Stem Cell Therapy for Patients With Multiple Sclerosis Failing Alternate Approved Therapy- A Randomized Study
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|ClinicalTrials.gov Identifier: NCT00273364|
Recruitment Status : Completed
First Posted : January 9, 2006
Results First Posted : August 12, 2020
Last Update Posted : August 12, 2020
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|Condition or disease||Intervention/treatment||Phase|
|Multiple Sclerosis||Procedure: Hematopoietic Stem Cell Therapy Drug: Standard treatment with a conventional drug||Phase 2|
To assess the efficacy of autologous PBSCT versus FDA approved standard of care ( i.e. interferon, glatiramer acetate, mitoxantrone, natalizumab, fingolimod, or tecfidera) for inflammatory multiple sclerosis (MS) failing failing alternate approved therapy. The endpoints to be considered in this study are:
2.1 Primary Endpoint:
Disease progression, defined as a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 6 months apart and not due to a non-MS disease process. Patients will be followed for 5 years after randomization.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||110 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Hematopoietic Stem Cell Therapy for Patients With Inflammatory Multiple Sclerosis Failing Alternate Approved Therapy: A Randomized Study|
|Actual Study Start Date :||November 16, 2005|
|Actual Primary Completion Date :||January 5, 2017|
|Actual Study Completion Date :||August 30, 2019|
Experimental: Hematopoietic Stem Cell Transplantation
Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG
Procedure: Hematopoietic Stem Cell Therapy
After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen
Other Name: stem cell infusion
Active Comparator: Standard therapy for MS
Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)
Drug: Standard treatment with a conventional drug
Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)
Other Name: standard of care
- Expanded Disability Status Scale (EDSS) Improvement [ Time Frame: Pre Treatment, 6 and 12 months Post Treatment ]The EDSS scale ranges from 0 to 10 in 0.5 increments that represent higher levels of disability. Improvement in EDSS is defined by both a 0.5 or 1.0 points sustained for more than 6 months.
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|Ages Eligible for Study:||18 Years to 55 Years (Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Age between18-55, inclusive.
- Diagnosis of MS using revised McDonald criteria of clinically definite MS (Appendix I).
- An EDSS score of 2.0 to 6.0 (Appendix II).
Inflammatory disease despite treatment with standard disease modifying therapy including at least 6 months of interferon or copaxone. Inflammatory disease is defined based on both MRI (gadolinium enhancing lesions) and clinical activity (acute relapses *treated with IV or oral high dose corticosteroids and prescribed by a neurologist). Minimum disease activity required for failure is defined as: a) two or more *steroid treated clinical relapses with documented new objective signs on neurological examination documented by a neurologist within the year prior to the study, or b) one *steroid treated clinical relapse within the year prior to study and evidence on MRI of active inflammation (i.e., gadolinium enhancement) within the last 12 months on an occasion separate from the clinical relapse (3 months before or after the clinical relapse).
- A steroid treated relapse will include a relapse that was severe enough to justify treatment but due to patient intolerance of steroids, or a history of non-response to steroids, they were offered but not used.
- 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 from the time of evaluation for eligibility until 6 months posttransplant (if on transplant) or until appropriate for non-transplant treatment (if on control arm). Effective birth control is defined as 1) abstinence defined as refraining from all acts of vaginal intercourse; 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 at one second (FEV1) / forced vital capacity (FVC) < 60% of predicted after bronchodilator therapy (if necessary)
- Diffusing capacity of lung for carbon monoxide (DLCO) < 50% of predicted (for the transplant arm)
- 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 MS
- Diagnosis of secondary progressive MS
- Platelet count < 100,000/ul, white blood cell count (WBC) < 1,500 cells/mm3
- Psychiatric illness, mental deficiency or cognitive dysfunction making compliance with treatment or informed consent impossible
- Active infection except asymptomatic bacteriuria
- Use of natalizumab (Tysabri) within the previous 6 months
- Use of fingolimod (Gilenya) within the previous 3 months
- Use of teriflunomide (Aubagio) within the previous 2 years unless cleared from the body (plasma concentration < 0.02mcg/ml) following elimination from the body with cholestyramine 8g three times a day for 11 days
- Prior treatment with CAMPATH (alemtuzumab)
- Prior treatment with mitoxantrone
- Any hereditary neurological disease such as Charcot-Marie-Tooth disease (CMT) or Spinocerebellar ataxia (SCA) are contraindications
Use of tecfidera within the previous 3 months
- For patients who clearly have inflammatory disease, an exception can be made if agreed upon by study PI and at least two study neurologists.
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): NCT00273364
|United States, Illinois|
|Northwestern University, Feinberg School of Medicine|
|Chicago, Illinois, United States, 60611|
|Principal Investigator:||Richard Burt, MD||Northwestern University|
Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||Richard Burt, MD, MD, Northwestern University|
|Other Study ID Numbers:||
|First Posted:||January 9, 2006 Key Record Dates|
|Results First Posted:||August 12, 2020|
|Last Update Posted:||August 12, 2020|
|Last Verified:||April 2019|
stem cell, autoimmune disease, multiple sclerosis
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Immune System Diseases