Sequential Therapy With WLL/Inhaling GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis

NCT03316651 — Phase 2, Unknown, 60 participants, Dai Huaping

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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

Registry	NCT03316651
Status	Unknown
Phase	Phase 2
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	60 (Estimated)
Sponsor	Dai Huaping (Other)
Conditions	Pulmonary Alveolar Proteinosis, Treatment
Interventions	GM-CSF (Drug)
Dates	start 2016-08-01, primary completion 2019-12-01 (estimated), completion 2020-12-01 (estimated), first posted 2017-10-20, last updated 2017-10-20
Locations	1 site in China

-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.

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+Arms and interventions

Arm	Type	Interventions	Description
GM-CSF	Experimental	Drug: GM-CSF	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.

GM-CSF (Drug): 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.

+Outcome measures

Primary outcomes

Time to the first relapse (the date: day/month/year) [Up to 2 years]: 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 outcomes

Relapse rate [Up to 2 years]
FEV1 difference [Up to 2 years]

+Eligibility

Sex: All, ages: 18 Years to any

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.

+Locations (1)

Facility	City	Country	Status
China Japan Friendship Hispital	Beijing	China	Recruiting