Status:
RECRUITING
Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations
Lead Sponsor:
University Hospital, Lille
Collaborating Sponsors:
Ministry of Health, France
Conditions:
Lymphatic Malformation
Pediatric
Eligibility:
All Genders
Up to 18 years
Phase:
PHASE2
Brief 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 v...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
June 25 2018
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 1 2026
Estimated Enrollment :
28 Patients enrolled
Trial Details
Trial ID
NCT03243019
Start Date
June 25 2018
End Date
February 1 2026
Last Update
December 16 2025
Active Locations (2)
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1
Hôpital Jeanne de Flandres, CHU
Lille, France
2
Hu Robert Debre Aphp - Paris
Paris, France