Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations

Recruiting now · Phase 2

Conditions studied: Lymphatic Malformation, Pediatric

In brief

To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period. The success rate is determined by volume reduction superior to 1/5e of the initial volume measured by MRI, impact on QOL and reduction of bleeding in case of mucosal involvement.

Key facts

Study ID

NCT03243019

Run by

University Hospital, Lille

People needed

28

Starts

2018-06-25

Expected to finish

2027-02-01

Last updated by the study team

2026-05-22

Who can join

Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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 is running

• Hôpital Jeanne de Flandres, CHU — Lille, France (enrolling)
• Hu Robert Debre Aphp - Paris — Paris, France (enrolling)

Full record on ClinicalTrials.gov

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