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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06363760
• Other study ID #: EDIT-301-LTFU-001
• Status: Not yet recruiting
• Start date: June 4, 2024
• Est. completion date: August 2040

Study information

• Verified date: April 2024
• Source: Editas Medicine, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 54
• Est. completion date: August 2040
• Est. primary completion date: August 2040
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 50 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• beta-Thalassemia
• Hemoglobinopathies
• Sickle Cell Disease
• Thalassemia
• Transfusion-dependent Beta-Thalassemia

Intervention

Other:
• Safety and efficacy assessments
Assessed throughout the duration of the study.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Editas Medicine, Inc.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse events of special interest (AESI)		up to 15 years post EDIT-301 infusion
Primary	Adverse events of interest (AEI)		up to 15 years post EDIT-301 infusion
Primary	All-cause mortality		up to 15 years post EDIT-301 infusion
Primary	Adverse events (AEs) related to EDIT-301		up to 15 years post EDIT-301 infusion
Primary	Serious adverse events (SAEs)		up to 15 years post EDIT-301 infusion
Secondary	Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion		up to 15 years post EDIT-301 infusion
Secondary	Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion		up to 15 years post EDIT-301 infusion
Secondary	Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants		up to 15 years post EDIT-301 infusion
Secondary	Proportion of TDT participants with sustained transfusion independence (TI) over time		up to 15 years post EDIT-301 infusion
Secondary	Proportion of TDT participants with sustained transfusion reduction (TR) over time		up to 15 years post EDIT-301 infusion
Secondary	Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants		up to 15 years post EDIT-301 infusion
Secondary	SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time		up to 15 years post EDIT-301 infusion
Secondary	SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time		up to 15 years post EDIT-301 infusion
Secondary	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
Secondary	Average HbF (pg) per RBC over time for SCD participants		up to 15 years post EDIT-301 infusion
Secondary	Complete Blood Count (CBC) red cell indices assay over time for SCD participants		up to 15 years post EDIT-301 infusion
Secondary	Average HbF (pg) per F-cell over time for SCD participants		up to 15 years post EDIT-301 infusion
Secondary	HbF and HbS concentration (g/dL) over time for SCD participants		up to 15 years post EDIT-301 infusion
Secondary	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
Secondary	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
Secondary	HbF concentration (g/dL) over time for TDT participants		up to 15 years post EDIT-301 infusion
Secondary	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.	up to 15 years post EDIT-301 infusion
Secondary	Proportion of TDT participants receiving iron chelation therapy over time.		up to 15 years post EDIT-301 infusion