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Phase I Pilot Study of Total-Body Irradiation, Anti-Thymocyte Globulin and Cyclophosphamide Followed By Syngeneic or Autologous Peripheral Blood Stem Cell Transplantation in Patients With Multiple Sclerosis

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ClinicalTrials.gov Identifier: NCT00014755
Recruitment Status : Completed
First Posted : April 11, 2001
Last Update Posted : June 24, 2005
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:

OBJECTIVES: I. Determine the toxicity of total-body irradiation, anti-thymocyte globulin, and cyclophosphamide followed by syngeneic or autologous peripheral blood stem cell (PBSC) transplantation in patients with multiple sclerosis.

II. Determine the disease response of patients treated with this regimen. III. Determine the safety and efficacy of filgrastim (G-CSF) for PBSC mobilization in this patient population.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Drug: anti-thymocyte globulin Drug: cyclophosphamide Drug: filgrastim Drug: prednisone Procedure: peripheral blood stem cell transplantation Procedure: irradiation Phase 1

Detailed Description:

PROTOCOL OUTLINE: This is a multicenter study. Patients receive oral prednisone on days 0-10. Beginning on day 1, patients undergoing autologous peripheral blood stem cell (PBSC) transplantation receive filgrastim (G-CSF) subcutaneously daily until leukapheresis is completed. Leukapheresis begins on approximately day 4 and continues until adequate CD34+ PBSC are collected.

PBSC are collected from syngeneic donors in a similar manner. Patients undergo total-body irradiation twice daily on days -5 and -4. Patients receive cyclophosphamide IV on days -3 and -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. Patients undergo autologous or syngeneic PBSC transplantation on day 0. Following PBSC transplantation, patients receive oral prednisone on days 7-30 and G-CSF IV daily beginning on day 0 and continuing until blood counts recover.

Patients are followed at 30, 80, and 90 days, monthly for 6 months, and then at 1 and 2 years.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 35 participants
Primary Purpose: Treatment
Study Start Date : December 1997

Resource links provided by the National Library of Medicine

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

Diagnosis of rapidly progressive multiple sclerosis (MS) by Proser criteria and at high risk for a fatal outcome or severe disability with one of the following:

  • Primary progressive disease
  • Relapsing/remitting disease with 2 or more attacks in 2 years
  • Secondary progressive disease

Extended disability status scale (EDSS) between 5.0 and 8.0 with deterioration in the EDSS of 1 or more points over the past year

More than 60 days since relapse of MS

No evidence of myelodysplasia

Sibling donor proven to be an identical twin by ABO typing, HLA typing, and VNTR analysis (for syngeneic transplantation)

--Prior/Concurrent Therapy--

Radiotherapy: No prior total-lymphoid irradiation

Other: No other concurrent investigational agents

--Patient Characteristics-- Hepatic: No hepatic impairment that would preclude high-dose immunosuppressive therapy

Renal: No renal impairment that would preclude high-dose immunosuppressive therapy

Cardiovascular: No cardiac impairment that would preclude high-dose immunosuppressive therapy

Pulmonary: No pulmonary impairment that would preclude high-dose immunosuppressive therapy


  • No neurologic impairment that would preclude high-dose immunosuppressive therapy
  • No active uncontrolled infection
  • No active malignancy
  • No other illness that would severely limit life expectancy
  • No medical or psychiatric conditions that would preclude study
  • No history of hypersensitivity to murine proteins or E. coli-derived proteins
  • No demonstrated lack of compliance with prior medical care
  • Able to undergo an MRI scan
  • HIV negative
  • Not pregnant or nursing

Information from the National Library of Medicine

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): NCT00014755

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United States, California
City of Hope National Medical Center
Duarte, California, United States, 91010
United States, Colorado
University of Colorado Cancer Center
Denver, Colorado, United States, 80262
United States, Missouri
Washington University Barnard Cancer Center
Saint Louis, Missouri, United States, 63110
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198-3330
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Fred Hutchinson Cancer Center
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Study Chair: Richard Nash Fred Hutchinson Cancer Center
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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ClinicalTrials.gov Identifier: NCT00014755    
Other Study ID Numbers: 199/15796
First Posted: April 11, 2001    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: April 2001
Keywords provided by Office of Rare Diseases (ORD):
multiple sclerosis
neurologic and psychiatric disorders
rare disease
Additional relevant MeSH terms:
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Multiple Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Antilymphocyte Serum
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal