Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis

NCT ID: NCT03482752

Last Updated: 2024-07-03

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-04-16
• Study Completion Date: 2021-01-14

Brief Summary

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.

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Molgramostim nebulizer solution (300 μg)

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

Group Type: EXPERIMENTAL

Molgramostim

Intervention Type: DRUG

300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.

Interventions

Molgramostim

300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.

Intervention Type: DRUG

Other Intervention Names

Recombinant human GM-CSF

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Savara Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Francesco Bonella, Prof.

Role: PRINCIPAL_INVESTIGATOR

Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany

Locations

Dept. Of Respiratory Diseases & Allergy

Aarhus, , Denmark

CHU Rennes Hospital Pontchaillou, Service de Pneumologie

Rennes, , France

Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH

Essen, , Germany

Asklepios Fachkliniken München - Gauting Klinik für Pneumologie

Gauting, , Germany

Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin

Heidelberg, , Germany

Universitätsklinikum Schleswig-Holstein Zentralklinikum Lübeck Medizinische Klinik III - Pneumologie

Lübeck, , Germany

Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens

Athens, , Greece

Rabin Medical Center Institute of Pulomonary Medicine

Tel Aviv, , Israel

S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo

Pavia, , Italy

St. Antonius Hospital

Nieuwegein, , Netherlands

Pavlov first Saint Petersburg State Medical Univerisity

Saint Petersburg, , Russia

Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital

Istanbul, , Turkey (Türkiye)

Dept. Of Intensive Care Unit Royal Brompton Hospital London

London, , United Kingdom

Countries

Denmark; France; Germany; Greece; Israel; Italy; Netherlands; Russia; Turkey (Türkiye); United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2017-004078-32

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

SAV006-03

Identifier Type: -

Identifier Source: org_study_id