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 @ 4:07 AM
Ignite Modification Date:
2025-12-25 @ 4:07 AM
NCT ID:
NCT04207320
Eligibility Criteria:
Inclusion Criteria:
* Hemoglobin SS, SC, S-β0 Thalassemia, or SO-Arab Sickle Cell Disease
* Between the ages of 2 and 25 years (Stage 1: 10-25 years; Stage II: 2-25 years)
* Lack a fully matched family donor or fully matched unrelated donor register in the National Marrow Donor Program
* Partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait) phenotype
* SCD with Severe Phenotype, defined by the following criteria: Neurologic manifestations of sickle disease including cerebral vascular accident (CVA), transient ischemic event (TIA) or abnormal MRI findings suggestive of silent infarct; Two or more episodes of acute chest syndrome (ACS) requiring admission for transfusional or respiratory support including supplemental oxygen within \[two years\] of enrollment in study despite hydroxyurea therapy. Patients who cannot tolerate hydroxyurea and who experience multiple episodes of ACS will also be eligible; History of severe vaso-occlusive (VOC) disease requiring hospitalization and intravenous narcotics on 3 or more occasions per year over the two years prior to enrollment despite hydroxyurea therapy. Patients who cannot tolerate hydroxyurea and who experience multiple episodes of VOC will also be eligible; Other severe phenotype as evidenced by end organ dysfunction related to sickle cell disease.
Exclusion Criteria:
* Karnofsky or Lansky score \< 60%
* Acute hepatitis or evidence of moderate or severe portal fibrosis on biopsy. (Biopsy will be obtained if patient has been on chronic transfusion therapy \> 6 months or has a ferritin \> 1000 ng/ml) or AST or ALT \>5 times the upper limit of normal
* Severe renal impairment (as evidenced by creatinine clearance of \<50ml/minute glomerular filtration rate (GFR) \< 50% predicted normal)
* Cardiac function that demonstrates shortening fraction less than 26% by cardiac echocardiogram or pulmonary hypertension.
* Pregnant Female.
* Lactating female.
* Pulmonary function with baseline O2 saturation \<85% or Diffusing Capacity for Carbon Monoxide (DLCO) on pulmonary function testing (PFT) with a DLCO \<40%.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
2 Years
Maximum Age:
25 Years
Study:
NCT04207320
Study Brief:
Protocol Section:
NCT04207320