CRISPR breakthrough: One-Shot gene therapy aims to free kids from sickle cell pain crises
NCT ID NCT05329649
Summary
This study is testing a one-time treatment called CTX001 in children with severe sickle cell disease who don't respond well to the standard medication, hydroxyurea. The treatment uses the patient's own modified stem cells (a type of gene therapy using CRISPR technology) to try to stop the painful blockages in blood vessels (crises) that define the disease. The main goal is to see if children can go a full year without a single severe pain crisis after receiving the treatment.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HEMOGLOBINOPATHIES are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
-
Levine Children's Hospital - Hematology
Charlotte, North Carolina, 28203, United States
-
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
-
St.Mary's Hospital - Haematology Dept
London, United Kingdom
-
The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, 19104, United States
-
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
-
University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Conditions
Explore the condition pages connected to this study.