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:
2025-12-24 @ 3:25 PM
Ignite Modification Date:
2025-12-24 @ 3:25 PM
NCT ID:
NCT00744692
Eligibility Criteria:
Inclusion Criteria:
* 0-21 years of age with a diagnosis of a immunodeficiency, congenital marrow failure syndrome, inborn error of metabolism, or hereditary anemia
* Appropriately matched related or unrelated umbilical cord blood unit with a cell dose ≥ 3 x 10e7cells/kg
* Performance score (lansky or karnofsky) greater than or equal to 70
* Adequate organ function (Creatinine ≤ 2.0 mg/dl and creatinine clearance ≥ 50 ml/min/1.73 m2; Hepatic transaminases (ALT/AST) ≤ 4 x normal; Shortening fraction \>26% or ejection fraction \>40% or \> 80% of normal value for age; Pulmonary function tests demonstrating CVC or FEV1/FVC of \>60% of predicted for age.)
* Informed consent
* Not pregnant or breast feeding
* Minimum life expectancy of at least 6 months
* HIV negative
* No uncontrolled infections at the time of cytoreduction
* Disease specific inclusion criteria
Exclusion Criteria:
* Patients with hemoglobinopathies \> 3 years of age
* UCB unit with a total nucleated cell count \< 3 x 10e7/kg or \> 2 antigen mismatching
* Available HLA-matched related living donor able to donate without previous UCB donation
* Allogeneic hematopoietic stem cell transplant within the previous 6 months
* Any active malignancy, MDS, or any history of malignancy
* Severe acquired aplastic anemia
* DLCO \< 60% of normal value for age; requirement for supplemental oxygen
* Uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms)
* Pregnancy or nursing mother
* HIV/HTLV seropositive, Hep B surface antigen positive, or HCV RNA positive by PCR
* Any condition that precludes serial follow-up
Healthy Volunteers:
False
Sex:
ALL
Maximum Age:
21 Years
Study:
NCT00744692
Study Brief:
Protocol Section:
NCT00744692