Ireland
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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
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)

  • Proportion 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) — 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) — Screening through up to 24 months post EDIT-301 infusion

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
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