A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Running, not enrolling · Phase 1/Phase 2
Conditions studied: Sickle Cell Disease, Hemoglobinopathies
In brief
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
Key facts
- Study ID
- NCT04853576
- Run by
- Editas Medicine, Inc.
- People needed
- 45
- Starts
- 2021-05-04
- Expected to finish
- 2025-08-01
- Last updated by the study team
- 2025-01-31
Who can join
Age: 12 and older, up to 50. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCSF Benioff Children's Hospital — Oakland, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Smilow Cancer Hospital — New Haven, Connecticut, United States
- Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States
- Children's Healthcare of Atlanta — Atlanta, Georgia, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- University of Mississippi Medical Center — Jackson, Mississippi, United States
- Hackensack University Medical Center — Hackensack, New Jersey, United States
- Columbia University Medical Center - Department of Pediatrics — New York, New York, United States
- Columbia University Medical Center — New York, New York, United States
- The University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
- Atrium Health — Charlotte, North Carolina, United States
- University Hospitals Rainbow Babies & Children's Hospital — Cleveland, Ohio, United States
- Cleveland Clinic — Cleveland, Ohio, United States
- Nationwide Children's Hospital — Columbus, Ohio, United States
- The James Cancer Hospital — Columbus, Ohio, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Medical University of South Carolina — Charleston, South Carolina, United States
- Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers — Nashville, Tennessee, United States
- Texas Oncology - Baylor Charles A. Sammons Cancer Center — Dallas, Texas, United States
- Cook Children's — Fort Worth, Texas, United States
- Ottawa Hospital Research Institute — Ottawa, Ontario, Canada
- Princess Margaret Cancer Centre — Toronto, Ontario, Canada
- Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec, Canada
Full record on ClinicalTrials.gov
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