Rapamycin for cervico‑facial lymphatic malformations in children
Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
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
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 28 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | University Hospital, Lille Academic / other |
| Locations | 2 sites (Lille and 1 other locations) |
| Trial ID | NCT03243019 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Lille and 1 other locations
- Hôpital Jeanne de Flandres, CHU — Lille, France (Recruiting)
- Hu Robert Debre Aphp - Paris — Paris, France (Recruiting)
Study contacts
- Principal investigator: Pierre Fayoux, MD — University Hospital, Lille
- Study coordinator: Pierre Fayoux, MD
- Email: pierre.fayoux@chru-lille.fr
- Phone: 3 20 44 50 67
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.