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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03482752
Registration number
NCT03482752
Ethics application status
Date submitted
21/03/2018
Date registered
29/03/2018
Date last updated
3/02/2021
Titles & IDs
Public title
Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
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Scientific title
An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
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Secondary ID [1]
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SAV006-03
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Universal Trial Number (UTN)
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Trial acronym
IMPALA-X
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Autoimmune Pulmonary Alveolar Proteinosis
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Condition category
Condition code
Respiratory
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Other respiratory disorders / diseases
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Inflammatory and Immune System
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Molgramostim
Treatment: Drugs: Molgramostim
300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of adverse events
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Assessment method [1]
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Timepoint [1]
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36 months
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Primary outcome [2]
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Number of serious adverse events
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Assessment method [2]
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Timepoint [2]
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36 months
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Primary outcome [3]
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Number of adverse drug reactions
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Assessment method [3]
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Timepoint [3]
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36 months
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Primary outcome [4]
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Number of adverse events leading to treatment discontinuation
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Assessment method [4]
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Timepoint [4]
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36 months
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Secondary outcome [1]
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Alveolar-arterial oxygen gradient
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Assessment method [1]
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Difference in oxygen tension between lungs and blood
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Timepoint [1]
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36 months
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Secondary outcome [2]
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6-minute walk distance
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Assessment method [2]
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Timepoint [2]
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36 months
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Secondary outcome [3]
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St Georges Respiratory Questionnaire total score
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Assessment method [3]
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Respiratory-specific questionnaire measuring impact on overall health, daily life, and perceived well-being. Scores range from 0 to 100, with higher scores indicating more limitations.
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Timepoint [3]
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36 months
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Secondary outcome [4]
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Frequency of whole lung lavages during the trial
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Assessment method [4]
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Timepoint [4]
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36 months
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Secondary outcome [5]
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Diffusion capacity of the lung for carbon monoxide
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Assessment method [5]
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Timepoint [5]
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36 months
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Secondary outcome [6]
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Forced expiratory volume in one second
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Assessment method [6]
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Timepoint [6]
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36 months
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Secondary outcome [7]
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Forced vital capacity
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Assessment method [7]
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Timepoint [7]
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36 months
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Secondary outcome [8]
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Arterial oxygen tension
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Assessment method [8]
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Timepoint [8]
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36 months
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Secondary outcome [9]
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Pulmonary alveolar proteinosis Disease Severity Score
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Assessment method [9]
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Disease-specific score graded based on symptoms of PAP and oxygenation of blood. Scores range from 1 to 5, where 5 indicates most severe disease.
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Timepoint [9]
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36 months
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Secondary outcome [10]
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Need for oxygen supplement therapy
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Assessment method [10]
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Timepoint [10]
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36 months
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Secondary outcome [11]
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Number of subjects not requiring treatment for pulmonary alveolar proteinosis
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Assessment method [11]
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Timepoint [11]
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36 months
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Secondary outcome [12]
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Time off treatment for pulmonary alveolar proteinosis
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Assessment method [12]
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Timepoint [12]
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36 months
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Eligibility
Key inclusion criteria
- Completer of the IMPALA trial.
- Females who have been post menopausal for >1 year, or females of child-bearing
potential who are not pregnant or lactating and are using acceptable contraceptive
methods.
- Males agreeing to use using acceptable contraceptive methods.
- Willing and able to provide signed informed consent.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Treatment with GM-CSF products other than molgramostim nebuliser solution within three
months of Baseline.
- Treatment with any investigational medicinal product other than inhaled molgramostim
within four weeks of Baseline.
- History of allergic reactions to GM-CSF.
- Connective tissue disease, inflammatory bowel disease or other autoimmune disorder
requiring treatment associated with significant immunosuppression, e.g. more than 10
mg/day systemic prednisolone.
- Previous experience of severe and unexplained side effects during aerosol delivery of
any kind of medicinal product.
- History of, or present, myeloproliferative disease or leukaemia.
- Apparent pre-existing concurrent pulmonary fibrosis.
- Any other serious medical condition which in the opinion of the investigator would
make the subject unsuitable for the trial.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/04/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
14/01/2021
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Sample size
Target
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Accrual to date
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Final
62
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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Denmark
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State/province [1]
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Århus
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Country [2]
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France
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State/province [2]
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Rennes
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Country [3]
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Germany
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State/province [3]
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Essen
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Country [4]
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Germany
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State/province [4]
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Gauting
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Country [5]
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Germany
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State/province [5]
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Heidelberg
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Country [6]
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Germany
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State/province [6]
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Lübeck
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Country [7]
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Greece
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State/province [7]
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Athens
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Country [8]
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Israel
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State/province [8]
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Tel Aviv
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Country [9]
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Italy
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State/province [9]
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Pavia
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Country [10]
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Netherlands
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State/province [10]
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Nieuwegein
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Country [11]
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Russian Federation
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State/province [11]
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Saint Petersburg
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Country [12]
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Turkey
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State/province [12]
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Istanbul
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Country [13]
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United Kingdom
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State/province [13]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Savara Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Clinical trial for subjects with autoimmune pulmonary alveolar proteinosis (aPAP) who have
completed the IMPALA trial (NCT02702180).
At the Baseline visit, eligible subjects may continue or re-start treatment with 300 µg
inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor;
GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once
daily, and seven days off treatment.
Subject will be treated with inhaled molgramostim for up to 36 months.
During the trial, whole lung lavage will be applied as rescue therapy.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03482752
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
Name
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Francesco Bonella, Prof.
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Address
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Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03482752
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