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)
Facility
City
Region
Status
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
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.
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