Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

NCT00006054 | Terminated

• 3 other identifiers: 199/15104UMN-MT-1995-26UMN-MT-9526
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

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

Keywords

Chediak-Higashi syndrome; Langerhans cell histiocytosis; Wiskott-Aldrich syndrome; 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

Interventions

anti-thymocyte globulin DRUG

busulfan DRUG

cyclophosphamide DRUG

cyclosporine DRUG

etoposide DRUG

methotrexate DRUG

methylprednisolone DRUG

prednisone DRUG

Allogeneic Bone Marrow Transplantation PROCEDURE

Eligibility Criteria

• Age: Up to 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

* 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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Fairview University Medical Center: lead

Study Sites (1)

Fairview University Medical Center

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Immunologic Deficiency Syndromes; Chediak-Higashi Syndrome; Common Variable Immunodeficiency; Graft vs Host Disease; Lymphoproliferative Disorders; Lymphohistiocytosis, Hemophagocytic; Bruton type agammaglobulinemia; Wiskott-Aldrich Syndrome; Granulomatous Disease, Chronic; Hyper-IgM Immunodeficiency Syndrome, Type 1; Severe Combined Immunodeficiency; Leukocyte-Adhesion Deficiency Syndrome; Histiocytosis, Langerhans-Cell; Hereditary Complement Deficiency Diseases; Genetic Diseases, Inborn; Hematologic Diseases; Histiocytosis; Immune System Diseases; Communicable Diseases; Primary Immunodeficiency Diseases; Rare Diseases

Interventions

Antilymphocyte Serum; Busulfan; Cyclophosphamide; Cyclosporine; Etoposide; Methotrexate; Methylprednisolone; Prednisone

Condition Hierarchy (Ancestors)

Albinism; Eye Diseases, Hereditary; Eye Diseases; Phagocyte Bactericidal Dysfunction; Leukocyte Disorders; Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lymphatic Diseases; Immunoproliferative Disorders; Histiocytosis, Non-Langerhans-Cell; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Lymphopenia; Leukopenia; Cytopenia; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Hyper-IgM Immunodeficiency Syndrome; Dysgammaglobulinemia; Blood Protein Disorders; Infant, Newborn, Diseases; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Infections

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Podophyllotoxin; Tetrahydronaphthalenes; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Glucosides; Glycosides; Carbohydrates; Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Pregnadienediols

Study Officials

• K. Scott Baker

  Fairview University Medical Center

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: July 5, 2000
• First Posted: July 6, 2000
• Study Start: March 1, 2000
• Primary Completion: December 1, 2002
• Study Completion: December 1, 2002
• Last Updated: October 15, 2009

Record last verified: 2003-10

Locations

US (1)