One-Time gene therapy aims to free patients from sickle cell pain crises

NCT ID NCT05456880

Ongoing Disease control Sponsor: Beam Therapeutics Inc. Source: ClinicalTrials.gov ↗

Summary

This study is testing a single treatment that uses a patient's own blood stem cells, which are edited in a lab to potentially correct the underlying cause of sickle cell disease. The goal is to see if this one-time procedure is safe and can reduce the severe, painful episodes (crises) that patients experience. The study is enrolling a small group of young people with severe forms of the disease.

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

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Contacts and locations

Show contact details

Locations

Boston Children's Hospital

Boston, Massachusetts, 02215, United States
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital

Atlanta, Georgia, 30329, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Columbia University Irving Medical Center

New York, New York, 10032, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Henry Ford Cancer Center

Detroit, Michigan, 48202, United States
Mayo Clinic Florida

Jacksonville, Florida, 32224, United States
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
St Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
The Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203, United States
The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States
University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States
University of Miami

Miami, Florida, 33136-1005, United States
University of Minnesota

Minneapolis, Minnesota, 55455, United States
Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.

SICKLE CELL DISEASE