This study is accepting new patients by invitation only

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

a study on Sickle Cell Anemia Beta Thalassemia Hemoglobinopathy Anemia

Summary

Eligibility
for people ages 12-50 (full criteria)
Location
at UCSF
Dates
study started
completion around

Description

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.

Details

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.

Keywords

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

Eligibility

You can join if…

Open to people ages 12-50

  • 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 CAN'T join 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.

Locations

  • UCSF Benioff Children's Hospital
    Oakland California 94609 United States
  • Children's Hospital Colorado
    Aurora Colorado 80045 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Editas Medicine, Inc.
ID
NCT06363760
Study Type
Observational
Participants
Expecting 54 study participants
Last Updated