Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia

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