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

The recruitment status of this study is unknown because the information has not been verified recently.

Verified October 2003 by Office of Rare Diseases (ORD). Recruitment status was Active, not recruiting

First Received on June 6, 2001. Last Updated on June 23, 2005 History of Changes

Sponsor:	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

Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

MedlinePlus related topics: Anemia Aplastic Anemia Bone Marrow Transplantation

Drug Information available for: Cyclophosphamide Prednisolone Prednisolone acetate Depo-medrol Cyclosporine Medrol veriderm Cyclosporin Methylprednisolone Prednisolone sodium phosphate Prednisolone Sodium Succinate Methylprednisolone Sodium Succinate Methylprednisolone hemisuccinate 6-Methylprednisolone

U.S. FDA Resources

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

No publications provided

ClinicalTrials.gov Identifier:	NCT00017654 History of Changes
Other Study ID Numbers:	199/14983, NU-96AA1T, NU-96H
Study First Received:	June 6, 2001
Last Updated:	June 23, 2005
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

Additional relevant MeSH terms:

Anemia
Anemia, Aplastic
Graft vs Host Disease
Hematologic Diseases
Bone Marrow Diseases
Immune System Diseases
Antilymphocyte Serum
Cyclophosphamide
Cyclosporins
Cyclosporine
Methylprednisolone Hemisuccinate
Prednisolone
Methylprednisolone acetate
Prednisolone acetate
Methylprednisolone

Prednisolone phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Enzyme Inhibitors
Antifungal Agents
Anti-Infective Agents

ClinicalTrials.gov processed this record on February 12, 2012