Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis (IMPALA-X)

Savara

Status and phase: Terminated; Phase 3

Conditions: Autoimmune Pulmonary Alveolar Proteinosis

Treatments: Drug: Molgramostim

Study type: Interventional

Funder types: Industry

Identifiers

NCT03482752

2017-004078-32 (EudraCT Number)

SAV006-03

Details and patient eligibility

About

SAV006-03 is an open-label extension study for participants who had completed the IMPALA study.

At the baseline visit, eligible participants 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.

Participants will be treated with inhaled molgramostim for up to 36 months.

During the trial, whole lung lavage will be applied as rescue therapy.

Enrollment: 60 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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60 participants in 1 patient group

Molgramostim nebulizer solution (300 μg)

Experimental group

Description:

Open-label treatment with molgramostim nebulizer solution (300 μg) administered intermittently (repetitive cycles of 7 days of treatment followed by 7 days off-treatment).

Treatment:

Drug: Molgramostim