Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Pulmonary Alveolar Proteinosis |
Drug: rhGM-CSF Procedure: Whole Lung Lavage(WLL) |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: 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. |
- Improvements in double pulmonary diffuse lesions （Chest CT score ） [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Clinical symptoms observation: shod of breath, cough (according to each score standard） [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
- Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Improvements in pulmonary function [ Time Frame: 6 months ] [ Designated as safety issue: No ]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). [ Time Frame: 6 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 40 |
| Study Start Date: | January 2012 |
| Estimated Study Completion Date: | October 2014 |
| Estimated Primary Completion Date: | June 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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:
|
|
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:
|
|
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:
|
|
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:
Procedure: Whole Lung Lavage(WLL)
using double lumen endotracheal tube (DLT) to selectively lavage one lung
Other Names:
|
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.
Eligibility| Ages Eligible for Study: | 17 Years to 80 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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 LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT01983657
| Contact: Huiping Li, Dr | 86-13817389991 | liw2013@126.com |
| China | |
| Shanghai Pulmonary Hospital | Recruiting |
| Shanghai, China, 200433 | |
| Contact: Huiping Li, Doctor 86-13817389991 liw2013@126.com | |
| Principal Investigator: Huiping Li, Doctor | |
| Principal Investigator: | Huiping Li, Dr | Shanghai Pulmonary Hospital, Shanghai, China |
More Information
No publications provided
| 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 |
| Study First Received: | November 7, 2013 |
| Last Updated: | November 14, 2013 |
| Health Authority: | China: Food and Drug Administration |
Keywords provided by Shanghai Pulmonary Hospital, Shanghai, China:
|
Pulmonary Pulmonary Alveolar Proteinosis PAP |
Additional relevant MeSH terms:
|
Pulmonary Alveolar Proteinosis Lung Diseases Respiratory Tract Diseases |
ClinicalTrials.gov processed this record on February 27, 2015