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

November 14, 2013 updated by: Huiping Li, Shanghai Pulmonary Hospital, Shanghai, China

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

40

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Shanghai, China, 200433
        • Recruiting
        • Shanghai Pulmonary Hospital
        • Contact:
        • Principal Investigator:
          • Huiping Li, Doctor

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

17 years to 80 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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:
  • GM-CSF
  • Granulocyte Macrophage-Colony Stimulating Factor
  • TOPLEUCON
  • Recombinant 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:
  • GM-CSF
  • Granulocyte Macrophage-Colony Stimulating Factor
  • TOPLEUCON
  • Recombinant 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:
  • GM-CSF
  • Granulocyte Macrophage-Colony Stimulating Factor
  • TOPLEUCON
  • Recombinant 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:
  • GM-CSF
  • Granulocyte Macrophage-Colony Stimulating Factor
  • TOPLEUCON
  • Recombinant 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Improvements in double pulmonary diffuse lesions （Chest CT score ）
Time Frame: 6 months
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical symptoms observation: shod of breath, cough (according to each score standard）
Time Frame: 6 months
6 months
Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change
Time Frame: 6 months
6 months
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).
6 months
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
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shanghai Pulmonary Hospital, Shanghai, China

Investigators

  • Principal Investigator: Huiping Li, Dr, Shanghai Pulmonary Hospital, Shanghai, China

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2012

Primary Completion (Anticipated)

June 1, 2014

Study Completion (Anticipated)

October 1, 2014

Study Registration Dates

First Submitted

November 7, 2013

First Submitted That Met QC Criteria

November 7, 2013

First Posted (Estimate)

November 14, 2013

Study Record Updates

Last Update Posted (Estimate)

November 18, 2013

Last Update Submitted That Met QC Criteria

November 14, 2013

Last Verified

November 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 20120401

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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