Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.

Pulmonary Alveolar Proteinosis Clinical Trial

— PAP  

Official title:

Study of Subcutaneous Injection of Low-dose Recombinant Granulocyte Macrophage-Colony Stimulating Factor (rhGM-CSF) +/- Whole Lung Lavage(WLL) in Pulmonary Alveolar Proteinosis.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01983657
• Other study ID #: 20120401
• Status: Recruiting
• Phase: Phase 2
• First received: November 7, 2013
• Last updated: November 14, 2013
• Start date: January 2012
• Est. completion date: October 2014

Study information

• Verified date: November 2013
• Source: Shanghai Pulmonary Hospital, Shanghai, China
• Contact: Huiping Li, Dr
• Phone: 86-13817389991
• Email: liw2013[@]126.com
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP.

Description:

The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP. During the observation, study visits will occur at the end of each month. During the 1-year follow-up period which is lasting 6 months after the treatment, all participants will be required to check the various efficacy indicators.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: October 2014
• Est. primary completion date: June 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 17 Years to 80 Years

Eligibility

Inclusion Criteria:

• Diagnosed PAP patients

• Aged 17-80

• Signed informed consent

Exclusion Criteria:

• Secondary PAP

• Received whole lung lavage therapy within 4 weeks before enrollment

• Received previous GM-CSF therapy within 6 months before enrollment

• WBC=12,000/ul

• fever=38?

• Severe edema, severe liver, kidney, lung and cardiovascular disease.

• Pregnant,planning to get pregnant or nursing

• Inability to express the subjective discomfort

• Serious drug allergy history, E.coli preparation or rhGM-CSF serious allergy history

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Pulmonary Alveolar Proteinosis

Intervention

Drug:
• rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.

Procedure:
• Whole Lung Lavage(WLL)
using double lumen endotracheal tube (DLT) to selectively lavage one lung

Locations

Country	Name	City	State
China	Shanghai Pulmonary Hospital	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Shanghai Pulmonary Hospital, Shanghai, China

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvements in double pulmonary diffuse lesions (Chest CT score )		6 months	No
Secondary	Clinical symptoms observation: shod of breath, cough (according to each score standard)		6 months	Yes
Secondary	Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change		6 months	No
Secondary	Improvements in pulmonary function	Pulmonary function tests include residual volume/total lung capacity(RV/TLC), forced vital capacity(FVC), forced expiratory volume in one second/forced vital capacity(FEV1/FVC), diffusing capacity of carbon monoxide(DLCO).	6 months	No
Secondary	Improvements in arterial blood gas, including alveolar-arterial oxygen difference(A-aDO2), partial pressure of oxygen(PaO2), partial pressure of carbon dioxide in artery(PaCO2), arterial oxygen saturation(SaO2).		6 months	No