Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis (IMPALA-X)
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|ClinicalTrials.gov Identifier: NCT03482752|
Recruitment Status : Completed
First Posted : March 29, 2018
Last Update Posted : February 3, 2021
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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.
|Condition or disease||Intervention/treatment||Phase|
|Autoimmune Pulmonary Alveolar Proteinosis||Drug: Molgramostim||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||62 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open Label, Non-controlled|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients|
|Actual Study Start Date :||April 16, 2018|
|Actual Primary Completion Date :||January 14, 2021|
|Actual Study Completion Date :||January 14, 2021|
- Drug: Molgramostim
300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.Other Name: Recombinant human GM-CSF
- Number of adverse events [ Time Frame: 36 months ]
- Number of serious adverse events [ Time Frame: 36 months ]
- Number of adverse drug reactions [ Time Frame: 36 months ]
- Number of adverse events leading to treatment discontinuation [ Time Frame: 36 months ]
- Alveolar-arterial oxygen gradient [ Time Frame: 36 months ]Difference in oxygen tension between lungs and blood
- 6-minute walk distance [ Time Frame: 36 months ]
- St Georges Respiratory Questionnaire total score [ Time Frame: 36 months ]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.
- Frequency of whole lung lavages during the trial [ Time Frame: 36 months ]
- Diffusion capacity of the lung for carbon monoxide [ Time Frame: 36 months ]
- Forced expiratory volume in one second [ Time Frame: 36 months ]
- Forced vital capacity [ Time Frame: 36 months ]
- Arterial oxygen tension [ Time Frame: 36 months ]
- Pulmonary alveolar proteinosis Disease Severity Score [ Time Frame: 36 months ]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.
- Need for oxygen supplement therapy [ Time Frame: 36 months ]
- Number of subjects not requiring treatment for pulmonary alveolar proteinosis [ Time Frame: 36 months ]
- Time off treatment for pulmonary alveolar proteinosis [ Time Frame: 36 months ]
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03482752
|Dept. Of Respiratory Diseases & Allergy|
|CHU Rennes Hospital Pontchaillou, Service de Pneumologie|
|Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH|
|Asklepios Fachkliniken München - Gauting Klinik für Pneumologie|
|Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin|
|Universitätsklinikum Schleswig-Holstein Zentralklinikum Lübeck Medizinische Klinik III - Pneumologie|
|Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens|
|Rabin Medical Center Institute of Pulomonary Medicine|
|Tel Aviv, Israel|
|S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo|
|St. Antonius Hospital|
|Pavlov first Saint Petersburg State Medical Univerisity|
|Saint Petersburg, Russian Federation|
|Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital|
|Istanbul, Turkey, 34020|
|Dept. Of Intensive Care Unit Royal Brompton Hospital London|
|London, United Kingdom|
|Principal Investigator:||Francesco Bonella, Prof.||Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany|
|Responsible Party:||Savara Inc.|
|Other Study ID Numbers:||
|First Posted:||March 29, 2018 Key Record Dates|
|Last Update Posted:||February 3, 2021|
|Last Verified:||February 2021|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Pulmonary Alveolar Proteinosis
Respiratory Tract Diseases
Immune System Diseases
Physiological Effects of Drugs