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 @ 4:13 AM
Ignite Modification Date:
2025-12-25 @ 4:13 AM
NCT ID:
NCT01745120
Eligibility Criteria:
Inclusion criteria:
* Participants between 12 and 35 years of age, inclusive, at the time of consent/assent, and able to provide written consent/assent, if applicable.
* Diagnosis of β-thalassemia major and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years.
* Eligible for allogeneic bone marrow transplant.
* Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
Exclusion criteria:
* Positive for presence of human immunodeficiency virus type 1 or 2 (HIV 1 and HIV 2).
* A white blood cell (WBC) count \<3 × 10\^9/L, and / or platelet count \<100 × 10\^9/L if not due to hypersplenism.
* Uncorrected bleeding disorder.
* Any prior or current malignancy or myeloproliferative or immunodeficiency disorder.
* Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis).
* Receipt of an allogeneic transplant.
* Advanced liver disease, including persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value \>3 × the upper limit of normal, liver biopsy demonstrating cirrhosis, extensive bridging fibrosis, or active hepatitis.
* Kidney disease with a calculated creatinine clearance \<30% normal value.
* Uncontrolled seizure disorder.
* Diffusion capacity of carbon monoxide (DLco) \<50% of predicted (corrected for hemoglobin).
* A cardiac T2\* \<10 ms by magnetic resonance imaging (MRI).
* Any other evidence of severe iron overload that, in the Investigator's opinion, warrants exclusion.
* Clinically significant pulmonary hypertension, as defined by the requirement for ongoing pharmacologic treatment or the consistent or intermittent use of supplemental home oxygen.
* Participation in another clinical study with an investigational drug within 30 days of Screening.
* Any prior or current malignancy or myeloproliferative disorder.
* Prior receipt of gene therapy.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
12 Years
Maximum Age:
35 Years
Study:
NCT01745120
Study Brief:
Protocol Section:
NCT01745120