One-Shot CRISPR cure trial for devastating blood diseases
NCT ID NCT05477563
Summary
This study is testing a single treatment that uses CRISPR gene editing on a patient's own blood stem cells. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in people with severe beta-thalassemia and stop painful crises in those with severe sickle cell disease. The trial will enroll 26 participants to closely monitor the safety and effectiveness of this potential cure.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
RECRUITINGRome, Italy
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King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
RECRUITINGAl Mathar Ash Shamali, Saudi Arabia
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Levine Children's Hospital - Hematology
RECRUITINGCharlotte, North Carolina, 28203, United States
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New York Presbyterian Hospital - Morgan Stanley Children's Hospital
RECRUITINGNew York, New York, 10032, United States
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TriStar Medical Group Children's Specialists - Pediatric Oncology
RECRUITINGNashville, Tennessee, 37203, United States
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University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
RECRUITINGDüsseldorf, Germany