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

NCT06363760 · tracked via the Priya Life Science Canada tracker

Sponsor

Editas Medicine, Inc

Phase

Observational

Started

2024-06-17

Last updated

2025-04-02

Condition(s) studied

Sickle Cell DiseaseTransfusion-dependent Beta-ThalassemiaHemoglobinopathies

Investigational drug(s) / intervention(s)

Safety and efficacy assessments

Safety and efficacy assessments: Assessed throughout the duration of the study.

Study 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.

Eligibility

Sex

ALL

Min age

12 Years

Max age

50 Years

Healthy volunteers

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.

Primary outcome measure(s)

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

Trial sites (18)

Facility	City	Region
UCSF Benioff Children's Hospital	Oakland	California
Children's Hospital Colorado	Aurora	Colorado
Smilow Cancer Hospital	New Haven	Connecticut
Johns Hopkins All Children's Hospital	St. Petersburg	Florida
Children's Healthcare of Atlanta	Atlanta	Georgia
University of Minnesota	Minneapolis	Minnesota
Columbia University Medical Center - Department of Pediatrics	New York	New York
Columbia University Medical Center	New York	New York
Atrium Health	Charlotte	North Carolina
Cleveland Clinic	Cleveland	Ohio
Nationwide Children's Hospital	Columbus	Ohio
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
Medical University of South Carolina	Charleston	South Carolina
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers	Nashville	Tennessee
Texas Oncology - Baylor Charles A. Sammons Cancer Center	Dallas	Texas
Cook Children's	Fort Worth	Texas
Ottawa Hospital Research Institute	Ottawa	Ontario
Princess Margaret Cancer Centre-University Health Network	Toronto	Ontario

More Editas Medicine, Inc. trials in Canada

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) Active, not recruiting

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) Active, not recruiting

Other trials for the same condition

Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation Active, not recruiting

Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor Recruiting

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) Active, not recruiting

The BENeFiTS Trial in Beta Thalassemia Intermedia Enrolling by invitation

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06363760 on ClinicalTrials.gov ↗ ← All trials in Canada