Advertisement

Topics

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

2014-08-27 03:17:18 | BioPortfolio

Summary

This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1). Subjects will apply either a Polyvinylidene fluoride (PVDF) coating (Skincerity) containing rapamycin or the PVDF coating alone nightly to fibromatous lesions for a duration of six months.

The primary goal of this study is to evaluate the safety of the topical product in patients with TSC and NF1. The secondary goal of this study is to evaluate the effectiveness of the topical product for treatment of cutaneous fibromatous lesions.

Study Design

Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Conditions

Tuberous Sclerosis

Intervention

Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin

Location

The University of Texas Health Science Center
Houston
Texas
United States
77030

Status

Recruiting

Source

The University of Texas Health Science Center, Houston

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:17:18-0400

Clinical Trials [441 Associated Clinical Trials listed on BioPortfolio]

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, ...

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.

Dose Ranging Study of an Ocular Sirolimus (Rapamycin) Formulation in Patients With Diabetic Macular Edema

The purpose of this study is to determine the safety and efficacy of an ocular sirolimus (rapamycin) formulation at various doses in patients with diabetic macular edema.

Phase 2 Study of an Ocular Sirolimus (Rapamycin) Formulation in Combination With Lucentis® in Patients With Age-Related Macular Degeneration

The purpose of this study is to determine the safety and efficacy of an ocular sirolimus (rapamycin) formulation in combination with Lucentis in patients with sub-foveal choroidal neovascu...

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...

PubMed Articles [363 Associated PubMed Articles listed on BioPortfolio]

Measurement of sirolimus concentrations in human blood using an automated electrochemiluminescence immunoassay (ECLIA): a multicenter evaluation.

Therapeutic drug monitoring (TDM) of sirolimus is essential in transplant recipients. We evaluated the performance of a new electrochemiluminescence immunoassay (ECLIA) for measuring sirolimus concent...

Prolonged activity and toxicity of sirolimus in a patient with metastatic renal perivascular epithelioid cell tumor: a case report and literature review.

Perivascular epithelioid cell tumor (PEComa) is a family of mesenchymal tumors. Conventional chemotherapy has little activity in this disease, but case reports are available on the activity of mammali...

Effect of sirolimus on liver cirrhosis and hepatic encephalopathy of common bile duct-ligated rats.

Cirrhosis is often associated with portal hypertension and portal-systemic collateral vessels formation attributed to angiogenesis, which leads to severe complications as hepatic encephalopathy. Sirol...

Sirolimus for management of complex vascular anomalies - A proposed dosing regimen for very young infants.

Neonates with vascular anomalies causing airway compromise and other complications require early initiation of medical therapy. Sirolimus has emerged as a safe and effective treatment, but standard re...

The SABRE Trial (Sirolimus Angioplasty Balloon for Coronary In-Stent Restenosis): Angiographic Results and 1-Year Clinical Outcomes.

The aim of this first-in-human study was to assess the safety and effectiveness of the Virtue sirolimus-eluting balloon in a cohort of patients with in-stent restenosis (ISR).

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.

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.-

A 12-KDa tacrolimus binding protein that is found associated with and may modulate the function of calcium release channels. It is a peptidyl-prolyl cis/trans isomerase which is inhibited by both tacrolimus (commonly called FK506) and SIROLIMUS.

Members of a family of highly conserved proteins which are all cis-trans peptidyl-prolyl isomerases (PEPTIDYLPROLYL ISOMERASE). They bind the immunosuppressant drugs CYCLOSPORINE; TACROLIMUS and SIROLIMUS. They possess rotamase activity, which is inhibited by the immunosuppressant drugs that bind to them.

More From BioPortfolio on "Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)"

Advertisement
Quick Search
Advertisement
Advertisement

 

Relevant Topics

Alzheimer's Disease
Of all the types of Dementia, Alzheimer's disease is the most common, affecting around 465,000 people in the UK. Neurons in the brain die, becuase  'plaques' and 'tangles' (mis-folded proteins) form in the brain. People with Al...

Nutrition
Within medicine, nutrition (the study of food and the effect of its components on the body) has many different roles. Appropriate nutrition can help prevent certain diseases, or treat others. In critically ill patients, artificial feeding by tubes need t...


Searches Linking to this Trial