EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
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Phase
Phase 1/2
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
9estimated
Sites
8
Countries
Canada, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-04-29 | actual | When the study began enrolling |
| Primary completion | Sep 2025 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Dec 2025 | estimated | The whole study's end, after follow-up |
| First posted | 2022-07-06 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-04-02 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Transfusion Dependent Beta Thalassemia
- Hemoglobinopathies
- Thalassemia Major
- Thalassemia Intermedia
Intervention
- Genetic: EDIT-301 — also filed as renizgamglogene autogedtemcel, reni-cel
Primary outcomes
what the primary-completion date above is the date OFProportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days)
measured EDIT-301 infusion (Day 0) to 42 days post EDIT-301 infusion
Frequency and severity of adverse events (AEs) (incidence of AEs and Grade 3 or higher serious adverse events, using National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] v.5.0)
measured Screening through up to 24 months post EDIT-301 infusion
Publications
- PMID 41931048 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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