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Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations (RAPAMALYMPH)

U

University Hospital, Lille

Status and phase

Enrolling
Phase 2

Conditions

Pediatric
Lymphatic Malformation

Treatments

Device: MRI
Biological: Rapamycin dosage
Drug: rapamycin

Study type

Interventional

Funder types

Other

Identifiers

NCT03243019
2012_67
2013-002800-15 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment

28 estimated patients

Sex

All

Ages

Under 18 years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

28 participants in 1 patient group

SIROLIMUS
Experimental group
Treatment:
Biological: Rapamycin dosage
Device: MRI
Drug: rapamycin

Trial contacts and locations

2

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Central trial contact

Pierre Fayoux, MD

Data sourced from clinicaltrials.gov

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