Brief Title
Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia
Brief Summary
OBJECTIVES:
I. Determine the effect of supplementation with donor T-cell depleted, CD34+ peripheral blood
stem cells on durable engraftment and incidence of graft-versus-host disease in patients with
aplastic anemia undergoing allogeneic bone marrow transplantation.
Detailed Description
PROTOCOL OUTLINE: Bone marrow and peripheral blood stem cells (PBSC) are harvested from a HLA
identical or 1 antigen mismatched related donor. PBSC are selected for CD34+ cells and T
cells are depleted.
Patients receive cyclophosphamide IV over 2 hours on days -6 to -3 and anti-thymocyte
globulin IV with methylprednisolone IV over 10-12 hours on days -5 to -3. T-cell depleted
PBSC and bone marrow are infused on day 0. Patients receive cyclosporine IV over 12-24 hours
on days -1 to 120 followed by a taper and methylprednisolone IV on days 7-64 for
graft-versus-host disease prophylaxis.
Patients are followed every 30 days for 1 year, every 60 days for 2 years, and then as needed
for a minimum of 3 years.
Study Type
Interventional
Condition
Graft Versus Host Disease
Intervention
anti-thymocyte globulin
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
3
Start Date
April 2001
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Diagnosis of life-threatening or transfusion-dependent marrow or immune-mediated
failure involving one or more hematopoietic cell lines evidenced by one or more of the
following: Granulocyte count less than 500/mm3 Platelet count less than 20,000/mm3
Absolute reticulocyte count less than 60,000/mm3
- Immune-mediated anemia and/or thrombocytopenia must have failed corticosteroids and
cyclosporine
- Paroxysmal nocturnal hemoglobinuria with progressive marrow failure allowed
- Either congenital or acquired severe granulocytopenia with a history of life
threatening infections No diagnosis of Fanconi's anemia
- HLA identical or 1 antigen mismatched related donor meeting the following criteria: No
prior or concurrent malignancy except localized basal cell or squamous cell skin
cancer (malignancies judged to be cured by local surgery, such as head and neck cancer
or stage I breast cancer, considered on an individual basis) Negative pregnancy test
--Patient Characteristics--
- Hepatic: Hepatitis B surface antigen negative Bilirubin no greater than 2.0 mg/dL
Transaminases no greater than 2 times upper limit of normal (in the absence of
Gilbert's disease)
- Renal: Creatinine no greater than 2.0 mg/dL
- Cardiovascular: No history of coronary artery disease LVEF at least 40% by MUGA
- Pulmonary: FEV1 at least 50% predicted FVC at least 50% predicted DLCO at least 50%
predicted
- Other: No uncontrolled diabetes mellitus No other illness that would preclude
aggressive chemotherapy No prior or concurrent malignancy except localized basal cell
or squamous cell skin cancer (malignancies judged to be cured by local surgery, such
as head and neck cancer or stage I breast cancer, considered on an individual basis)
No psychiatric illness or mental deficiency that would preclude study Not pregnant or
nursing Fertile patients must use effective contraception HIV negative
Gender
All
Ages
15 Years - 55 Years
Accepts Healthy Volunteers
No
Contacts
Richard K. Burt, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00017654
Organization ID
199/14983
Secondary IDs
NU-96AA1T
Study Sponsor
Northwestern Memorial Hospital
Study Sponsor
Richard K. Burt, Study Chair, Northwestern Memorial Hospital
Verification Date
October 2003