Back to resultsSequential Therapy With WLL/Inhaling GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis
Primary Purpose
Pulmonary Alveolar Proteinosis, Treatment
Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
GM-CSF
Sponsored by
About this trial
This is an interventional treatment trial for Pulmonary Alveolar Proteinosis focused on measuring pulmonary alveolar proteinosis, granulocyte-macrophage colony-stimulating factor, whole lung lavage
Eligibility Criteria
Inclusion Criteria:
- Comfirmed diagnosis of autoimmune pulmonary alveolar proteinosis
- Disease severity score (DSS) is 4-5
Exclusion Criteria:
- The acute respiratory infection
- Heart failure (such as cardiogenic pulmonary edema)
- The serious liver and kidney dysfunction (creatinine or ALT were equal to or more than 2 times of the upper limit of normal range);
- Pregnancy;
- The patients with hereditary and secondary factors (inhalation of dust, hematological diseases, autoimmune diseases, etc.);
- DSS is 1-3;
- The patient is allergic to the drugs that be used in our research;
- The patients with poor compliance, or suffering from mental illness;
- The patients have not signed informed consent;
- They were treated with whole lung lavage or regular GM-CSF therapy (treatment for more than 2 weeks) within 3 months before the enrollment.
Sites / Locations
- China Japan Friendship HispitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
GM-CSF
Arm Description
After the patients were randomly divided into two groups, they will receive whole lung lavage (WLL), and then one of the two groups with continue the next step as follows: Induction period: The time of beginning is 1 week after whole lung lavage, aerosolized GM-CSF was given for 7 days (150ug bid), and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle, a total of 6 cycles (3 months) were known as the induction period. Maintenance period: maintenance period came up after the induction period. The dose of aerosolized GM-CSF was reduced to 150ug/d for three times a week, and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle and maintenance period lasted for 9 months.
Outcomes
Primary Outcome Measures
Time to the first relapse (the date: day/month/year)
The standard of relapse: the condition of the disease after GM-CSF inhalation meeted the standard of effective treatment or the stability criteria, and then the condition deteriorated and meeted the deteriorate standard. The standard of effective treatment: oxygenation improvement (an increase in PaO2 of more than 10mmHg and / or a reduction in A-aDO2 more than 10mmHg). The stability criteria: an increase or a reduction in PaO2 of less than 10mmHg and / a reduction or an increase in A-aDO2 of less than 10mmHg. The deteriorate standard: deteriorate oxygenation (a reduction in PaO2 of more than 10mmHg and / or an increase in A-aDO2 of more than 10mmHg).
Secondary Outcome Measures
Relapse rate
FEV1 difference
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03316651
Brief Title
Sequential Therapy With WLL/Inhaling GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis
Official Title
A Multicenter Clinical Study of the Sequential Therapy With Whole Lung Lavage/Inhaling Granulocyte-macrophage Colony Stimulating Factor in Adult Patients With Severe Autoimmune Pulmonary Alveolar Proteinosis in China
Study Type
Interventional
2. Study Status
Record Verification Date
September 2017
Overall Recruitment Status
Unknown status
Study Start Date
August 2016 (Actual)
Primary Completion Date
December 2019 (Anticipated)
Study Completion Date
December 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Dai Huaping
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of the study is to evaluate the effectiveness of the sequential therapy with whole lung Lavage (WLL)/inhaling granulocyte-macrophage colony stimulating factor, compared to WLL only, for adult patients with severe autoimmune pulmonary alveolar proteinosis in China over a two-year period.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Alveolar Proteinosis, Treatment
Keywords
pulmonary alveolar proteinosis, granulocyte-macrophage colony-stimulating factor, whole lung lavage
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
GM-CSF
Arm Type
Experimental
Arm Description
After the patients were randomly divided into two groups, they will receive whole lung lavage (WLL), and then one of the two groups with continue the next step as follows:
Induction period: The time of beginning is 1 week after whole lung lavage, aerosolized GM-CSF was given for 7 days (150ug bid), and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle, a total of 6 cycles (3 months) were known as the induction period.
Maintenance period: maintenance period came up after the induction period. The dose of aerosolized GM-CSF was reduced to 150ug/d for three times a week, and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle and maintenance period lasted for 9 months.
Intervention Type
Drug
Intervention Name(s)
GM-CSF
Intervention Description
After the patients were randomly divided into two groups, they will receive whole lung lavage (WLL), and then one of the two groups with continue the next step as follows:
Induction period: The time of beginning is 1 week after whole lung lavage, aerosolized GM-CSF was given for 7 days (150ug bid), and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle, a total of 6 cycles (3 months) were known as the induction period.
Maintenance period: maintenance period came up after the induction period. The dose of aerosolized GM-CSF was reduced to 150ug/d for three times a week, and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle and maintenance period lasted for 9 months.
Primary Outcome Measure Information:
Title
Time to the first relapse (the date: day/month/year)
Description
The standard of relapse: the condition of the disease after GM-CSF inhalation meeted the standard of effective treatment or the stability criteria, and then the condition deteriorated and meeted the deteriorate standard. The standard of effective treatment: oxygenation improvement (an increase in PaO2 of more than 10mmHg and / or a reduction in A-aDO2 more than 10mmHg). The stability criteria: an increase or a reduction in PaO2 of less than 10mmHg and / a reduction or an increase in A-aDO2 of less than 10mmHg. The deteriorate standard: deteriorate oxygenation (a reduction in PaO2 of more than 10mmHg and / or an increase in A-aDO2 of more than 10mmHg).
Time Frame
Up to 2 years
Secondary Outcome Measure Information:
Title
Relapse rate
Time Frame
Up to 2 years
Title
FEV1 difference
Time Frame
Up to 2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Comfirmed diagnosis of autoimmune pulmonary alveolar proteinosis
Disease severity score (DSS) is 4-5
Exclusion Criteria:
The acute respiratory infection
Heart failure (such as cardiogenic pulmonary edema)
The serious liver and kidney dysfunction (creatinine or ALT were equal to or more than 2 times of the upper limit of normal range);
Pregnancy;
The patients with hereditary and secondary factors (inhalation of dust, hematological diseases, autoimmune diseases, etc.);
DSS is 1-3;
The patient is allergic to the drugs that be used in our research;
The patients with poor compliance, or suffering from mental illness;
The patients have not signed informed consent;
They were treated with whole lung lavage or regular GM-CSF therapy (treatment for more than 2 weeks) within 3 months before the enrollment.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Huaping Dai
Phone
0086-13901293597
Email
daihuaping@sina.com
Facility Information:
Facility Name
China Japan Friendship Hispital
City
Beijing
ZIP/Postal Code
100029
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Huaping Dai
Phone
0086-13901293597
Email
daihuaping@sina.com
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Sequential Therapy With WLL/Inhaling GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis
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