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Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed By Donor Bone Marrow Transplant, Cyclophosphamide, Mycophenolate Mofetil, and Tacrolimus in Treating Patients With Immunodeficiency or Noncancerous Inherited Disorders

Complete title: HLA-haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-dose Cyclophosphamide, TBI and Fludarabine; Postgrafting Immunosuppression Will Consist of a Single Low Dose of Cyclophosphamide, MMF and Tacrolimus

Research Study Number       2032.00
    
Principal Investigator       Lauri Burroughs, MD
    
Phase       II

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Research Study Description

This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant, cyclophosphamide, mycophenolate mofetil, and tacrolimus in treating patients with immunodeficiency or noncancerous inherited disorders. Giving low doses of chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclophosphamide, mycophenolate mofetil, and tacrolimus after transplant may stop this from happening.

Eligibility Criteria (must meet the following to participate in this study)

Ages Eligible for Study: up to 54 Years

Genders Eligible for Study: Both

Inclusion Criteria:

- Primary immunodeficiency disorder or other nonmalignant inherited disease (except Fanconi anemia) treatable by allogeneic HCT

- Patients with pre-existing medical conditions or other factors that renders them at high risk for regimen related toxicity or ineligible for conventional myeloablative HCT and who do not have HLA-matched related or unrelated donors

- Patients with a related donor who is identical for one HLA haplotype

- Acquired aplastic anemia: Severe aplastic anemia (SAA) is defined as follows:

- * Bone marrow cellularity < 25%, or marrow cellularity < 50% but with < 30% residual hematopoietic cells

- * Two out of three of the following (in peripheral blood): neutrophils < 0.5 x 10^9/L; platelets < 20 x 10^9/L; reticulocytes < 20 x 10^9/L

- * SAA diagnostic criteria may be applied to assessment at initial diagnosis or follow-up assessments

- DONOR: Related donors who are identical for one HLA haplotype (bone marrow will be the only allowed stem cell source)

Other eligibility criteria may apply.

Exclusions (conditions that would prevent participation in this study)

- Fanconi anemia

- Suitably HLA-matched related or unrelated donors

- Patients with metabolic storage diseases who have severe central nervous system (CNS) involvement of disease, defined as IQ score < 70

- Cardiac ejection fraction < 30% (or, if unable to obtain ejection fraction, shortening fraction < 26%) on multiple-gated acquisition (MUGA) scan or cardiac echo, symptomatic coronary artery disease, or other cardiac failure requiring therapy

- Patients with a history of, or current cardiac disease should be evaluated with appropriate cardiac studies and/or cardiology consult; patients with a shortening fraction of < 26% must be seen by cardiology for approval

- Poorly controlled hypertension despite anti-hypertensive medications

- Patients with clinical or laboratory evidence of liver disease will need to be evaluated for the cause of the liver disease, its clinical severity in terms of liver function and the degree of portal hypertension; patients will be excluded if they are found to have fulminant liver failure, cirrhosis of the liver with evidence of portal hypertension, bridging fibrosis, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin > 3 mg/dl, or symptomatic biliary disease

- Seropositive for human immunodeficiency virus (HIV)

- Females who are pregnant (beta- human chorionic gonadotropin [B-HCG]+) or breast-feeding

- Fertile men or women who are unwilling to use contraceptives during HCT and up to 12 months post-treatment

- Patients with fungal pneumonia with radiological progression after receipt of amphotericin formulation or mold-active azoles for greater than 1 month will not be eligible for this protocol

- DONOR: Donor-recipient pairs in which the HLA-mismatch is only in the host-versus-graft (HVG) direction; patients are homozygous and donor is heterozygous

- DONOR: Donors who are not expected to meet the minimum target dose of marrow cells (1 x 10^8 nucleated cells/kg recipient Ideal Body Weight)

- DONOR: HIV-positive donors

- DONOR: A positive anti-donor cytotoxic cross match is absolute donor exclusion

- DONOR: < 6 months old and > 75 years old

Other exclusion criteria may apply.



Research Study Number       2032.00
    
Contact       Seattle Cancer Care Alliance Intake Office
    
Telephone       800-804-8824 / 206-288-1024
    
   

Keywords
Bone Marrow and Hematopoietic Stem Cell Transplant (BMT and HSCT); Immunodeficiency Syndromes; Severe Combined Immunodeficiency Syndrome (SCID); Non-malignant Condition

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