Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 18
SponsorUniversity Hospital, Lille
About this trial
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.
Eligibility criteria
Qualifiers
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%
Disqualifiers
Non-respect of inclusion criteria
Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period
renal failure
Liver failure
Trial design
Treatments tested in this trial
- rapamycin
- MRI
- Rapamycin dosage
Treatment groups
28 Participants
are divided into 1 treatment groupSponsors and collaborators
University Hospital, Lille
Lead sponsor
Ministry of Health, France
Collaborator
Source ClinicalTrials.gov