High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS.
Richard A Nash, George J Hutton, Michael K Racke...
https://pubmed.ncbi.nlm.nih.gov/28148635Actively Recruiting
Phase 3
Age: 18Years - 55Years
All Genders
ID04047628
A Multicenter Randomized Controlled Trial Comparing Best Available Therapy to Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis
Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2026-01-06
156
Participants Needed
22
Research Sites
156 weeks
Total Duration
On this page
Sponsors
N
National Institute of Allergy and Infectious Diseases (NIAID)
Lead Sponsor
I
Immune Tolerance Network (ITN)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating treatment options for people with relapsing multiple sclerosis (MS) that remains active despite current therapies. This phase 3 randomized controlled trial compares autologous hematopoietic stem cell transplantation (AHSCT) with the best available therapy (BAT) for treatment-resistant relapsing MS. MS is an immune disease that damages nerve fibers in the brain and spinal cord, causing symptoms like vision loss, weakness, sensory changes, and memory problems. The study aims to see if AHSCT better controls the disease compared to standard medicines over time. Participants will be randomly assigned to one of two groups: the AHSCT group or the BAT group. The AHSCT group undergoes a three-step process including mobilization and collection of their own stem cells, high-dose chemotherapy to destroy immune cells, and reinfusion of their stored stem cells to rebuild their immune system. The BAT group will receive one of several approved medicines for relapsing MS, chosen by the study doctor. Both treatments are closely monitored throughout the study. Participants will be followed for six years after starting treatment. They will have regular assessments including brain MRI scans, neurologic exams, and blood tests to track MS relapses, disability changes, brain volume, and other health measures. Researchers will also monitor for serious side effects and infections. The main outcome is how long participants remain free from MS relapses during the first three years. This long-term follow-up aims to provide a detailed understanding of how these treatments affect disease activity and safety over time.
CONDITIONS
Brief Title
Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)
Who Can Participate
Age: 18Years - 55Years
All Genders
Eligibility Criteria
You may qualify if you...
- Age 18 to 55 years at screening
- Diagnosis of multiple sclerosis by 2017 McDonald Criteria
- Expanded Disability Status Scale (EDSS) score of 6.0 or less at randomization
- Brain MRI showing T2 abnormalities meeting dissemination in space criteria
- Highly active relapsing MS with at least 2 disease activity episodes in past 36 months
- At least one episode of disease activity after one month of FDA-approved MS treatment
- At least one disease activity episode within 12 months before screening
- Clinical MS relapse confirmed by neurologist or approved by study committee
- MRI evidence of active lesions on brain or spinal cord
- Eligible for at least one high efficacy best available therapy (BAT) drug
- Completed COVID-19 vaccination series at least 14 days before randomization
- Positive for varicella zoster virus antibodies or received Shingrix vaccine
- Insurance approval for treatment with at least one BAT drug
- Able to comply with study procedures and give informed consent
- Females and males with female partners must use contraception
- Willingness to substitute MARINOL if randomized to AHSCT if using marijuana
You will not qualify if you...
- Diagnosis of primary progressive MS
- History of neuromyelitis optica spectrum disorder or MOG antibody disease
- Treatment with investigational agents in past 3 months or 5 half-lives
- Acute MS relapse or high-dose steroid treatment within one month before randomization
- Starting any BAT drug between screening and randomization
- Evidence or history of progressive multifocal leukoencephalopathy (PML)
- History or presence of blood disorders like myelodysplastic syndrome or hemoglobinopathy
- Past or current hepatitis B or C infection
- Liver disease including cirrhosis or elevated liver enzymes
- Positive COVID-19 test within 14 days before randomization
- HIV infection
- Positive tuberculosis test
- Active infections including fungal or opportunistic
- Recent hospitalization for infection unless cleared by specialist
- Recent live vaccine within 6 weeks before randomization
- Significant heart diseases or valve problems
- Left ventricular ejection fraction below 50%
- Impaired kidney function with eGFR below 60
- Lung function below 70% predicted
- Poorly controlled diabetes with HbA1c over 8%
- History of malignancy except some treated skin or cervical cancers
- Moderate to severe autoimmune rheumatologic diseases
- Active peptic ulcer disease
- Prior hematopoietic stem cell transplant or solid organ transplant
- Pregnancy or breastfeeding
- Inability to accept irreversible sterility as side effect
- Severe psychiatric or cognitive issues interfering with consent
- Allergies to rabbit or E coli proteins
- Metal or electronic implants preventing MRI with contrast
- History of cerebrovascular disorders
- Other neurological disorders affecting assessments
- Medical conditions increasing treatment risk or incompatible with study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 72 months (6 years)
Participants will be randomized to receive either autologous hematopoietic stem cell transplantation (AHSCT) or best available therapy (BAT). AHSCT includes mobilization and collection of stem cells, high-dose chemotherapy conditioning, and infusion of the collected stem cells. BAT involves disease-modifying therapy selected by the site investigator from approved medications for multiple sclerosis.
Visits scheduled according to treatment arm protocols
Follow-up
Duration - Up to 72 months (6 years)
Participants will be monitored for multiple sclerosis relapse-free survival, disease activity, disability changes, and adverse events following treatment.
Regular follow-up visits as per study schedule
Trial Site Locations
Total: 22 locations
1
Stanford Multiple Sclerosis Center
Palo Alto, California, United States, 94304
Actively Recruiting
2
Rocky Mountain Multiple Sclerosis Center, University of Colorado School of Medicine
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Northwestern University
Evanston, Illinois, United States, 60208
Actively Recruiting
4
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States, 01655
Actively Recruiting
5
University of Minnesota Multiple Sclerosis Center
Minneapolis, Minnesota, United States, 55455
Active, Not Recruiting
6
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
7
John L. Trotter Multiple Sclerosis Center, Washington University School of Medicine in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
8
Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Siinai
New York, New York, United States, 10029
Actively Recruiting
9
Rochester Multiple Sclerosis Center, University of Rochester
Rochester, New York, United States, 14620
Not Yet Recruiting
10
Duke University Medical Center
Durham, North Carolina, United States, 27710
Active, Not Recruiting
11
University of Cincinnati (UC) Waddell Center for Multiple Sclerosis
Cincinnati, Ohio, United States, 45219
Actively Recruiting
12
Mellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
13
Multiple Sclerosis Center, Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
14
Penn Comprehensive MS Center, Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
15
University of Texas Southwestern Medical Center: Division of Multiple Sclerosis and Neuroimmunology
Dallas, Texas, United States, 75390
Actively Recruiting
16
Maxine Mesigner Multiple Sclerosis Comprehensive Care Center, Baylor College of Medicine Medical Center
Houston, Texas, United States, 77030
Actively Recruiting
17
University of Virginia
Charlottesville, Virginia, United States, 22903
Actively Recruiting
18
Virginia Commonwealth University Multiple Sclerosis Treatment and Research Center
Richmond, Virginia, United States, 23219
Actively Recruiting
19
Clinical Research Division, Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Actively Recruiting
20
Multiple Sclerosis Center, Swedish Neuroscience Institute
Seattle, Washington, United States, 98122
Actively Recruiting
21
Multiple Sclerosis Center at Northwest Hospital
Seattle, Washington, United States, 98133
Actively Recruiting
22
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
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How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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Published Research Related To This Trial
High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for relapsing-remitting multiple sclerosis (HALT-MS): a 3-year interim report.
Richard A Nash, George J Hutton, Michael K Racke...
https://pubmed.ncbi.nlm.nih.gov/25546364Autologous Hematopoietic Cell Transplantation for Treatment-Refractory Relapsing Multiple Sclerosis: Position Statement from the American Society for Blood and Marrow Transplantation.
Jeffrey A Cohen, Laura E Baldassari, Harold L Atkins...
https://pubmed.ncbi.nlm.nih.gov/30794930