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Enrolling by Invitation

NCT Number: NCT06363760

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

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia Transfusion-dependent Beta-Thalassemia beta-Thalassemia

Age range

12 year–50 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Ottawa Hospital Research Institute, Ottawa, Ontario, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Safety and efficacy assessments

Other

Assessed throughout the duration of the study.

Primary outcomes

  1. Adverse events of special interest (AESI)

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

  2. Adverse events of interest (AEI)

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

  3. All-cause mortality

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

  4. Adverse events (AEs) related to EDIT-301

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

  5. Serious adverse events (SAEs)

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

Secondary outcomes

  1. 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

  2. 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

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

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

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

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

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

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

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

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

  7. 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

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

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

  9. 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

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

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

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

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

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

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

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

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

  14. 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

  15. 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

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

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

  17. Iron overload of TDT participants

    Time frame: 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.

  18. Proportion of TDT participants receiving iron chelation therapy over time.

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

Sponsors and collaborators

Lead sponsor

Editas Medicine, Inc.

Industry

Registry information

Important dates

Study start
2024
Primary completion
2040
Study completion
2040
First posted
Apr 12, 2024
Registry last updated
Apr 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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