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

NCT ID: NCT06363760

Last Updated: 2025-04-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

54 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-06-17

Study Completion Date

2040-08-31

Brief Summary

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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

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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

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Sickle Cell Disease Transfusion-dependent Beta-Thalassemia Hemoglobinopathies

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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All participants

All participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001)

Safety and efficacy assessments

Intervention Type OTHER

Assessed throughout the duration of the study.

Interventions

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Safety and efficacy assessments

Assessed throughout the duration of the study.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Participant must have received an EDIT-301 infusion as part of a clinical study.
* Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.

Exclusion Criteria

* Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.
Minimum Eligible Age

12 Years

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Editas Medicine, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCSF Benioff Children's Hospital

Oakland, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Smilow Cancer Hospital

New Haven, Connecticut, United States

Site Status

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Columbia University Medical Center - Department of Pediatrics

New York, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Atrium Health

Charlotte, North Carolina, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers

Nashville, Tennessee, United States

Site Status

Texas Oncology - Baylor Charles A. Sammons Cancer Center

Dallas, Texas, United States

Site Status

Cook Children's

Fort Worth, Texas, United States

Site Status

Ottawa Hospital Research Institute

Ottawa, Ontario, Canada

Site Status

Princess Margaret Cancer Centre-University Health Network

Toronto, Ontario, Canada

Site Status

Countries

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United States Canada

Other Identifiers

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EDIT-301-LTFU-001

Identifier Type: -

Identifier Source: org_study_id

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