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

N/AEnrolling By InvitationNCT06363760

Editas Medicine, Inc.54 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Sickle Cell Disease Transfusion-dependent Beta-Thalassemia Hemoglobinopathies

Interventions

Safety and efficacy assessmentsOTHER

Assessed throughout the duration of the study.

Eligibility

Sex: ALLMin age: 12 YearsMax age: 50 Years

Medical Language ↔ Plain English

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.

Locations (18)

UCSF Benioff Children's Hospital

Oakland, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Outcomes

Primary Outcomes

Adverse events of special interest (AESI)

Time frame: up to 15 years post EDIT-301 infusion

Adverse events of interest (AEI)

Time frame: up to 15 years post EDIT-301 infusion

All-cause mortality

Time frame: up to 15 years post EDIT-301 infusion

Adverse events (AEs) related to EDIT-301

Time frame: up to 15 years post EDIT-301 infusion

Serious adverse events (SAEs)

Time frame: up to 15 years post EDIT-301 infusion

Secondary Outcomes

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

Time frame: up to 15 years post EDIT-301 infusion

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

Time frame: up to 15 years post EDIT-301 infusion

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

Time frame: up to 15 years post EDIT-301 infusion

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

Time frame: up to 15 years post EDIT-301 infusion

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

Time frame: up to 15 years post EDIT-301 infusion

Data from ClinicalTrials.gov

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Smilow Cancer Hospital

New Haven, Connecticut, United States

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

University of Minnesota

Minneapolis, Minnesota, United States

Columbia University Medical Center - Department of Pediatrics

New York, New York, United States

Columbia University Medical Center

New York, New York, United States

Atrium Health

Charlotte, North Carolina, United States

Cleveland Clinic

Cleveland, Ohio, United States

...and 8 more locations

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

Time frame: up to 15 years post EDIT-301 infusion

SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time

Time frame: up to 15 years post EDIT-301 infusion

SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time

Time frame: up to 15 years post EDIT-301 infusion

SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time

Time frame: up to 15 years post EDIT-301 infusion

Average HbF (pg) per RBC over time for SCD participants

Time frame: up to 15 years post EDIT-301 infusion

Complete Blood Count (CBC) red cell indices assay over time for SCD participants

Time frame: up to 15 years post EDIT-301 infusion

Average HbF (pg) per F-cell over time for SCD participants

Time frame: up to 15 years post EDIT-301 infusion

HbF and HbS concentration (g/dL) over time for SCD participants

Time frame: up to 15 years post EDIT-301 infusion

The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.

Time frame: up to 15 years post EDIT-301 infusion

Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g/dL of all Hb variants, excluding HbA) over time for TDT participants.

Time frame: up to 15 years post EDIT-301 infusion

HbF concentration (g/dL) over time for TDT participants

Time frame: up to 15 years post EDIT-301 infusion

Iron overload of TDT participants

As measured by a) Liver iron concentration (LIC) as assessed by R2\* magnetic resonance imaging (MRI) for the first 3 years in this study; b) Cardiac iron concentration (CIC) as assessed by T2\* MRI for the first 3 years of this study; c) Serum ferritin levels over time.

Time frame: up to 15 years post EDIT-301 infusion

Proportion of TDT participants receiving iron chelation therapy over time.

Time frame: up to 15 years post EDIT-301 infusion