Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia (Mafia)
Fanconi Anemia, Severe Aplastic Anemia
About this trial
This is an interventional treatment trial for Fanconi Anemia focused on measuring Allogeneic Stem Cell Transplant, Fanconi Anemia, Severe Aplastic Anemia, fludarabine, campath, anti-CD45
Eligibility Criteria
Inclusion Criteria:
Diagnosis of Fanconi Anemia or other suspected DNA breakage/chromosomal instability syndromes, such as dyskeratosis congenita or Nijmegen breakage syndrome of all ages are eligible.
Diagnosis of Fanconi anemia confirmed by studies of peripheral blood or bone marrow sensitivity to mitomycin C or DEB or clinical evidence of other DNA breakage/chromosomal instability syndrome as determined by genetic testing or clinical diagnosis by a geneticist
Severe aplasia anemia as evidenced by a hypocellular bone marrow and at least 1 of the 3 criteria below: ANC < 500/mm3 Hemoglobin < 10 gm/dl with reticulocyte count < 1% Platelet count < 50,000/mm3
Availability of an HLA matched or mismatched (up to one haplotype) family member who has been documented not to have Fanconi anemia or of an unrelated HLA matched stem cell donor. Fully matched is defined at 6/6 match by high resolution DR based DNA typing.
Life expectancy greater than 6 weeks limited by diseases other than FA
Creatinine 2X normal for age or less
Karnofsky score 70% or more
Exclusion Criteria:
Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction less than 25%).
Patients with known allergy to rat serum products.
Patients with a severe infection that on evaluation by the Principal Investigator precludes ablative chemotherapy or successful transplantation.
Patients with severe personality disorder or mental illness.
Patients with documented HIV positivity.
Pregnant
NOTE: Patients who would be excluded from the protocol strictly for laboratory abnormalities can be included at the investigator's discretion after approval by the CCGT Protocol Review Committee and the FDA Reviewer.
Sites / Locations
- Methodist Hospital
- Texas Children's Hospital
Arms of the Study
Arm 1
Experimental
Single Arm Study: Stem Cell Transplant
CAMPATH-1H Anti-CD45 Fludarabine Stem Cell Infusion