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A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
NCT04853576 · View on ClinicalTrials.gov ↗
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04853576
The ClinicalTrials.gov registry entry for NCT04853576 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 45 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Editas Medicine, which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04853576 reports 20 study locations spanning 15 distinct geographic areas — top geographies include Ohio, New York, North Carolina. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04853576?
The interventions under investigation include: EDIT-301 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04853576?
This trial is sponsored by Editas Medicine, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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