Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis

• Status: Completed
• Trial end date: 2021-01-14
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Savara Inc.
• Treatments: Molgramostim; Sargramostim

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.