korea, Republic of, United Kingdom, Netherlands, Australia, Romania, Japan, Portugal, Canada, Greece, Spain, Italy, Poland, Belgium, France, Germany, Ireland, Turkey
-
IRCCS Policlinico S. MatteoAgenzia Italiana del FarmacoCompleted
-
Savara Inc.CompletedAutoimmune Pulmonary Alveolar ProteinosisUnited States, United Kingdom, Denmark, Switzerland, Australia, Portugal, Italy, Greece, Netherlands, Japan, Germany, France, Israel, Korea, Republic of, Russian Federation, Slovakia, Spain, Turkey
-
Children's Hospital Medical Center, CincinnatiRare Diseases Clinical Research Network; National Center for Advancing Translational...CompletedAutoimmune Pulmonary Alveolar ProteinosisUnited States
-
Peking Union Medical College HospitalThe Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical...UnknownAutoimmune Pulmonary Alveolar ProteinosisChina
-
Niigata University Medical & Dental HospitalCompletedPulmonary Alveolar Proteinosis, AutoimmuneJapan
Clinical Trials on Placebo
-
SamA Pharmaceutical Co., LtdUnknownAcute Bronchitis | Acute Upper Respiratory Tract InfectionKorea, Republic of
-
National Institute on Drug Abuse (NIDA)CompletedCannabis UseUnited States
-
AstraZenecaParexel; Spandauer Damm 130; 14050; Berlin, GermanyCompletedMale Subjects With Type II Diabetes (T2DM)Germany
-
Heptares Therapeutics LimitedCompletedPharmacokinetics | Safety IssuesUnited Kingdom
-
GlaxoSmithKlineCompletedPulmonary Disease, Chronic ObstructiveUnited Kingdom, Netherlands
-
ItalfarmacoCompletedBecker Muscular DystrophyNetherlands, Italy
-
Shijiazhuang Yiling Pharmaceutical Co. LtdXuanwu Hospital, BeijingCompleted
-
GlaxoSmithKlineCompletedInfections, BacterialUnited States
-
West Penn Allegheny Health SystemCompletedAsthma | Allergic RhinitisUnited States