Hematopoietic Stem Cell Therapy for Patients With Multiple Sclerosis
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ClinicalTrials.gov Identifier: NCT00278655 |
Recruitment Status :
Terminated
First Posted : January 18, 2006
Results First Posted : May 1, 2014
Last Update Posted : May 1, 2014
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Multiple Sclerosis | Biological: Hematopoietic stem cell transplantation | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 21 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Hematopoietic Stem Cell Therapy for Patients With Inflammatory Multiple Sclerosis Failing Interferon Therapy: A Phase II Multi-Center Trial |
Study Start Date : | June 2003 |
Actual Primary Completion Date : | December 2011 |
Actual Study Completion Date : | May 2012 |

Arm | Intervention/treatment |
---|---|
Experimental: Hematopoietic stem cell transplantation
All participants will undergo hematopoietic stem cell transplantation after receiving conditioning regimen.
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Biological: Hematopoietic stem cell transplantation
Autologous hematopoietic stem cell transplantation |
- Disease Progression [ Time Frame: 3 years after transplant ]Data are reporting number of participants with disease progression. Disease progression is defined as a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 3 months apart.
- Survival [ Time Frame: three years ]
Data are reporting the number of participants who survived three years after the transplant
Survival of 21 participants was evaluated at three years after the transplant

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Ages Eligible for Study: | 18 Years to 50 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age between 18-50, inclusive.
- Diagnosis of Multiple Sclerosis (MS) using Poser criteria (Appendix A).
- An Expanded Disability Status Scale (EDSS) of 2.0 - 5.5 (Appendix B).
- Inflammatory disease despite primary disease modifying therapy with at least 3 months of interferon. Failure is defined as two or more clinical relapses with documented neurologic changes within the year prior to the study. (NOTE: Relapses must have required treatment with corticosteroids. Sensory only relapses are excluded.) Failure may also be defined as one relapse within the year prior to study if there is evidence on MRI of active inflammation (i.e., gadolinium enhancement).
Exclusion Criteria:
- Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
- Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis.
- Positive pregnancy test.
- Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an intrauterine device (IUD); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam.
- Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
- Forced expiratory volume in 1 second (FEV1) / forced vital capacity (FVC) < 60% of predicted after bronchodilator therapy (if necessary).
- Diffusing capacity of the lung for carbon monoxide (DLCO) < 50% of predicted.
- Resting left ventricular ejection fraction (LVEF) < 50 %.
- Bilirubin > 2.0 mg/dl.
- Serum creatinine > 2.0 mg/dl.
- Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins, or to iron compounds/medications.
- Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams.
- Diagnosis of primary progressive multipole sclerosis (MS).
- Platelet count < 100,000/ul.
- Psychiatric illness, mental deficiency or cognitive dysfunction making compliance with treatment or informed consent impossible.
- Active infection except asymptomatic bacteruria.

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): NCT00278655
Principal Investigator: | Richard Burt, MD | Northwestern University |
Responsible Party: | Richard Burt, MD, MD, Northwestern University |
ClinicalTrials.gov Identifier: | NCT00278655 |
Other Study ID Numbers: |
DIAD MS.Auto2002 |
First Posted: | January 18, 2006 Key Record Dates |
Results First Posted: | May 1, 2014 |
Last Update Posted: | May 1, 2014 |
Last Verified: | March 2014 |
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 |