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Scientists investigate why rare lung disease develops in children

NCT ID NCT04811274

Knowledge-focused Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at 20 children with a rare lung condition called pulmonary alveolar proteinosis (PAP) caused by changes in the MARS gene. Researchers wanted to understand if the lung cleaning cells (macrophages) are not working properly. They grew these cells in a lab from blood samples and tested how well they cleaned up fatty material. This was a research-only study, not a treatment trial.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

20 people

The number who actually took part.

Started

Jun 2021

Finished

Nov 2021

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Minor patients with alveolar proteinosis by mutations of the MARS gene and minor patients without alveolar proteinosis, hospitalized for their care at Necker Enfants Malades hospital and for whom a blood sample and a bronchoscopy with bronchoalveolar lavage must be performed for their care.

Ages

Up to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Minors from 0 to 17 years hospitalized for their care at Necker Enfants Malades hospital and for whom a blood sample and a bronchoscopy with bronchoalveolar lavage must be performed as part of their care * Information and consent of the holders of parental authority and the patient Exclusion Criteria: \- Refusal of holders of parental authority or patient

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Conditions

The condition(s) this trial relates to.

pulmonary alveolar proteinosis severe early-onset pulmonary alveolar proteinosis due to MARS deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Genetic mutation MARS Pulmonary alveolar proteinosis (PAP)

Contacts and locations

Locations

  • Hôpital Necker-Enfants Malades

    Paris, 75015, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.