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Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) | Clinical Trials | Clareo Health

RECRUITINGPHASE3

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

A Multicenter Randomized Controlled Trial of Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis (ITN077AI)

NCT04047628•National Institute of Allergy and Infectious Diseases (NIAID)

View on ClinicalTrials.gov

Summary

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).

Detailed Description

Participant recruitment for this six-year research study focuses on multiple sclerosis (MS) that has remained active despite treatment. This study will compare high dose immunosuppression followed by autologous hematopoietic stem cell transplantation (AHSCT) to best available therapy (BAT) in the treatment of relapsing MS. MS is a disease caused by one's own immune cells. Normally, immune cells fight infection. In MS, immune cells called T cells, or chemical products made by immune cells, react against the covering or coat (myelin) of nerve fibers in the brain and spinal cord. This leads to stripping the coat from certain nerve fibers (demyelination), and this causes neurologic problems. MS can cause loss of vision, weakness or incoordination, loss or changes in sensation, problems with thinking or memory, problems controlling urination, and other disabilities. Most individuals with MS first have immune attacks (called relapses) followed by periods of stability. Over time, MS can have episodes of new and worsening symptoms, ranging from mild to disabling. This is called relapsing MS. Relapsing MS includes relapsing remitting MS (RRMS) and secondary progressive MS (SPMS). There are medicines (drugs) to decrease relapses, but these are neither considered to be curative nor, to induce prolonged remissions without continuing therapy. More than a dozen medicines have been approved for the treatment of relapsing forms of MS. These medicines differ in how safe they are and how well they work. Despite availability of an increasing number of effective medicines, some individuals with relapsing MS do not respond to treatment. Research is being conducted to find other treatments. High dose immunosuppression followed by autologous hematopoietic stem cell transplantation (AHSCT) has been shown to help relapsing MS in cases where medicines did not work. AHSCT involves collecting stem cells, which are produced in the bone marrow. These stem cells are "mobilized" to leave the bone marrow and move into the blood where they can be collected and stored. Participants will then receive chemotherapy intended to kill immune cells. One's own stored (frozen) stem cells are then given back, through an infusion. This "transplant" of one's stem cells allows the body to form new immune cells in order to restore their immune system. New research suggest that MS might be better controlled with AHSCT than with medicines.

Eligibility

Age

18 - 55 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Age 18 to 55 years, inclusive, at the time of the screening Visit -2.
•2. Diagnosis of MS according to the 2017 McDonald Criteria139.
•3. EDSS ≤ 6.0 at the time of randomization (Day 0).
•4. T2 abnormalities on brain MRI that fulfill the 2017 McDonald MRI criteria for dissemination in space139. A detailed MRI report or MRI images must be available for review by the site neurology investigator.
•5. Highly active treatment-resistant relapsing MS, defined as ≥ 2 episodes of disease activity in the 36 months prior to the screening visit (Visit -2). The two disease activity episodes will be a clinical MS relapse or MRI evidence of MS disease activity and must meet all the criteria described below:
•1. At least one episode of disease activity must occur following ≥ 1 month of treatment with one of the following: (i) an oral DMT approved by the FDA for the treatment of relapsing MS, or (ii) a monoclonal antibody approved by the FDA for the treatment of relapsing MS, or (iii) rituximab. Qualifying DMTs include: dimethyl fumarate, diroximel fumarate, monomethyl fumarate, teriflunomide, cladribine, daclizumab, ponesimod, siponimod, ozanimod, fingolimod, rituximab, ocrelizumab, natalizumab, alemtuzumab, ublituximab, and ofatumumab, and
•2. At least one episode of disease activity must have occurred within the 12 months prior to the screening visit (Visit -2), and
•3. At least one episode of disease activity must be a clinical MS relapse (see item c.i. below). The other episode(s) must occur at least one month before or after the onset of the clinical MS relapse, and must be either another clinical MS relapse or MRI evidence of disease activity (see item c.ii. below):
•i. Clinical MS relapse must be confirmed by a neurologist's assessment and documented contemporaneously in the medical record. If the clinical MS relapse is not documented in the medical record, it must be approved by the study adjudication committee (see Section 3.5), and ii. MRI evidence of disease activity must include ≥ 1 unique active lesion on one or more brain or spinal cord MRIs. Detailed MRI reports or MRI images must be available for review by the site neurology investigator. A unique active lesion is defined as either of the following:
•1\. A gadolinium-enhancing lesion, or 2. A new non-enhancing T2 lesion compared to a reference scan obtained not more than 36 months prior to the screening visit (Visit -2).
+10 more criteria

Exclusion Criteria

•1. Diagnosis of primary progressive MS according to the 2017 McDonald criteria.
•2. History of neuromyelitis optica spectrum disorder or MOG antibody disease.
•3. Prior treatment with an investigational agent within 3 months or 5 half-lives, whichever is longer. Agents authorized by the FDA for prevention or treatment of COVID-19 are not considered investigational.
•4. Either of the following within one month prior to randomization (Day 0):
•1. Onset of acute MS relapse, or
•2. Treatment with intravenous methylprednisolone 1000 mg/day for 3 days or equivalent.
•5. Initiation of any BAT DMT (see Section 5.2.1) between Visit -2 and randomization (Day 0).
•6. Brain MRI or cerebrospinal fluid (CSF) examination indicating a diagnosis of progressive multifocal leukoencephalopathy (PML).
•7. History of cytopenia consistent with the diagnosis of myelodysplastic syndrome (MDS).
•8. Presence of unexplained cytopenia, polycythemia, thrombocythemia or leukocytosis.
+34 more criteria

Locations (22)

Stanford Multiple Sclerosis Center

Palo Alto, California, United States

Rocky Mountain Multiple Sclerosis Center, University of Colorado School of Medicine

Aurora, Colorado, United States

Northwestern University

Evanston, Illinois, United States

University of Massachusetts Memorial Medical Center

Worcester, Massachusetts, United States

University of Minnesota Multiple Sclerosis Center

Minneapolis, Minnesota, United States

Mayo Clinic

Rochester, Minnesota, United States

John L. Trotter Multiple Sclerosis Center, Washington University School of Medicine in St. Louis

St Louis, Missouri, United States

Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Siinai

New York, New York, United States

Rochester Multiple Sclerosis Center, University of Rochester

Rochester, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Key Dates

Start Date

December 19, 2019

Primary Completion Date

October 1, 2026

Completion Date

October 1, 2029

Last Updated

January 6, 2026

Enrollment

156

ESTIMATED participants

Conditions

Relapsing Multiple SclerosisRelapsing Remitting Multiple SclerosisSecondary Progressive Multiple Sclerosis

Interventions

Autologous Hematopoietic Stem Cell Transplantation

PROCEDURE

Best Available Therapy (BAT)

BIOLOGICAL

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Secondary Progressive Multiple Sclerosis (SPMS)

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RECRUITING

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Multiple SclerosisRemitting-Relapsing Multiple Sclerosis+4 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 6, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

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