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Could a simple amino acid ease a devastating lung condition?

NCT ID NCT03887169

Disease control 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested whether giving methionine (an amino acid) by mouth or feeding tube is safe and tolerable for children with a severe, inherited lung disease called pulmonary alveolar proteinosis. The disease is caused by a faulty MARS gene that prevents cells from using methionine properly. Only 3 children took part, and the goal was to see if the supplement could improve breathing and reduce the need for lung washes.

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.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

3 people

The number who actually took part.

Started

Sep 2019

Finished

Jun 2020

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.

Ages

Up to 18 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: * Minor Patient with alveolar proteinosis by double mutation Ala393Thr and SER567LEU of the MARS gene, genetically proven. * Patient in need of prolonged hospitalization in Necker for treatment of bronchial-alveolar washes in the context of care. * Patient for which methionine can be administered orally or by enteral probe (Nasogastric or gastrostomy probe) * Signed Informed consent form by parents / legal guardian Exclusion Criteria: * Patient with alveolar proteinosis by other mutations of the MARS gene * Patient with alveolar proteinosis secondary to another etiology or without identified cause * Refusal to participate in the study * High blood pressure requiring drug treatment * Heart failure * Known hypersensitivity to one of the substances used or potentially used in the study: methionine, vitamins B6, B12, B9 and C * Pre-Hypermethioninemia (Methioninemia \> + 2 DS of normal for age) whatever the cause

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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.

Mutation ALA393THR of the mars gene MUTATIONSER567LEU of the mars gene Pulmonary alveolar proteinosis

Contacts and locations

Locations

  • Hôpital Necker-Enfants Malades

    Paris, Île-de-France Region, 75015, France

More trials for these conditions

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