A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion Save

Date Added
August 8th, 2017
PRO Number
Pro00068449
Researcher
John Lazarchick

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Keywords
Blood Disorders, Drug Studies, Rare Diseases
Summary

This is a first-in-humans gene therapy study for subjects diagnosed with severe hemophilia A. A one time infusion of the gene therapy product (BAX888) will be given with the hope of eliminating or reducing the need for prophylactic and/or on-demand use of FVIII concentrate therapy. Up to 10 subjects will be enrolled study-wide with up to two subjects enrolled at MUSC. This study will require weekly visits to the study clinic after initial infusion for the first 15 weeks followed by monthly visits for the first year. Compensation will be provided.

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

Safety, tolerability, and pharmacokinetics study of single and multiple subcutaneous doses of turoctocog alfa pegol in patients with haemophilia A Save

Date Added
March 14th, 2017
PRO Number
Pro00063209
Researcher
John Lazarchick

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Keywords
Blood Disorders, Drug Studies
Summary

This trial is a first-in-human clinical trial with subcutaneous injections of turoctocog alfa pegol (N8-GP). You must be male, age 12 or older with a diagnosis of hemophilia A to participate. The purpose of the trial is to investigate the potential effect of N8-GP
on prevention of bleeds in previously treated patients with severe hemophilia A. This study would require approximately 10 visits to the research clinic over 5 months. Compensation will be provided for time and travel.

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

A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Hemophilia A Save

Date Added
March 26th, 2013
PRO Number
Pro00022822
Researcher
John Lazarchick

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Keywords
Blood Disorders
Summary

The purpose of this research trial is to find out how effective a new Factor VIII (FVIII) product called N8-GP is in prevention and treatment of bleeding episodes, and to find out if there are any unwanted effects related to its use.

Institution
MUSC
Recruitment Contact
Lisa Kuisel
843-792-0560
kuisel@musc.edu

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