Home › Trials › NCT03243019
Clinical trial Recruiting at 2 sites Phase 2 Interventional Ages Any age to 18

Rapamycin for cervico‑facial lymphatic malformations in children

Official title: Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis

Run by University Hospital, Lille · NCT03243019 · Data updated 2026-11-12

This trial will test whether six months of oral rapamycin can shrink cervico‑facial lymphatic malformations in children, improve quality of life, and reduce bleeding.

Quick facts

PhasePhase 2
Study typeInterventional
AgesAny age to 18 Years
SexAll
Enrollment28 (estimated)
SponsorUniversity Hospital, Lille
Academic / other
Locations2 sites (Lille and 1 other locations)
StatusRecruiting
StartedJune 25, 2018
Main results expectedFebruary 2027
Study endsFebruary 2027
Registry record updatedMay 22, 2026
Trial IDNCT03243019 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Children (0–18 years) with polycystic suprahyoid or mediastinal cervico‑facial lymphatic malformations causing chronic pain or respiratory/swallowing impairment, for whom curative surgery is not possible or carries high risk, and who meet basic lab and performance status criteria are the intended candidates.

Probably not a fit

Patients whose malformations are readily treatable with low‑risk curative procedures, those with renal failure, on incompatible immunosuppressive therapy without washout, or who fail required laboratory thresholds are unlikely to benefit from enrollment.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2, interventional study gives pediatric patients with extended cervico‑facial lymphatic malformations oral rapamycin for a six‑month treatment period and uses MRI to measure lesion volume changes. Success is defined primarily as more than a one‑fifth (20%) reduction in initial lesion volume on MRI, along with improvements in quality of life and reduced mucosal bleeding when present. Patients must have lesions not amenable to curative treatment or where surgery carries high morbidity, meet basic laboratory safety thresholds, and be aged 0–18 years. Participating centers are in Lille and Paris, France, with scheduled imaging and clinical follow‑up during and after treatment.

Why it matters

Potential benefit: If successful, oral rapamycin could reduce lesion size, relieve breathing or swallowing problems, lower bleeding risk, and improve quality of life for affected children.

How similar studies have gone: Prior small and nonrandomized studies of sirolimus/rapamycin in vascular and lymphatic malformations have reported promising reductions in lesion size and symptom improvement, though larger controlled data are limited.

Who can join: the full rules

Show full inclusion / exclusion criteria 
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

Where it's running

  • Hôpital Jeanne de Flandres, CHU
    Lille, France     Recruiting
  • Hu Robert Debre Aphp - Paris
    Paris, France     Recruiting

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov ↗ Find more trials like this →
Browse more
Lymphatic Malformation 9University Hospital, Lille 119
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.