Autoimmune Pulmonary Alveolar ProteinosisNCT06546098
Molgramostim Nebulizer Solution Expanded Access Program Protocol
A study testing Molgramostim nebulizer solution in people with Autoimmune Pulmonary Alveolar Proteinosis, run by Savara Inc. at 12 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Pulmonary Alveolar Proteinosis
- What is being tested?
- Molgramostim nebulizer solution
- How many people
- Not given
- Where
- 12 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 11Show fewer
- 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.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
- Available
University of Maryland School of Medicine
Baltimore, Maryland, 21201
Valeriya Rodionova, MD, 410-328-2835, vrodionova@som.umaryland.edu
- Available
The Medical University of South Carolina
Charleston, South Carolina, 29425
Zerlinna Teague, 843-792-0965, recruitment@musc.edu
- Available
University of Virginia Health
Charlottesville, Virginia, 22902
Heather M Haughey, Ph.D., 434-243-4008, UVAJEDITrials@uvahealth.org
- Available
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Brenna Carey, MS, PhD, 513-636-8916, Brenna.Carey@cchmc.org
- Available
UT Southwestern Medical Center
Dallas, Texas, 88915
Uma Gude, uma.gude@utsouthwestern.edu
- Available
Duke University Hospital
Durham, North Carolina, 27708
Wendy Curry, 919-668-8281, Wendy.curry@duke.edu
- Available
University Florida Health
Gainesville, Florida, 32610
Ali Ataya, MD, Ali.Ataya@medicine.ufl.edu
- Available
University of California
Los Angeles, California, 90095
Maria C Granone, MD, CCRP, mgranone@mednet.ucla.edu
- Available
AGH Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
Marvin R Balaan, MD, FCCP, 412-463-3298, marvin.balaan@ahn.org
- Available
WakeMed Raleigh
Raleigh, North Carolina, 27610
Kevin Davidson, MD
Rhonda Norton, CCRC, 919-350-5252, rnorton@wakemed.org
- Available
UW Medicine
Seattle, Washington, 98195
Lisa Batten, 206-310-7517, lsetya@uw.edu
- Available
Washington University School of Medicine
St Louis, Missouri, 63110
Kristine Kempf, RN, 314-273-8131, kempf@wustl.edu
About this study
Official title: Expanded Access to Inhaled Molgramostim Nebulizer Solution in Adults With Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
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.
Read more
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.
Read the 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 macrophages with impaired surfactant catabolism. The latter causes accumulation of surfactant in the alveoli, which has a negative impact on gas exchange between lung and blood. The clinical course of autoimmune PAP varies among patients with continuously slowly progressive disease in most patients, spontaneous improvement in a small percentage (5-7%), and rapid progression and/or pulmonary fibrosis, respiratory failure, and death in others. The most common cause of death is respiratory failure followed by secondary pulmonary bacterial infections. Estimated 5-year mortality rates vary between 10-30%, with overall disease-specific survival rates at 5 years exceeding 80%.
There are currently no approved pharmacological treatments for aPAP in most of the world. Whole lung lavage (WLL) is the primary treatment option currently available for most aPAP patients. However, its invasive nature, limited access, and the variable effect of WLLs due to lack of standardization emphasize that there is an unmet need for a non-invasive, safe and well-tolerated, easily accessible and effective treatment for aPAP patients.
The rationale for treating aPAP patients with molgramostim nebulizer solution is based on the capacity of GM-CSF to promote differentiation and mobilization of different myeloid leukocyte subsets including neutrophils, tissue macrophages/dendritic cells or their circulating precursors. It is crucially involved in anti-microbial pulmonary host defense and ameliorates lung injury by increasing the size and activation of the alveolar macrophage pool. GM-CSF also contributes to the proliferation of megakaryocytic and erythroid progenitors and plays a key role in surfactant homeostasis, by maturation of alveolar macrophages.
Molgramostim is a non-glycosylated recombinant human granulocyte macrophage colony stimulating factor (rhGM-CSF) produced by using recombinant DNA technology via a bacterial (E. coli) expression system. Molgramostim is formulated in a sterile nebulizer solution (molgramostim nebulizer solution) which is supplied in vials containing 300 µg of molgramostim in 1.2 mL solution and is administered by inhalation via an investigational eFlow Nebulizer System (PARI Pharma GmbH).
Results of a completed randomized, placebo-controlled clinical trial support the safety, tolerability, and efficacy of inhaled molgramostim nebulizer solution as a treatment for autoimmune PAP patients.
- Being tested
- Molgramostim nebulizer solution
- Keywords
- surfactant accumulation, autoimmune, alveolar proteinosis, GM-CSF
- Medical subject headings
- Pulmonary Alveolar Proteinosis, Acquired, Pulmonary Alveolar Proteinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Molgramostim nebulizer solutionDrug
Also called Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF)
Solution for inhalation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (12)Papers the sponsor has linked to this study.
- Other trials for Autoimmune Pulmonary Alveolar ProteinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Savara Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06546098
- Sponsor's protocol ID
- SAV006-06
- Status last verified
- Jul 2026
- First submitted
- 5 Aug 2024
- Last updated
- 26 Oct 2026
- Results posted
- No
Publications
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.
PubMed 19733489Hamilton JA. Colony-stimulating factors in inflammation and autoimmunity. Nat Rev Immunol. 2008 Jul;8(7):533-44. doi: 10.1038/nri2356.
PubMed 18551128Inaba 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.
PubMed 1460426Khan A, Agarwal R. Pulmonary alveolar proteinosis. Respir Care. 2011 Jul;56(7):1016-28. doi: 10.4187/respcare.01125. Epub 2011 Apr 15.
PubMed 21496372Kumar 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.
PubMed 29397349McCarthy 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.
PubMed 30064481McCarthy 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.
PubMed 35227171Metcalf D. The molecular biology and functions of the granulocyte-macrophage colony-stimulating factors. Blood. 1986 Feb;67(2):257-67.
PubMed 3002522Park 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.
PubMed 3014035Trapnell 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.
PubMed 19796925Trapnell 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.
PubMed 30846703Trapnell 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.
PubMed 32897035
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access to Inhaled Molgramostim Nebulizer Solution in Adults With Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
- ClinicalTrials.gov ID
- NCT06546098
- Acronym / sponsor ID
- SAV006-06
- Keywords
- surfactant accumulation, autoimmune, alveolar proteinosis, GM-CSF
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Savara Inc.Industry (pharma, biotech, devices)
- First posted
- 9 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 macrophages with impaired surfactant catabolism. The latter causes accumulation of surfactant in the alveoli, which has a negative impact on gas exchange between lung and blood. The clinical course of autoimmune PAP varies among patients with continuously slowly progressive disease in most patients, spontaneous improvement in a small percentage (5-7%), and rapid progression and/or pulmonary fibrosis, respiratory failure, and death in others. The most common cause of death is respiratory failure followed by secondary pulmonary bacterial infections. Estimated 5-year mortality rates vary between 10-30%, with overall disease-specific survival rates at 5 years exceeding 80%.
There are currently no approved pharmacological treatments for aPAP in most of the world. Whole lung lavage (WLL) is the primary treatment option currently available for most aPAP patients. However, its invasive nature, limited access, and the variable effect of WLLs due to lack of standardization emphasize that there is an unmet need for a non-invasive, safe and well-tolerated, easily accessible and effective treatment for aPAP patients.
The rationale for treating aPAP patients with molgramostim nebulizer solution is based on the capacity of GM-CSF to promote differentiation and mobilization of different myeloid leukocyte subsets including neutrophils, tissue macrophages/dendritic cells or their circulating precursors. It is crucially involved in anti-microbial pulmonary host defense and ameliorates lung injury by increasing the size and activation of the alveolar macrophage pool. GM-CSF also contributes to the proliferation of megakaryocytic and erythroid progenitors and plays a key role in surfactant homeostasis, by maturation of alveolar macrophages.
Molgramostim is a non-glycosylated recombinant human granulocyte macrophage colony stimulating factor (rhGM-CSF) produced by using recombinant DNA technology via a bacterial (E. coli) expression system. Molgramostim is formulated in a sterile nebulizer solution (molgramostim nebulizer solution) which is supplied in vials containing 300 µg of molgramostim in 1.2 mL solution and is administered by inhalation via an investigational eFlow Nebulizer System (PARI Pharma GmbH).
Results of a completed randomized, placebo-controlled clinical trial support the safety, tolerability, and efficacy of inhaled molgramostim nebulizer solution as a treatment for autoimmune PAP patients.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Molgramostim nebulizer solutionAlso: Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF)Solution for inhalation | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- Background: PubMed 19733489Bogunovic 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.
- Background: PubMed 18551128Hamilton JA. Colony-stimulating factors in inflammation and autoimmunity. Nat Rev Immunol. 2008 Jul;8(7):533-44. doi: 10.1038/nri2356.
- Background: PubMed 1460426Inaba 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.
- Background: PubMed 21496372Khan A, Agarwal R. Pulmonary alveolar proteinosis. Respir Care. 2011 Jul;56(7):1016-28. doi: 10.4187/respcare.01125. Epub 2011 Apr 15.
- Background: PubMed 29397349Kumar 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.
- Background: PubMed 30064481McCarthy 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.
- Background: PubMed 35227171McCarthy 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.
- Background: PubMed 3002522Metcalf D. The molecular biology and functions of the granulocyte-macrophage colony-stimulating factors. Blood. 1986 Feb;67(2):257-67.
- Background: PubMed 3014035Park 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.
- Background: PubMed 19796925Trapnell 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.
- Background: PubMed 30846703Trapnell 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.
- Background: PubMed 32897035Trapnell 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Molgramostim nebulizer solution in people with Autoimmune Pulmonary Alveolar Proteinosis, run by Savara Inc. at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yasmine Wasfi, MD., Ph.D. (512-851-1364, yasmine.wasfi@savarapharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06546098, which also lists 12 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.