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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03243019
Recruitment Status : Recruiting
First Posted : August 8, 2017
Last Update Posted : October 16, 2019
Sponsor:
Collaborator:
Ministry of Health, France
Information provided by (Responsible Party):
University Hospital, Lille

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.


Condition or disease Intervention/treatment Phase
Lymphatic Malformation Pediatric Drug: rapamycin Device: MRI Biological: Rapamycin dosage Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 28 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
Actual Study Start Date : June 25, 2018
Estimated Primary Completion Date : February 2022
Estimated Study Completion Date : February 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: SIROLIMUS Drug: rapamycin
oral administration

Device: MRI
cervicofacial MRI

Biological: Rapamycin dosage
Biological dosage of Rapamycin level




Primary Outcome Measures :
  1. Response rate to rapamycin [ Time Frame: At 3 months ]
    Volumetric assessment by MRI. A response is considered as positive if volume decrease is superior to 1/5th of the initial volume.


Secondary Outcome Measures :
  1. Kinetic of rapamycin response [ Time Frame: At 3, 6 and 12 months ]
    MRI assessment of the volume

  2. Efficacy of rapamycin on clinical symptoms [ Time Frame: At 3, 6 and 12 months ]
    Clinical and fiberscopy evaluation by scoring

  3. Pediatric Quality of Life Inventory (PedsQL 4) Scales [ Time Frame: Baseline, at 3, 6 and 12 months ]
    Assesses health-related quality of life among children with chronic and acute diseases

  4. Biological response to rapamycin [ Time Frame: Baseline and at 6 months ]
    biological effect of mTOR blockage by measuring pAKT, p70S6 kinase, pMEK, and VEGF C, VEGFR3

  5. Rapamycin side effects [ Time Frame: Monthly during 1 years ]
    Side effect assessment using the NCI-CTC 3.0 scale



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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient from 1 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
  • concomitant immuno-suppressor treatment
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03243019


Contacts
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Contact: Pierre Fayoux, MD 3 20 44 50 67 ext +33 pierre.fayoux@chru-lille.fr

Locations
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France
Hôpital Jeanne de Flandres, CHU Recruiting
Lille, France
Principal Investigator: pierre Fayoux, MD         
Sponsors and Collaborators
University Hospital, Lille
Ministry of Health, France
Investigators
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Principal Investigator: Pierre Fayoux, MD University Hospital, Lille

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Responsible Party: University Hospital, Lille
ClinicalTrials.gov Identifier: NCT03243019    
Other Study ID Numbers: 2012_67
2013-002800-15 ( EudraCT Number )
First Posted: August 8, 2017    Key Record Dates
Last Update Posted: October 16, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University Hospital, Lille:
cervico-facial
rapamycin
Additional relevant MeSH terms:
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Lymphangioma
Lymphatic Abnormalities
Congenital Abnormalities
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
Lymphatic Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs