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

• Sponsor: Dai Huaping (Other)
• Overall Status: Unknown status
• CT.gov ID: NCT03316651
• Collaborator: (none)
• Enrollment: 60
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 52
• Patients Per Site Per Month: 1.2

Study Details

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
• Anticipated 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: Aug 1, 2016
• Anticipated Primary Completion Date: Dec 1, 2019
• Anticipated Study Completion Date: Dec 1, 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) [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 [Up to 2 years]

2. FEV1 difference [Up to 2 years]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	China Japan Friendship Hispital	Beijing		China	100029

Sponsors and Collaborators

• Dai Huaping

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• 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: Oct 20, 2017
• Last Update Posted: Oct 20, 2017
• Last Verified: Sep 1, 2017

• Individual Participant Data (IPD) Sharing Statement: No
• 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, Deputy Director of Center for Respiratory Diseases, China-Japan Friendship Hospital: pulmonary alveolar proteinosis; granulocyte-macrophage colony-stimulating factor; whole lung lavage
• Additional relevant MeSH terms: Pulmonary Alveolar Proteinosis

Study Results

No Results Posted as of Oct 20, 2017