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Could a daily dab of sirolimus ease tongue cysts?

NCT ID NCT04128722

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a sirolimus solution applied once daily to the tongue in 12 children and adults with mild to moderate lymphatic malformations. The goal was to see if it reduces severity of symptoms like oozing, bleeding, and pain over 12 weeks compared to no treatment. Researchers used expert ratings of photos to measure improvement.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
sirolimus (topical solution)
What this could lead to
If it works, this could offer a simple, non-invasive treatment option for tongue lymphatic malformations, reducing oozing, bleeding, and pain without the need for systemic drugs.
What could go wrong
This is a small, early-phase trial with only 12 participants, so results may not apply to everyone. The treatment may not improve symptoms significantly, and side effects from topical use are not yet well understood.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

12 people

The number who actually took part.

Started

Feb 2020

Finished

Jan 2025

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

5 years and older

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: * Participants ≥ 5 years of age * Lingual microcystic lymphatic malformation that does not require systemic treatment, assessed by clinical examination and head-and-neck MRI imaging prior to study enrolment, with or without underlying syndromic malformation (CLAPO for instance) * Participants covered by or having the rights to social security * Written informed consent obtained from participant and participant's legal representative if participant is under 18 * Ability for participant to comply with the requirements of the study Exclusion Criteria: * Patients with a lymphatic malformation requiring a continued background therapy (involving deep organs) * Secondary lymphatic malformations (lymphangiectasia post-radiotherapy, etc) * Previous treatment with systemic or topical mTOR (mammilian target of rapamycin) inhibitors within 12 months before inclusion (half-life of oral sirolimus is 60 days in adults). * Previous treatment with oral or topical steroids within 10 days before inclusion (half-life of corticosteroids is 12-36 hours) * Immunosuppression (immunosuppressive disease or immunosuppressive treatment) * Ongoing neoplasia * Active chronic infectious disease (Hepatitis-B virus, Hepatitis-C virus, HIV, etc) * Local necrosis * Local fungal, viral (herpes simplex virus, varicella zoster virus, etc) or bacterial infection on the site of the LMLM (based on clinical examination) * Known allergy to one of the components of the sirolimus solution * Soy bean or Peanut allergy * Pregnant or breastfeeding women * Women of child-bearing potential (including teenagers) not using a reliable contraceptive method until the end of the study and three month after the end of the study or sirolimus discontinuation. * Already involved in another therapeutic trial

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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

  • Hospital NECKER -AP-HP - Dermatology

    Paris, 75015, France

  • Univsersity of TOURS _ Service de Dermatologie

    Tours, Indre et Loire, 37044, France