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Recruiting NCT03243019

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

Phase II Interventional Lymphatic Malformation Pediatric

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: rapamycin, MRI, Rapamycin dosage.
Who it may be relevant to
Registry conditions: Lymphatic Malformation, Pediatric. Basic parameters: up to 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
France
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis

Overview

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.

Interventions

  • Drug rapamycin
    oral administration
  • Device MRI
    cervicofacial MRI
  • Biological Rapamycin dosage
    Biological dosage of Rapamycin level

Primary outcome measures

  • Response rate to rapamycin [Time frame: At 3 months]
Secondary outcome measures (5)
  • Kinetic of rapamycin response [Time frame: At 3, 6 and 12 months]
  • Efficacy of rapamycin on clinical symptoms [Time frame: At 3, 6 and 12 months]
  • Pediatric Quality of Life Inventory (PedsQL 4) Scales [Time frame: Baseline, at 3, 6 and 12 months]
  • Biological response to rapamycin [Time frame: Baseline and at 6 months]
  • Rapamycin side effects [Time frame: Monthly during 1 years]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

France · 2 centers
  • Hôpital Jeanne de Flandres, CHU — Lille
  • Hu Robert Debre Aphp - Paris — Paris

Identifiers

NCT: NCT03243019 · 2012_67 · 2013-002800-15

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗