Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

June 23, 2005 updated by: Fairview University Medical Center

OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders.

II. Determine the quality of life, absence of adverse effects (e.g., graft versus host disease and B cell lymphoproliferative disease), and completeness of recovery of their underlying condition in these patients with this treatment regimen.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

PROTOCOL OUTLINE: Patients receive IV or oral busulfan every 6 hours on days -9 to -6; cyclophosphamide IV on days -5 to -2; anti-thymocyte globulin IV on days -4 to -2; and allogeneic bone marrow transplantation (BMT) on day 0.

Patients with class 3 thalassemia (liver edge greater than 2 cm below costal margin, a history of inconsistent chelation, and portal fibrosis) receive a less intensive conditioning regimen consisting of oral busulfan every 6 hours on days -7 to -4; anti-thymocyte globulin IV on days -5 to -1 and days 1-5; cyclophosphamide IV on days -3 to -2; and allogeneic BMT on day 0.

Patients are followed at day 28, and then at 3, 6, 12, and 24 months.

Study Type

Interventional

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Fairview University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Severe hemoglobinopathy including, but not limited to the following:

Homozygous beta thalassemia Other beta chain mutation as demonstrated by hemoglobin electrophoresis Sickle cell anemia (age 16 to 50 years) Evidence of one or more the following prior complications: Stage I-II sickle cell lung disease Sickle cell nephropathy (moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal for age) Acute chest syndrome requiring blood transfusions More than 3 debilitating pain episodes per year for 3 years lasting more than 4 hours each Any combination of episodes of acute chest syndrome and painful episodes adding up to 3 episodes per year for 3 consecutive years Requirement for chronic transfusions with alloimmunization (more than 2 antibodies) Sickle cell anemia (age under 16 years) Evidence of one or more the following prior complications: Prior stroke or hemorrhage Any neurologic event lasting more than 24 hours Abnormal cerebral MRI and cerebral arteriogram MRI angiographic impaired neuropsychologic testing Stage I-II sickle cell lung disease Sickle cell nephropathy (moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal for age) Significant visual impairment in at least one eye with bilateral proliferative retinopathy Acute chest syndrome with history of recurrent hospitalizations or exchange transfusions Osteonecrosis of multiple joints with destructive changes More than 3 debilitating pain episodes per year or priapism Requirement for chronic transfusions with alloimmunization

OR

Bone marrow failure syndrome unresponsive to therapy, including but not limited to the following: Congenital pure red cell aplasia (Diamond Blackfan anemia) Confirmed by bone marrow aspirate More than 6 transfusions per year despite steroid therapy Kostmann's neutropenia Confirmed by bone marrow aspirate Unable to maintain absolute neutrophil count greater than 750/mm3 or recurrent life threatening infections or more than 4 hospitalizations per year despite therapy with filgrastim (G-CSF) No myelodysplasia or aplastic anemia

Must have a related donor with at least a 5 out of 6 HLA antigen match

--Patient Characteristics--

Age: Sickle cell anemia patients 0 to 50; All other patients under 18

Performance status: Karnofsky 70-100%

Hepatic: Bilirubin no greater than 3.0 mg/dL with a direct fraction no greater than 2.0 mg/dL ALT no greater than 150 U/L No moderate or severe portal fibrosis No active hepatitis

Renal: Glomerular filtration rate at least 30% predicted (except with sickle cell anemia) No renal dysfunction

Cardiovascular: Left ejection fraction at least 50% No cardiac compromise

Other: No severe, stable neurologic impairment HIV negative

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Fairview University Medical Center

Investigators

  • Study Chair: Paul J. Orchard, Fairview University Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2000

Study Registration Dates

First Submitted

June 2, 2000

First Submitted That Met QC Criteria

June 2, 2000

First Posted (Estimate)

June 5, 2000

Study Record Updates

Last Update Posted (Estimate)

June 24, 2005

Last Update Submitted That Met QC Criteria

June 23, 2005

Last Verified

July 1, 2004

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 199/15101
  • UMN-MT-1994-18
  • UMN-MT-9418

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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