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Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02835742
Recruitment Status : Completed
First Posted : July 18, 2016
Last Update Posted : February 7, 2019
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Pulmonary Alveolar Proteinosis, Autoimmune Drug: Sargramostim Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 78 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
Actual Study Start Date : September 1, 2016
Actual Primary Completion Date : August 2017
Actual Study Completion Date : June 2018

Resource links provided by the National Library of Medicine


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



Primary Outcome Measures :
  1. Change value of AaDO2 between baseline and 24 weeks [ Time Frame: 24 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age over 16 years and below 80 years (as of the date of registration).
  2. Can provide signed informed consent.
  3. 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).
  4. Autoimmune pulmonary alveolar proteinosis diagnosed by both HR-CT and biopsy and/or BAL as well as GM-CSF antibodies in serum positive.
  5. 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:

  1. Diagnosed as secondary or hereditary pulmonary alveolar proteinosis
  2. WBC of 12,000/mm3 or more
  3. Fever of 38 degree celsius or more
  4. Severe edema
  5. History of malignant disease within recent 5 years (not applied to the treated cases of uterine carcinoma in situ and local basal cell carcinoma)
  6. Complication of cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc with severe condition.
  7. 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.
  8. History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.
  9. Treatment with other cytokines
  10. Pregnant or possibly pregnant women, lactating women, and women who desire to become pregnant during the study period
  11. 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)
  12. Severe liver dysfunction (AST > 100 IU/L and/or ALT > 100 IU/L and/or T-bil >3.0mg/dL)
  13. Severe renal dysfunction (Ccr < 30 mL/min, calculated by Cockcroft-Gault (CG) formula)
  14. Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product
  15. Treatment with oral or intravenous administration or inhalation of corticosteroids.
  16. Treatment with other inhaled drugs.
  17. Previously treated with GM-CSF before the start of the study.
  18. Demonstrate hypersensitivity to GM-CSF agent.
  19. 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02835742


Locations
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Japan
Niigata University Med & Dental Hospital
Niigata, Japan
Sponsors and Collaborators
Niigata University Medical & Dental Hospital
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Niigata University Medical & Dental Hospital
ClinicalTrials.gov Identifier: NCT02835742    
Other Study ID Numbers: PAGE
First Posted: July 18, 2016    Key Record Dates
Last Update Posted: February 7, 2019
Last Verified: February 2019
Additional relevant MeSH terms:
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Pulmonary Alveolar Proteinosis
Respiratory Tract Diseases
Lung Diseases
Sargramostim
Immunologic Factors
Physiological Effects of Drugs