Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations
Recruiting
Phase 2Interventional Study
Lymphatic MalformationPediatric
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
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Review the details below, then apply to join this clinical trial.
At a Glance
Age
Up to 18
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
28 (estimated)
Sponsor
University Hospital, Lille · Other
Who this trial is looking for
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Answer a few quick questions to see if you may meet the eligibility requirements.
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.
Trial Locations
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Eligibility 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 ,mo…
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
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