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 @ 2:00 AM
Ignite Modification Date:
2025-12-25 @ 2:00 AM
NCT ID:
NCT03597594
Eligibility Criteria:
Inclusion Criteria - Transplant Recipient
* Age ≥2 months old at the time of chemotherapy administration
* A proven mutation as defined by direct sequencing of patient DNA
* Has a suitable matched sibling donor or matched unrelated donor (8/8) or single haplotype matched (≥3 of 6) family member donor
* Patient must fulfill pre-transplant evaluation:
* Left ventricular ejection fraction \>40% and no evidence of uncorrected congenital malformation with clinical symptomatology
* Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/min/1.73m2 or serum Creatinine ≤1.2mg/dL
* Resting pulse oximetry ≥90% on room or ≥95% on oxygen supplementation
* Lansky (age-dependent) performance score ≥50
* Bilirubin ≤3 times the upper limit of normal for age
* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age
Exclusion Criteria - Transplant Recipient
* Positive for HIV infection by genome PCR
* Presence of active malignancy
* A social situation indicating that the family may not be able to comply with protocol procedures and recommended medical care
* Presence of a medical condition indicating that survival will be dismal such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive infection that is refractory to medical therapy
Inclusion Criteria - Matched Sibling Donor and Haplocompatible Donor
* Fully matched sibling donor (8/8), or matched unrelated donor (8/8), or at least single haplotype matched (≥3 of 6) family member
* At least 1 year old (MSD) and at least 18 years of age (Haplocompatible)
* HIV negative
* Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female)
* Not breast feeding
* Regarding donation eligibility, is identified as either:
* Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR
* Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
2 Months
Study:
NCT03597594
Study Brief:
Protocol Section:
NCT03597594