Ignite Creation Date:
2024-05-06 @ 2:43 PM
Last Modification Date:
2024-10-26 @ 1:36 PM
Study NCT ID:
NCT04406870
Status:
NOT_YET_RECRUITING
Last Update Posted:
2020-05-29
First Post:
2020-05-17
Brief Title:
Sirolimus in the Treatment for Infantile Hepatic HemangioendotheliomaIEEH
Sponsor:
Shanghai Childrens Medical Center
Organization:
Shanghai Childrens Medical Center
Study Overview
Official Title:
Sirolimus in the Treatment for Propranolol-resistant Infantile Hepatic Hemangioendothelioma
Status:
NOT_YET_RECRUITING
Status Verified Date:
2020-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PRL-SRL-IHHE
Brief Summary:
Infantile hepatic hemangioendothelioma IHHE is an infantile hemangioma involving the liverSince 2008 propranolol has been used for the treatment of hemangioma and some researchers have also started to report the use of propranolol for the treatment of infantile hepatic hemangioma in children Sirolimus can be used in patients with vascular malformations such as hemangiomas IHHE is also an infantile hemangioma involving the liverthus sirolimus may paly the role in treatment of IHHEThe clinical trial explores the efficacy of sequential treatment of sirolimus to refractory IHHE patients resistant with propranolol to improve the therapeutic effect reduce the side effects of traditional treatment methods hormones interferon and reduce the number of operations and interventions and to provide a clinical basis for the application of the new therapeutics model of IHHE of propranolol sequential sirolimus treatment
Detailed Description:
According to the World Health OrganizationWHO classification of digestive system tumors in the fourth edition in 2010 infantile hepatic hemangioendothelioma IHHE is an infantile hemangioma involving the liver It is a benign tumor which can be subgrouped into focal multifocal and diffusing in children as well as combining vascular malformations of the skin brain digestive tract and other organsSince 2008 propranolol has been used for the treatment of hemangioma and some researchers have also started to report the use of propranolol for the treatment of infantile hepatic hemangioma in childrenAnd more and more clinicians unanimously recommend propranolol as first-line medication for the treatment of IHHEHowever there are few solutions to propranolol-resistant IHHE Sirolimus is a serinethreonine kinase which plays a pivotal role in cell mortality angiogenesis and cell growth Sirolimus can be used in patients with vascular malformations such as hemangiomas Children with vascular malformations received 01mgkg of sirolimus orally every day and maintained the blood concentration at 8-15ngml The lesions gradually disappear with time going on Therefore sirolimus can be used as a second-line medicine for refractory hemangioma and vascular malformationInterestingly the investigators previous study which is retrospective analysis of 30 patients has indicated the effective rate of propranolol alone in treating infantile hepatic endothelial tumor IHHE was 577 while that of sequential treatment combined with sirolimus was 846So this clinical trial is going to explore the efficacy of sequential treatment of sirolimus to refractory IHHE patients resistant with propranololto improve the therapeutic effectfurtherly reduce the side effects of traditional treatment methods hormones interferon as well as the number of operations and interventions and to provide a clinical guide for the novel therapeutics model of propranolol combined with sirolimus for sequential treatment of infantile hepatic hemangioendothelioma
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None