Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Editas Medicine, Inc.
This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive
Age
18–35
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
