Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| 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. |
Drug: Sargramostim
|
| 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. |
Drug: Placebo
|
Outcome Measures
Primary Outcome Measures
- Change value of AaDO2 between baseline and 24 weeks [24 weeks]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Age over 16 years and below 80 years (as of the date of registration).
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Can provide signed informed consent.
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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).
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Autoimmune pulmonary alveolar proteinosis diagnosed by both HR-CT and biopsy and/or BAL as well as GM-CSF antibodies in serum positive.
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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:
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Diagnosed as secondary or hereditary pulmonary alveolar proteinosis
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WBC of 12,000/mm3 or more
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Fever of 38 degree celsius or more
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Severe edema
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History of malignant disease within recent 5 years (not applied to the treated cases of uterine carcinoma in situ and local basal cell carcinoma)
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Complication of cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc with severe condition.
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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.
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History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.
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Treatment with other cytokines
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Pregnant or possibly pregnant women, lactating women, and women who desire to become pregnant during the study period
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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)
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Severe liver dysfunction (AST > 100 IU/L and/or ALT > 100 IU/L and/or T-bil >3.0mg/dL)
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Severe renal dysfunction (Ccr < 30 mL/min, calculated by Cockcroft-Gault (CG) formula)
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Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product
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Treatment with oral or intravenous administration or inhalation of corticosteroids.
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Treatment with other inhaled drugs.
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Previously treated with GM-CSF before the start of the study.
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Demonstrate hypersensitivity to GM-CSF agent.
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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).
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Niigata University Med & Dental Hospital | Niigata | Japan |
Sponsors and Collaborators
- Niigata University Medical & Dental Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- PAGE