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Enrolling by invitation NCT06363760

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

— Observational Sickle Cell Disease Transfusion-dependent Beta-Thalassemia Hemoglobinopathies

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Safety and efficacy assessments.
Who it may be relevant to
Registry conditions: Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies. Basic parameters: 12 years — 50 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Canada
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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.

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.

Interventions

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

Primary outcome measures

  • 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 outcome measures (12)
  • 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]
  • 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]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

United States · 16 centers
  • UCSF Benioff Children's Hospital — Oakland
  • Children's Hospital Colorado — Aurora
  • Smilow Cancer Hospital — New Haven
  • Johns Hopkins All Children's Hospital — St. Petersburg
  • Children's Healthcare of Atlanta — Atlanta
  • University of Minnesota — Minneapolis
  • Columbia University Medical Center - Department of Pediatrics — New York
  • Columbia University Medical Center — New York
  • … and 8 more centers
Canada · 2 centers
  • Ottawa Hospital Research Institute — Ottawa
  • Princess Margaret Cancer Centre-University Health Network — Toronto

Identifiers

NCT: NCT06363760 · EDIT-301-LTFU-001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗