Phase I Study of High-Dose Cyclophosphamide and Total Body Irradiation With T Lymphocyte-Depleted Autologous Peripheral Blood Stem Cell or Bone Marrow Rescue in Patients With Multiple Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00017628
Recruitment Status : Completed
First Posted : June 6, 2001
Last Update Posted : June 24, 2005
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:


I. Determine the efficacy, in terms of disease progression, of high-dose cyclophosphamide and total body irradiation with T lymphocyte-depleted autologous peripheral blood stem cell or bone marrow rescue in patients with multiple sclerosis.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Drug: cyclophosphamide Drug: filgrastim Drug: methylprednisolone Procedure: Autologous Stem Cell Transplantation Phase 1

Detailed Description:

PROTOCOL OUTLINE: Following an induction course of cyclophosphamide IV, patients receive filgrastim (G-CSF) subcutaneously (SC) daily until the completion of peripheral blood stem cell (PBSC) harvesting. PBSCs are collected over several days. Patients who do not mobilize sufficient cells undergo bone marrow harvest. Harvested PBSCs or bone marrow then undergo T-lymphocyte depletion.

Patients receive cyclophosphamide IV over 1 hour on days -6 and -5 and methylprednisolone IV daily on days -4 to -1. Patients also undergo total body irradiation twice a day on days -4 to -1. Lymphocyte-depleted PBSCs or bone marrow is reinfused on day 0. Patients receive G-CSF SC daily beginning on day 0 and continuing until blood counts have recovered for 3 days.

Patients are followed at 1, 2, 3, 6, and 12 months and then annually for 5 years.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Primary Purpose: Treatment
Study Start Date : April 2001

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Ages Eligible for Study:   up to 59 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

Diagnosis of multiple sclerosis Kurtzke score of 4.0-7.5 Increase of 1.0 point over the past 12 months More than 3 relapses in 24 months despite conventional disease modifying therapy Failure to stabilize active clinical progression with a 3-day regimen of methylprednisolone IV

--Prior/Concurrent Therapy--

Chemotherapy: No prior cladribine

Radiotherapy: No prior radiotherapy to greater than 10 cm2 of lung tissue No prior craniospinal irradiation No prior total lymphoid irradiation

--Patient Characteristics--

Hematopoietic: Absolute neutrophil count at least 1,000/mm3 Platelet count at least 100,000/mm3 Hemoglobin at least 9.0 g/dL

Hepatic: Hepatitis B antigen negative Bilirubin no greater than 2.0 mg/dL Transaminases no greater than 2 times upper limit of normal

Renal: Creatinine no greater than 2.0 mg/dL Cardiovascular: No history of coronary artery disease Resting LVEF at least 45%

Pulmonary: FEV1/FVC at least 75% predicted DLCO at least 50% predicted


  • Not pregnant
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • HIV negative
  • No uncontrolled diabetes mellitus
  • No other concurrent medical illness that would preclude study
  • No concurrent psychiatric illness or mental deficiency that would preclude study
  • No prior malignancy except localized basal cell or squamous cell skin cancer (malignancies judged to be cured by local surgical therapy such as head and neck cancer or stage I breast cancer, are considered on an individual basis)
  • No presence of body shrapnel, metal fragments, or unremovable devices such as a pacemaker or aneurysm clip

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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 identifier (NCT number): NCT00017628

United States, Illinois
Northwestern Memorial Hospital
Chicago, Illinois, United States, 60611
Rush-Presbyterian-St. Luke's Medical Center
Chicago, Illinois, United States, 60612
Sponsors and Collaborators
Northwestern Memorial Hospital
Study Chair: Richard K. Burt Northwestern Memorial Hospital Identifier: NCT00017628     History of Changes
Other Study ID Numbers: 199/14975
First Posted: June 6, 2001    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: July 2004

Keywords provided by Office of Rare Diseases (ORD):
multiple sclerosis
neurologic and psychiatric disorders
rare disease

Additional relevant MeSH terms:
Multiple Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Methylprednisolone Hemisuccinate
Prednisolone acetate
Methylprednisolone acetate
Prednisolone hemisuccinate
Prednisolone phosphate
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
Autonomic Agents
Peripheral Nervous System Agents