Clinical trial NCT03482752: Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
Official title: An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Autoimmune Pulmonary Alveolar Proteinosis
Lead sponsor: Savara Inc.
Sponsor class: INDUSTRY
First posted: 2018-03-29
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label, non-controlled.
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Number of Treatment-emergent Adverse Events (TEAEs)
timeFrame: 139 weeks
measure: Number of Serious TEAEs
timeFrame: 139 weeks
measure: Number of Treatment-emergent Adverse Drug Reactions (ADRs)
timeFrame: 139 weeks
measure: Number of TEAEs Leading to Treatment Discontinuation
timeFrame: 139 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Århus
country: Denmark
facility: Dept. Of Respiratory Diseases & Allergy
city: Rennes
country: France
facility: CHU Rennes Hospital Pontchaillou, Service de Pneumologie
city: Essen
country: Germany
facility: Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH
city: Gauting
country: Germany
facility: Asklepios Fachkliniken München - Gauting Klinik für Pneumologie
city: Heidelberg
country: Germany
facility: Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin
city: Lübeck
country: Germany
facility: Universitätsklinikum Schleswig-Holstein Zentralklinikum Lübeck Medizinische Klinik III - Pneumologie
city: Athens
country: Greece
facility: Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens
city: Tel Aviv
country: Israel
facility: Rabin Medical Center Institute of Pulomonary Medicine
city: Pavia
country: Italy
facility: S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo
city: Nieuwegein
country: Netherlands
facility: St. Antonius Hospital
city: Saint Petersburg
country: Russian Federation
facility: Pavlov first Saint Petersburg State Medical Univerisity
city: Istanbul
country: Turkey
facility: Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital
city: London
country: United Kingdom
facility: Dept. Of Intensive Care Unit Royal Brompton Hospital London
Study officials:
affiliation: Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany
name: Francesco Bonella, Prof.
role: PRINCIPAL_INVESTIGATOR
References: