Title
Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
Phase
Phase 3Lead Sponsor
Savara Inc.Study Type
InterventionalStatus
Completed No Results PostedIndication/Condition
Autoimmune Pulmonary Alveolar ProteinosisIntervention/Treatment
MolgramostimStudy Participants
62Clinical 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.
300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.
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.
