Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations (RAPAMALYMPH)
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|ClinicalTrials.gov Identifier: NCT03243019|
Recruitment Status : Recruiting
First Posted : August 8, 2017
Last Update Posted : October 16, 2019
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|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||28 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Biological: Rapamycin dosage
Biological dosage of Rapamycin level
- 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.
- Kinetic of rapamycin response [ Time Frame: At 3, 6 and 12 months ]MRI assessment of the volume
- Efficacy of rapamycin on clinical symptoms [ Time Frame: At 3, 6 and 12 months ]Clinical and fiberscopy evaluation by scoring
- 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
- 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
- Rapamycin side effects [ Time Frame: Monthly during 1 years ]Side effect assessment using the NCI-CTC 3.0 scale
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
|Contact: Pierre Fayoux, MD||3 20 44 50 67 ext +email@example.com|
|Hôpital Jeanne de Flandres, CHU||Recruiting|
|Principal Investigator: pierre Fayoux, MD|
|Principal Investigator:||Pierre Fayoux, MD||University Hospital, Lille|