RecruitingPHASE3INTERVENTIONAL

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

This study is investigating an inhaled medicine called molgramostim for children and teenagers aged 6 to 18 who have a rare lung condition called autoimmune pulmonary alveolar proteinosis, or aPAP. aPAP causes a build-up of material in the lungs, making it hard to breathe. The main goals are to find out if molgramostim can help improve breathing and activity levels, and if it is safe for young people with aPAP. All participants in this study will receive molgramostim, which they will take using a nebulizer at home every day for about a year. They will also have regular check-ups at a clinic.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Savara Inc.

Enrolment target

Start

22 Oct 2025

Estimated completion

01 Dec 2027

Autoimmune Pulmonary Alveolar Proteinosis

What is this study about?

This study is exploring a new treatment option for a rare lung condition called Autoimmune Pulmonary Alveolar Proteinosis (aPAP) in children and teenagers aged 6 to 18. aPAP happens when tiny air sacs in the lungs get blocked with a fatty, protein-rich substance, making it difficult to breathe properly and get enough oxygen.

The medicine being tested is called molgramostim, and it's given as an inhaled treatment. The researchers want to understand if this medicine can make a difference in how well children with aPAP can breathe and how active they can be in their daily lives. They also want to make sure the treatment is safe for this age group.

Since aPAP is a rare condition, finding effective and safe treatments is very important. This study hopes to provide valuable information that could help doctors better manage aPAP in young patients in the future.

Key takeaways

This study is testing an inhaled medicine (molgramostim) for children and teens with aPAP.
It aims to see if the medicine improves breathing and is safe.
Participants take the medicine daily for a year and have regular check-ups.
The total study time is about 56 weeks.
Regular medical support will be provided, and extra treatment for worsening symptoms is allowed.

Who may be eligible?

To be considered for this study, individuals must be between 6 and 18 years old. They need to have a confirmed diagnosis of autoimmune pulmonary alveolar proteinosis (aPAP). This diagnosis would usually be based on tests like lung biopsies, fluid samples from the lungs, or special CT scans of the chest, along with a positive blood test for specific antibodies.

Also, their lung function, specifically how well their lungs can transfer oxygen, needs to be below a certain level. This helps ensure the study is focused on those who might benefit most from a new treatment.

However, some conditions would mean someone couldn't join. For example, if they have a different type of lung proteinosis (not autoimmune), or if they’ve recently had a significant lung wash procedure (called lung lavage) within the last month.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 6 and 18 years old?
Has a doctor confirmed you have autoimmune pulmonary alveolar proteinosis (aPAP)?
Did a blood test show positive for anti-GM-CSF antibodies?
Has your lung function (DLCO) been measured as 70% or less?
Have you *not* had a full lung wash (WLL) in the last month?
Do you *not* have another type of lung proteinosis (not autoimmune)?

Answer every question to see your result.

What does participation involve?

If you join this study, you would take molgramostim every day for 12 months using a nebulizer, which turns liquid medicine into a fine mist you can breathe in. You would visit the clinic about every 12 weeks for check-ups and tests to see how you are doing. You would also be asked to keep a simple diary noting if and when you use any oxygen.

The entire study period, including initial screening, the treatment phase, and a follow-up after treatment, will last for a maximum of 56 weeks (just over a year). During the study, if your aPAP gets much worse, doctors can still perform a lung wash procedure as a 'rescue' treatment to help you.

Potential risks and benefits

Taking part in this study could potentially offer some benefits if molgramostim helps to improve your breathing and activity levels. However, as with any medicine, there might be side effects or risks that the study team will discuss fully with you. There's no guarantee that the treatment will work. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik
Verified postcode
München, Germany· Recruiting

Common questions

What is autoimmune pulmonary alveolar proteinosis (aPAP)?

It's a rare lung condition where a fatty substance builds up in the air sacs of your lungs, making it hard to breathe.

What is molgramostim?

It's an experimental medicine given by an inhaler (nebulizer) that the study is testing for aPAP.

How long will I take the medicine?

You will take molgramostim once a day for about 12 months (48 weeks).

Will I have to go to the hospital often?

You'll have clinic visits about every 12 weeks for check-ups and tests, which is roughly once every three months.

What if my aPAP gets worse during the study?

Doctors can perform a lung wash procedure if needed to help with worsening symptoms, even while you're in the study.

How to find out more

Yasmine Wasfi, MD, PhD,

yasmine.wasfi@savarapharma.com 1 512 851 1364 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.