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Clinical Trials in the USA / NCT05444894
Active, not recruiting Phase 1/2

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

NCT05444894 · tracked via the Priya Life Science USA tracker
Sponsor
Editas Medicine, Inc.
Phase
Phase 1/2
Started
2022-04-29
Last updated
2025-04-02

Condition(s) studied

Transfusion Dependent Beta ThalassemiaHemoglobinopathiesThalassemia MajorThalassemia Intermedia

Investigational drug(s) / intervention(s)

EDIT-301

EDIT-301: Administered by intravenous infusion after myeloablative conditioning with busulfan.

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
35 Years
Healthy volunteers
No
Key Inclusion Criteria: Diagnosis of Transfusion Dependent B-Thalassemia as defined by: * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and * History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent * Clinically stable and eligible to undergo autologous HSCT * Karnofsky Performance Status ≥ 70 Key Exclusion Criteria: * Available 10/10 human leukocyte antigen (HLA)-matched related donor * Prior HSCT or contraindications to autologous HSCT * Participants with associated a history of α-thalassemia and \> 1 alpha chain deletion, or alpha multiplications as documented in medical records * Participants with a history of other inherited hemoglobinopathy or thalassemic mutation (Hb S, C, D or other) as documented in medical records * Prior receipt of gene therapy * Inadequate bone marrow function, as defined by white blood cell count of \< 3 x 10\^9/L or a platelet count \< 100 x 10\^9/L (without hypersplenism), per investigator judgement * Inadequate organ function * Advanced liver disease * Any prior or current malignancy, or immunodeficiency disorder, * Immediate family member with a known or suspected Familial Cancer Syndrome * Clinically significant and active bacterial, viral, fungal, or parasitic infection

Primary outcome measure(s)

Trial sites (8)

FacilityCityRegionStatus
University of California San Francisco Oakland California
University of Minnesota Minneapolis Minnesota
Columbia University Medical Center - Department of Pediatrics New York New York
Columbia University Medical Center New York New York
Cleveland Clinic Cleveland Ohio
Children's Hospital of Philadelphia Philadelphia Pennsylvania
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers Nashville Tennessee
Princess Margaret Cancer Centre-University Health Network Toronto Ontario

More Editas Medicine, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05444894 on ClinicalTrials.gov ↗ ← All trials in the USA