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

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

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.

Study Overview

• Status: Unknown
• Conditions: Treatment; Pulmonary Alveolar Proteinosis
• Intervention / Treatment: Drug: GM-CSF

• Study Type: Interventional
• Enrollment (Anticipated): 60
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100029
    • Recruiting
    • China Japan Friendship Hispital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	Up to 2 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Relapse rate	Up to 2 years
FEV1 difference	Up to 2 years

Collaborators and Investigators

• Sponsor: Dai Huaping

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 1, 2016
• Primary Completion (ANTICIPATED): December 1, 2019
• Study Completion (ANTICIPATED): December 1, 2020

Study Registration Dates

• First Submitted: August 27, 2017
• First Submitted That Met QC Criteria: October 17, 2017
• First Posted (ACTUAL): October 20, 2017

Study Record Updates

• Last Update Posted (ACTUAL): October 20, 2017
• Last Update Submitted That Met QC Criteria: October 17, 2017
• Last Verified: September 1, 2017

More Information

Terms related to this study

• Keywords: granulocyte-macrophage colony-stimulating factor; pulmonary alveolar proteinosis; whole lung lavage
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Pulmonary Alveolar Proteinosis
• Other Study ID Numbers: ChinaJapanFH001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No