Self-Management for Youth Living with Sickle Cell Disease: SMYLS Multi-site Trial

Date Added
April 4th, 2023
PRO Number
Pro00127137
Researcher
Shannon Phillips

List of Studies


Keywords
Adolescents, Blood Disorders, Minorities, Pain, Pediatrics, Rare Diseases
Summary

The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms. 272 teens and adults with SCD will be enrolled in this study which is being conducted at the Medical University of South Carolina in Charleston SC., East Carolina University in Greenville NC., University of Miami in Miami FL., and the University of Alabama in Birmingham AL.

Institution
MUSC
Recruitment Contact
Shannon Phillips
843-792-9379
phillipss@musc.edu

A Phase 2a, double-blind, randomised, placebo-controlled, efficacy, and safety study of multiple doses of VIT-2763 in subjects with sickle cell disease (ViSion Serenity)

Date Added
February 28th, 2023
PRO Number
Pro00123111
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

The purpose of this study is to find out if VIT-2763 an experimental drug being developed by Vifor, the sponsor of this study, is safe for the treatment of improving iron deficiency in sickle cell diseased subjects. That means it can prevent hemolysis (the breakdown of red blood cells) and pain crisis in patients with sickle cell disease. You will be receiving VIT-2763 or placebo (study medication). A placebo looks like VIT-2763 in a capsule, but does not contain any active substance.

The total duration of your participation in the study will be up to 16 weeks. This involves a screening period of up to 4-weeks (28 days) during which the study doctor will check whether you can participate, 8 weeks of treatment and a 4-week follow-up after the treatment. You will have at least 7 visits(V1-V7) to the study site similar to a standard of care visit and may include 12-lead ECG and assessments of pain episodes.

Institution
MUSC
Recruitment Contact
Cheryl Alston
843-876-8652
alstonch@musc.edu

A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease

Date Added
February 1st, 2023
PRO Number
Pro00121710
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study will assess the appropriate dosing and evaluate the safety of NDec in patients with sickle cell disease. Patients may or may not be currently treated with Hydroxyurea to participate in this study. This study is for male and female patients above the age of 18 years who weight between 110 to 308 pounds. Patients must experience at least 2 but no more than 10 Vaso-occlusive crises within a 12-month period. This is an oral medication that is take twice a week and they will participate in this study for approximately 52 weeks.

Institution
MUSC
Recruitment Contact
Cheryl Alston
(843) 876-8652
alstonch@musc.edu

InSep in the Diagnosis and Prognosis of Emergency Department Patients with Suspected Infection and Suspected Sepsis (SEPSIS-SHEILD)"

Date Added
January 24th, 2023
PRO Number
Pro00121117
Researcher
Gary Headden

List of Studies


Keywords
Blood Disorders
Summary

Researchers want to find out more about how to manage and treat suspected infections and sepsis. Sepsis is a life-threatening condition in which your body is fighting a severe infection.

This study is a collection of health information, blood samples and possibly a nose swab. The samples collected will depend on your doctor's recommendation.

Institution
MUSC
Recruitment Contact
Robert Houck
18436931860
houckr@musc.edu

A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis

Date Added
January 11th, 2023
PRO Number
Pro00123694
Researcher
Kelli Williams

List of Studies


Keywords
Blood Disorders, Cancer/Other, Immune System
Summary

The purpose of this study is determine the optimal dose, efficacy and safety of an investigational drug (a new drug not yet approved by the U.S. Food and Drug Administration) in adults with Advanced Systemic Mastocytosis. The investigational drug is known as CGT9486 and will be taken daily orally. Participation in the study is expected to be approximately 6 years.

Institution
MUSC
Recruitment Contact
Natalie Naylon
8437925824
naylon@musc.edu

A Prospective, Multicenter, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of PMX Cartridge in Addition to Standard Medical Care for Patients with Endotoxemic Septic Shock

Date Added
December 7th, 2022
PRO Number
Pro00122530
Researcher
Blaithin McMahon

List of Studies


Keywords
Blood Disorders, Hypertension/ High Blood Pressure, Infectious Diseases, Kidney
Summary

The purpose of this study is to compare the safety and efficacy of the PMX cartridge ( Toramyxin) (in Addition to Standard Medical Care for Patients with Endotoxemic Septic Shock:
Eligible and consented subjects will be randomized to receive either the PMX cartridge (administered twice for 1½ to 2 hours per treatment session approximately 24 hours apart) plus standard medical care or standard medical care alone. For all subjects in whom treatment has been initiated, a follow-up visit (if they are still in the hospital) or a telephone call will be completed at Day 28 (or later) to determine their mortality status. In surviving subjects, a follow-up visit or telephone call to determine their mortality status will also take place at approximately three months (i.e. Day 90) and 12 months after the subject was randomized.

Institution
MUSC
Recruitment Contact
Marcie Pregulman
843-792-8166
pregulma@musc.edu

REAL Answers (Registry Expansion Analysis to Learn Answers)

Date Added
November 16th, 2022
PRO Number
Pro00124358
Researcher
Cathy Melvin

List of Studies