Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis

• Register: ClinicalTrials.gov
• Last refreshed on: 16 February 2021
• Main ID: NCT03482752
• Date of registration: 21/03/2018
• Prospective Registration: Yes
• Primary sponsor: Savara Inc.
• Public title: Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis IMPALA-X
• Scientific title: An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
• Date of first enrolment: April 16, 2018
• Target sample size: 62
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03482752
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Denmark, France, Germany, Greece, Israel, Italy, Netherlands, Russian Federation, Turkey, United Kingdom

Contacts

• Name: Francesco Bonella, Prof.
• Affiliation: Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany

Key inclusion & exclusion criteria

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.

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Autoimmune Pulmonary Alveolar Proteinosis

Intervention(s)

Drug: Molgramostim

Primary Outcome(s)

Number of serious adverse events [Time Frame: 36 months]

Number of adverse events [Time Frame: 36 months]

Number of adverse events leading to treatment discontinuation [Time Frame: 36 months]

Number of adverse drug reactions [Time Frame: 36 months]

Secondary Outcome(s)

St Georges Respiratory Questionnaire total score [Time Frame: 36 months]

6-minute walk distance [Time Frame: 36 months]

Alveolar-arterial oxygen gradient [Time Frame: 36 months]

Number of subjects not requiring treatment for pulmonary alveolar proteinosis [Time Frame: 36 months]

Pulmonary alveolar proteinosis Disease Severity Score [Time Frame: 36 months]

Frequency of whole lung lavages during the trial [Time Frame: 36 months]

Need for oxygen supplement therapy [Time Frame: 36 months]

Forced expiratory volume in one second [Time Frame: 36 months]

Forced vital capacity [Time Frame: 36 months]

Time off treatment for pulmonary alveolar proteinosis [Time Frame: 36 months]

Arterial oxygen tension [Time Frame: 36 months]

Diffusion capacity of the lung for carbon monoxide [Time Frame: 36 months]

Secondary ID(s)

SAV006-03

Source(s) of Monetary Support

Please refer to primary and secondary sponsors