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

NCT03316651 | Unknown Phase 2

• 1 other identifier: ChinaJapanFH001
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

Pulmonary Alveolar Proteinosis; Treatment

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Relapse rate

  Up to 2 years

• FEV1 difference

  Up to 2 years

Study Arms (1)

GM-CSF

EXPERIMENTAL

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

Interventions

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.

GM-CSF

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Comfirmed diagnosis of autoimmune pulmonary alveolar proteinosis
• Disease severity score (DSS) is 4-5

You may not qualify if:

• 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.

Sponsors & Collaborators

• Dai Huaping: lead

Study Sites (1)

China Japan Friendship Hispital

Beijing, 100029, China

RECRUITING

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Central Study Contacts

Huaping Dai

CONTACT

0086-13901293597; daihuaping@sina.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Deputy Director of Center for Respiratory Diseases

Study Record Dates

• First Submitted: August 27, 2017
• First Posted: October 20, 2017
• Study Start: August 1, 2016
• Primary Completion: December 1, 2019
• Study Completion: December 1, 2020
• Last Updated: October 20, 2017

Record last verified: 2017-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)