Clinical Trial: Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies
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)
Detailed Summary: 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.
Sponsor: Children's Hospital Medical Center, Cincinnati
Current Primary Outcome:
- Evaluation of Disease Response - Volumetric MRI [ Time Frame: Baseline, 3, 6, and 12 months ]
- Evaluation of Disease Response - Quality of Life and Pain Assessments [ Time Frame: Baseline, 3, 6, 12 months ]
- Evaluation of Disease Response - Clinical Criteria and Functional Impairment [ Time Frame: baseline, 3, 6, 12 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Tissue (only baseline) and Serum Sample analysis [ Time Frame: baseline, 6, 12 months ]
Original Secondary Outcome: Same as current
Information By: Children's Hospital Medical Center, Cincinnati
Dates:
Date Received: September 10, 2009
Date Started: October 2009
Date Completion: October 2019
Last Updated: February 3, 2015
Last Verified: January 2015
