Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders
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|ClinicalTrials.gov Identifier: NCT00006056|
Recruitment Status : Unknown
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was: Active, not recruiting
First Posted : July 6, 2000
Last Update Posted : June 24, 2005
OBJECTIVES: I. Determine the efficacy of unrelated donor hematopoietic stem cell transplantation in the treatment of patients with life threatening hemophagocytic disorders.
II. Determine the rate of disease free survival, incidence of graft failure, and incidence of graft versus host disease in these patients after undergoing this treatment regimen.
|Condition or disease||Intervention/treatment||Phase|
|Chediak-Higashi Syndrome Graft Versus Host Disease X-Linked Lymphoproliferative Syndrome Familial Erythrophagocytic Lymphohistiocytosis Hemophagocytic Lymphohistiocytosis Virus-Associated Hemophagocytic Syndrome||Drug: anti-thymocyte globulin Drug: busulfan Drug: cyclophosphamide Drug: cyclosporine Drug: etoposide Drug: filgrastim Drug: methotrexate Procedure: allogeneic hematopoietic stem cell transplantation||Not Applicable|
PROTOCOL OUTLINE: Patients receive oral busulfan twice a day on days -9 to -6; cyclophosphamide IV over 1 hour on days -5 to -2; etoposide IV over 4 hours on days -5 to -3; and anti-thymocyte globulin IV twice a day on days -2 and -1 and days 1 and 2. Patients undergo allogeneic hematopoietic stem cell transplantation on day 0. Filgrastim (G-CSF) is administered subcutaneously beginning on day 1 and continuing until blood counts recover. Patients receive graft versus host disease prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1-4 hours (orally once the patients resumes eating) every 12 hours (every 8 hours for pediatric patients) starting on or prior to day -3 and continuing up to 1 year.
Patients are followed at days 28 and 100, at 6 months and 1 year, and then annually for 5 years.
|Study Type :||Interventional (Clinical Trial)|
|Enrollment :||40 participants|
|Study Start Date :||March 2000|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00006056
|United States, Minnesota|
|Fairview University Medical Center|
|Minneapolis, Minnesota, United States, 55455|
|Study Chair:||K. Scott Baker||Fairview University Medical Center|