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New hope for rare lung disease: investigational drug now available through expanded access

NCT ID NCT06546098

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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jul 29, 2026 · Updated 4 times

Summary

This program offers adults with autoimmune pulmonary alveolar proteinosis (aPAP) access to an investigational inhaled medication called molgramostim. aPAP is a rare lung condition where surfactant builds up, making breathing difficult. The drug aims to help the lungs clear this buildup and improve oxygen levels. Patients receive daily doses and are monitored every three months.

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.

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

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: Eligible patients must: 1. Be ≥18 years of age at the time of signing the informed consent. 2. Agree to use a highly effective form of contraception (see Section 3.5). 3. Have a positive serum anti-granulocyte macrophage colony-stimulating factor (GM CSF) autoantibody test result confirming aPAP. 4. Have a history of pulmonary alveolar proteinosis (PAP), based on examination of a lung biopsy, bronchoalveolar lavage (BAL) cytology, or a high-resolution computed tomogram (HRCT) of the chest. 5. Have at least one symptom of aPAP, including but not limited to dyspnea (at rest or with exertion), cough, or fatigue. 6. Be capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. 7. Be willing and able to comply with the visit schedule and treatment plan specified in the protocol, as judged by the physician. Exclusion Criteria: Eligible patients must not: 1. Have a diagnosis of hereditary or secondary PAP, or a metabolic disorder of surfactant production. 2. Require a whole lung lavage (WLL) at the time of screening (patient may be eligible 1 week post WLL). 3. Have received GM-CSF treatment within 1 month prior to the screening visit. 4. Have been treated with any investigational product within 5 half-lives or 3 months (whichever is longer) prior to the screening visit. 5. Have a history of allergic reactions to GM-CSF or any of the excipients in the nebulizer solution. 6. Be using significant (e.g., more than 10 mg/day systemic prednisolone) immunosuppression. 7. Have a history of severe and unexplained side effects during aerosol delivery of any medicinal product. 8. Have a history or current diagnosis of a myeloproliferative disease or leukemia. 9. Have any other medical condition which in the opinion of the physician would make the patient unsuitable for treatment with molgramostim nebulizer solution. 10. Be pregnant or breastfeeding, or planning to become pregnant during treatment with molgramostim nebulizer solution.

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Conditions

The condition(s) this trial relates to.

autoimmune pulmonary alveolar proteinosis pulmonary alveolar proteinosis

As listed by the trial registrant

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

Autoimmune pulmonary alveolar proteinosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    9 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AGH Allegheny General Hospital

    AVAILABLE

    Pittsburgh, Pennsylvania, 15212, United States

  • Cincinnati Children's Hospital Medical Center

    AVAILABLE

    Cincinnati, Ohio, 45229, United States

  • Duke University Hospital

    AVAILABLE

    Durham, North Carolina, 27708, United States

  • UT Southwestern Medical Center

    AVAILABLE

    Dallas, Texas, 88915, United States

  • University Florida Health

    AVAILABLE

    Gainesville, Florida, 32610, United States

  • University of California

    AVAILABLE

    Los Angeles, California, 90095, United States

  • University of Maryland School of Medicine

    AVAILABLE

    Baltimore, Maryland, 21201, United States

  • WakeMed Raleigh

    AVAILABLE

    Raleigh, North Carolina, 27610, United States

  • Washington University School of Medicine

    AVAILABLE

    St Louis, Missouri, 63110, United States

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