NCT04853576

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 45 (estimated)
• Sponsor: Editas Medicine, Inc. · Industry
• Sex: All
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. Planned study subjects will be comprised of male and female adult and adolescent subjects with severe SCD, from 12 to 50 years of age, inclusive.

Conditions

• Sickle Cell Disease
• Hemoglobinopathies

Interventions

Type	Name	Description
GENETIC	EDIT-301	Administered by IV infusion after myeloablative conditioning with busulfan.

Timeline

• Start date: 2021-05-04
• Primary completion: 2025-08-01
• Completion: 2025-08-01
• First posted: 2021-04-21
• Last updated: 2025-01-31

Locations

24 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study