Active not recruitingPHASE3INTERVENTIONAL

Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

This study is testing a new inhaled medication called molgramostim for people with a rare lung disease called autoimmune pulmonary alveolar proteinosis (aPAP). The main goal is to find out if this medicine, given as a daily inhaler, is safe and effective in improving lung health compared to a dummy treatment (placebo). 160 adults with aPAP will join. For the first 48 weeks, half will get the study drug and half will get the placebo. After this, everyone will receive the active molgramostim for another 96 weeks. This helps us understand the long-term effects. We hope this research leads to a better treatment option for aPAP.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Savara Inc.

Enrolment target

164

Start

19 May 2021

Estimated completion

30 May 2027

Autoimmune Pulmonary Alveolar Proteinosis

What is this study about?

Autoimmune pulmonary alveolar proteinosis, or aPAP, is a rare lung condition where a substance similar to fat and protein builds up in the air sacs of your lungs. This buildup makes it hard for your lungs to work properly, leading to symptoms like shortness of breath and coughing. Currently, treatments can be invasive, like washing out the lungs. This study is exploring a new approach using an inhaled medicine called molgramostim.

Molgramostim is a type of protein that naturally helps stimulate the immune system and support lung function. In this trial, it's given as an inhaled mist that you breathe in. We want to see if this inhaled medicine can help clear the protein buildup in the lungs and improve how people with aPAP feel and breathe. This is a "Phase 3" study, which means it's one of the final steps before a new treatment might be approved for wider use.

The study is important because it could offer a less invasive and more convenient treatment option for people living with aPAP. By comparing molgramostim to a placebo (a treatment that looks the same but has no active medicine), we can carefully assess if the changes observed are truly due to the new drug. Learning more about this medicine could potentially improve the lives of many people with this challenging condition.

Key takeaways

Tests a new inhaled medicine (molgramostim) for aPAP.
Compares molgramostim to a placebo first, then all participants get the active drug.
Aims to reduce lung buildup and improve breathing.
Whole lung lavage is allowed if condition worsens.
Study lasts up to 3 years with regular monitoring.

Who may be eligible?

To join this study, you need to be an adult (18 years or older, or 20 in Japan) and have a confirmed diagnosis of autoimmune pulmonary alveolar proteinosis (aPAP). This diagnosis needs to be backed up by a special blood test that checks for specific antibodies, as well as evidence from lung scans (like a CT scan), a lung biopsy, or a lung fluid test.

Your lung function will also be assessed. Specifically, your ability to take oxygen from the air into your blood (measured by something called DLCO) must be 70% or less at the start of the study. You also need to show some difficulty with exercise, meaning you can't manage very strenuous activity. You should be able to stop using extra oxygen for short periods during some tests.

Both men and women can participate. If you're a woman who can still have children, or if you're a male participant, you'll need to use reliable birth control methods during the study and for 30 days afterwards. This is to ensure the safety of any potential pregnancies, as the effects of the study drug on unborn babies are not fully known.

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 18 years or older?
Do you have a confirmed diagnosis of autoimmune pulmonary alveolar proteinosis (aPAP)?
Is your specific lung function test (DLCO) 70% or less?
Can you temporarily stop using supplemental oxygen for some tests?
Are you willing to use reliable birth control during the study if needed?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last a maximum of 156 weeks (about three years). It starts with a screening period of about 6 weeks to check if you're suitable. After that, you'll enter the first treatment phase, which lasts 48 weeks. During this time, you'll be randomly assigned to either receive the inhaled molgramostim or a placebo (dummy treatment) once daily. Neither you nor your study doctor will know which one you're getting, ensuring the results are fair.

After these 48 weeks, everyone will receive the active inhaled molgramostim once daily for a longer period of 96 weeks. Throughout the study, you'll have regular visits to the clinic for check-ups, lung function tests, and to make sure you're doing well. If your aPAP gets much worse during the study, your doctor can still give you a standard treatment called whole lung lavage.

Once your treatment phases are complete, there will be a final safety check-up about 4 weeks later. The medicines are taken by inhalation, meaning you'll breathe them in using a special device. You'll be carefully monitored for any side effects throughout your time in the study.

Potential risks and benefits

Participating in this study might offer several potential benefits. You would receive a new treatment (molgramostim) that could potentially improve your lung function and aPAP symptoms, and you'd have close medical monitoring. However, there are no guarantees that you will personally benefit. Potential risks include side effects from the study medication, which will be monitored closely. Some people might experience discomfort or side effects from the tests and procedures involved. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (54)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University Of Arkansas For Medical Services
Verified postcode
Little Rock, United States
UCLA David Geffen School of Medicine
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Los Angeles, United States
National Jewish Health
Verified postcode
Denver, United States
Yale University
Verified postcode
New Haven, United States
University of Florida Health
Verified postcode
Gainesville, United States
Emory University
Verified postcode
Atlanta, United States
Loyola University
Verified postcode
Maywood, United States
University of Maryland Medical Center
Verified postcode
Baltimore, United States
Washington University in St. Louis
Verified postcode
St Louis, United States
Duke University Medical Center
Verified postcode
Durham, United States
Wake Med Health & Hospital
Verified postcode
Raleigh, United States
Cincinnati Children's Hospital Medical Center
Verified postcode
Cincinnati, United States

Common questions

What is autoimmune pulmonary alveolar proteinosis (aPAP)?

It's a rare lung condition where a fatty-protein substance builds up in your lungs, making it hard to breathe.

What is molgramostim?

It's an inhaled medicine being tested to see if it can help clear the buildup in the lungs for people with aPAP.

Will I definitely get the new medicine?

For the first 48 weeks, you have a 50/50 chance of getting molgramostim or a dummy treatment. After that, everyone gets molgramostim.

How long will the study last if I join?

The maximum time you'd be involved in the study, including screening and follow-up, is about three years (156 weeks).

Could I still have standard treatment if my condition worsens?

Yes, if your aPAP gets worse during the study, you can still receive whole lung lavage, which is a standard treatment.