Unrelated HSCT in Patients With Fanconi Anemia
Withdrawn before enrolling · Not applicable
Conditions studied: Fanconi Anemia
In brief
The protocol is designed for the compassionate treatment of patients with Fanconi Anemia who do not have an HLA-matched sibling donor. The purpose of this study is to determine the likelihood of engraftment in Fanconi Anemia patients using total body irradiation (TBI), cyclophosphamide (CY), fludarabine (FLU) and antithymocyte globulin (ATG) followed by an unrelated donor hematopoietic cell transplant with T-cell depletion using the CliniMACS device.
Key facts
- Study ID
- NCT02127905
- Run by
- Neena Kapoor, M.D.
- People needed
- 0
- Starts
- 2011-03-01
- Expected to finish
- 2016-03-01
- Last updated by the study team
- 2017-07-11
Who can join
Age: 0 and older, up to 21. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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:
- platelet count <20 x 109/L
- ANC <5 x 108/L
- Hgb <8 g/dL with at least one of the following:
- <!-- -->
- transfusion dependence
- 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.
You may not qualify if…
- 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.
Where it is running
- Children's Hospital Los Angeles — Los Angeles, California, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.