Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide
Clinical Trials in the USA / NCT06363760
Enrolling by invitation Observational

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 USA 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
No
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)

FacilityCityRegionStatus
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 the USA

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

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease Active, not recruiting
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease Recruiting
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease Active, not recruiting
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD) Active, not recruiting
A Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis Starting soon
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 the USA