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

ConditionsLymphatic MalformationPediatric
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 18
SponsorUniversity Hospital, Lille

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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%

Disqualifiers

Non-respect of inclusion criteria

Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period

renal failure

Liver failure

Trial design

Design model

Single group

Treatments tested in this trial

  • rapamycin

    Drug

    oral administration

  • MRI

    Device

    cervicofacial MRI

  • Rapamycin dosage

    Biological/Vaccine

    Biological dosage of Rapamycin level

Treatment groups

28 Participants
are divided into 1 treatment group
Group A: SIROLIMUSExperimental treatment 3 interventions

Trial outcomes

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.

Time frame
At 3 months

Secondary outcomes

1

Kinetic of rapamycin response

MRI assessment of the volume

Time frame
At 3, 6 and 12 months
2

Efficacy of rapamycin on clinical symptoms

Clinical and fiberscopy evaluation by scoring

Time frame
At 3, 6 and 12 months
3

Pediatric Quality of Life Inventory (PedsQL 4) Scales

Assesses health-related quality of life among children with chronic and acute diseases

Time frame
Baseline, at 3, 6 and 12 months
4

Biological response to rapamycin

biological effect of mTOR blockage by measuring pAKT, p70S6 kinase, pMEK, and VEGF C, VEGFR3

Time frame
Baseline and at 6 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University Hospital, Lille

Lead sponsor

Ministry of Health, France

Collaborator

Source ClinicalTrials.gov
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