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

Primary Purpose

Pulmonary Alveolar Proteinosis

Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
rhGM-CSF
Whole Lung Lavage(WLL)
Sponsored by
Shanghai Pulmonary Hospital, Shanghai, China
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pulmonary Alveolar Proteinosis focused on measuring Pulmonary, Pulmonary Alveolar Proteinosis, PAP

Eligibility Criteria

17 Years - 80 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Shanghai Pulmonary HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Active Comparator

Arm Label

D1

D2

D3

D4

Arm Description

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.

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).

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).

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).

Outcomes

Primary Outcome Measures

Improvements in double pulmonary diffuse lesions （Chest CT score ）

Secondary Outcome Measures

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

Full Information

First Posted
November 7, 2013
Last Updated
November 14, 2013
Sponsor
Shanghai Pulmonary Hospital, Shanghai, China
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1. Study Identification

Unique Protocol Identification Number
NCT01983657
Brief Title
Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.
Acronym
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.
Study Type
Interventional

2. Study Status

Record Verification Date
November 2013
Overall Recruitment Status
Unknown status
Study Start Date
January 2012 (undefined)
Primary Completion Date
June 2014 (Anticipated)
Study Completion Date
October 2014 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Shanghai Pulmonary Hospital, Shanghai, China

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Alveolar Proteinosis
Keywords
Pulmonary, Pulmonary Alveolar Proteinosis, PAP

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
D1
Arm Type
Experimental
Arm Description
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.
Arm Title
D2
Arm Type
Experimental
Arm Description
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).
Arm Title
D3
Arm Type
Experimental
Arm Description
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).
Arm Title
D4
Arm Type
Active Comparator
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
rhGM-CSF
Other Intervention Name(s)
TOPLEUCON, GM-CSF, Recombinant Granulocyte Macrophage-Colony Stimulating Factor, Granulocyte Macrophage-Colony Stimulating Factor
Intervention Description
GM-CSF will be given subcutaneously according to the rule in different groups.
Intervention Type
Procedure
Intervention Name(s)
Whole Lung Lavage(WLL)
Other Intervention Name(s)
Whole Lung Lavage, WLL
Intervention Description
using double lumen endotracheal tube (DLT) to selectively lavage one lung
Primary Outcome Measure Information:
Title
Improvements in double pulmonary diffuse lesions （Chest CT score ）
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Clinical symptoms observation: shod of breath, cough (according to each score standard）
Time Frame
6 months
Title
Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change
Time Frame
6 months
Title
Improvements in pulmonary function
Description
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).
Time Frame
6 months
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
17 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Huiping Li, Dr
Phone
86-13817389991
Email
liw2013@126.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Huiping Li, Dr
Organizational Affiliation
Shanghai Pulmonary Hospital, Shanghai, China
Official's Role
Principal Investigator
Facility Information:
Facility Name
Shanghai Pulmonary Hospital
City
Shanghai
ZIP/Postal Code
200433
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Huiping Li, Doctor
Phone
86-13817389991
Email
liw2013@126.com
First Name & Middle Initial & Last Name & Degree
Huiping Li, Doctor

12. IPD Sharing Statement

Citations:
PubMed Identifier
31898493
Citation
Zhang F, Weng D, Su Y, Yin C, Shen L, Zhang Y, Zhou Y, Li Q, Hu Y, Li H. Therapeutic effect of subcutaneous injection of low dose recombinant human granulocyte-macrophage colony-stimulating factor on pulmonary alveolar proteinosis. Respir Res. 2020 Jan 2;21(1):1. doi: 10.1186/s12931-019-1261-1.
Results Reference
derived

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

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