The ASCEND Trial: A Multicenter, Double Blinded Vehicle Controlled Study of TMB-001, a Proprietary Isotretinoin Ointment, in the Treatment of RXLI (X-linked) or ARCI (Lamellar) Ichthyosis; Preceded by a Voluntary Maximal Use Study; Both Studies in Subjects 6 Years of Age and Above

Date Added
August 10th, 2022
PRO Number
Pro00120937
Researcher
Lara Wine Lee

List of Studies


Keywords
Adolescents, Drug Studies, Pediatrics, Skin
Summary

This study aims to evaluate the safety and efficacy of isotretinoin ointment (TMB-001 0.05%) in treating subjects > or = to 6 years of age with lamellar ichthyosis, and aims to assess the bodily absorption of the cream across application frequencies. Subjects with ARCI/RXLI will be randomized 2:1, to either receive the TMB-001 0.05% isotretinoin ointment or a vehicle ointment - applied daily - for 3 weeks. Subsequently, dosing will be increased to twice daily for 9 weeks. If significant improvement is observed at the end of the 9 weeks, subjects will be randomized 1:1 to receive the TMB-001 0.05% ointment, either applied daily or twice-daily, for 12 weeks. Prior to the Phase III trial, subjects will have the option to participate in a 14 day treatment period with TMB-001 0.05% - twice daily - followed by continued treatment with TMB-001 0.05% twice daily for 10 weeks.

Institution
MUSC
Recruitment Contact
Courtney Rowley
843-792-9784
rowle@musc.edu

Open-label Study to Evaluate the Pharmacokinetics, Safety, and Immunogenicity of Ustekinumab in Pediatric Participants

Date Added
March 21st, 2022
PRO Number
Pro00118773
Researcher
Lara Wine Lee

List of Studies


Keywords
Pediatrics, Skin
Summary

The purpose of this study is to find out how long ustekinumab stays in and acts in participant's who are diagnosed with pediatric psoriasis and juvenile psoriatic arthritis. This is measured by blood tests. Another purpose is to find out if ustekinumab can cause side effects, which are unexpected or unwanted reactions from taking a drug. About 75 children will take part in this study worldwide. Participants will be in the study for a maximum of about 16 weeks.

Institution
MUSC
Recruitment Contact
Wounmee Yaka
8437924091
yaka@musc.edu

Daily Topical Rapamycin Therapy for the Treatment of Vitiligo

Date Added
March 1st, 2022
PRO Number
Pro00115924
Researcher
Ahmad Aleisa

List of Studies

Keywords
Adolescents, Autoimmune disease, Pediatrics, Skin
Summary

In current Dermatology practice, options for vitiligo remain limited. The purpose of this study is to determine if once daily dosed topical rapamycin is effective for the treatment of patients with vitiligo. Participants will apply either 0.1% topical rapamcyin or 0.001% topical rapamycin for six months to a lesion on one side of the body, and topical placebo to a corresponding lesion on the opposite side of the body. The study also aims to evaluate patient satisfaction and identify any adverse effects on these dosing regimens.

Institution
MUSC
Recruitment Contact
Chelsea Shope
8437549577
shopec@musc.edu

A Double Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of PRA023 in Subjects with Systemic Sclerosis Associated with Interstitial Lung Disease (SSc-ILD)

Date Added
February 22nd, 2022
PRO Number
Pro00117883
Researcher
Richard Silver

List of Studies


Keywords
Autoimmune disease, Drug Studies, Rare Diseases, Scleroderma, Skin
Summary

The purpose of this study is to test whether a drug called PRA023/MK7240 (the study drug) is a good treatment for patients with Systemic Sclerosis associated with Interstitial Lung Disease (SSc-ILD). The study drug PRA023/7240 is an investigational drug that is given by infusion every 4 weeks. An investigational drug is not approved by The US Food and Drug Administration. It can only be used in a research study like this one. In this study, PRA023/MK7240 will be compared with a placebo (dummy drug). The placebo will be a saline solution that does not have any study drug in it. The comparison with the placebo helps to determine whether the effects seen in your body is because of the PRA023/MK7240 or not. This is a randomized study meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. This will be done with the help of a computer-based program and you will have 50% chance of receiving either the study drug or placebo. The study is double-blinded study and 50 weeks long, meaning you and your study doctor will not know what you are receiving, the study drug or placebo.

The study is sponsored by Prometheus Biosciences, Inc., a subsidiary of Merck & Co., Inc. The study is being done at approximately 25 sites across the United States. The main portion of the study will require 15 visits to the MUSC main campus and will have the following procedures completed over the course of your participation: blood draw, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High Resolution Computed Tomography (HRCT)), electrocardiogram, as well as asked to complete surveys. If you complete the initial blinded treatment period of 50 weeks, the study doctor will discuss whether you are eligible to enter the open label period of the study, meaning no placebo. If you are eligible and agree, you will receive 500 mg of study drug once every 4 weeks for an additional 52 weeks. Compensation is available for participation.

Institution
MUSC
Recruitment Contact
Brittany Frasier
843-792-8613
frasibri@musc.edu

Multicenter Phenotype-Genotype Analysis of Vascular Anomalies and Related Syndromes

Date Added
December 24th, 2021
PRO Number
Pro00116848
Researcher
Lara Wine Lee

List of Studies


Keywords
Genetics, Pediatrics, Skin, Vascular
Summary

The purpose of this research study is to develop a better understanding of the cause and natural history of vascular anomalies and related syndromes. This study is being done in order to develop a better understanding of the cause of vascular anomalies in order to to improve care for people who are affected by these anomalies and related syndromes.

This study is being done at the University of Wisconsin-Madison (UW-Madison) and other sites in North America and Europe. A total of about 1000 people will participate in this study. About 20 – 30 people will take part in the study here at the Medical University of South Carolina.

Institution
MUSC
Recruitment Contact
Chelsea Shope
8437549577
shopec@musc.edu

A Randomized, Double-blind, Placebo-controlled, Repeat-dose, Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis

Date Added
September 14th, 2021
PRO Number
Pro00110634
Researcher
Richard Silver

List of Studies


Keywords
Autoimmune disease, Drug Studies, Rare Diseases, Scleroderma, Skin
Summary

The purpose of this study is to test whether a drug called HZN-825 (the study drug) is an appropriate treatment for patients with Systemic Sclerosis (SSc). HZN-825 is an investigational drug that taken by mouth as a pill. An investigational drug is not approved by The US Food and Drug Administration and can only be used in a research study, like this one.

In this study, HZN-825 will be compared with a placebo (a pill that looks like the study pill but does not have any study drug in it). The comparison with the placebo helps to determine whether the effects seen in your body is because of HZN-825 or not. This is a randomized study meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. This will be done with the help of a computer-based program, and you will have 67% chance of receiving HZN-825. The study is double-blinded, meaning you and your study doctor will not know what you are receiving, the study drug or placebo. Should an emergency arise, and it is determined necessary, your study doctor will be able to find out what medication group you have been assigned.

The study is sponsored by Horizon Therapeutics Ireland DAC. The study is being done at approximately 135 sites across the world. The study is 60 weeks long will require 12 visits to the MUSC main campus. During study participation, the following procedures will be completed: blood draw, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High-Resolution Computed Tomography (HRCT)), electrocardiogram, as well as being asked to complete surveys about your overall health and wellbeing. Compensation is available for participation

Institution
MUSC
Recruitment Contact
Brittany Frasier
843-792-8613
frasibri@musc.edu

Fibrotic Mechanisms in Systemic Sclerosis

Date Added
August 17th, 2021
PRO Number
Pro00111928
Researcher
Deanna Baker Frost

List of Studies


Keywords
Autoimmune disease, Skin
Summary

We plan to obtain skin biopsies from patients with systemic sclerosis and healthy donors to grow cells from biopsies. With those cells, we will use them in experiments to see why the cells from systemic sclerosis patients have more fibrosis compared to patients without systemic sclerosis.

Institution
MUSC
Recruitment Contact
Deanna Baker Frost
8437928461
bakerde@musc.edu

A Phase 1/2a, Controlled, Randomized, Multicenter Study Evaluating the Efficacy, Safety, Tolerability of StrataGraft Skin Tissue Overlay of Meshed Autograft (SOMA) in Treatment of Full-Thickness Thermal Burns

Date Added
July 6th, 2021
PRO Number
Pro00109484
Researcher
Steven Kahn

List of Studies


Keywords
Skin, Surgery
Summary

The objective of this study is to evaluate the efficacy of augmenting traditional autografting with Stratagraft. Stratagraft is a synthetic human-tissue based regenerative skin device, which was designed to decrease the amount of donor tissue needed to treat severe burn wounds. Patients who participate in this study are asked to complete assessments, such as wound photography, wound healing progression, and scar progression, at multiple timepoints during a 1 year period.

Institution
MUSC
Recruitment Contact
Jason Hirsch
843-792-8824
hirschj@musc.edu

Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

Date Added
April 6th, 2021
PRO Number
Pro00106369
Researcher
Chelsea Shope

List of Studies

Keywords
Adolescents, Pediatrics, Skin, Vascular
Summary

In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.

Institution
MUSC
Recruitment Contact
Alexandra Richmond
7577776673
ritteral@musc.edu

A Social Genomics Model to Explore Loneliness and Systemic Inflammation in an Older Adult Population with Chronic Venous Leg and Diabetic Foot Ulcers.

Date Added
August 28th, 2020
PRO Number
Pro00102546
Researcher
Teresa Kelechi

List of Studies


Keywords
Inflammation, Non-interventional, Psychiatry, Skin
Summary

Chronic wounds such as venous leg ulcers (CVLUs) and Diabetic Foot Ulcers affect millions of individuals worldwide, causing considerable suffering, disability and poor quality of life. The objective of this study is to assess stressors, symptoms, and biomarkers associated with lonely and non-lonely adults living with CVLUs and DFUs. The results from this study are expected to improve our understanding of the mechanisms in the body that are common to loneliness and inflammation and lead towards to the development of a tool that can predict wound healing potential among persons with chronic wounds.

Institution
MUSC
Recruitment Contact
Margaret Prentice
843 792 4771
prenticm@musc.edu



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