Actively Recruiting
Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
Led by University Hospital, Lille · Updated on 2026-05-22
28
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
Sponsors
U
University Hospital, Lille
Lead Sponsor
M
Ministry of Health, France
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the use of Rapamycin to treat extended cervicofacial lymphatic malformations in children and adolescents up to 18 years old. This Phase 2 study aims to measure how well Rapamycin reduces the volume of these malformations, improves quality of life, and decreases bleeding when mucosal areas are involved. The treatment outcomes are assessed by MRI scans and clinical evaluations over time. Participants receive Rapamycin orally for a period of 6 months. The study monitors response rates at 3 months and continues to observe clinical symptoms and quality of life impacts up to 12 months. Biological tests measure Rapamycin levels and side effects are tracked monthly throughout one year. MRI imaging is used to measure the malformation volume and follow treatment effectiveness. During the study, participants undergo regular clinical assessments and complete Pediatric Quality of Life Inventory (PedsQL 4) scales at baseline, 3, 6, and 12 months. Blood tests evaluate biological responses and safety. Researchers monitor side effects monthly for one year to ensure participant safety. The total follow-up lasts at least one year, allowing detailed tracking of treatment effects and participant well-being.
CONDITIONS
Brief Title
Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient aged 0 to 18 years with poly-cystic suprahyoid or mediastinal lymphatic malformation
- Chronic pain or functional respiratory or swallowing impairment with a CDS score less than 8
- No possible curative treatment or curative treatment poses high risk of morbidity, mortality, or functional and cosmetic impairment
- Karnofsky score greater than 50% if over 10 years old, or Lansky score greater than 50% if 10 years old or younger
- Blood counts and chemistry within specified limits: neutrophils ≥1.0 x 10^9/L, platelets ≥100 x 10^9/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 pregnancy test if relevant
- Social security affiliation
- At least 2 months since any previous procedure on the malformation
You will not qualify if you...
- Failure to meet inclusion criteria
- Use of other immunosuppressive therapy or long-term corticosteroids without a 28-day washout
- Renal failure
- Liver failure
- Digestive diseases causing rapamycin malabsorption
- Uncontrolled or severe infectious disease
- Use of medications that interfere with or inhibit CYP3A4 enzyme activity or use of cisapride and metoclopramide
- Concurrent use of mTOR inhibitors
- Allergy to peanuts or soya
- Inability to provide informed consent
- No social security affiliation
- Refusal to sign consent or participate
- Ongoing pregnancy or breastfeeding
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 months
Participants receive oral rapamycin to treat cervico-facial lymphatic malformations and have periodic assessments including MRI and biological dosage of rapamycin levels.
Monthly visits for rapamycin side effect monitoring and assessments at baseline, 3, 6, and 12 months
Trial Site Locations
Total: 2 locations
1
Hôpital Jeanne de Flandres, CHU
Lille, France
Actively Recruiting
2
Hu Robert Debre Aphp - Paris
Paris, France
Actively Recruiting
Research Team
P
Pierre Fayoux, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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