NCT06363760

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 54 (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 long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.

Detailed description

This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.

Conditions

• Sickle Cell Disease
• Transfusion-dependent Beta-Thalassemia
• Hemoglobinopathies

Interventions

Type	Name	Description
OTHER	Safety and efficacy assessments	Assessed throughout the duration of the study.

Timeline

• Start date: 2024-06-17
• Primary completion: 2040-08-01
• Completion: 2040-08-01
• First posted: 2024-04-12
• Last updated: 2025-04-02

Locations

18 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study