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 @ 3:28 AM
Ignite Modification Date:
2025-12-25 @ 3:28 AM
NCT ID:
NCT02833805
Eligibility Criteria:
Inclusion Criteria:
* Confirmed diagnosis of inherited or acquired severe aplastic anemia (SAA)
* One of the following available donors:
1. HLA-haploidentical relative
2. If recipient is \>= 40 years old, may use HLA-matched related donor
3. For recipients with inherited bone marrow failure syndromes (IBMFS) with clear evidence of same disorder in potential related donors, may use 10/10 matched unrelated donor
* Recipient and/or legal guardian must sign protocol informed consent
* Donor must be willing to donate bone marrow
* Left ventricular ejection fraction (LVEF) \>= 40%. For recipients \< 13 years old, shortening fraction \>= 26% may be used instead.
* Bilirubin \< 3 x upper limit of normal (ULN) for age, unless patient has Gilbert's disease
* aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 5 x ULN for age
* For patients \>= 13 years old: estimated creatinine clearance \> 50 mL/min using Cockcroft-Gault formula and actual body weight
* For patients \>= 1 but \< 13 years old: glomerular filtration rate (GFR) estimated by updated Schwartz formula \>= 90 mL/min/1.73 m\^2. If estimated GFR is \< 90 mL/min/1.73 m\^2, 24-hour measured creatinine clearance must be \> 50 mL/min/1.73 m\^2.
* For patients \>= 8 years old, diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) \> 40%; forced expiratory volume at one second (FEV1) \> 50%; forced vital capacity (FVC) \> 50%
* For patients \< 8 years old or unable to undergo pulmonary function testing: no evidence of dyspnea at rest; no need for supplemental oxygen; oxygen saturation \> 92% on room air
* Karnofsky/Lansky status (depending on age) \>= 70%
* Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time. If unwilling, they must agree to complete abstinence.
Exclusion Criteria:
* Previous administration of immunosuppressive therapy for SAA.
* Fanconi anemia. At minimum, this diagnosis must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow in patients \< 30 years old.
* Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on bone marrow examination
* Presence of anti-donor antibodies
* Prior allogeneic stem cell transplant
* Prior solid organ transplant
* Uncontrolled bacterial, viral, or fungal infection
* HIV seropositivity
* Active hepatitis B or C infection determined by serology and/or nucleic acid testing (NAT)
* Pregnancy or active breastfeeding
* Prior malignancies except: resected basal carcinoma or treated cervical carcinoma in situ; cancer treated with curative intent \> 5 years previously. Other prior cancers will not be allowed unless approved by the PI.
Healthy Volunteers:
False
Sex:
ALL
Study:
NCT02833805
Study Brief:
Protocol Section:
NCT02833805