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Can a daily inhaled protein restore lung function in a rare autoimmune disease?

NCT ID NCT04544293

Disease control Sponsor: Savara Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 26, 2026 · Last updated Aug 27, 2026 · Updated 1 time

Summary

This trial tests whether inhaling molgramostim—a lab-made version of a protein that helps lung cells clear out protein buildup—can improve lung function in adults with autoimmune pulmonary alveolar proteinosis (aPAP). In this condition, the immune system blocks a key signal, causing protein to fill the air sacs and make breathing difficult. Participants receive either the inhaled treatment or a placebo once daily for 48 weeks, followed by an open-label period where everyone gets the treatment. The main goal is to see if the treatment improves the lungs' ability to transfer oxygen, measured by a standard breathing test.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Inhaled molgramostim (a lab-made version of a natural protein that helps lung cells clear protein buildup)
What this could lead to
If it works, this could become a daily inhaled treatment to improve breathing and lung function in people with autoimmune pulmonary alveolar proteinosis.
What could go wrong
This is a phase 3 trial, but results are not yet known. The treatment may not improve lung function for everyone, and inhaled therapy can cause side effects like cough or chest discomfort.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

164 people

The number who actually took part.

Started

May 2021

Expected to finish

May 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Subject must be ≥18 years of age, at the time of signing the informed consent (≥20 in Japan). 2. A serum anti-GM-CSF autoantibody test result confirming autoimmune PAP. 3. History of PAP, based on examination of a lung biopsy, bronchoalveolar lavage (BAL) cytology, or a high-resolution computed tomogram (HRCT) of the chest. 4. A diffusing capacity for carbon monoxide of 70% predicted or lower adjusted for hemoglobin (%DLCOadj) at the screening and baseline visits. 5. Change in %DLCO adj of \<15% points during the screening period. 6. Demonstrated functional impairment in the treadmill exercise test (defined as a peak metabolic equivalent (MET) ≤8). 7. Willing and able to come off supplemental oxygen use prior to and during the treadmill exercise test, the DLCO assessment, and the arterial blood gas sampling. 8. Resting oxygen saturation (SpO2) \>85% during 15 minutes without use of supplemental oxygen at the screening visits. 9. Male or female 10. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. 1. Male subjects: Males agreeing to use condoms during and until 30 days after last dose of trial treatment, or males having a female partner who is using adequate contraception as described below. 2. Female subjects: 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, vasectomized partner, sexual abstinence\*), during and until 30 days after last dose of trial treatment. Females of childbearing potential must have a negative serum pregnancy test at the screening visits, and a negative urine pregnancy test at Baseline visit (Visit 3) and must not be lactating. 11. Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. 12. 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. Exclusion Criteria: 1. Diagnosis of hereditary or secondary PAP, or a metabolic disorder of surfactant production. 2. Whole lung lavage (WLL) performed within 3 months prior to baseline. 3. Requirement for WLL at screening or baseline. 4. GM-CSF treatment within 6 months prior to baseline. 5. Treatment with rituximab within 6 months prior to baseline. 6. Treatment with plasmapheresis within 6 weeks prior to baseline. 7. Treatment with any investigational medicinal product within 5 half-lives or 3 months (whichever is longer) prior to baseline. 8. Previously randomized in this trial. 9. History of allergic reactions to GM-CSF or any of the excipients in the nebulizer solution. 10. Inflammatory or autoimmune disease of a severity that necessitates significant (e.g. more than 10 mg/day systemic prednisolone) immunosuppression. 11. Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product. 12. History of, or present, myeloproliferative disease or leukemia. 13. Apparent pre-existing concurrent pulmonary fibrosis. 14. Acute or unstable cardiac or pulmonary disease that may be aggravated by exercise. 15. Known active infection (viral, bacterial, fungal, or mycobacterial) that may affect the efficacy evaluation in the trial. 16. Physical disability or other condition that precludes safe and adequate exercise testing. 17. Any other serious medical condition which in the opinion of the Investigator would make the subject unsuitable for the trial. 18. Pregnant, planning to become pregnant during the trial, or breastfeeding woman. For France only: including as further defined by French Health Code L-1121-5. 19. For France only: Any subject considered to be "vulnerable" on account of, e.g., mental or physical disability, socio-economic situation, or subjects deprived of their liberty. For France only: including as further defined by French Health Code L1121-8-1.

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Conditions

The condition(s) this trial relates to.

autoimmune pulmonary alveolar proteinosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Autoimmune pulmonary alveolar proteinosis

Contacts and locations

Locations

  • Aichi Medical University Hospital

    Nagakute, 480-1195, Japan

  • Asan Medical Center

    Seoul, 05505, South Korea

  • Asklepios Fachkliniken Muenchen-Gauting

    Muenchen-Gauting, Bavaria, 82131, Germany

  • Attikon University Hospital

    Athens, 12462, Greece

  • CHU Pontchaillou

    Rennes, Brittany Region, 35033, France

  • Chiba University Hospital - Respiratory Medicine

    Chiba, Chiba, 260-8677, Japan

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Ege University Hospital - Department of Pulmonology

    Bornova, İzmir, 35100, Turkey (Türkiye)

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, Lombardy, 27100, Italy

  • Health Sciences University Gulhane Training and Research Hospital

    Ankara, Ankara, 6010, Turkey (Türkiye)

  • Hokkaido University Hospital

    Sapporo, Hokkaidô, 060-8648, Japan

  • Hospital Pulido Valente

    Lisbon, Lisbon District, 1769-001, Portugal

  • Hospital São João

    Porto, Porto District, 4200-319, Portugal

  • Hospital Universitario de Bellvitge

    Barcelona, Catalonia, 08907, Spain

  • Hôpital Erasme

    Brussels, Brussels Capital, 1070, Belgium

  • Hôpital Louis Pradel

    Bron, Auvergne-Rhône-Alpes, 69500, France

  • Institutul de Pneumoftiziologie "Marius Nasta"

    Bucharest, Bucharest, 050159, Romania

  • Instytut Gruzlicy i Chorob Pluc

    Warsaw, Masovian Voivodeship, 01-138, Poland

  • Kanagawa Cardiovascular and Respiratory Center

    Yokohama, Kanagawa, 236-0051, Japan

  • Kumamoto University Hospital

    Kumamoto, Kumamoto, 860-8556, Japan

  • Kyorin University Hospital

    Mitaka, Tokyo, 181-8611, Japan

  • Loyola University

    Maywood, Illinois, 60153, United States

  • National Hospital Organization Kinki-Chuo Chest medical Center

    Sakai, Osaka, 591-8555, Japan

  • National Jewish Health

    Denver, Colorado, 80206, United States

  • Royal Brompton and Harefield NHS Foundation Trust

    London, London, SW3 6NP, United Kingdom

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050, Australia

  • Ruhrlandklinik Westdeutsches Lungenzentrum

    Essen, North Rhine-Westphalia, 45239, Germany

  • Saitama Red Cross Hospital

    Saitama, Saitama, 330-8553, Japan

  • Samsung Medical Center

    Seoul, 06351, South Korea

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Severance Hospital - Yonsei University Health System - Pulmonary

    Seoul, 03722, South Korea

  • St Antonius Hospital

    Nieuwegein, Utrecht, 3435CM, Netherlands

  • St Joseph's Healthcare Hamilton Research

    Hamilton, Ontario, L8N 4A6, Canada

  • St. Vincent's University Hospital

    Dublin, DO4 T6F4, Ireland

  • Thoraxklinik Heidelberg gGmbH am Universitätsklinikum Heidelberg

    Heidelberg, Baden-Wurttemberg, 69126, Germany

  • Tohoku University Hospital - Respiratory Tract Medicine

    Sendai, Miyagi, 980-8574, Japan

  • UCLA David Geffen School of Medicine

    Los Angeles, California, 90095, United States

  • UT Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • UZ Leuven - Campus Gasthuisberg - Pneumologie

    Leuven, Vlaams Brabant, 3000, Belgium

  • University Institute of Cardiology and Respirology of Quebec

    Québec, Quebec, G1V 4G5, Canada

  • University Of Arkansas For Medical Services

    Little Rock, Arkansas, 72205, United States

  • University of Calgary

    Calgary, Alberta, T2N 4N1, Canada

  • University of Florida Health

    Gainesville, Florida, 32610, United States

  • University of Maryland Medical Center

    Baltimore, Maryland, 21201, United States

  • University of Pennsylvania Perelman School of Medicine - Pulmonology

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

  • Wake Med Health & Hospital

    Raleigh, North Carolina, 27610, United States

  • Washington University in St. Louis

    St Louis, Missouri, 63110, United States

  • Westmead Hospital

    Westmead, New South Wales, 2145, Australia

  • Yale University

    New Haven, Connecticut, 06520, United States

  • Yedikule Chest Disease and Surgery Training and Research Hospital

    Istanbul, Istanbul, 34020, Turkey (Türkiye)

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