Could a common drug shrink rare growths in Kids' faces and necks?
NCT ID NCT03243019
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether the drug rapamycin can shrink large, hard-to-treat lymphatic malformations in the head and neck area of children. Over 6 months, 28 kids will take oral rapamycin, and doctors will measure changes using MRI scans and quality-of-life surveys. The goal is to see if the drug reduces the size of the growths by at least one-fifth and eases symptoms like pain or trouble breathing.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- rapamycin
- What this could lead to
- If successful, this could offer a non-surgical treatment option for children with severe lymphatic malformations, reducing pain and improving breathing and swallowing.
- What could go wrong
- This is a small, early-phase trial with only 28 participants. Rapamycin may not shrink the malformations enough, and side effects like infections or mouth sores are possible.
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 28 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2018
- Expected to finish
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Feb 2027
An estimate. End dates often move.
- Lead sponsor
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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
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Up to 18 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: * Patient from 0 to 18 years of age, presenting with poly-cystic suprahyoid or mediastinal lymphatic. * with chronic pain or functional respiratory or swallowing impairment with a CDS score \< 8 * Curative treatment is not possible or associated with a high risk of morbidity ,mortality and functional and cosmetic impairment * Karnofsky Score (\> 10 years of age) or Lansky score (≤10 years of age) \> 50% * Biology * Neutrophils count≥1.0 x 109/L * Platelets count ≥ 100 x 109/L * Hemoglobin ≥ 8 g/dL * Bilirubin ≤ 1,5 ULN * Transaminases \< 2,5 ULN * Serum albumin ≥ 2 g/dL. * LDL cholesterol \<160 mg/dL * Triglycerides \< 150 mg/dL * Negative test of pregnancy if relevant * Social security affiliation * At least 2 months after a previous procedure on the malformation Exclusion Criteria: * Non-respect of inclusion criteria * Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period * renal failure * Liver failure * Digestive disease leading to rapamycin malabsorption * uncontrolled or severe infectious disease * Patients requiring treatment interfering with CYP3A4 isoenzyme (rifampicin, rifabutin, carbamazepine, phenobarbital, phenytoin) or inhibiting CYP3A4 isoenzyme's activity (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin, Diltiazem, Verapamil, nicardipine, clotrimazole, fluconazole , troleandomycin, bromocriptine, cimetidine, danazol, protease inhibitors) -patients requiring treatment by cisapride and metoclopramide * Concomitant administration of mTOR inhibitor * Peanuts or soya allergy * Impossibility to receive informed consent * Absence of social security affiliation * refusal to sign consent * Ongoing pregnancy or breastfeeding * refusal to participate
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Hu Robert Debre Aphp - Paris
RECRUITINGParis, France
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Hôpital Jeanne de Flandres, CHU
RECRUITINGLille, France
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