Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations

NCT03243019 | Recruiting Phase 2

• 2 other identifiers: 2012_672013-002800-15
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 2

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.

Conditions

Lymphatic Malformation; Pediatric

Keywords

cervico-facial; rapamycin

Outcome Measures

Primary Outcomes (1)

• Response rate to rapamycin

  Volumetric assessment by MRI. A response is considered as positive if volume decrease is superior to 1/5th of the initial volume.

  At 3 months

Secondary Outcomes (5)

• Kinetic of rapamycin response

  At 3, 6 and 12 months

• Efficacy of rapamycin on clinical symptoms

  At 3, 6 and 12 months

• Pediatric Quality of Life Inventory (PedsQL 4) Scales

  Baseline, at 3, 6 and 12 months

• Biological response to rapamycin

  Baseline and at 6 months

• Rapamycin side effects

  Monthly during 1 years

Study Arms (1)

SIROLIMUS

EXPERIMENTAL

Drug: rapamycin; Device: MRI; Biological: Rapamycin dosage

Interventions

rapamycin DRUG

oral administration

SIROLIMUS

MRI DEVICE

cervicofacial MRI

SIROLIMUS

Rapamycin dosage BIOLOGICAL

Biological dosage of Rapamycin level

SIROLIMUS

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital, Lille: lead
• Ministry of Health, France: collaborator

Study Sites (2)

Hôpital Jeanne de Flandres, CHU

Lille, France

RECRUITING

Hu Robert Debre Aphp - Paris

Paris, France

RECRUITING

MeSH Terms

Conditions

Lymphatic Abnormalities

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Pierre Fayoux, MD

  University Hospital, Lille

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Pierre Fayoux, MD

CONTACT

3 20 44 50 67; pierre.fayoux@chru-lille.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2017
• First Posted: August 8, 2017
• Study Start: June 25, 2018
• Primary Completion: February 1, 2026
• Study Completion: February 1, 2026
• Last Updated: December 16, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)