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


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Ages Eligible for Study: | 18 Years to 60 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--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
Other:
- 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

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
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 |
Study Chair: | Richard Nash | Fred Hutchinson Cancer Center |
ClinicalTrials.gov Identifier: | NCT00014755 |
Other Study ID Numbers: |
199/15796 FHCRC-1164.00 |
First Posted: | April 11, 2001 Key Record Dates |
Last Update Posted: | June 24, 2005 |
Last Verified: | April 2001 |
multiple sclerosis neurologic and psychiatric disorders rare disease |
Multiple Sclerosis Sclerosis Pathologic Processes Demyelinating Autoimmune Diseases, CNS Autoimmune Diseases of the Nervous System Nervous System Diseases Demyelinating Diseases Autoimmune Diseases Immune System Diseases Prednisone Cyclophosphamide 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 Glucocorticoids Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Antineoplastic Agents, Hormonal |