Stem Cell Transplant for Immunologic or Histiocytic Disorders - Full Text View

Sponsored by:	Masonic Cancer Center, University of Minnesota
Information provided by:	Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:	NCT00176865

Purpose

This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD).

Condition	Intervention	Phase
Hemophagocytic Lymphohistiocytosis X-Linked Lymphoproliferative Disorders Chediak-Higashi Syndrome Griscelli Syndrome Immunologic Deficiency Syndromes Langerhans-Cell Histiocytosis	Procedure: Stem Cell Transplant Drug: Fludarabine, melphalan, ATG or Campath	Phase II

Genetics Home Reference related topics: Chediak-Higashi syndrome

Drug Information available for: Fludarabine Fludarabine monophosphate Campath Alemtuzumab Melphalan Sarcolysin Melphalan hydrochloride

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study
Official Title:	Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:

To demonstrate the safety and the ability to establish stable mixed chimerism (>10% donor cells at day 100) using a nonmyeloablative preparative regimen in a phase 2 pilot trial [ Time Frame: day +100 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Determine the incidence of chimerism at 100 days, 6 months and 1 year [ Time Frame: 100 days, 6 months and 1 year ] [ Designated as safety issue: Yes ]
Determine the incidence of grade 2-4 and 3-4 acute GVHD at 100 days [ Time Frame: 100 days ] [ Designated as safety issue: Yes ]
Determine the incidence of chronic GVHD at 6 months and 1 year [ Time Frame: 6 months and 1 year ] [ Designated as safety issue: Yes ]
Compare the quality of life (QOL) at 1, 2 and 5 years with the pre-transplant assessment [ Time Frame: 1, 2 and 5 years ] [ Designated as safety issue: No ]
Determine overall survival at 100 days and 1 year [ Time Frame: 100 days and 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment:	30
Study Start Date:	August 2002
Estimated Study Completion Date:	August 2012
Estimated Primary Completion Date:	August 2010 (Final data collection date for primary outcome measure)

Intervention Details:

Reduced intensity chemotherapy followed by infusion of hematopoietic stem cells

all drugs are given IV. Fludarabine x 5 days and melphalan x 1 day

Detailed Description:

Prior to transplantation, subjects will receive Melphalan, Fludarabine and Anti-Thymocyte Globulin (ATG) or Campath (for subjects unable to tolerate ATG). These three drugs are being given to subjects to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via IV catheter.

After stem cell transplantation, subjects will be given CSA and MMF to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.

Eligibility

Ages Eligible for Study:	up to 35 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients with immunodeficiencies or histiocytic disorders 0-35 years of age with an acceptable stem cell donor and disease characteristic defined by the following:

Patients with histocytic disorders (hemophagocytic lymphohistiocytosis of any etiology and refractory Langerhans cell histiocytosis) who do not meet eligibility criteria for a myeloablative transplant procedure
Patients with immunodeficiency disorders in whom residual immune function may not require a fully myeloablative preparative regimen or patient is ineligible for standard myeloablative preparative regimen (any form of severe combined immunodeficiency [SCID], or other immunodeficiency with T cell defect)
Patients with immunodeficiency disorders that have had poor outcome with myeloablative stem cell transplants (including, but not limited to, common variable immunodeficiency [CVID], Wiskott Aldrich Syndrome [WAS] if > 5 years of age, ataxia telangiectasia)
Patients with immunodeficiencies or histocytic disorders that require a second stem cell transplant (SCT) for any reason

Exclusion Criteria:

Karnofsky or Lansky performance score <70
Glomerular filtration rate (GFR)<30% predicted
Cardiac function <50% normal by echocardiogram
Serum creatinine > 2x normal for age/weight
Pregnant or lactating females
Active serious infection that has not had an adequate course of therapy pre-SCT. Any patient with AIDS or AIDS-related complex (ARC) or HIV seropositivity

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00176865

Contacts

Contact: Angela Smith, MD

612-273-2800

smith719@umn.edu

Locations

United States, Minnesota
Masonic Cancer Center University of Minnesota	Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Angela Smith, MD 612-273-2800 smith719@umn.edu
Principal Investigator: Angela Smith, MD

Sponsors and Collaborators

Masonic Cancer Center, University of Minnesota

Investigators

Principal Investigator:

Angela Smith, MD

Masonic Cancer Center, University of Minnesota

More Information

No publications provided

Responsible Party:	Masonic Cancer Center, University of Minnesota ( Angela Smith, MD )
Study ID Numbers:	0207M29448, MT2002-12
Study First Received:	September 12, 2005
Last Updated:	April 21, 2009
ClinicalTrials.gov Identifier:	NCT00176865 History of Changes
Health Authority:	United States: Institutional Review Board

Keywords provided by Masonic Cancer Center, University of Minnesota:

Stem cell transplant
immunodeficiency
donor lymphocyte infusion

Study placed in the following topic categories:

Melphalan
Immunologic Factors
Leukocyte Disorders
Histiocytosis X
Respiratory Tract Diseases
Histiocytosis
Alemtuzumab
Alkylating Agents
Chediak-Higashi Syndrome
Langerhans Cell Histiocytosis
Lung Diseases, Interstitial
Letterer-Siwe Disease
Immunoproliferative Disorders
Lymphohistiocytosis, Hemophagocytic

Hematologic Diseases
Fludarabine monophosphate
Histiocytosis, Langerhans-Cell
Immunosuppressive Agents
Immunologic Deficiency Syndromes
Antilymphocyte Serum
Lymphatic Diseases
Lung Diseases
Hemophagocytic Lymphohistiocytosis
Antineoplastic Agents, Alkylating
Fludarabine
Lymphoproliferative Disorders
X-linked Lymphoproliferative Syndrome

Additional relevant MeSH terms:

Melphalan
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Physiological Effects of Drugs
Leukocyte Disorders
Pathologic Processes
Histiocytosis
Respiratory Tract Diseases
Therapeutic Uses
Syndrome
Alemtuzumab
Alkylating Agents
Chediak-Higashi Syndrome
Phagocyte Bactericidal Dysfunction

Lung Diseases, Interstitial
Lymphohistiocytosis, Hemophagocytic
Disease
Reticuloendotheliosis
Immunoproliferative Disorders
Immune System Diseases
Hematologic Diseases
Histiocytosis, Langerhans-Cell
Immunosuppressive Agents
Pharmacologic Actions
Immunologic Deficiency Syndromes
Lymphatic Diseases
Lung Diseases
Myeloablative Agonists
Antineoplastic Agents, Alkylating

ClinicalTrials.gov processed this record on May 07, 2009