Safety and effectiveness of Pradaxa oral pellet formulation for treatment of acute venous thromboembolic events (VTE) and/or for risk reduction of recurrence of VTE in pediatric patients aged 3 months to less than 12 years in a real world setting: a prospective non-interventional study conducted in the United States

Date Added
September 23rd, 2024
PRO Number
Pro00138643
Researcher
Shayla Bergmann

List of Studies


Keywords
Pediatrics
Summary

A study in the United States that looks at the safety and effectiveness of Pradaxa Pellets in children aged 3 months to less than 12 years who need treatment of a blood clot or who have had a blood clot and are at risk of developing another blood clot. This study is for children prescribed Pradaxa oral pellet as a standard of care.
Research visits will occur when your child starts taking Pradaxa Pellets and at 6 weeks, 3 months, 6 months, and 12 months. During those visits questions about your child's medical history and current medication and health during the study will be asked.

Institution
MUSC
Recruitment Contact
Layla Al Sarraf
843-876-5203
alsarral@musc.edu

An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)

Date Added
May 9th, 2022
PRO Number
Pro00114464
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with Sickle Cell Disease (SCD) in a real-world setting.

Participants who are currently taking or have been prescribed and initiating standard of care treatment with Oxbryta are eligible to participate. This registry will collect data that is recorded in participants' medical records and other off site sources. Study data will be collected a regular intervals and entered in case reports (CRFs) by electronic capture system (EDC) by the study staff. Participants will be on the study for 5 years after their first dose of Oxbryta treatment or until they choose to withdraw from the study. Participants who stop Oxbryta treatment prior to the 5 years will continue to be monitored at regular intervals unless they request to withdraw from study. Participants quality of life (QoL) questionnaires will be collected throughout the 5 years of the study.

Participants safety and tolerability will be assessed throughout the study data collection period by the study doctor and reported to the Sponsor.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease

Date Added
July 13th, 2021
PRO Number
Pro00107924
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

Vaso-occulsive crisis is a complication of Sickle cell disease in which the red blood cells (RBC) change shape, causing congestion within the blood vessels that leads to pain and tissue damage.

The study medication FT-4202, an oral tablet, is believed to reduce the rate of sickle cell polymerization and improve RBC membrane function, thereby reduction sickling of RBCs and their hemolysis (breakdown of red cells) that causes vascular obstruction and anemia.

This study will consist of a 52-week, randomized (volunteers are selected by chance to receive study either study medication or placebo) , placebo controlled (a placebo is a look-alike pill that contains no active medication). There will be 17 study visits.

The study is followed by a 52-week open label extension study in which all participants will receive study medication. There will be 11 study visits.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease

Date Added
February 28th, 2017
PRO Number
Pro00062418
Researcher
Shayla Bergmann

List of Studies


Keywords
Drug Studies, Pediatrics
Summary

This is a study to determine the use of recombinant Von Willebrand Factor (rVWF) in the treatment and control of nonsurgical bleeding episodes and bleeding during elective and emergency surgery in children with severe Von Willebrand Disease. The study will last approximately 14 months and will involve regular visits to a research clinic.

Institution
MUSC
Recruitment Contact
Lauren Card
843-792-5935
cardl@musc.edu



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