Ignite Creation Date:
2024-05-06 @ 12:26 PM
Last Modification Date:
2024-10-26 @ 12:59 PM
Study NCT ID:
NCT03767660
Status:
UNKNOWN
Last Update Posted:
2018-12-19
First Post:
2018-11-24
Brief Title:
Efficacy of Rapamycin Sirolimus in the Treatment of BRBNS Hereditary or Sporadic Venous Malformation
Sponsor:
Peking Union Medical College Hospital
Organization:
Peking Union Medical College Hospital
Study Overview
Official Title:
Efficacy of Rapamycin Sirolimus in the Treatment of Blue Rubber Bleb Nevus Syndrome Hereditary or Sporadic Venous Malformation
Status:
UNKNOWN
Status Verified Date:
2018-12
Last Known Status:
RECRUITING
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:
None
Brief Summary:
A prospective nonrandomized open-label single-arm clinical trial to study efficacy of rapamycin sirolimus in the treatment of Blue Rubber Bleb Nevus Syndrome hereditary or sporadic venous malformation
Detailed Description:
Blue rubber bleb nevus syndrome BRBNS and venous malformation are mainly caused by somatic mutation of TEK and PIK3CA which activates the PI3KAKT signaling pathway As an important protein kinase downstream of the PI3KAKT pathway mTOR can serve as a potential therapeutic target for BRBNS Experiments of mice have shown that rapamycin inhibited the progression of venous malformation lesions There are a few human cases reported using rapamycin treatment The investigators study is designed to be a prospective nonrandomized open-label single-arm clinical trial to investigate its efficacy and safety
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?:
None
Is an FDA AA801 Violation?:
None