NCT02243228

Brief Summary

The purpose of the study is to evaluate if inhaled granulocyte-macrophage colony stimulating factor delay the increase in alveolar-arterial oxygen difference, compared to no treatment, for adult patients with mild-to-moderate autoimmune pulmonary alveolar proteinosis in China over a two-year period.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
42

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Aug 2014

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2014

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

September 14, 2014

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 17, 2014

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2017

Completed
Last Updated

September 17, 2014

Status Verified

September 1, 2014

Enrollment Period

3 years

First QC Date

September 14, 2014

Last Update Submit

September 16, 2014

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Keywords

granulocyte-macrophage colony stimulating factor(GM-CSF)Autoimmune pulmonary alveolar proteinosis (PAP)

Outcome Measures

Primary Outcomes (1)

  • A-aDO2 difference

    2 years

Secondary Outcomes (4)

  • Time to relapse

    up to 2 years

  • FEV1 difference

    2 years

  • 6 minutes walking distance difference

    2 years

  • Severe adverse event

    2 years

Study Arms (1)

GM-CSF, nebulizer

EXPERIMENTAL

After the patients were randomly divided into two groups, they will be treated by GM-CSF (using nebulizer, 150ug bid) every other week for 6 months.

Drug: GM-CSF

Interventions

GM-CSFDRUG
Also known as: HUABEI JIMUXIN
GM-CSF, nebulizer

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Adult autoimmune PAP subjects will be included: 1) a positive PAS stain from BALF or lung biopsy; 2) high level of serum anti-GM-CSF antibody (\>2.39ug/ml, the cut-off point in our hospital); 3) age above 18 years old; 4) exclude hereditary and secondary PAP.
  • Able to give written informed consent and comply with the requirements of the study.
  • Patients are not eligible for the whole lung lavage (WLL), decided by clinicians.
  • Eligibility for GM-CSF inhalation: 1) Disease severity score (DSS) is 1-3; 2) No treatment with GM-CSF therapy or WLLin the 3 months prior to enrollment. Definition of DSS2: 1, no symptom and PaO2\>=70mmHg；2, PaO2\>=70mmHg with symptoms；3, PaO2\>=60 and \<70mmHg; 4, PaO2\>=50 and \<60mmHg; 5, PaO2\<50mmHg.

You may not qualify if:

  • Patients will be observed for 3 months and all APAP patients who resolved spontaneously will be excluded from our study.
  • PAP resulting from another condition (e.g. occupational exposure to silica, underlying HIV, respiratory infections, myeloproliferative disorder or leukaemia);
  • A normal or low-titre serum anti-GM-CSF antibody (≤2.39ug/ml);
  • History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies；
  • Chronic lung disease associated with already existing respiratory failure (such as pulmonary emphysema or fibrosis);
  • Other serious medical conditions which, in the opinion of the investigator, would make the patient unsuitable for the study.
  • Pregnancy.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

Related Publications (1)

  • Tian X, Yang Y, Chen L, Sui X, Xu W, Li X, Guo X, Liu L, Situ Y, Wang J, Zhao Y, Meng S, Song W, Xiao Y, Xu KF. Inhaled granulocyte-macrophage colony stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis - a six month phase II randomized study with 24 months of follow-up. Orphanet J Rare Dis. 2020 Jul 2;15(1):174. doi: 10.1186/s13023-020-01450-4.

    PMID: 32615994DERIVED

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis, Acquired

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor

Intervention Hierarchy (Ancestors)

Colony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Officials

  • Kai-Feng Xu, M.D.

    Peking Union Medical College Hosptial

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Kai-Feng Xu, M.D.

CONTACT

10-69155039kaifeng.xu@gmail.com

Xin-Lun Tian, M.D.

CONTACT

10-69155039xinlun_t@sina.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

September 14, 2014

First Posted

September 17, 2014

Study Start

August 1, 2014

Primary Completion

August 1, 2017

Study Completion

August 1, 2017

Last Updated

September 17, 2014

Record last verified: 2014-09

Locations

CN(1)