Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations (RAPAMALYMPH)

University Hospital, Lille

Status and phase: Enrolling; Phase 2

Conditions: Pediatric; Lymphatic Malformation

Treatments: Device: MRI; Biological: Rapamycin dosage; Drug: rapamycin

Study type: Interventional

Funder types: Other

Identifiers

NCT03243019

2012_67

2013-002800-15 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 28 estimated patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

28 participants in 1 patient group

SIROLIMUS

Experimental group

Treatment:

Biological: Rapamycin dosage

Device: MRI

Drug: rapamycin