Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 1:46 AM
Ignite Modification Date:
2025-12-25 @ 1:46 AM
NCT ID:
NCT05444894
Eligibility Criteria:
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
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
18 Years
Maximum Age:
35 Years
Study:
NCT05444894
Study Brief:
Protocol Section:
NCT05444894