Study of Inhaled Molgramostim for Adults 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 this condition, the lungs fill with a protein-rich material, making it difficult to breathe. The study is testing a treatment called Molgradex, which is a solution for inhalation containing the active substance molgramostim. This treatment is compared to a placebo, which looks like the treatment but does not contain the active substance.

The purpose of the study is to investigate how effective Molgradex is in treating aPAP compared to the placebo. Participants in the study will inhale the solution once daily. The study will last for several weeks, during which participants will have regular check-ups to monitor their health and the effects of the treatment. The study aims to see if there is an improvement in lung function and overall well-being of the participants.

Throughout the study, participants will undergo various assessments to track changes in their condition. These assessments will help determine if Molgradex can improve breathing and quality of life for those with aPAP. The study is designed to provide valuable information on the potential benefits of this treatment for people living with this rare lung disease.

1 beginning of the trial

Upon joining the clinical trial, participants will receive detailed information about the study and will be asked to sign an informed consent form. This form confirms understanding of the trial’s requirements and agreement to participate.

Participants must be at least 18 years old, or 20 years old if in Japan, and must have a confirmed diagnosis of autoimmune pulmonary alveolar proteinosis (aPAP).

2 baseline assessments

Participants will undergo initial assessments to establish baseline health data. This includes a test to measure lung function, known as DLCO, which evaluates how well the lungs transfer gas from inhaled air to the bloodstream.

Additional tests may include a treadmill exercise test and blood sampling, during which participants must refrain from using supplemental oxygen.

3 treatment phase

Participants will be randomly assigned to receive either the molgramostim nebulizer solution or a placebo. The treatment is administered once daily through inhalation.

The duration of the treatment phase is up to 48 weeks, with regular monitoring and assessments to track progress and any changes in health status.

4 follow-up assessments

Throughout the trial, participants will have scheduled visits to assess changes in lung function and overall health. These visits will occur at specified intervals, such as at 24 weeks and 48 weeks.

Participants will complete questionnaires to evaluate their quality of life and activity levels, using tools like the SGRQ (St. George’s Respiratory Questionnaire).

5 end of trial

At the conclusion of the trial, participants will undergo final assessments to determine the overall impact of the treatment.

Participants will be informed about the results of the study and any potential next steps regarding their health care.

Who Can Join the Study?

Must be 18 years or older to participate. In Japan, must be 20 years or older.
Must be able to provide written consent to participate in the study.
Must be willing and able to attend scheduled visits, follow the treatment plan, and complete tests and procedures as required by the study.
Must have a positive test result for anti-GM-CSF autoantibodies, which confirms autoimmune PAP.
Must have a history of PAP confirmed by a lung biopsy, bronchoalveolar lavage (BAL) cytology, or a high-resolution CT scan of the chest.
Must have a DLCO (a lung function test) result of 70% predicted or lower at the first screening and baseline visits.
Must have a change in % predicted DLCO of less than 15% points during the screening period.
Must be willing and able to stop using supplemental oxygen before and during certain tests, such as the treadmill exercise test, DLCO assessment, and blood gas sampling.
Must have a resting SpO2 (oxygen saturation) of more than 85% for 15 minutes without supplemental oxygen at the screening visits.
Can be male or female.
Must agree to use contraception if of childbearing potential, consistent with local regulations. This includes:
- Men: Agree to use condoms during and until 30 days after the last dose of trial treatment, or have a female partner using adequate contraception.
- Women: Must be post-menopausal for more than 1 year, or if of childbearing potential, must use a highly effective method of contraception during and until 30 days after the last dose of trial treatment. Must have a negative pregnancy test at screening and baseline and must not be breastfeeding.

Who Cannot Join the Study?

Patients with any other autoimmune disease cannot participate. An autoimmune disease is when the body’s defense system attacks its own cells.
Patients who have had a lung transplant are not eligible. A lung transplant is a surgery to replace a diseased lung with a healthy one from a donor.
Patients with a history of cancer in the last 5 years cannot join. Cancer is a disease where cells in the body grow uncontrollably.
Patients who are currently pregnant or breastfeeding are not allowed to participate.
Patients who have participated in another clinical trial within the last 30 days are excluded.
Patients with a known allergy to the study medication or its ingredients cannot take part.
Patients with severe heart disease are not eligible. Heart disease refers to various conditions that affect the heart’s function.
Patients with uncontrolled high blood pressure are excluded. High blood pressure is when the force of the blood against the artery walls is too high.
Patients with severe liver or kidney disease cannot participate. The liver and kidneys are organs that help filter waste from the body.
Patients with a history of substance abuse in the last 2 years are not eligible. Substance abuse refers to the harmful use of substances like drugs or alcohol.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Bellvitge University Hospital	L'hospitalet De Llobregat	Spain
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium

Other Sites

Site Name	City	Country
Asklepios Klinik Gauting GmbH	Gauting	Germany
Ruhrlandklinik Westdeutsches Lungenzentrum Am Universitaetsklinikum Essen gGmbH	Essen	Germany
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Institutul De Pneumoftiziologie Marius Nasta	Bucharest	Romania
Centre Hospitalier Universitaire De Rennes	Rennes	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Nrmbqxad Iptkfyehx Or Twzjarppsksz Ape Lbvn Dkjwzgae	Warsaw	Poland
Sj Vvfwyzykqvaiqzj Ujcvrkayys Hrxfvolo	Dublin	Ireland

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	19.05.2021
France	Not recruiting	19.05.2021
Germany	Not recruiting	19.05.2021
Ireland	Not recruiting	19.05.2021
Italy	Not recruiting	19.05.2021
Poland	Not recruiting	19.05.2021
Portugal	Not recruiting	19.05.2021
Romania	Not recruiting	19.05.2021
Spain	Not recruiting	19.05.2021

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). This condition affects the lungs, making it hard for them to work properly. Molgramostim is inhaled through a nebulizer, which turns the medication into a mist that you can breathe in. The goal of using molgramostim in this trial is to see if it can improve lung function and help people with aPAP breathe better.

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 accumulation occurs due to an autoimmune process where the body mistakenly attacks a protein called GM-CSF, which is crucial for clearing the surfactant from the alveoli. As the disease progresses, the buildup of this material can impair the lungs’ ability to exchange gases, leading to breathing difficulties. Patients may experience symptoms such as shortness of breath, cough, and fatigue. Over time, the disease can lead to reduced lung function and decreased exercise capacity. The progression of the disease can vary, with some individuals experiencing a slow worsening of symptoms.

Trial ID:

2024-511052-41-00

Protocol code:

SAV00605

NCT ID:

NCT04544293

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of Inhaled Molgramostim for Adults with Autoimmune Pulmonary Alveolar Proteinosis

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