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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 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.

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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).

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

FieldValue
Enrollment Target 45 participants
Start Date 2021-05-04
Est. Completion 2025-08
Phase Phase 1

Sponsor

Editas Medicine

3 total trials

What the Registry Record Tells You About NCT04853576

The ClinicalTrials.gov registry entry for NCT04853576 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 45 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (85% lower). The listed sponsor is Editas Medicine, which has 3 total studies on file at ClinicalTrials.gov.

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 reports 20 study locations spanning 15 distinct geographic areas - top geographies include 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.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.