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

Shanghai Pulmonary Hospital, Shanghai, China

Status and phase: Unknown; Phase 2

Conditions: Pulmonary Alveolar Proteinosis

Treatments: Drug: rhGM-CSF; Procedure: Whole Lung Lavage(WLL)

Study type: Interventional

Funder types: Other

Identifiers

NCT01983657

20120401

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 estimated patients

Sex: All

Ages: 17 to 80 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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40 participants in 4 patient groups

D1

Experimental group

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.

Treatment:

Drug: rhGM-CSF

D2

Experimental group

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).

Treatment:

Drug: rhGM-CSF

D3

Experimental group

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).

Treatment:

Drug: rhGM-CSF

D4

Active Comparator group

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).

Treatment:

Procedure: Whole Lung Lavage(WLL)

Drug: rhGM-CSF