IMPALA-X: Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Outcome Measures
Primary Outcome Measures
- Number of adverse events [36 months]
- Number of serious adverse events [36 months]
- Number of adverse drug reactions [36 months]
- Number of adverse events leading to treatment discontinuation [36 months]
Secondary Outcome Measures
- Alveolar-arterial oxygen gradient [36 months]
Difference in oxygen tension between lungs and blood
- 6-minute walk distance [36 months]
- St Georges Respiratory Questionnaire total score [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 [36 months]
- Diffusion capacity of the lung for carbon monoxide [36 months]
- Forced expiratory volume in one second [36 months]
- Forced vital capacity [36 months]
- Arterial oxygen tension [36 months]
- Pulmonary alveolar proteinosis Disease Severity Score [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 [36 months]
- Number of subjects not requiring treatment for pulmonary alveolar proteinosis [36 months]
- Time off treatment for pulmonary alveolar proteinosis [36 months]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Completer of the IMPALA trial.
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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.
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Males agreeing to use using acceptable contraceptive methods.
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Willing and able to provide signed informed consent.
Exclusion Criteria:
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Treatment with GM-CSF products other than molgramostim nebuliser solution within three months of Baseline.
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Treatment with any investigational medicinal product other than inhaled molgramostim within four weeks of Baseline.
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History of allergic reactions to GM-CSF.
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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.
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Previous experience of severe and unexplained side effects during aerosol delivery of any kind of medicinal product.
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History of, or present, myeloproliferative disease or leukaemia.
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Apparent pre-existing concurrent pulmonary fibrosis.
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Any other serious medical condition which in the opinion of the investigator would make the subject unsuitable for the trial.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Dept. Of Respiratory Diseases & Allergy | Århus | Denmark | ||
| 2 | CHU Rennes Hospital Pontchaillou, Service de Pneumologie | Rennes | France | ||
| 3 | Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH | Essen | Germany | ||
| 4 | Asklepios Fachkliniken München - Gauting Klinik für Pneumologie | Gauting | Germany | ||
| 5 | Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin | Heidelberg | Germany | ||
| 6 | Universitätsklinikum Schleswig-Holstein Zentralklinikum Lübeck Medizinische Klinik III - Pneumologie | Lübeck | Germany | ||
| 7 | Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens | Athens | Greece | ||
| 8 | Rabin Medical Center Institute of Pulomonary Medicine | Tel Aviv | Israel | ||
| 9 | S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo | Pavia | Italy | ||
| 10 | St. Antonius Hospital | Nieuwegein | Netherlands | ||
| 11 | Pavlov first Saint Petersburg State Medical Univerisity | Saint Petersburg | Russian Federation | ||
| 12 | Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital | Istanbul | Turkey | 34020 | |
| 13 | Dept. Of Intensive Care Unit Royal Brompton Hospital London | London | United Kingdom |
Sponsors and Collaborators
- Savara Inc.
Investigators
- Principal Investigator: Francesco Bonella, Prof., Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- SAV006-03