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

• ClinicalTrials.gov Identifier: NCT03316651
• Recruitment Status: Unknown
• First Posted: October 20, 2017
• Last Update Posted: October 20, 2017
• Sponsor: Dai Huaping
• Information provided by (Responsible Party): Dai Huaping, China-Japan Friendship Hospital

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.

Condition or disease	Intervention/treatment	Phase
Pulmonary Alveolar Proteinosis; Treatment	Drug: GM-CSF	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 60 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: August 2016
• Estimated Primary Completion Date: December 2019
• Estimated Study Completion Date: December 2020

Arms and Interventions

Arm

Intervention/treatment

Experimental: 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.

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.

Outcome Measures

Primary Outcome Measures :

1. Time to the first relapse (the date: day/month/year) [ Time Frame: 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 :

1. Relapse rate [ Time Frame: Up to 2 years ]
2. FEV1 difference [ Time Frame: Up to 2 years ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Contacts

Contact: Huaping Dai; 0086-13901293597; daihuaping@sina.com

Locations

China

China Japan Friendship Hispital; Recruiting

Beijing, China, 100029

Contact: Huaping Dai 0086-13901293597 daihuaping@sina.com

Sponsors and Collaborators

Dai Huaping

More Information

• Responsible Party: Dai Huaping, Deputy Director of Center for Respiratory Diseases, China-Japan Friendship Hospital
• ClinicalTrials.gov Identifier: NCT03316651
• Other Study ID Numbers: ChinaJapanFH001
• First Posted: October 20, 2017
• Last Update Posted: October 20, 2017
• Last Verified: September 2017

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Dai Huaping, China-Japan Friendship Hospital:

pulmonary alveolar proteinosis; granulocyte-macrophage colony-stimulating factor; whole lung lavage

Additional relevant MeSH terms:

Pulmonary Alveolar Proteinosis; Lung Diseases; Respiratory Tract Diseases