NCT03243019 Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations
| NCT ID | NCT03243019 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | University Hospital, Lille |
| Condition | Lymphatic Malformation |
| Study Type | INTERVENTIONAL |
| Enrollment | 28 participants |
| Start Date | 2018-06-25 |
| Primary Completion | 2026-02 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 28 participants in total. It began in 2018-06-25 with a primary completion date of 2026-02.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 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
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
Contact & Investigator
Pierre Fayoux, MD
PRINCIPAL INVESTIGATOR
University Hospital, Lille
Frequently Asked Questions
Who can join the NCT03243019 clinical trial?
This trial is open to participants of all sexes, up to 18 Years, studying Lymphatic Malformation. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03243019 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT03243019 currently recruiting?
Yes, NCT03243019 is actively recruiting participants. Contact the research team at pierre.fayoux@chru-lille.fr for enrollment information.
Where is the NCT03243019 trial being conducted?
This trial is being conducted at Lille, France, Paris, France.
Who is sponsoring the NCT03243019 clinical trial?
NCT03243019 is sponsored by University Hospital, Lille. The principal investigator is Pierre Fayoux, MD at University Hospital, Lille. The trial plans to enroll 28 participants.