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

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Editas Medicine

Status

Begins enrollment this month

Conditions

Hemoglobinopathies
Sickle Cell Disease
Transfusion-dependent Beta-Thalassemia

Treatments

Other: Safety and efficacy assessments

Study type

Observational

Funder types

Industry

Identifiers

NCT06363760
EDIT-301-LTFU-001

Details and patient eligibility

About

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.

Full description

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.

Enrollment

54 estimated patients

Sex

All

Ages

12 to 50 years old

Volunteers

No 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.

Trial design

54 participants in 1 patient group

All participants
Description:
All participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001)
Treatment:
Other: Safety and efficacy assessments

Trial contacts and locations

0

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Data sourced from clinicaltrials.gov

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