Study on Inhaled Molgramostim for Children with Autoimmune Pulmonary Alveolar Proteinosis

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What is this study about?

This clinical trial is focused on studying a rare lung condition called Autoimmune Pulmonary Alveolar Proteinosis (aPAP) in children and teenagers. This condition affects the lungs by causing a build-up of proteins and fats, making it difficult to breathe. The study will use a treatment called Molgradex, which is a solution for inhalation containing the active substance molgramostim. This medication is designed to help clear the build-up in the lungs and improve breathing.

The purpose of the study is to evaluate how effective and safe inhaled Molgradex is for young participants with aPAP. Participants in the study will inhale the medication over a period of time, and their progress will be monitored. The study will look at changes in lung function and overall quality of life over 24 and 48 weeks. Participants will have regular check-ups to assess their lung health and any side effects they might experience.

Throughout the study, various health measurements will be taken, such as oxygen levels in the blood and the distance participants can walk in six minutes. The study will also monitor the presence of specific antibodies related to aPAP and any potential side effects, including hypersensitivity and chest pain. The goal is to gather information that could lead to better treatment options for children and teenagers with this rare lung condition.

1 joining the study

Upon joining the study, participants will be informed about the purpose and procedures of the trial. Participants must be between 6 and less than 18 years old and have a confirmed diagnosis of autoimmune pulmonary alveolar proteinosis (aPAP).

Participants will undergo initial assessments, including a lung function test and a blood test to confirm eligibility.

2 treatment initiation

Participants will begin treatment with Molgradex, a solution for inhalation. This medication is administered through inhalation, which means breathing it in through the mouth using a special device.

The treatment will be administered regularly as per the study protocol, and participants will be instructed on how to use the inhalation device properly.

3 ongoing assessments

Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment. These check-ups will include lung function tests and assessments of oxygen levels in the blood.

Participants will also be asked to complete questionnaires about their quality of life and any symptoms they experience.

4 mid-study evaluation

At approximately 24 weeks into the study, participants will undergo a detailed evaluation to assess changes in lung function and overall health.

This evaluation will help determine the effectiveness of the treatment at this stage.

5 continuation of treatment

Participants will continue the inhalation treatment with Molgradex for a total of 48 weeks, with ongoing monitoring and assessments.

Regular follow-up visits will ensure that any side effects or concerns are addressed promptly.

6 final evaluation

At the end of the 48-week treatment period, participants will have a final evaluation to assess the overall impact of the treatment on their condition.

This will include a comprehensive review of lung function, oxygen levels, and quality of life.

7 post-study follow-up

After completing the study, participants may be asked to attend follow-up visits to monitor their health and gather additional data for the study.

These visits are important to ensure the long-term safety and effectiveness of the treatment.

Who Can Join the Study?

Be between 6 years and less than 18 years old at the time of signing the consent forms.
Have a history of pulmonary alveolar proteinosis, which is a lung condition, confirmed by a lung biopsy, a special lung wash test called bronchoalveolar lavage, or a detailed chest scan known as a high-resolution computed tomography (CT) scan.
Have a positive result on a blood test for anti-GM-CSF autoantibodies, which are specific proteins in the blood that confirm the presence of the condition, before the screening process.
Have a lung function test result, called hemoglobin-adjusted diffusing capacity of the lung for carbon monoxide (DLCO), that is 70% or less of what is predicted for healthy lungs at the screening.

Who Cannot Join the Study?

Participants who have any other serious health conditions that could interfere with the study.
Participants who are currently taking any medications that might affect the study results.
Participants who have had a major surgery within the last 3 months.
Participants who are pregnant or breastfeeding.
Participants who have a history of drug or alcohol abuse.
Participants who have participated in another clinical trial within the last 30 days.
Participants who have a known allergy to the study medication or its ingredients.
Participants who are unable to follow the study procedures or attend the required visits.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Ludwig Maximilian University Of Munich	Munich	Germany

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	01.06.2025

Trial locations

Investigated drugs:

Molgramostim

Molgramostim is a medication used in this clinical trial to help treat a condition called autoimmune pulmonary alveolar proteinosis (aPAP) in children. This condition affects the lungs and makes it hard for them to work properly. Molgramostim is inhaled, which means it is breathed in through the mouth so it can go directly to the lungs. The goal of using this medication is to improve lung function and help the children breathe better by reducing the symptoms of aPAP. The trial is focused on understanding how effective and safe this treatment is for children aged 6 to less than 18 years old.

Autoimmune Pulmonary Alveolar Proteinosis – Autoimmune Pulmonary Alveolar Proteinosis is a rare lung disease characterized by the accumulation of a protein-rich substance in the alveoli, the tiny air sacs in the lungs. This buildup occurs due to an immune system malfunction that affects the clearance of surfactant, a substance that helps keep the alveoli open. As the disease progresses, the accumulation of material in the alveoli can lead to impaired gas exchange, causing difficulty in breathing and reduced oxygen levels in the blood. Over time, individuals may experience symptoms such as shortness of breath, fatigue, and a persistent cough. The condition can vary in severity, with some individuals experiencing mild symptoms and others having more significant respiratory issues. The progression of the disease can be slow, and symptoms may fluctuate over time.

Trial ID:

2024-512039-66-00

Protocol code:

SAV006-04

NCT ID:

NCT06431776

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Inhaled Molgramostim for Children with Autoimmune Pulmonary Alveolar Proteinosis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?