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

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

An Open-label, Expanded Access Protocol for Pediatric Patients with Sickle Cell Disease Who Have No Alternative Treatment Options

Date Added
April 13th, 2021
PRO Number
Pro00107737
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study is intended to provide open-label, early access to voxelotor for pediatric patients with Sickle Cell Disease between the ages of 4 to 11 years old who have no alternative treatment options. Voxelotor is approved by the Food and Drug Administration in patients ages 12 and up with Sickle Cell Disease. Voxelotor has not been approved by the Food and Drug Administration for patients less than 12 years of age. Participants will follow their standard of care schedule and receive supply of medication every 3 months during study period.

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

A PHASE 3B, PROSPECTIVE, OPEN-LABEL, UNCONTROLLED, MULTICENTER STUDY ON LONG-TERM SAFETY AND EFFICACY OF rVWF IN PEDIATRIC AND ADULT SUBJECTS WITH SEVERE VON WILLEBRAND DISEASE (VWD)

Date Added
October 27th, 2020
PRO Number
Pro00099367
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study will evaluate if Food and Drug Administration (FDA) approved recombinant von Willebrand Factor (rVWF) is safe and effective with long term use in adult and pediatric/adolescent patients with von Willebrand Disease (VWD). Dosing is an optional prophylactic (preventative) dosing on a weekly basis, or on demand (OD) for bleeding episodes and in the management of surgical bleeding. This study may last up to 3 years. You will have clinic visits in 3 month intervals during this time.

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

A phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis

Date Added
August 14th, 2018
PRO Number
Pro00079784
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study will assess the appropriate dosing and evaluate the safety of crizanlizumab in pediatric sickle cell disease patients. The study is for male and female subjects between the ages of 6 months to 17 years old who have experienced at least one pain crisis within a 12 month period. The drug is given via an IV infusion in an outpatient setting and has the potential to reduce the amount of sickle cell pain crisis a participant may experience. Participants can expected to participant in this study for up to 2 years.

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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