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

Phase 2

• Conditions: Pulmonary Alveolar Proteinosis; Treatment
• Interventions: Drug: GM-CSF

• Registration Number: NCT03316651
• Lead Sponsor: Dai Huaping

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-08
• Study First Submitted: 2017-08-27
• Status Verified: 2017-09
• Study First Submitted QC: 2017-10-17
• Last Update Submitted: 2017-10-17
• Study First Posted: 2017-10-20
• Last Update Posted: 2017-10-20
• Primary Completion: 2019-12
• Study Completion: 2020-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 60

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
GM-CSF	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.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Relapse rate	Up to 2 years
FEV1 difference	Up to 2 years

Trial Locations

Locations (1)

China Japan Friendship Hispital; 🇨🇳 Beijing, China