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EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCT05444894 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2025-04-02

No results posted yet for this study

Summary

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

Conditions

  • Transfusion Dependent Beta Thalassemia
  • Hemoglobinopathies
  • Thalassemia Major
  • Thalassemia Intermedia

Interventions

GENETIC

EDIT-301

Administered by intravenous infusion after myeloablative conditioning with busulfan.

Sponsors & Collaborators

  • Editas Medicine, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-04-29
Primary Completion
2025-09-30
Completion
2025-12-31
FDA Drug
Yes

Countries

  • United States
  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05444894 on ClinicalTrials.gov