Not yet recruitingRare disease

Immunomodulatory Treatment with Prednisone, Azithromycin and Hydroxychloroquine in Patients with Surfactant‑Related Gene Mutation Interstitial Lung Disease

Verified siteRegistered drugNo placebo

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What is this trial about?

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

The study focuses on Interstitial Lung Disease that occurs because of a change in a surfactant-related gene. The treatment being tested combines three medicines taken by mouth: prednisone (a steroid that reduces inflammation), azithromycin (an antibiotic that can also affect the immune system), and hydroxychloroquine (a drug that modifies immune activity).

The purpose of the study is to determine whether this triple therapy can slow the loss of lung function over a year. Participants will receive the medication regimen for 12 months and will return for check‑ups roughly every three months. During these visits, lung capacity will be measured with a breathing test (PFT), the chest will be imaged using a CT scan, and heart rhythm will be recorded with an ECG. An eye exam will also be performed once during the year.

Safety will be closely monitored, and any side effects will be recorded. Participants will also complete a questionnaire about overall well‑being at several points during the study, and information about hospital visits, lung transplants, and disease flare‑ups will be collected throughout the follow‑up period.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Baseline assessment
After joining the study you will go to the clinic for the first assessment. this includes a lung function test that measures forced vital capacity (how much air you can exhale), a chest ct scan (detailed picture of the lungs), and a set of questions about your health and quality of life. you will also be asked about any current medicines and medical history.
Step 2
Start medication
You will begin taking three oral medicines every day for a total of twelve months:
prednisone 10 mg once daily – a steroid that reduces inflammation in the lungs.
azithromycin 250 mg once daily – an antibiotic that may help modify the immune response.
hydroxychloroquine 400 mg once daily – a drug that can affect the immune system.
The study staff will give you instructions on how to take each pill and will provide a calendar to track your doses.
Step 3
3‑month visit
Three months after starting the medicines you will return to the clinic.
You will complete a quality of life questionnaire that asks about symptoms, daily activities, and overall well‑being.
An ecg (electrocardiogram – a simple test that records the electrical activity of the heart) will be performed because hydroxychloroquine and azithromycin can affect the heart rhythm.
The staff will ask you to report any side effects such as stomach upset, skin changes, or vision problems.
Step 4
6‑month visit
Six months after enrollment you will have another clinic visit.
A repeat lung function test will be done to measure forced vital capacity again.
A follow‑up chest ct scan will be performed to look at changes in the lungs.
You will fill out the same quality of life questionnaire as before.
Another ecg will be recorded to monitor heart rhythm.
Any new or worsening side effects should be reported to the study team.
Step 5
9‑month visit
At nine months you will attend a routine visit.
You will again answer the quality of life questionnaire and undergo an ecg to check heart rhythm.
The staff will review your medication adherence and ask about any adverse symptoms.
Step 6
12‑month visit
One year after starting treatment you will complete the final study visit.
The visit includes a lung function test measuring forced vital capacity, a chest ct scan, and the quality of life questionnaire.
An ecg will be performed, and an ophthalmological exam (eye exam) will be done to detect any eye changes that can be caused by hydroxychloroquine.
The study team will collect information about any hospitalizations, lung transplants, or disease worsening that occurred during the year.

Who can join the trial?

3 criteria

Age 18 years or older but younger than 80 years.
Must have a genetic change (called a variant) that experts have judged to be disease‑causing (pathogenic) or probably disease‑causing, in one of these genes: SFTPA1, SFTPA2, SFTPC, NKX2‑1, or SFTPB; or must have two such variants in the ABCA3 or SFTPB genes. This judgment is made by a team of genetic specialists (a genetic multidisciplinary discussion).
Must have interstitial lung disease (a condition where the tissue around the air sacs of the lungs becomes scarred) that involves more than 10 % of the total lung volume on a CT scan (a detailed X‑ray picture) taken within the past 2 years.

Who cannot join the trial?

5 criteria

Contraindication to azithromycin, hydroxychloroquine or prednisone – this means you have a medical reason that makes it unsafe for you to take any of these medicines (azithromycin is an antibiotic, hydroxychloroquine affects the immune system, and prednisone is a steroid that reduces inflammation).
Pregnancy or breastfeeding – if you are currently pregnant or feeding a baby, you cannot join because the study drugs might affect the baby.
Participation in another interventional study (a clinical trial that tests a medicinal product, medical device, or any other experimental treatment involving people) – you may only be in one study at a time.
Being deprived of liberty (for example, in prison) or under a legal protection measure (such as a court‑ordered guardianship) – these situations prevent participation.
Lack of any health insurance system affiliation – you must have health insurance coverage to be eligible.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Assistance Publique Hopitaux De Paris
Verified
Paris, France
Centre Hospitalier Universitaire De Lille
Verified
Lille, France
Centre Hospitalier Universitaire De Rennes
Verified
Rennes, France

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Investigated drugs

Prednisone
is a medicine taken by mouth that reduces inflammation and suppresses the immune system. In this study it is used to calm the over‑active immune response that can damage the lungs in people with interstitial lung disease linked to surfactant‑related gene changes.
Azithromycin
is an oral antibiotic that also has effects on the immune system. In the trial it is given to help control any hidden infections and to provide additional anti‑inflammatory benefits that may improve lung function.
Hydroxychloroquine
is a pill that was originally used for malaria but is now known to modify the immune system. In this research it is added to the treatment plan to further reduce abnormal immune activity that contributes to lung scarring.

What is already known about the treatment

Prednisolone
Prednisolone is taken by mouth as a tablet and is an approved medication widely used in clinical practice. It is mainly prescribed to treat inflammation, asthma, and autoimmune disorders. It works by attaching to steroid receptors inside cells, which lowers the production of chemicals that cause swelling and pain. It belongs to the corticosteroid class of drugs.
Prednisone
Prednisone is also taken orally as a tablet and is an approved drug commonly used for inflammatory and immune‑related conditions. After swallowing, the body changes it into the active form prednisolone, which then reduces swelling and discomfort. Its action is similar to other steroids, binding to cellular receptors to decrease inflammatory signals. It is classified as a corticosteroid.
Azithromycin
Azithromycin is taken by mouth in tablet form and is an approved antibiotic used for many lung and throat infections. It is also sometimes used for its mild anti‑inflammatory effects in chronic lung diseases. The medicine stops bacteria from making essential proteins, which stops them from growing, and it can calm down inflammation in the airways. It belongs to the macrolide antibiotic group.
Hydroxychloroquine
Hydroxychloroquine is taken orally as a tablet and is an approved drug for malaria prevention as well as for lupus and rheumatoid arthritis. It is being studied for its ability to calm an overactive immune system in lung disease. The drug interferes with cell signaling pathways that trigger immune responses, thereby reducing inflammation. It is classified as an antimalarial and immune‑modulating agent.

Investigated diseases

Interstitial Lung Disease - Interstitial Lung Disease is a group of disorders that cause scarring and inflammation in the tissue surrounding the tiny air sacs of the lungs. The scarring makes the lung tissue stiff, reducing its ability to expand and absorb oxygen. Over time, the disease can cause a gradual decline in breathing capacity and may lead to persistent cough and shortness of breath. In cases linked to a surfactant‑related gene variant, the abnormal surfactant can trigger early inflammation and faster accumulation of scar tissue. The condition typically progresses slowly but can accelerate, leading to worsening lung function over months to years.