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The purpose of this multi-center study is to study the effects of AVR-RD-02 as a potential therapy people who have type 1 Gaucher disease, a long-term progressive disorder that can damage the liver, spleen and bone marrow. Conventional treatments, enzyme replacement therapy and substrate reduction therapy, have limitations and negative side effects. The AVR-RD-02 method collects the patient’s stem cells and genetically modifies them in the laboratory, adding a lentiviral vector capable of delivering and inserting a DNA sequence into the patient’s cells following intravenous infusion of the modified stem cells. The DNA is capable of producing normal GCase enzymes, which can mature and replace cells that are responsible for Gaucher disease.
The study is accepting people ages 18 through 50 who have type 1 Gaucher disease.
Males and Females: Ages 18 through 50
Participants will receive screening and baseline examinations to determine eligibility and to prepare them for stem cell collection. Following collection, participants will undergo a conditioning period to prepare them to have their modified stem cells transplanted via intravenous infusion. In the year-long post-transplant period, 15 assessments will occur, starting daily for 7 days and then over increasingly extended intervals.
Visits: About 30
Duration: 68 weeks
Study Description at National Institutes of Health
Genetic and Genomic Medicine Research
Damara Ortiz, MD
For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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