Molgramostim Nebulizer Solution Expanded Access Program Protocol

NCT06546098 | Unknown

• 1 other identifier: SAV006-06
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 6

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, people with aPAP can experience persistent cough, overwhelming fatigue, unintentional changes in weight, chest or back pain, suddenly feeling out of shape, and general discomfort. Currently, there are no approved medications for aPAP in the United States, but the symptoms of aPAP can be treated with whole lung lavage (WLL). WLL is an invasive procedure that temporarily removes surfactant, and it can result in serious consequences like trauma to the lung, a collapsed lung, and prolonged requirement for artificial ventilation. Savara is studying an investigational drug called molgramostim nebulizer solution to see if it activates the cells that help clear surfactant from the lungs, which improves oxygen transfer from the lungs to the bloodstream. Molgramostim nebulizer solution is administered by inhalation using a hand-held nebulizer. In clinical trials, molgramostim nebulizer solution has shown improvements in gas exchange and patient reported outcomes. This expanded access program will make molgramostim nebulizer solution available to adult patients with diagnosed aPAP. Access must be obtained through the treating physician. Patients will dose molgramostim nebulizer solution 300 micrograms (mcg) once daily and be followed by their physician every 3 months to assess their clinical status and report any adverse events.

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Keywords

surfactant accumulation; autoimmune; alveolar proteinosis; GM-CSF

Interventions

Molgramostim nebulizer solution DRUG

Solution for inhalation

Also known as: Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Savara Inc.: lead

Study Sites (6)

University of California

Los Angeles, California, 90095, United States

AVAILABLE

University Florida Health

Gainesville, Florida, 32610, United States

AVAILABLE

University of Maryland School of Medicine

Baltimore, Maryland, 21201, United States

AVAILABLE

Washington University School of Medicine

St Louis, Missouri, 63110, United States

AVAILABLE

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

AVAILABLE

UT Southwestern Medical Center

Dallas, Texas, 88915, United States

AVAILABLE

Related Publications (12)

• Bogunovic M, Ginhoux F, Helft J, Shang L, Hashimoto D, Greter M, Liu K, Jakubzick C, Ingersoll MA, Leboeuf M, Stanley ER, Nussenzweig M, Lira SA, Randolph GJ, Merad M. Origin of the lamina propria dendritic cell network. Immunity. 2009 Sep 18;31(3):513-25. doi: 10.1016/j.immuni.2009.08.010. Epub 2009 Sep 10.

  PMID: 19733489 BACKGROUND

• Hamilton JA. Colony-stimulating factors in inflammation and autoimmunity. Nat Rev Immunol. 2008 Jul;8(7):533-44. doi: 10.1038/nri2356.

  PMID: 18551128 BACKGROUND

• Inaba K, Inaba M, Romani N, Aya H, Deguchi M, Ikehara S, Muramatsu S, Steinman RM. Generation of large numbers of dendritic cells from mouse bone marrow cultures supplemented with granulocyte/macrophage colony-stimulating factor. J Exp Med. 1992 Dec 1;176(6):1693-702. doi: 10.1084/jem.176.6.1693.

  PMID: 1460426 BACKGROUND

• Khan A, Agarwal R. Pulmonary alveolar proteinosis. Respir Care. 2011 Jul;56(7):1016-28. doi: 10.4187/respcare.01125. Epub 2011 Apr 15.

  PMID: 21496372 BACKGROUND

• Kumar A, Abdelmalak B, Inoue Y, Culver DA. Pulmonary alveolar proteinosis in adults: pathophysiology and clinical approach. Lancet Respir Med. 2018 Jul;6(7):554-565. doi: 10.1016/S2213-2600(18)30043-2. Epub 2018 Feb 1.

  PMID: 29397349 BACKGROUND

• McCarthy C, Avetisyan R, Carey BC, Chalk C, Trapnell BC. Prevalence and healthcare burden of pulmonary alveolar proteinosis. Orphanet J Rare Dis. 2018 Jul 31;13(1):129. doi: 10.1186/s13023-018-0846-y.

  PMID: 30064481 BACKGROUND

• McCarthy C, Carey BC, Trapnell BC. Autoimmune Pulmonary Alveolar Proteinosis. Am J Respir Crit Care Med. 2022 May 1;205(9):1016-1035. doi: 10.1164/rccm.202112-2742SO.

  PMID: 35227171 BACKGROUND

• Metcalf D. The molecular biology and functions of the granulocyte-macrophage colony-stimulating factors. Blood. 1986 Feb;67(2):257-67.

  PMID: 3002522 BACKGROUND

• Park LS, Friend D, Gillis S, Urdal DL. Characterization of the cell surface receptor for human granulocyte/macrophage colony-stimulating factor. J Exp Med. 1986 Jul 1;164(1):251-62. doi: 10.1084/jem.164.1.251.

  PMID: 3014035 BACKGROUND

• Trapnell BC, Carey BC, Uchida K, Suzuki T. Pulmonary alveolar proteinosis, a primary immunodeficiency of impaired GM-CSF stimulation of macrophages. Curr Opin Immunol. 2009 Oct;21(5):514-21. doi: 10.1016/j.coi.2009.09.004. Epub 2009 Sep 30.

  PMID: 19796925 BACKGROUND

• Trapnell BC, Nakata K, Bonella F, Campo I, Griese M, Hamilton J, Wang T, Morgan C, Cottin V, McCarthy C. Pulmonary alveolar proteinosis. Nat Rev Dis Primers. 2019 Mar 7;5(1):16. doi: 10.1038/s41572-019-0066-3.

  PMID: 30846703 BACKGROUND

• Trapnell BC, Inoue Y, Bonella F, Morgan C, Jouneau S, Bendstrup E, Campo I, Papiris SA, Yamaguchi E, Cetinkaya E, Ilkovich MM, Kramer MR, Veltkamp M, Kreuter M, Baba T, Ganslandt C, Tarnow I, Waterer G, Jouhikainen T; IMPALA Trial Investigators. Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis. N Engl J Med. 2020 Oct 22;383(17):1635-1644. doi: 10.1056/NEJMoa1913590. Epub 2020 Sep 7.

  PMID: 32897035 BACKGROUND

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis, Acquired; Pulmonary Alveolar Proteinosis

Interventions

Colony-Stimulating Factors; regramostim

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Central Study Contacts

Yasmine Wasfi, MD., Ph.D.

CONTACT

512-851-1364; yasmine.wasfi@savarapharma.com

Michele Rhee, MBA, MPH

CONTACT

617-807-0488; michele.rhee@savarapharma.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2024
• First Posted: August 9, 2024
• Last Updated: January 7, 2026

Record last verified: 2026-01

Locations

US (6)