Pulmonary Alveolar ProteinosisPhase 2PAPNCT01983657
Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.
A Phase 2 study testing rhGM-CSF and Whole Lung Lavage(WLL) in people with Pulmonary Alveolar Proteinosis, run by Shanghai Pulmonary Hospital, Shanghai, China at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 17 to 80 years
- Conditions the study is for
- Pulmonary Alveolar Proteinosis
- What is being tested?
- rhGM-CSF and Whole Lung Lavage(WLL)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- Jan 2012to Oct 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 17 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosed PAP patients
- Aged 17-80
- Signed informed consent
Who cannot join
- 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.
Show all 9Show fewer
- 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 locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Shanghai Pulmonary Hospital
Shanghai, 200433
Huiping Li, Doctor, 86-13817389991, liw2013@126.com
Huiping Li, Doctor
About this study
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.
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.
Read the 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.
- Conditions
- Pulmonary Alveolar Proteinosis
- Being tested
- rhGM-CSF, Whole Lung Lavage(WLL)
- Keywords
- Pulmonary, Pulmonary Alveolar Proteinosis, PAP
- Medical subject headings
- Pulmonary Alveolar Proteinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhGM-CSFDrug
Also called TOPLEUCON, GM-CSF, Recombinant Granulocyte Macrophage-Colony Stimulating Factor, Granulocyte Macrophage-Colony Stimulating Factor
GM-CSF will be given subcutaneously according to the rule in different groups.
Whole Lung Lavage(WLL)Procedure or surgery
Also called Whole Lung Lavage, WLL
using double lumen endotracheal tube (DLT) to selectively lavage one lung
| Study group | Role | What they receive |
|---|---|---|
| D1 | Experimental | 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. Receives: Drug: rhGM-CSF |
| D2 | Experimental | 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). Receives: Drug: rhGM-CSF |
| D3 | Experimental | 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). Receives: Drug: rhGM-CSF |
| D4 | Active comparator | 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). Receives: Drug: rhGM-CSF; Procedure: Whole Lung Lavage(WLL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvements in double pulmonary diffuse lesions （Chest CT score ）
Measured: 6 months
Secondary outcomes (4)
Clinical symptoms observation: shod of breath, cough (according to each score standard）
Measured: 6 months
Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change
Measured: 6 months
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).
Measured: 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).
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2013 (estimated)
- Study startJan 2012
- Primary completionJun 2014 (estimated)
- Study completionOct 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pulmonary Alveolar ProteinosisStudies of the same condition, open and under way.
- More trials run by Shanghai Pulmonary Hospital, Shanghai, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Pulmonary Hospital, Shanghai, China
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Huiping Li, Shanghai Pulmonary Hospital, Shanghai, China
- Investigators
- Huiping Li, Dr, Principal investigator, Shanghai Pulmonary Hospital, Shanghai, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01983657
- Sponsor's protocol ID
- 20120401
- Acronym
- PAP
- Status last verified
- Nov 2013
- First submitted
- 7 Nov 2013
- Last updated
- 18 Nov 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 31898493
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT01983657
- Acronym / sponsor ID
- PAP / 20120401
- Conditions
- Pulmonary Alveolar Proteinosis
- Keywords
- Pulmonary, Pulmonary Alveolar Proteinosis, PAP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Shanghai Pulmonary Hospital, Shanghai, ChinaUniversity, hospital or other
- Study start
- Jan 2012
- Primary completion
- Jun 2014 (estimated)
- Study completion
- Oct 2014 (estimated)
- First posted
- 14 Nov 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| D1 | Experimental | Drug: rhGM-CSFPatients 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. |
| D2 | Experimental | Drug: rhGM-CSFPatients 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). |
| D3 | Experimental | Drug: rhGM-CSFPatients 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). |
| D4 | Active comparator | Drug: rhGM-CSF, Procedure: Whole Lung Lavage(WLL)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). |
Who may qualify
- Ages
- 17 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosed PAP patients
- Aged 17-80
- Signed informed consent
Key exclusion criteria (9 listed)
- 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℃
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rhGM-CSFAlso: TOPLEUCON, GM-CSF, Recombinant Granulocyte Macrophage-Colony Stimulating Factor, Granulocyte Macrophage-Colony Stimulating FactorGM-CSF will be given subcutaneously according to the rule in different groups. | Drug | D1, D2, D3, D4 |
| Whole Lung Lavage(WLL)Also: Whole Lung Lavage, WLLusing double lumen endotracheal tube (DLT) to selectively lavage one lung | Procedure or surgery | D4 |
Primary outcome
Improvements in double pulmonary diffuse lesions （Chest CT score ）
Time frame: 6 months
4 secondary outcomes
Clinical symptoms observation: shod of breath, cough (according to each score standard）
Time frame: 6 months
Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change
Time frame: 6 months
Improvements in pulmonary function
Time frame: 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2013.
- Derived from this study: PubMed 31898493Zhang 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Huiping Li, Dr principal investigator
Shanghai Pulmonary Hospital, Shanghai, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rhGM-CSF and Whole Lung Lavage(WLL) in people with Pulmonary Alveolar Proteinosis, run by Shanghai Pulmonary Hospital, Shanghai, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Nov 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Huiping Li, Dr (86-13817389991, liw2013@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvements in double pulmonary diffuse lesions （Chest CT score ） (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01983657, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2013.