RecruitingRare disease

Study on Inhaled Molgramostim for Children with Autoimmune Pulmonary Alveolar Proteinosis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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.
Step 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.
Step 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.
Step 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.
Step 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.
Step 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.
Step 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 trial?

4 criteria

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 trial?

8 criteria

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.

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Ludwig Maximilian University Of Munich
Responsive
Munich, Germany

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Investigated drugs

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.

Molgramostim

What is already known about the treatment

Molgramostim – Molgramostim is administered as an inhaled medication, meaning it is breathed directly into the lungs. It is currently being studied in clinical trials for its effectiveness and safety in treating autoimmune pulmonary alveolar proteinosis (aPAP) in children aged 6 to under 18 years. This medication is primarily used to help clear the lungs of excess proteins that accumulate in this rare lung condition. At the molecular level, molgramostim works by stimulating certain cells in the immune system to help break down and remove these proteins. It is classified as a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), which is a type of protein that encourages the growth and function of white blood cells.

Investigated diseases

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.