Ignite Creation Date:
2024-05-05 @ 9:51 PM
Last Modification Date:
2024-10-26 @ 10:10 AM
Study NCT ID:
NCT00975819
Status:
UNKNOWN
Last Update Posted:
2015-02-05
First Post:
2009-09-10
Brief Title:
Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
Sponsor:
Childrens Hospital Medical Center Cincinnati
Organization:
Childrens Hospital Medical Center Cincinnati
Study Overview
Official Title:
A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies
Status:
UNKNOWN
Status Verified Date:
2015-01
Last Known Status:
ACTIVE_NOT_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:
The purpose of this study is to determine if the use of sirolimus in the treatment of children and young adults with complicated vascular anomalies will prove to be safe and provide objective response resulting in improved clinical status and quality of life
Funding Source - FDA OOPD Food and Drug Administration - Office of Orphan Products Development
Funding Source - FDA OOPD Food and Drug Administration - Office of Orphan Products Development
Detailed Description:
Patients with vascular anomalies VA have a spectrum of diseases that can be broadly classified into vascular tumors and malformations Complicated vascular anomalies can cause disfigurement chronic pain and organ dysfunction with significant morbidity and mortality Despite the severity of potential complications we lack uniform guidelines for the treatment and response to treatment of children and young adults with these diseases There are pre-clinical and clinical data supporting the essential regulatory function of the PI3KAktmTOR pathway in vascular growth and organization and suggest a therapeutic target for patients with complicated vascular anomalies The overall goal of this trial is to objectively determine the effectiveness and safety of the mTOR inhibitor Rapamycin in the treatment of children and young adults diagnosed with complicated vascular anomalies We propose a Phase 2 trial with the diagnostic therapeutic and response criteria experimentally determined in this study used as a framework for future Phase 3 clinical trials
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 5R01FD003712-04 | FDA | None | httpsreporternihgovquickSearch5R01FD003712-04 |