Could a malaria drug help kids with rare lung disease?

NCT ID NCT03822780

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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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 whether hydroxychloroquine, a medicine used for malaria and autoimmune conditions, can help children with a rare genetic lung disease that affects the lungs' natural cleaning fluid. 25 children took the drug, and researchers checked if their need for extra oxygen improved over 6 months. The goal was to see if the drug could control the disease and reduce breathing problems.

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Study facts

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

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

25 people

The number who actually took part.

Started

Jul 2017

Finished

Mar 2026

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

1 month 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: * Patients should be clinically stable for inclusion into the study * Mature newborn ≥ 37 weeks of gestation, Infants and children (≥2month and \< 18y) or previously preterm (≤ 37 weeks of gestation) babies or children(≥2month and \<18y) if chILD genetically diagnosed * chILD genetically diagnosed surfactant dysfunction disorders including patients with mutations in SFTPC, SFTPB, ABCA3, TTF1 (Nkx2-1), FOXF1 further extremely rare entities with specific mutations, for example in TBX4, NPC2, NPC1, NPB, COPA, LRBA and other genes * no HCQ treatment in the last 3 months * Ability of subject or/and legal representatives to understand character and individual consequences of clinical trial * Signed and dated informed consent of the subject (if subject has the ability) and the representatives (of underaged children) must be available before start of any specific trial procedures Exclusion Criteria: Subjects presenting with any of the following criteria will not be included in the trial: * chILD primarily related to developmental disorders * chILD primarily related to growth abnormalities reflecting deficient alveolarization * chILD related to chronic aspiration * chILD related to immunodeficiency * chILD related to abnormalities in lung vessel structure * chILD related to organ transplantation/organ rejection/GvHD * chILD related to recurrent infections * Acute severe infectious exacerbations * Known hypersensitivity to HCQ, or other ingredients of the tablets * Proven retinopathy or maculopathy * Glucose-6-phosphate-dehydrogenase deficiency resulting in favism or hemolytic anemia * Myasthenia gravis * Hematopoetic disorders * Participation in other clinical trials during the present clinical trial or not beyond the time of 4 half-lives of the medication used, at least one week * Hereditary galactose intolerance, lactase deficiency or glucose-galactose- malabsorption * Simultaneous prescription of other potentially nephrotoxic or hepatotoxic medication at the discretion of the treating physician

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's hospital of Fudan University

    Shanghai, Shanghai Municipality, 201102, China

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