Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis

Completed · Phase 2 · Has a placebo group

Conditions studied: Autoimmune Pulmonary Alveolar Proteinosis

In brief

This study evaluates inhaled molgramostim (recombinant human granulocyte macrophage-colony stimulating factor \[rhGM-CSF\]) in the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) patients. A third of the patients will receive inhaled molgramostim once daily for 24 weeks, a third will receive inhaled molgramostim intermittently (7 days on, 7 days off) for 24 weeks and a third will receive inhaled matching placebo for 24 weeks.

Key facts

Study ID

NCT02702180

Run by

Savara Inc.

People needed

139

Starts

2016-03-21

Expected to finish

2019-09-27

Last updated by the study team

2023-04-12

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• aPAP diagnosed by computed tomography, or by biopsy, or by Broncho Alveolar Lavage (BAL), and by increased GM-CSF autoantibodies in serum.
• Stable or progressive aPAP during a minimum period of 2 months prior to the Baseline visit.
• Arterial oxygen tension (PaO2) <75 mmHg/<10 kilo Pascal (kPa) at rest, OR desaturation of >4 percentage points on the 6MWT
• An alveolar-arterial oxygen difference [(A-a)DO2] of minimum 25 mmHg/3.33 kPa
• Female or male ≥18 years of age
• Females who have been post-menopausal for >1 year or females of childbearing potential after a confirmed menstrual period using a highly efficient method of contraception (i.e. a method with <1% failure rate such as combined hormonal contraception, progesterone-only hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner, sexual abstinence), during and until 30 days after last dose of double-blind trial treatment. Females of childbearing potential must have a negative serum pregnancy test at Screening (Visit 1) and a negative urine pregnancy test at dosing at Baseline (Visit 2) and must not be lactating
• Males agreeing to use condoms during and until 30 days after last dose of double-blind medication, or males having a female partner who is using adequate contraception as described above
• Willing and able to provide signed informed consent
• Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other trial procedures specified in the protocol as judged by the investigator

You may not qualify if…

• Diagnosis of hereditary or secondary PAP
• WLL within 1 month of Baseline
• Treatment with GM-CSF within 3 months of Baseline
• Treatment with rituximab within 6 months of Baseline
• Treatment with plasmapheresis within 3 months of Baseline
• Treatment with any investigational medicinal product within 4 weeks of Screening
• Concomitant use of sputum modifying drugs such as carbocysteine or ambroxol
• 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
• Known active infection (viral, bacterial, fungal or mycobacterial)
• Apparent pre-existing concurrent pulmonary fibrosis
• Any other serious medical condition which in the opinion of the investigator would make the participant unsuitable for the trial

Where it is running

• UCLA — Los Angeles, California, United States
• University of Florida — Gainesville, Florida, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Royal Prince Alfred Hospital — Sydney, New South Wales, Australia
• Aarhus University Hospital — Aarhus, Denmark
• CHU Rennes Hospital Pontchaillou — Rennes, France
• Westdeutsches Lungenzentrum am Universitätsklinikum Essen — Essen, Germany
• Asklepios Fachkliniken München - Gauting — Gauting, Germany
• Thoraxklinik am Universitätsklinikum Heidelberg — Heidelberg, Germany
• Universitätsklinikum Schleswig-Holstein Zentralklinikum, Lübeck Medizinische Klinik III, Pneumologie — Lübeck, Germany
• Attikon University Hospital — Athens, Greece
• Rabin Medical Center — Petah Tikva, Israel
• IRCCS Policlinico San Matteo — Pavia, Italy
• Niigata University Medical and Dental Hospital — Niigata, Japan
• National Hospital Organization Kinki-Chuo — Osaka, Japan
• Tohoku University Hospital — Sendai, Japan
• Aichi Medical University Hospital — Toyohashi, Japan
• Kanagawa Cardiovascular and Respiratory Center — Yokohama, Japan
• St. Antonius Hospital — Nieuwegein, Netherlands
• Hospital de dia de Pneumologia — Lisbon, Portugal
• Hospital Sao Joao — Porto, Portugal
• City Hospital St. Petersburg — Saint Petersburg, Russia
• II. Pulmonary Department National Institut for TB, Lung Diseases and Chest Surgery — Vyšné Hágy, Slovakia
• Seoul National University Hospital — Seoul, South Korea
• Asan Medical Center, Division of Pulmonary and Critical Care Medicine — Seoul, South Korea

Full record on ClinicalTrials.gov

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