🏛️ Verified NIH Record • Updated 2025-01-29
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) (NCT04853576)
🔵 Active, Not Recruiting Phase1 / Phase2 📋 Clinical Research Protocol Sponsor: Editas Medicine, Inc.
🩺 Plain-English Summary (8th-Grade Level)
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).
Who can join: Review the eligibility criteria below.
Location: UCSF Benioff Children's Hospital — Oakland, California
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (24 Sites)
- UCSF Benioff Children's Hospital — Oakland, California
- Children's Hospital Colorado — Aurora, Colorado
- Smilow Cancer Hospital — New Haven, Connecticut
- Johns Hopkins All Children's Hospital — St. Petersburg, Florida
- Children's Healthcare of Atlanta — Atlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- University of Mississippi Medical Center — Jackson, Mississippi
- Hackensack University Medical Center — Hackensack, New Jersey
- Columbia University Medical Center - Department of Pediatrics — New York, New York
- Columbia University Medical Center — New York, New York
- The University of North Carolina at Chapel Hill — Chapel Hill, North Carolina
- Atrium Health — Charlotte, North Carolina
- University Hospitals Rainbow Babies & Children's Hospital — Cleveland, Ohio
- Cleveland Clinic — Cleveland, Ohio
- Nationwide Children's Hospital — Columbus, Ohio
- The James Cancer Hospital — Columbus, Ohio
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Medical University of South Carolina — Charleston, South Carolina
- Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers — Nashville, Tennessee
- Texas Oncology - Baylor Charles A. Sammons Cancer Center — Dallas, Texas
- Cook Children's — Fort Worth, Texas
- Ottawa Hospital Research Institute — Ottawa, Ontario
- Princess Margaret Cancer Centre — Toronto, Ontario
- Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.