Autoimmune Pulmonary Alveolar ProteinosisPhase 3NCT06431776
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
A Phase 3 study testing Molgramostim in people with Autoimmune Pulmonary Alveolar Proteinosis, run by Savara Inc. at 1 site in Germany.
Trial summary
The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are: The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety… Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Autoimmune Pulmonary Alveolar Proteinosis
- What is being tested?
- Molgramostim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteGermany
- Study dates
- 22 Oct 2025to Dec 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Be ≥6 and <18 years of age, at the time of signing the informed consent and informed assent (if applicable).
- Have a history of pulmonary alveolar proteinosis, based on examination of a lung biopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram of the chest.
- Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP.
- Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening.
Who cannot join
- Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production.
- Have undergone treatment with Lung Lavage (WLL) within 1 month of Baseline
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Germany1 site, 1 recruiting
- Recruiting
Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik
München, 80337
Matthias Griese, MD, +49 8951603677, matthias.griese@med.uni-muenchen.de
Angelika Gold, +49 894400-57871, Angelika.Gold@med.uni-muenchen.de
About this study
Official title: An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are:
The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP.
This is an open-label study: all participants will receive treatment with molgramostim.
Patients will:
- Take molgramostim once daily via nebulizer every day for 12 months.
- Visit the clinic approximately every 12 weeks for checkups and tests.
- Keep a diary of any oxygen use.
Read the detailed description
This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP).
The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology.
The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP.
- Being tested
- Molgramostim
- Keywords
- alveolar proteinosis, autoimmune, lung lavage, GM-CSF, children
- 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.
MolgramostimDrug
Also called Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF).
Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds.
| Study group | Role | What they receive |
|---|---|---|
| molgramostim | Experimental | Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks. Receives: Drug: Molgramostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
DLCO
Change in Hb-adjusted % predicted DLCO from Baseline.
Measured: 24 weeks
Secondary outcomes (7)
DLCO
Change in Hb-adjusted % predicted DLCO from Baseline .
Measured: 48-weeks
6-minute walk distance
Absolute change from Baseline in 6-minute walk distance (6MWD)
Measured: 24-weeks
6-minute walk distance
Absolute change from Baseline in 6-minute walk distance (6MWD).
Measured: 48-weeks
PedsQL
Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score.
Measured: 24-weeks
PedsQL
Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score.
Measured: 48-weeks
Oxygen Saturation (SpO2)
Absolute change from Baseline in oxygen saturation (SpO2)
Measured: 24 weeks
Oxygen Saturation (SpO2)
Absolute change from Baseline in oxygen saturation (SpO2)
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2024
- Study start22 Oct 2025
- Primary completionJun 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label study
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Savara Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Matthias Griese, MD,, Principal investigator, Ludwig Maximilians Universität München
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06431776
- Sponsor's protocol ID
- SAV006-04
- Status last verified
- Feb 2026
- First submitted
- 22 May 2024
- Last updated
- 19 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
- ClinicalTrials.gov ID
- NCT06431776
- Acronym / sponsor ID
- SAV006-04
- Keywords
- alveolar proteinosis, autoimmune, lung lavage, GM-CSF, children
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Savara Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Oct 2025 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 29 May 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.
The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are:
The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP.
This is an open-label study: all participants will receive treatment with molgramostim.
Patients will:
- Take molgramostim once daily via nebulizer every day for 12 months.
- Visit the clinic approximately every 12 weeks for checkups and tests.
- Keep a diary of any oxygen use.
Detailed description
This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP).
The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology.
The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label study
| Arm | Type | Receives |
|---|---|---|
| molgramostim | Experimental | Drug: MolgramostimMolgramostim 300 mcg administered once daily via nebulizer for 48 weeks. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Be ≥6 and <18 years of age, at the time of signing the informed consent and informed assent (if applicable).
- Have a history of pulmonary alveolar proteinosis, based on examination of a lung biopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram of the chest.
- Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP.
- Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening.
Key exclusion criteria (2 listed)
- Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production.
- Have undergone treatment with Lung Lavage (WLL) within 1 month of Baseline
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 |
|---|---|---|
| MolgramostimAlso: Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF).Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds. | Drug | molgramostim |
Primary outcome
DLCO
Time frame: 24 weeks
Change in Hb-adjusted % predicted DLCO from Baseline.
7 secondary outcomes
DLCO
Time frame: 48-weeks
6-minute walk distance
Time frame: 24-weeks
6-minute walk distance
Time frame: 48-weeks
PedsQL
Time frame: 24-weeks
PedsQL
Time frame: 48-weeks
Oxygen Saturation (SpO2)
Time frame: 24 weeks
Oxygen Saturation (SpO2)
Time frame: 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2026.
- Individual participant dataSharing planned: No
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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.
Matthias Griese, MD, principal investigator
Ludwig Maximilians Universität München
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Molgramostim in people with Autoimmune Pulmonary Alveolar Proteinosis, run by Savara Inc. at 1 site in Germany.
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 4 inclusion and 2 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: looking for participants now, as of the sponsor's last update on 19 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yasmine Wasfi, MD, PhD, (1 512 851 1364, yasmine.wasfi@savarapharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DLCO (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Feb 2026.