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| Sponsored by: |
Fairview University Medical Center |
|---|---|
| Information provided by: | Office of Rare Diseases (ORD) |
| ClinicalTrials.gov Identifier: | NCT00006056 |
Purpose
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 | Intervention |
|---|---|
|
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 |
| Study Type: | Interventional |
| Study Design: | Treatment |
| Estimated Enrollment: | 40 |
| Study Start Date: | March 2000 |
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.
Eligibility| Ages Eligible for Study: | up to 55 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Patients diagnosed with any of the following active but stable, or nonactive/quiescent, hemophagocytic disorders:
X-linked lymphoproliferative disorder (XLP)
Two or more maternally related males manifesting at least one of the following XLP phenotypes:
Chediak-Higashi syndrome
Partial oculocutaneous albinism (hair, skin, eyes)
Frequent bacterial infections
Large peroxidase positive granules in leukocytes of peripheral blood or bone marrow
Positive family history or parental consanguinity is supportive of the diagnosis
May not have entered accelerated phase as defined by any of the following:
Viral associated hemophagocytic syndrome (VAHS)
Relapsed after prior therapy or supportive care
Diagnostic criteria as for HLH
No hemophagocytic disorders secondary to underlying malignancy
Patients 35 years of age and under must have a hematopoietic stem cell donor that is one of the following:
Patients 36 to 55 years of age must have a hematopoietic stem cell donor that is one of the following:
Patients receiving umbilical cord blood must have an unrelated donor with no more than two antigen HLA A, B, or DRB1 mismatches
--Patient Characteristics--
Performance status: Karnofsky 70-100% OR Age less than 16 years: Lansky 50-100%
Life expectancy: Not severly limited by another disease
Hepatic: SGOT less than 3 times normal Bilirubin less than 2.5 mg/dL
Renal: Creatinine normal OR Creatinine clearance or glomerular filtration rate greater than 50% normal
Cardiovascular: If symptomatic, ventricular ejection fraction must be greater than 40% and must improve with exercise OR Shortening fraction normal on echocardiogram
Pulmonary:
Other: HIV negative No significant active infections
Contacts and Locations| United States, Minnesota | |
| Fairview University Medical Center | |
| Minneapolis, Minnesota, United States, 55455 | |
| Study Chair: | K. Scott Baker | Fairview University Medical Center |
More Information
| Study ID Numbers: | 199/15106, UMN-MT-1997-08, UMN-MT-9708 |
| Study First Received: | July 5, 2000 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00006056 History of Changes |
| Health Authority: | Unspecified |
|
Chediak-Higashi syndrome X-linked lymphoproliferative syndrome disease-related problem/condition familial erythrophagocytic lymphohistiocytosis genetic diseases and dysmorphic syndromes graft versus host disease hematologic disorders |
hemophagocytic lymphohistiocytosis histiocytosis immunologic disorders and infectious disorders primary immunodeficiency disease rare disease virus-associated hemophagocytic syndrome |
|
Antimetabolites Cyclosporine Immunologic Factors Clotrimazole Miconazole Leukocyte Disorders Cyclophosphamide Etoposide phosphate Cyclosporins Graft Versus Host Disease Histiocytosis Antifungal Agents Methotrexate Alkylating Agents Chediak-Higashi Syndrome |
Etoposide Virus Associated Hemophagocytic Syndrome Immunoproliferative Disorders Lymphohistiocytosis, Hemophagocytic Hematologic Diseases Tioconazole Rare Diseases Folic Acid Antagonists Immunosuppressive Agents Immunologic Deficiency Syndromes Homologous Wasting Disease Folic Acid Virus Diseases Antilymphocyte Serum Lymphatic Diseases |
|
Antimetabolites Anti-Infective Agents Antimetabolites, Antineoplastic Cyclosporine Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Leukocyte Disorders Reproductive Control Agents Cyclophosphamide Cyclosporins Pathologic Processes Histiocytosis Antifungal Agents |
Therapeutic Uses Syndrome Abortifacient Agents Methotrexate Dermatologic Agents Alkylating Agents Chediak-Higashi Syndrome Nucleic Acid Synthesis Inhibitors Phagocyte Bactericidal Dysfunction Reticuloendotheliosis Lymphohistiocytosis, Hemophagocytic Disease Immunoproliferative Disorders Immune System Diseases Hematologic Diseases |
