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 @ 3:20 AM
Ignite Modification Date:
2025-12-25 @ 3:20 AM
NCT ID:
NCT02127905
Eligibility Criteria:
Inclusion Criteria:
* Patients must be \> 2 months and \< 21 years of age with a diagnosis of Fanconi anemia.
* Patients must have an HLA-A, B, DRB1 identical or 1 antigen mismatched related (non-sibling) or unrelated donor. Patients and donors will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. (Patients with a 2 antigen mismatched related donor will be eligible for the protocol but evaluated separately).
* Patients with FA must have high risk genotype or aplastic anemia (AA) or myelodysplastic syndrome without excess blasts.
* Aplastic anemia is defined as having at least one of the following:
1. platelet count \<20 x 109/L
2. ANC \<5 x 108/L
3. Hgb \<8 g/dL with at least one of the following:
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1. transfusion dependence
2. supportive care toxicity
* Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal anomalies.
* High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations)
* Adequate major organ function including:
* Cardiac: ejection fraction \>45%
* Renal: creatinine clearance \>40 mL/min.
* Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
* Karnofsky performance status \>70% or Lansky \>50%
* Women of child bearing age must be using adequate birth control and have a negative pregnancy test.
Exclusion Criteria:
* Available HLA-genotypically identical related donor.
* The harvested marrow (prior to TCD) should contain a minimum of 2.5 x 108 nucleated cells/kg recipient body weight with a goal of \>5.0 x 108 nucleated cells/kg recipient body weight.
* Positive lymphocytotoxic crossmatch against donor (T cells and B cells)
* History of gram negative sepsis or systemic fungal infection (proven or suspected based on radiographic studies).
* Myelodysplastic syndrome with excess blasts or leukemia.
* Active CNS leukemia at time of HCT.
* Malignant solid tumor (e.g. squamous cell carcinoma of the head/neck/cervix) within 2 years of HCT.
* Pregnant or lactating female.
* Prior radiation therapy preventing use of TBI 450 cGy.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
8 Weeks
Maximum Age:
21 Years
Study:
NCT02127905
Study Brief:
Protocol Section:
NCT02127905