Molgramostim Nebulizer Solution Expanded Access Program Protocol

Status:

AVAILABLE

Molgramostim Nebulizer Solution Expanded Access Program Protocol

Lead Sponsor:

Savara Inc.

Conditions:

Autoimmune Pulmonary Alveolar Proteinosis

Eligibility:

All Genders

18+ years

Brief Summary

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disorder in which a material called surfactant builds up in the lungs and makes it hard to breathe. In addition to shortness of breath, peopl...

Detailed Description

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disease mediated by autoantibodies targeting granulocyte macrophage colony-stimulating factor (GM-CSF), resulting in malfunctioning macrophag...

Eligibility Criteria

Inclusion

Eligible patients must:
Be ≥18 years of age at the time of signing the informed consent.
Agree to use a highly effective form of contraception (see Section 3.5).
Have a positive serum anti-granulocyte macrophage colony-stimulating factor (GM CSF) autoantibody test result confirming aPAP.
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.
Have at least one symptom of aPAP, including but not limited to dyspnea (at rest or with exertion), cough, or fatigue.
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.
Be willing and able to comply with the visit schedule and treatment plan specified in the protocol, as judged by the physician.

Exclusion

Eligible patients must not:
Have a diagnosis of hereditary or secondary PAP, or a metabolic disorder of surfactant production.
Require a whole lung lavage (WLL) at the time of screening (patient may be eligible 1 week post WLL).
Have received GM-CSF treatment within 1 month prior to the screening visit.
Have been treated with any investigational product within 5 half-lives or 3 months (whichever is longer) prior to the screening visit.
Have a history of allergic reactions to GM-CSF or any of the excipients in the nebulizer solution.
Be using significant (e.g., more than 10 mg/day systemic prednisolone) immunosuppression.
Have a history of severe and unexplained side effects during aerosol delivery of any medicinal product.
Have a history or current diagnosis of a myeloproliferative disease or leukemia.
Have any other medical condition which in the opinion of the physician would make the patient unsuitable for treatment with molgramostim nebulizer solution.
Be pregnant or breastfeeding, or planning to become pregnant during treatment with molgramostim nebulizer solution.

Key Trial Info

Start Date :

Trial Type :

EXPANDED_ACCESS

End Date :

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT06546098

Last Update

January 7 2026

Active Locations (6)

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Page 1 of 2 (6 locations)

University of California

Los Angeles, California, United States, 90095

University Florida Health

Gainesville, Florida, United States, 32610

University of Maryland School of Medicine

Baltimore, Maryland, United States, 21201