A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Public ClinicalTrials.gov record NCT04853576. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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).
Study identification
- NCT ID
- NCT04853576
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 45 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 12 Years to 50 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 4, 2021 (Actual)
- Primary completion
- Aug 2025 (Estimated)
- Completion
- Aug 2025 (Estimated)
- Last update posted
- Jan 31, 2025
2021 – 2025
United States locations
- U.S. sites
- 21
- U.S. states
- 15
- U.S. cities
- 18
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Smilow Cancer Hospital | New Haven | Connecticut | 06511 | — |
| Johns Hopkins All Children's Hospital | St. Petersburg | Florida | 33701 | — |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | — |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| Columbia University Medical Center - Department of Pediatrics | New York | New York | 10032 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| The University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | — |
| Atrium Health | Charlotte | North Carolina | 28204 | — |
| University Hospitals Rainbow Babies & Children's Hospital | Cleveland | Ohio | 44106 | — |
| Cleveland Clinic | Cleveland | Ohio | 44195 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| The James Cancer Hospital | Columbus | Ohio | 43210 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | — |
| Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers | Nashville | Tennessee | 37203 | — |
| Texas Oncology - Baylor Charles A. Sammons Cancer Center | Dallas | Texas | 75246 | — |
| Cook Children's | Fort Worth | Texas | 76104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04853576, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 31, 2025 · Synced Oct 27, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04853576 live on ClinicalTrials.gov.