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New hope for kids with rare lung condition: inhaled protein therapy in trial

NCT ID NCT06431776

Disease control Sponsor: Savara Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests an inhaled drug called molgramostim in children aged 6 to 18 with autoimmune pulmonary alveolar proteinosis (aPAP), a rare disease where protein builds up in the lungs and makes breathing hard. All 5 participants will take the drug daily via a nebulizer for 12 months. Researchers will check lung function, exercise ability, and quality of life to see if the treatment helps and is safe.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
molgramostim (a lab-made protein that helps immune cells clear protein buildup in the lungs)
What this could lead to
If it works, this could point toward a treatment for children with a rare lung disease that currently has limited options.
What could go wrong
This is a very small, early-phase trial with only 5 children, so results may not apply to everyone. The treatment requires daily nebulizer use for a year, and side effects are still being studied.
Why investors are watching

Savara Inc. is running a Phase 3 trial of inhaled molgramostim in 5 pediatric patients with autoimmune pulmonary alveolar proteinosis (aPAP). For a small company, this readout matters because it tests whether the drug improves breathing and activity in children, a group not covered by existing data, and a positive result could expand the drug's approved use.

If it works: A positive result could support regulatory approval for pediatric aPAP, broadening the drug's market beyond adults. That would give Savara a new patient population and a stronger commercial story.

If it fails: The trial has only 5 participants, so results may be hard to interpret, and the drug could fail to show benefit or cause safety problems. Trials often fail, and a negative or delayed readout could hurt the company's prospects.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 5 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

autoimmune pulmonary alveolar proteinosis pulmonary alveolar proteinosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Autoimmune pulmonary alveolar proteinosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik

    RECRUITING

    München, 80337, Germany

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