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Clinical Trial 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)


Clinical Trial 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 PI3K/Akt/mTOR 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 Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT00975819
Study type Interventional
Source Children's Hospital Medical Center, Cincinnati
Contact
Status Active, not recruiting
Phase Phase 2
Start date October 2009
Completion date October 2019

See also
  Status Clinical Trial Phase
Recruiting NCT05050149 - Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations Phase 2
Recruiting NCT06160739 - Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations
Not yet recruiting NCT06239480 - A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations Phase 3