Actively Recruiting

Phase 3
Age: 18Years - 55Years
All Genders
NCT04047628

Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)

Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2026-01-06

156

Participants Needed

22

Research Sites

510 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

This is a multi-center prospective rater-masked (blinded) randomized controlled trial of 156 participants, comparing the treatment strategy of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1 to 1 (1:1) ratio. All participants will be followed for 72 months after randomization (Day 0, Visit 0).

CONDITIONS

Official 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

Eligible

You may qualify if you...

  • Age 18 to 55 years, inclusive, at the time of screening
  • Diagnosed with multiple sclerosis according to the 2017 McDonald Criteria
  • Expanded Disability Status Scale (EDSS) score of 6.0 or less at randomization
  • Brain MRI showing T2 abnormalities meeting criteria for MS dissemination in space
  • Highly active, treatment-resistant relapsing MS with at least two disease activity episodes in the past 36 months
  • At least one disease activity episode occurring after one month of treatment with an approved oral or monoclonal antibody disease-modifying therapy (DMT) or rituximab
  • At least one disease activity episode within 12 months prior to screening
  • At least one clinical MS relapse confirmed by a neurologist
  • Eligibility for treatment with at least one high efficacy BAT DMT without prior use or contraindications
  • Completed COVID-19 vaccination series at least 14 days before randomization
  • Positive varicella zoster virus antibodies or received at least one dose of Shingrix vaccine at least 4 weeks before randomization
  • Insurance approval for treatment with at least one candidate BAT DMT
  • Ability to comply with study procedures and provide informed consent
  • Adherence to contraception requirements if of childbearing potential
  • Willingness to substitute Marinol if using marijuana and randomized to AHSCT
Not Eligible

You will not qualify if you...

  • Diagnosis of primary progressive MS
  • History of neuromyelitis optica spectrum disorder or MOG antibody disease
  • Prior treatment with investigational agents within 3 months or 5 half-lives
  • Acute MS relapse or high-dose steroid treatment within one month prior to randomization
  • Starting any BAT DMT between screening and randomization
  • Brain MRI or cerebrospinal fluid findings indicating progressive multifocal leukoencephalopathy (PML)
  • History or presence of blood disorders such as myelodysplastic syndrome, cytopenia, sickle cell anemia, or hemoglobinopathies
  • Evidence or history of hepatitis B or C infection, cirrhosis, or significant liver disease
  • Positive COVID-19 test within 14 days prior to randomization
  • HIV infection
  • Positive tuberculosis test
  • Active infections including viral, bacterial, fungal, or parasitic
  • Recent hospitalization for infections requiring intravenous treatment unless cleared
  • Receipt of live vaccines within 6 weeks of randomization
  • Significant heart disease including arrhythmias, coronary artery disease, heart valve disease, or low heart function
  • Impaired kidney or lung function
  • Poorly controlled diabetes
  • History of cancer except certain localized skin cancers or cervical carcinoma in situ
  • Moderate to severe autoimmune rheumatologic diseases requiring treatment
  • Active peptic ulcer disease
  • Prior autologous hematopoietic stem cell transplant or solid organ transplant
  • Positive pregnancy test or breastfeeding
  • Inability to accept irreversible sterility as a side effect
  • Severe psychiatric illness or cognitive dysfunction interfering with consent
  • Allergy to rabbit or E. coli proteins
  • Metallic or electronic implants preventing MRI with gadolinium
  • History of ischemic cerebrovascular disorders
  • Other neurological or systemic autoimmune disorders affecting participation
  • Any medical condition increasing treatment mortality risk or incompatible with trial participation

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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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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Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) | DecenTrialz