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NCT04853576 · ClinicalTrials.gov registry record · Phase 1
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
A Phase 1 study of Sickle Cell Disease and Hemoglobinopathies, sponsored by Editas Medicine.
- Active
- Registry status
- Phase 1
- Development phase
- 45
- Enrollment target
- 20
- Study locations
NCT04853576: Active Phase 1 study of Sickle Cell Disease and Hemoglobinopathies, sponsored by Editas Medicine.
NCT04853576 is a Phase 1 study of Sickle Cell Disease and Hemoglobinopathies that is active but no longer recruiting, run by Editas Medicine. The registered enrollment target is 45 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (85% lower). The trial reports 20 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04853576, a Phase 1 study of Sickle Cell Disease and Hemoglobinopathies, is active but no longer recruiting, sponsored by Editas Medicine.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 45 participants
- Enrollment target
- 20
- Study locations
Study Summary
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).
Conditions Studied
Interventions
- GENETIC EDIT-301
Study Locations (20)
Ohio
- University Hospitals Rainbow Babies & Children's Hospital - Cleveland
- Cleveland Clinic - Cleveland
- Nationwide Children's Hospital - Columbus
- The James Cancer Hospital - Columbus
New York
- Columbia University Medical Center - Department of Pediatrics - New York
- Columbia University Medical Center - New York
North Carolina
- The University of North Carolina at Chapel Hill - Chapel Hill
- Atrium Health - Charlotte
California
- UCSF Benioff Children's Hospital - Oakland
Colorado
- Children's Hospital Colorado - Aurora
Connecticut
- Smilow Cancer Hospital - New Haven
Florida
- Johns Hopkins All Children's Hospital - St. Petersburg
Georgia
- Children's Healthcare of Atlanta - Atlanta
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2021-05-04 |
| Est. Completion | 2025-08 |
| Phase | Phase 1 |
What the registry record for NCT04853576 still lists
NCT04853576 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 45 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (85% lower).
The record links to 2 conditions, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which EDIT-301 is the first listed.
NCT04853576 names 20 study sites across 15 states, led by Ohio, New York, North Carolina.
Frequently Asked Questions
What is clinical trial NCT04853576 about?
NCT04853576 is a clinical study titled "A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)". 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).
What is the current status of trial NCT04853576?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 45 participants. The study started on 2021-05-04. Estimated completion is 2025-08.
What conditions does trial NCT04853576 study?
This clinical trial studies the following conditions: Sickle Cell Disease, Hemoglobinopathies.
What interventions are being tested in trial NCT04853576?
The interventions under investigation include: EDIT-301 (GENETIC).
Who is sponsoring clinical trial NCT04853576?
This trial is sponsored by Editas Medicine, which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04853576 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Sickle Cell Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04853576's enrollment target sits among peer trials
45 116th of 213 higher than 95 of 213 other Sickle Cell Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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