An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)

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, randomized, placebo-controlled, doubleblind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment

Date Added
September 8th, 2020
PRO Number
Pro00098276
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

For patients with thrombotic thrombocytopenic purpura (aTTP), research involving SHP655 added to current standard of care treatment for aTTP may be effective by reducing the severity of TTP episodes, length time on therapy and reducing clinical complications of the disease. rADAMTS-13 is an essential protein that is inactivated by antibodies. The study drug, SHP655, is given by intravenous injection (IV). It is an essential synthetic protein used to replace decreased volumes of the rADAMTS-13 protein which will prevent or lessen the symptoms of aTTP. The study will last approximately 1.5 years with the Treatment phase occurring during hospitalization for the treatment of aTTP illness and the follow-up phase that will consist of study visits occurring every week for 4 visits, then biweekly for the 2 visits with a final completion visit 1 month after the last follow-up visit lasting for a 3-month period. The length of time for each visit should last approximately 2 hours. If a recurrence or relapse of aTTP occurs during the follow-up phase, subjects will not receive additional study drug, but subjects will be followed on a bi-weekly visit schedule until remission is achieved or 4 months after the initial remission, whichever is sooner.

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

A Phase 1/2 randomized, double-blind, placebo-controlled, multicenter, ascending dose, safety and Pharmacokinetic/Pharmacodynamic PK/PD study of SHP655 (rADAMTS13) in sickle cell disease at baseline health and during acute vaso-occlusive crisis.

Date Added
August 25th, 2020
PRO Number
Pro00101087
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

Sickle cell disease (SCD) is a inherited disease that can cause sudden, severe pain. The management of this pain is accomplished through analgesic medications. This study for for male and female subjects between the ages of 18 and 65 years. This study will assess the appropriate dose and the evaluate the safety of SHP 655 in SCD patients at a baseline health state which is Part A of this study. The study medication is given by infusion as a single dose. SHP 655 is believed to increase blood flow and decrease blood cells from being trapped in low blood flow areas such as joints which can lead to tissue death.

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

A Prospective Phase II, Open-Label, Single-arm, Multicenter, Study to Assess Efficacy and Safety of SEG101 (crizanlizumab), in Sickle Cell Disease Patients with Priapism (SPARTAN)

Date Added
February 11th, 2020
PRO Number
Pro00091529
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study is to evaluate the effectiveness of crizanlizumab-an monoclonal antibody on male patients between 16 to 65 years of age with Sickle cell disease experiencing vaso-occlusive crisis (VOC) priapism. The study will review the number of VOC-priapism events, their duration of the episodes and requirement of opioid treatment. Male patients may also take Hydroxyruea (HU) during study but must be receiving HU for at least 14 weeks before screening and continue HU during study.

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, Randomized, Double-Blind, Placebo Controlled Study of Voxelotor (GBT440) in Pediatric Participants with Sickle Cell Disease Study (HOPE Kids 2)

Date Added
May 8th, 2018
PRO Number
Pro00076380
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study will assess the safety and efficacy of voxelotor with long term, daily oral dosing compared to placebo in pediatric participants (ages 9 months to 12 years old) with SCD as measured by improvement in anemia. Participants can expected to be in this study for about 52 weeks with at least 12 visits to the study center.

Institution
MUSC
Recruitment Contact
Joannie Hayes
843-876-8767
hayesj@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

A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults with Severe Sickle Cell Disease

Date Added
December 6th, 2016
PRO Number
Pro00056206
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders, Transplant
Summary

This study is meant to compare transplant to standard care (regular care) for sickle cell patients. By comparing the health outcomes for patients who receive bone marrow transplant to those patients who receive standard of care, this study will be able to determine whether the two treatments are the same, or if one is better than the other.

Institution
MUSC
Recruitment Contact
Brandi Day
843-792-3379
dayb@musc.edu



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