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

NCT06363760 | Enrolling By Invitation FDA Drug

• 1 other identifier: EDIT-301-LTFU-001
• Study Type: observational
• Target: 54
• Locations: 2 countries
• Sites: 18

Brief 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.

Conditions

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

Keywords

Sickle Cell Disease; Sickle Cell Anemia; Sickling Disorder Due to Hemoglobin S; Beta-Thalassemia; Thalassemia Major; Thalassemia Intermedia; Hemoglobinopathies; CRISPR-Cas12a; Autologous CD34+; Genetic Diseases, Inborn; Hematologic Diseases; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic

Outcome Measures

Primary Outcomes (5)

• Adverse events of special interest (AESI)

  up to 15 years post EDIT-301 infusion

• Adverse events of interest (AEI)

  up to 15 years post EDIT-301 infusion

• All-cause mortality

  up to 15 years post EDIT-301 infusion

• Adverse events (AEs) related to EDIT-301

  up to 15 years post EDIT-301 infusion

• Serious adverse events (SAEs)

  up to 15 years post EDIT-301 infusion

Secondary Outcomes (18)

• Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion

  up to 15 years post EDIT-301 infusion

• Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion

  up to 15 years post EDIT-301 infusion

• Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants

  up to 15 years post EDIT-301 infusion

• Proportion of TDT participants with sustained transfusion independence (TI) over time

  up to 15 years post EDIT-301 infusion

• Proportion of TDT participants with sustained transfusion reduction (TR) over time

  up to 15 years post EDIT-301 infusion

Study Arms (1)

All participants

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

Other: Safety and efficacy assessments

Interventions

Safety and efficacy assessments OTHER

Assessed throughout the duration of the study.

All participants

Eligibility Criteria

• Age: 12 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The study population will be subjects currently enrolled in any one of the two parent studies ( EM-SCD-301-001 or EM-301-BThal-001)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Editas Medicine, Inc.: lead

Study Sites (18)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Smilow Cancer Hospital

New Haven, Connecticut, 06511, United States

Location

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55410, United States

Location

Columbia University Medical Center - Department of Pediatrics

New York, New York, 10032, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Atrium Health

Charlotte, North Carolina, 28204, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

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

Nashville, Tennessee, 37203, United States

Location

Texas Oncology - Baylor Charles A. Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

Cook Children's

Fort Worth, Texas, 76104, United States

Location

Ottawa Hospital Research Institute

Ottawa, Ontario, K1H 8L6, Canada

Location

Princess Margaret Cancer Centre-University Health Network

Toronto, Ontario, M5G 2M9, Canada

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobinopathies; beta-Thalassemia; Genetic Diseases, Inborn; Hematologic Diseases; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic

Interventions

Safety

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Thalassemia

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2024
• First Posted: April 12, 2024
• Study Start: June 17, 2024
• Primary Completion (Estimated): August 1, 2040
• Study Completion (Estimated): August 1, 2040
• Last Updated: April 2, 2025

Record last verified: 2025-03

Locations

CA (2); US (16)