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

• Status: Recruiting
• Trial end date: 2022-02-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Hospital, Lille
• Collaborator: Ministry of Health, France
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Patient from 1 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

• concomitant immuno-suppressor treatment

• 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