In short
Currently, there are 2 clinical trials investigating treatments for Alveolar Proteinosis, specifically focusing on the autoimmune form of this rare lung condition. Both trials are testing an inhaled medication called molgramostim (Molgradex) to help clear protein buildup in the lungs and improve breathing. One trial focuses on children and teenagers, while the other involves adult patients across multiple European countries.
Clinical trial locations
- Belgium
- France
- Germany
- Ireland
- Italy
- Poland
- Portugal
- Romania
- Spain
Study on Inhaled Molgramostim for Children with Autoimmune Pulmonary Alveolar Proteinosis
This trial is studying how well an inhaled treatment called Molgradex works for children and teenagers with autoimmune pulmonary alveolar proteinosis. This rare lung condition causes a buildup of proteins and fats in the lungs, making breathing difficult.
Main inclusion criteria: Children and teenagers between 6 and 17 years old can participate if they have a confirmed diagnosis of autoimmune pulmonary alveolar proteinosis through a lung biopsy, special lung wash test, or detailed chest scan. They must also have a positive blood test showing anti-GM-CSF autoantibodies and a lung function test result showing their lungs are working at 70% or less of what would be expected for healthy lungs.
Main exclusion criteria: Participants cannot join if they have other serious health conditions that could interfere with the study, are taking medications that might affect results, have had major surgery in the last 3 months, are pregnant or breastfeeding, have a history of drug or alcohol abuse, participated in another clinical trial within 30 days, have allergies to the study medication, or cannot follow study procedures and attend required visits.
Focus of the trial: The study aims to evaluate how effective and safe inhaled Molgradex is for young people with this condition. Participants will inhale the medication regularly over 48 weeks while attending check-ups to monitor lung function, oxygen levels, and quality of life. Researchers will measure changes at 24 and 48 weeks, including oxygen levels in the blood, walking distance in six minutes, and monitoring for side effects such as hypersensitivity and chest pain.
Investigational drug: The trial uses molgramostim, delivered through an inhaled solution. This medication is a type of protein called granulocyte-macrophage colony-stimulating factor that helps the immune system break down and remove excess proteins that accumulate in the lungs. It is breathed directly into the lungs using a special inhalation device.
Study of Inhaled Molgramostim for Adults with Autoimmune Pulmonary Alveolar Proteinosis
This clinical trial is testing whether Molgradex, an inhaled treatment, can help adults with autoimmune pulmonary alveolar proteinosis. In this condition, the lungs fill with protein-rich material that makes breathing difficult. The treatment is compared against a placebo, which looks identical but contains no active medication.
Main inclusion criteria: Adults aged 18 or older can participate if they have a confirmed diagnosis of autoimmune pulmonary alveolar proteinosis and a positive blood test for anti-GM-CSF autoantibodies. Their lung function test must show 70% or less of predicted capacity, and they must be able to maintain oxygen levels above 85% for 15 minutes without supplemental oxygen. Participants must agree to use proper contraception if of childbearing potential, and women must have a negative pregnancy test and not be breastfeeding.
Main exclusion criteria: People cannot join if they have other autoimmune diseases, have had a lung transplant, had cancer in the last 5 years, are pregnant or breastfeeding, participated in another trial within 30 days, have allergies to the study medication, have severe heart disease, uncontrolled high blood pressure, severe liver or kidney disease, or have a history of substance abuse in the last 2 years.
Focus of the trial: The study investigates whether Molgradex can improve lung function and overall well-being compared to placebo. Participants inhale the solution once daily for up to 48 weeks, with regular assessments to track changes in breathing ability and quality of life. Follow-up visits occur at specified intervals, including at 24 and 48 weeks, where participants complete questionnaires and undergo lung function tests.
Investigational drug: The trial uses molgramostim, administered through a nebulizer that turns the medication into a breathable mist. This granulocyte-macrophage colony-stimulating factor stimulates certain white blood cells to help clear protein buildup from the lungs, supporting the immune system's function.
Summary
Both ongoing clinical trials focus on testing the same medication, molgramostim, for treating autoimmune pulmonary alveolar proteinosis in different age groups. The pediatric trial is conducted in Germany, while the adult trial spans multiple European countries including Spain, Portugal, Belgium, France, Italy, Germany, Ireland, Romania, and Poland. This geographic spread in the adult trial suggests a broader effort to gather diverse patient data across Europe.
Both trials use similar approaches, evaluating the inhaled treatment over extended periods of up to 48 weeks, with key assessment points at 24 and 48 weeks. The concentration of research on molgramostim as the primary investigational drug indicates this treatment pathway is a significant focus for this rare lung condition. The trials aim to provide important data on both safety and effectiveness for patients who currently have limited treatment options.
