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Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

This study has been terminated.
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00006054
First received: July 5, 2000
Last updated: October 14, 2009
Last verified: October 2003
  Purpose

OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies.

II. Determine relevant outcomes of this treatment in these patients including quality of survival, extent of morbidity and mortality from complications of the treatment (e.g., graft versus host disease, regimen related toxicities, B- cell lymphoproliferative disease), and completeness of functional immunoreconstitution.


Condition Intervention
Immunologic Deficiency Syndromes
Chediak-Higashi Syndrome
Common Variable Immunodeficiency
Graft Versus Host Disease
X-Linked Lymphoproliferative Syndrome
Familial Erythrophagocytic Lymphohistiocytosis
Hemophagocytic Lymphohistiocytosis
X-linked Agammaglobulinemia
Wiskott-Aldrich Syndrome
Chronic Granulomatous Disease
X-linked Hyper IgM Syndrome
Severe Combined Immunodeficiency
Leukocyte Adhesion Deficiency Syndrome
Virus-Associated Hemophagocytic Syndrome
Drug: anti-thymocyte globulin
Drug: busulfan
Drug: cyclophosphamide
Drug: cyclosporine
Drug: etoposide
Drug: methotrexate
Drug: methylprednisolone
Drug: prednisone
Procedure: Allogeneic Bone Marrow Transplantation

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


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

Study Start Date: March 2000
Study Completion Date: December 2002
Primary Completion Date: December 2002 (Final data collection date for primary outcome measure)
Detailed Description:

PROTOCOL OUTLINE: Patients with severe combined immunodeficiency (SCID) using a matched sibling donor receive allogeneic bone marrow or umbilical cord blood transplantation on day 0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50.

Patients with SCID using donors other than histocompatible siblings, Wiskott Aldrich syndrome using a histocompatible sibling donor, Wiskott Aldrich syndrome and under 5 years of age using donors other than histocompatible siblings, X-linked CD40 ligand deficiency using a histocompatible sibling donor, X-linked CD40 ligand deficiency and under 5 years of age using donors other than histocompatible siblings, other primary immunodeficiencies without manifestations of hemophagocytosis using a histocompatible sibling donor, or other primary immunodeficiencies without manifestations of hemophagocytosis and under 5 years of age using donors other than histocompatible siblings receive busulfan IV over 2 hours every 6 hours on days -9 to -6, cyclophosphamide IV on days -5 to -2, and antithymocyte globulin (ATG) twice daily on days -4 to -1. Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50.

Patients with hemophagocytic lymphohistiocytosis, Chediak Higashi syndrome, X-linked lymphoproliferative syndrome, severe progressive Langerhans cell histiocytosis, or other primary immunodeficiencies with complications of hemophagocytosis receive busulfan IV over 2 hours every 6 hours on days -9 to -6, cyclophosphamide IV over 2 hours on days -5 to -2, etoposide IV over 22 hours on days -5 to -3, and ATG IV twice daily on days -2, -1, 1, and 2. Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50.

Patients with Wiskott Aldrich syndrome or other primary immunodeficiencies without manifestations of hemophagocytosis, who are over 5 years of age and using donors other than histocompatible siblings, receive busulfan IV over 2 hours every 6 hours on days -6 and -5, cyclophosphamide IV over 2 hours on days -4 and -3, total body irradiation on day -2, and ATG IV over 2 hours twice daily on days -2, -1, 2, and 3. Allogeneic bone marrow or umbilical cord blood transplantation takes place on days 0 and 1. Patients receive GVHD prophylaxis with methylprednisolone IV every 12 hours on days 2-21, oral prednisone every 12 hours on days 22-100 and then tapered off over days 101 to 128, and cyclosporine IV over 2 hours every 8-12 hours on days -3 to 100.

All patients are followed as determined by their primary physician.

  Eligibility

Ages Eligible for Study:   up to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria
  • Severe combined immunodeficiency All ages with histocompatible sibling donors or with other donors OR Wiskott Aldrich syndrome All ages with histocompatible sibling donors or with other donors OR X-linked CD40 ligand deficiency All ages with histocompatible sibling donors OR Under 5 years of age with donors other than histocompatible siblings OR Other primary immunodeficiencies without manifestations of hemophagocytosis All ages with histocompatible sibling donors or with other donors OR Hemophagocytic lymphohistiocytosis (HLH) Familial erythrophagocytic lymphohistiocytosis (FEL), familial HLH (FHLH), recurrent virus-associated hemophagocytic syndrome (VAHS) All ages with related or unrelated donors OR Chediak Higashi syndrome All ages with related or unrelated donors OR X-linked lymphoproliferative syndrome All ages with related or unrelated donors OR Other primary immunodeficiencies with complication of hemophagocytosis All ages with related or unrelated donors OR Severe progressive Langerhans cell histiocytosis All ages with related or unrelated donors
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00006054

Locations
United States, Minnesota
Fairview University Medical Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Fairview University Medical Center
Investigators
Study Chair: K. Scott Baker Fairview University Medical Center
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00006054     History of Changes
Other Study ID Numbers: 199/15104, UMN-MT-1995-26, UMN-MT-9526
Study First Received: July 5, 2000
Last Updated: October 14, 2009
Health Authority: Unspecified

Keywords provided by Office of Rare Diseases (ORD):
Wiskott-Aldrich syndrome
Chediak-Higashi syndrome
Langerhans cell histiocytosis
X-linked agammaglobulinemia
X-linked hyper IgM syndrome
X-linked lymphoproliferative syndrome
chronic granulomatous disease
common variable immunodeficiency
complement deficiency
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
leukocyte adhesion deficiency syndrome
primary immunodeficiency disease
rare disease
severe combined immunodeficiency
virus-associated hemophagocytic syndrome

Additional relevant MeSH terms:
Wiskott-Aldrich Syndrome
Agammaglobulinemia
Chediak-Higashi Syndrome
Common Variable Immunodeficiency
Genetic Diseases, X-Linked
Graft vs Host Disease
Granuloma
Granulomatous Disease, Chronic
Hyper-IgM Immunodeficiency Syndrome
Hyper-IgM Immunodeficiency Syndrome, Type 1
Immunologic Deficiency Syndromes
Leukocyte-Adhesion Deficiency Syndrome
Lymphohistiocytosis, Hemophagocytic
Lymphoproliferative Disorders
Severe Combined Immunodeficiency
Syndrome
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Blood Protein Disorders
DNA Repair-Deficiency Disorders
Disease
Dysgammaglobulinemia
Genetic Diseases, Inborn
Hematologic Diseases
Hemorrhagic Disorders
Histiocytosis
Histiocytosis, Non-Langerhans-Cell
Immune System Diseases
Immunoproliferative Disorders
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on November 25, 2014