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

Not yet recruiting

• Conditions: Hemoglobinopathies; Sickle Cell Disease; Transfusion-dependent Beta-Thalassemia
• Interventions: Other: Safety and efficacy assessments

• Registration Number: NCT06363760
• Lead Sponsor: Editas Medicine, Inc.

Brief 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.

Detailed 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.

Study Timeline

• Status Verified: 2024-04
• Study First Submitted: 2024-04-09
• Study First Submitted QC: 2024-04-09
• Last Update Submitted: 2024-04-09
• Study First Posted: 2024-04-12
• Last Update Posted: 2024-04-12
• Study Start: 2024-06-04
• Primary Completion: 2040-08
• Study Completion: 2040-08

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 54

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.

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
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
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
All-cause mortality	up to 15 years post EDIT-301 infusion

Secondary Outcome Measures

Name	Time	Method
SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time	up to 15 years post EDIT-301 infusion
HbF concentration (g/dL) over time for TDT participants	up to 15 years post EDIT-301 infusion
Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion	up to 15 years post EDIT-301 infusion
Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion	up to 15 years post EDIT-301 infusion
Complete Blood Count (CBC) red cell indices assay over time for SCD participants	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.	up to 15 years post EDIT-301 infusion
Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants	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.	up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion reduction (TR) over time	up to 15 years post EDIT-301 infusion
Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants	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	up to 15 years post EDIT-301 infusion
HbF and HbS concentration (g/dL) over time for SCD participants	up to 15 years post EDIT-301 infusion
Proportion of TDT participants receiving iron chelation therapy over time.	up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion independence (TI) over time	up to 15 years post EDIT-301 infusion
Average HbF (pg) per RBC over time for SCD participants	up to 15 years post EDIT-301 infusion
SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time	up to 15 years post EDIT-301 infusion
Average HbF (pg) per F-cell over time for SCD participants	up to 15 years post EDIT-301 infusion
Iron overload of TDT participants	up to 15 years post EDIT-301 infusion	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.

Trial Locations

Locations (1)

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States