ClinicalTrials.gov record NCT06431776

Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.

Age6 years old to 18 years old

SexAll

LocationGermany

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label study gives inhaled molgramostim to pediatric participants with autoimmune pulmonary alveolar proteinosis (aPAP). All participants receive daily nebulized molgramostim for up to 48 weeks, with clinic visits about every 12 weeks. Whole lung lavage (WLL) is allowed as a rescue treatment if aPAP worsens.

What it is studying. The study is testing whether daily inhaled molgramostim affects breathing tests (primary: DLCO) and activity measures, and is assessing the safety of molgramostim in pediatric participants with aPAP. It also measures other outcomes such as 6-minute walk distance, pediatric quality of life (PedsQL), and oxygen saturation.

What the researchers hope to learn. Scientific goals: to measure change in hemoglobin-adjusted percent predicted DLCO at 24 weeks (primary outcome), to assess DLCO again at 48 weeks, and to evaluate changes in 6-minute walk distance, PedsQL scores, oxygen saturation, and safety over the treatment period.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: Molgramostim

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

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.

Exclusion criteria

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After a 4-week screening period, participants will inhale molgramostim 300 mcg once daily using a study nebulizer for 48 weeks, followed by a 4-week safety follow-up. They will come to the clinic about every 12 weeks for checkups and tests, including breathing tests (DLCO), 6-minute walk tests, oxygen saturation checks, and quality-of-life questionnaires. Participants will keep a diary of any oxygen use, and lung lavage (whole‑lung lavage) may be used as a rescue treatment if aPAP worsens. Key assessments at 24 and 48 weeks will measure lung diffusing capacity and walking distance while the study team monitors safety throughout the study.

How the study is structured. This is an interventional, open-label, single-group study: every enrolled child receives molgramostim (nebulized) once daily. The study includes a 4-week screening period, a 48-week treatment period, and a 4-week safety follow-up after treatment ends. There is no placebo or randomized comparison and no masking. Clinic visits occur roughly every 12 weeks; whole lung lavage (WLL) may be used if needed as a rescue therapy.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The listed sites are in Germany. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on pulmonary alveolar proteinosis

Clinical trials5 trials listed for pulmonary alveolar proteinosisEvery trial the registry lists for pulmonary alveolar proteinosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in pulmonary alveolar proteinosis researchLatest monthly update: September 2026. Includes: ECMO during lavage. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Oct 22, 2025
Primary completion: June 2027 (estimated)
Study completion: December 2027 (estimated)
Target enrollment: 5 participants (estimated)
Last updated on registry: Feb 19, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Savara Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Yasmine Wasfi, MD, PhD, 📞 1 512 851 1364 ✉ yasmine.wasfi@savarapharma.com
Brian Maurer 📞 1 512 774 5786 ✉ brian.mauer@savarapharma.com

Participating sites (1)

Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und KinderpoliklinikRecruiting
München, 80337 · Germany
- Matthias Griese, MD · +49 8951603677 · matthias.griese@med.uni-muenchen.de
- Angelika Gold · +49 894400-57871 · Angelika.Gold@med.uni-muenchen.de

Terms used on this page · explanations by MedicalUpdates.ai

open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent: A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
biopsy: Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
diffusing capacity: A breathing test that measures how well the lungs pass oxygen from the air into the blood.
at Screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Baseline: Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
single arm: All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
computed tomography: An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
follow up: The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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