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

This study is ongoing, but not recruiting participants.

Sponsored by: Northwestern Memorial Hospital
Information provided by: Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier: NCT00017654
  Purpose

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.


Condition Intervention
Graft Versus Host Disease
Aplastic Anemia
Drug: anti-thymocyte globulin
Drug: cyclophosphamide
Drug: cyclosporine
Drug: methylprednisolone
Procedure: Allogeneic Bone Marrow Transplantation

MedlinePlus related topics:   Anemia    Bone Marrow Transplantation   

Drug Information available for:   Cyclophosphamide    Methylprednisolone    Cyclosporin    Cyclosporine   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:   3
Study Start Date:   April 2001

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.

  Eligibility
Ages Eligible for Study:   15 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00017654

Locations
United States, Illinois
Northwestern Memorial Hospital    
      Chicago, Illinois, United States, 60611

Sponsors and Collaborators
Northwestern Memorial Hospital

Investigators
Study Chair:     Richard K. Burt     Northwestern Memorial Hospital    
  More Information


Study ID Numbers:   199/14983, NU-96AA1T, NU-96H
First Received:   June 6, 2001
Last Updated:   June 23, 2005
ClinicalTrials.gov Identifier:   NCT00017654
Health Authority:   Unspecified

Keywords provided by Office of Rare Diseases (ORD):
aplastic anemia  
disease-related problem/condition  
graft versus host disease  
hematologic disorders  
rare disease  

Study placed in the following topic categories:
Cyclosporine
Clotrimazole
Hematologic Diseases
Graft versus host disease
Methylprednisolone
Miconazole
Tioconazole
Anemia
Rare Diseases
Methylprednisolone acetate
Prednisolone acetate
Cyclophosphamide
Cyclosporins
Homologous wasting disease
Antilymphocyte Serum
Prednisolone
Anemia, Aplastic
Graft vs Host Disease
Bone Marrow Diseases
Aplastic anemia
Methylprednisolone Hemisuccinate

Additional relevant MeSH terms:
Anti-Inflammatory Agents
Anti-Infective Agents
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Physiological Effects of Drugs
Hormones, Hormone Substitutes, and Hormone Antagonists
Antiemetics
Hormones
Neuroprotective Agents
Therapeutic Uses
Antifungal Agents
Alkylating Agents
Dermatologic Agents
Antineoplastic Agents, Hormonal
Immune System Diseases
Gastrointestinal Agents
Enzyme Inhibitors
Immunosuppressive Agents
Protective Agents
Glucocorticoids
Pharmacologic Actions
Autonomic Agents
Myeloablative Agonists
Antineoplastic Agents, Alkylating
Peripheral Nervous System Agents
Antirheumatic Agents
Central Nervous System Agents

ClinicalTrials.gov processed this record on December 03, 2008




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