Advertisement

Topics

Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies

2014-08-27 03:19:24 | BioPortfolio

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.

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

Conditions

Kaposiform Hemangioendotheliomas

Intervention

sirolimus

Location

Cincinnati Children's Hospital Medical Center
Cincinnati
Ohio
United States
45229

Status

Recruiting

Source

Children's Hospital Medical Center, Cincinnati

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:19:24-0400

Clinical Trials [304 Associated Clinical Trials listed on BioPortfolio]

Sirolimus Versus Sirolimus Plus Prednisolone for Kaposiform Hemangioendothelioma With Kasabach-Merritt Syndrome

Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm that occurs predominantly in infancy or early childhood. KHE has a nearly equal sex ratio. The annual incidence of KHE has...

A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors

In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk...

A Dose-Ranging Clinical Study to Assess the Safety and Efficacy of Subconjunctival Injection of Sirolimus in Patients With Dry Eye in the Controlled Adverse Environmental (CAE) Model

The purpose of this study is to determine the safety and efficacy of an ocular sirolimus (rapamycin) formulation compared to Placebo on the signs and symptoms of dry eye in the CAE Model.

Safety and Efficacy of Sirolimus for HIV Reservoir Reduction in Individuals on Suppressive ART

The purpose of this study is to find out about the safety of sirolimus in individuals with HIV infection who are also being treated with ART. The investigators want to learn whether siroli...

Sirolimus as Therapeutic Approach to Uveitis

The purpose of this study is to find out about the safety and effectiveness of the study drug, sirolimus, in patients with uveitis and to utilize the potential effectiveness of sirolimus, ...

PubMed Articles [43 Associated PubMed Articles listed on BioPortfolio]

First Intracardiac Kaposiform Hemangioendothelioma in an Infant Resolved With Sirolimus: A Case Report.

Kaposiform hemangioendothelioma (KHE) is a rare and aggressive vascular tumor that can be associated with a consumptive coagulopathy and thrombocytopenia (Kasabach-Merritt phenomenon). Only one case o...

Kaposiform hemangioendothelioma without cutaneous involvement.

We sought to characterize the clinical features and management of patients diagnosed as Kaposiform hemangioendothelioma (KHE) without cutaneous involvement.

Comparative Effects of Topical 0.2% Sirolimus for Angiofibromas in Adults and Pediatric Patients with Tuberous Sclerosis Complex.

Recent reports have suggested that the topical formulation of sirolimus is effective in treating facial angiofibromas in tuberous sclerosis complex (TSC). Here, we determined the safety and efficacy o...

Comparison of Corticosteroid and Vincristine in Treating Kaposiform Hemangioendothelioma and Tufted Angioma: A Systematic Review and Meta-Analysis.

 To evaluate the outcome and safety of corticosteroids and vincristine (VCR) in the treatment of kaposiform hemangioendothelioma (KHE) and tufted angioma (TA).

Development of Kasabach-Merritt phenomenon following vaccination: More than a coincidence?

Kasabach-Merritt phenomenon (KMP) occurred uniquely in patients with kaposiform hemangioendothelioma (KHE) and tufted angioma (TA). We report the clinical characteristics of two patients with KHE invo...

Medical and Biotech [MESH] Definitions

A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to IMMUNOPHILINS. Sirolimus is a potent immunosuppressant and possesses both antifungal and antineoplastic properties.

Rapidly growing vascular lesion along the midline axis of the neck, upper trunk, and extremities that is characterized by CONSUMPTION COAGULOPATHY; THROMBOCYTOPENIA; and HEMOLYTIC ANEMIA. It is often associated with infantile Kaposiform HEMANGIOENDOTHELIOMA and other vascular tumors such as tufted ANGIOMA.

A neoplasm derived from blood vessels, characterized by numerous prominent endothelial cells that occur singly, in aggregates, and as the lining of congeries of vascular tubes or channels. Hemangioendotheliomas are relatively rare and are of intermediate malignancy (between benign hemangiomas and conventional angiosarcomas). They affect men and women about equally and rarely develop in childhood. (From Stedman, 25th ed; Holland et al., Cancer Medicine, 3d ed, p1866)

A derivative of sirolimus and an inhibitor of TOR SERINE-THREONINE KINASES. It is used to prevent GRAFT REJECTION in heart and kidney transplant patients by blocking cell proliferation signals. It is also an ANTINEOPLASTIC AGENT.

A family of immunophilin proteins that bind to the immunosuppressive drugs TACROLIMUS (also known as FK506) and SIROLIMUS. EC 5.2.1.-

More From BioPortfolio on "Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies"

Advertisement
Quick Search
Advertisement
Advertisement

 

Relevant Topics

Pediatrics
Pediatrics is the general medicine of childhood. Because of the developmental processes (psychological and physical) of childhood, the involvement of parents, and the social management of conditions at home and at school, pediatrics is a specialty. With ...

Blood
Blood is a specialized bodily fluid that delivers necessary substances to the body's cells (in animals) – such as nutrients and oxygen – and transports waste products away from those same cells.  In vertebrates, it is composed of blo...

Pain
Pain is defined by the International Association for the Study of Pain as “an unpleasant sensory and emotional experience associated with actual or potential tissue damage or described in terms of such damage”. Some illnesses can be excruci...


Searches Linking to this Trial