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

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ClinicalTrials.gov Identifier: NCT01983657
Recruitment Status : Unknown
Verified November 2013 by Huiping Li, Shanghai Pulmonary Hospital, Shanghai, China.
Recruitment status was:  Recruiting
First Posted : November 14, 2013
Last Update Posted : November 18, 2013
Sponsor:
Information provided by (Responsible Party):
Huiping Li, Shanghai Pulmonary Hospital, Shanghai, China

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Pulmonary Alveolar Proteinosis Drug: rhGM-CSF Procedure: Whole Lung Lavage(WLL) Phase 2

Detailed 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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.
Study Start Date : January 2012
Estimated Primary Completion Date : June 2014
Estimated Study Completion Date : October 2014
Arms and Interventions
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Arm Intervention/treatment
Experimental: D1

Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day).

If the treatment is effective, participants may be entered into low-dose group(D1)(rhGM-CSF administration 1.25 ug/kg/d, qd, sc, for 2 months，then rhGM-CSF administration 1.25 ug/kg/d, qod, sc, for 3 months), when the chest CT absorption≥25% , and /or the PaO2 elevated by 5 mm Hg.

 Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
  • TOPLEUCON
  • GM-CSF
  • Recombinant Granulocyte Macrophage-Colony Stimulating Factor
  • Granulocyte Macrophage-Colony Stimulating Factor
Experimental: D2

Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day).

When the treatment is ineffective, and anti-GM-CSF antibody titers level ≥1:1000，the dose will be increased to 2.5 ug/kg/d. After 2 months, if the clinical response was optimal, that dose is continued for 3 months, and defined as group 2(D2).

 Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
  • TOPLEUCON
  • GM-CSF
  • Recombinant Granulocyte Macrophage-Colony Stimulating Factor
  • Granulocyte Macrophage-Colony Stimulating Factor
Experimental: D3

Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day).

When the treatment is ineffective, and anti-GM-CSF antibody titers level ≥1:1000，the dose will be increased to 2.5 ug/kg/d. After 2 months, if the clinical response was not optimal, the patients will receive whole lung lavage(WLL), who are defined as group 3(D3).

 Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
  • TOPLEUCON
  • GM-CSF
  • Recombinant Granulocyte Macrophage-Colony Stimulating Factor
  • Granulocyte Macrophage-Colony Stimulating Factor
Active Comparator: D4

Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day).

When the treatment is ineffective, and anti-GM-CSF antibody titers level ＜1:1000，the patients will receive whole lung lavage(WLL), then give rhGM-CSF administration (1.25 ug/kg/d) for 3 months, which belong to group4(D4).

 Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
  • TOPLEUCON
  • GM-CSF
  • Recombinant Granulocyte Macrophage-Colony Stimulating Factor
  • Granulocyte Macrophage-Colony Stimulating Factor
Procedure: Whole Lung Lavage(WLL)
using double lumen endotracheal tube (DLT) to selectively lavage one lung
Other Names:
  • Whole Lung Lavage
  • WLL


Outcome Measures
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Primary Outcome Measures :
  1. Improvements in double pulmonary diffuse lesions （Chest CT score ） [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Clinical symptoms observation: shod of breath, cough (according to each score standard） [ Time Frame: 6 months ]
  2. Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change [ Time Frame: 6 months ]
  3. Improvements in pulmonary function [ Time Frame: 6 months ]
    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).

  4. 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). [ Time Frame: 6 months ]

Eligibility Criteria
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Ages Eligible for Study:   17 Years to 80 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01983657


Contacts
Contact: Huiping Li, Dr 86-13817389991 liw2013@126.com

Locations
China
Shanghai Pulmonary Hospital Recruiting
Shanghai, China, 200433
Contact: Huiping Li, Doctor    86-13817389991    liw2013@126.com   
Principal Investigator: Huiping Li, Doctor         
Sponsors and Collaborators
Shanghai Pulmonary Hospital, Shanghai, China
Investigators
Principal Investigator: Huiping Li, Dr Shanghai Pulmonary Hospital, Shanghai, China
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party: Huiping Li, Professor,Chief of Dept. of Respiratory Medicine, Shanghai Pulmonary Hospital, Shanghai, China
ClinicalTrials.gov Identifier: NCT01983657     History of Changes
Other Study ID Numbers: 20120401
First Posted: November 14, 2013    Key Record Dates
Last Update Posted: November 18, 2013
Last Verified: November 2013

Keywords provided by Huiping Li, Shanghai Pulmonary Hospital, Shanghai, China:
Pulmonary
Pulmonary Alveolar Proteinosis
PAP

Additional relevant MeSH terms:
Pulmonary Alveolar Proteinosis
Lung Diseases
Respiratory Tract Diseases