Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
Phase 2
Completed
- Conditions
- Pulmonary Alveolar Proteinosis, Autoimmune
- Interventions
- Drug: Placebo
- Registration Number
- NCT02835742
- Lead Sponsor
- Niigata University Medical & Dental Hospital
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 78
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group1 Sargramostim 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. Group2 Placebo Treatments for Group 2 include placebo inhalation (Placebo BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles.
- Primary Outcome Measures
Name Time Method Change value of AaDO2 between baseline and 24 weeks 24 weeks
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Niigata University Med & Dental Hospital
🇯🇵Niigata, Japan