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)
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years to 55 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Time periods mentioned: 72 months
- Procedures mentioned: Infusions
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "All participants will be followed for 72 months after randomization (Day 0, Visit 0)."
- "One's own stored (frozen) stem cells are then given back, through an infusion."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study compares a transplant using a person's own blood-forming stem cells with the best available MS medicines. It is for adults ages 18 to 55 with relapsing multiple sclerosis (MS) that stays active despite treatment. Treatment is chosen by chance, and participants are followed for 6 years.
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).
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be 18 to 55 years old and have MS confirmed by study rules. Brain MRI scans must show MS changes that meet those rules.
- Have a score of 6 or less on the EDSS, a scale that measures disability from MS.
- Have at least 2 episodes of MS activity in the past 3 years, including 1 in the past year. At least 1 must be a confirmed symptom flare-up. Another flare-up or new active MRI spot must be at least 1 month apart from that flare-up.
- Have at least 1 episode of MS activity after taking a qualifying MS medicine for at least 1 month.
- Be able to take at least 1 of the study's best available MS medicines, with insurance approval. You must not have had MS activity while taking that medicine or used it in the past year.
- Complete the recommended COVID-19 vaccine series at least 14 days before treatment assignment. Also have antibodies against the chickenpox virus or get at least 1 Shingrix dose at least 4 weeks before assignment.
- Be able to follow study steps and give informed consent. Follow the study's birth control rules if you or your partner could become pregnant.
- If you use marijuana, be willing to switch to MARINOL if assigned to the stem cell transplant.
Who can't join
- Have primary progressive MS, neuromyelitis optica spectrum disorder, or MOG antibody disease. These are different forms of disease affecting the brain, spinal cord, or nerves to the eyes.
- Have a new MS flare-up or receive the listed high-dose steroid treatment within 1 month before assignment. Recent use of an experimental drug or starting a study comparison medicine during screening also rules you out.
- Have signs of PML, a serious brain infection; HIV; a positive TB test; or past or current hepatitis B or C. A positive COVID-19 test within 14 days before assignment also rules you out.
- Have an active infection, or have needed hospital care or injected infection medicines in the past 30 days without approval from an infection specialist. You also cannot have received a live vaccine within 6 weeks before assignment.
- Have certain blood disorders, unexplained high or low blood cell counts, sickle cell anemia, or another disorder of the protein that carries oxygen in blood.
- Have liver scarring or liver test results above study limits; serious heart disease; or heart, kidney, or lung function below study limits. Poorly controlled diabetes, with an HbA1c above 8%, also rules you out.
- Have a history of cancer, except certain treated skin or cervical cancers. Other cancers judged cured may be reviewed individually.
- Have or have had a moderate to severe autoimmune disease that needs treatment, such as lupus or rheumatoid arthritis. These diseases cause the body's defenses to attack its own tissues.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
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). 6\. Candidacy for treatment with at least one of the following high efficacy BAT DMTs: cladribine, natalizumab, alemtuzumab, ocrelizumab, ofatumumab, ublituximab and rituximab. Candidacy for treatment for each BAT DMT is defined as meeting all of the following: 1. No prior disease activity episode, as defined in Inclusion Criterion #5, with the candidate BAT DMT, and 2. No contraindication to the candidate BAT DMT, and 3. No treatment with the candidate BAT DMT in the 12 months prior to screening. 7\. Completion of COVID-19 vaccination series, according to the current Centers for Disease Control and Prevention (CDC) Advisory Committee on Immunization Practices (ACIP) recommendations, ≥ 14 days prior to randomization (Day 0). 8\. Positive for VZV antibodies, or completion of at least one dose of the varicella zoster glycoprotein E (gE) Shingrix vaccine at least 4 weeks prior to randomization (Day 0). 9\. Insurance approval for MS treatment with at least one candidate BAT DMT (see Inclusion Criterion #6). 10\. Ability to comply with study procedures and provide informed consent, in the opinion of the investigator. 11\. Females of childbearing potential (defined in Section 5.4.3.1) and males with female partners of childbearing potential are required to adhere to the contraception provisions of Section 5.4.3.1. 12\. For participants who use medicinal or recreational marijuana, willingness to substitute MARINOL® if randomized to AHSCT (Section 5.4.2.6). 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. 9. History of sickle cell anemia or other hemoglobinopathy. 10. Evidence of past or current hepatitis B or hepatitis C infection, including treated hepatitis B or hepatitis C. Hepatitis B surface antibody following hepatitis B immunization is not considered to be evidence of past infection. 11. Presence or history of mild to severe cirrhosis. 12. Hepatic disease with the presence of either of the following: 1. Total bilirubin ≥ 1.5 times the upper limit of normal (ULN) or total bilirubin ≥ 3.0 times the ULN in the presence of Gilbert's syndrome, or 2. Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥ 2.0 times the ULN. 13. Positive COVID-19 PCR test, or alternative nucleic acid amplification test (NAAT) per institutional standards, within 14 days prior to randomization (Day 0). 14. Evidence of HIV infection. 15. Positive QuantiFERON - TB Gold,TB Gold Plus, or T-SPOT®.TB test results. PPD tuberculin test may be substituted for QuantiFERON - TB Gold, TB Gold Plus, or T-SPOT®.TB test. 16. Active viral, bacterial, endoparasitic, or opportunistic infections. 17. Active invasive fungal infection. 18. Hospitalization for treatment of infections or parenteral (IV or IM) antibacterials, antivirals, antifungals, or antiparasitic agents within the 30 days prior to randomization (Day 0) unless clearance is obtained from an Infectious Disease specialist. 19. Receipt of live or live-attenuated vaccines within 6 weeks of randomization (Day 0). 20. Presence or history of clinically significant cardiac disease including: a. Arrhythmia requiring treatment with any antiarrhythmia therapy, with the exception of low dose beta blocker for intermittent premature ventricular contractions. b. Coronary artery disease with a documented diagnosis of either: i. Chronic exertional angina, or ii. Signs or symptoms of congestive heart failure. c. Evidence of heart valve disease, including any of the following: i. Moderate to severe valve stenosis or insufficiency, or ii. Symptomatic mitral valve prolapse, or iii. Presence of prosthetic mitral or aortic valve. 21. Left ventricular ejection fraction (LVEF) \< 50%. 22. Impaired renal function defined as eGFR \< 60 mL/min/1.73 m2, according to the CKD-EPI formula144. 23. Forced expiratory volume in one second (FEV1) \< 70% predicted (no bronchodilator). 24. Diffusing capacity of the lungs for carbon monoxide (DLCO) (corrected for Hgb) \< 70% predicted. 25. Poorly controlled diabetes mellitus, defined as HbA1c \> 8%. 26. History of malignancy, except adequately treated localized basal cell or squamous skin cancer, or carcinoma in situ of the cervix. Malignancies for which the participant is judged to be cured will be considered on an individual basis by the study adjudication committee (see Section 3.5). 27. Presence or history of any moderate to severe rheumatologic autoimmune disease requiring treatment, including but not limited to the following: systemic lupus erythematous, systemic sclerosis, rheumatoid arthritis, Sjogren's syndrome, polymyositis, dermatomyositis, mixed connective tissue disease, polymyalgia rheumatica, polychondritis, sarcoidosis, vasculitis syndromes, or unspecified collagen vascular disease. 28. Presence of active peptic ulcer disease, defined as endoscopic or radiologic diagnosis of gastric or duodenal ulcer. 29. Prior history of AHSCT. 30. Prior history of solid organ transplantation. 31. Positive pregnancy test or breastfeeding. 32. Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy. 33. Psychiatric illness, mental deficiency, or cognitive dysfunction severe enough to interfere with compliance or informed consent. 34. History of hypersensitivity to rabbit or Escherichia coli-derived proteins. 35. Any metallic material or electronic device in the body, or other condition that precludes the participant from undergoing MRI with gadolinium administration, as determined by the site radiologist. 36. Presence or history of ischemic cerebrovascular disorders, including but not limited to transient ischemic attack, subarachnoid hemorrhage, cerebral thrombosis, cerebral embolism, or cerebral hemorrhage. 37. Presence or history of other neurological disorders, including but not limited to CNS or spinal cord tumor; metabolic or infectious cause of myelopathy; genetically-inherited progressive CNS disorder; CNS sarcoidosis; or systemic autoimmune disorders potentially causing progressive neurologic disease or affecting ability to perform the study assessments. 38. Presence of any medical comorbidity that the investigator determines will significantly increase the risk of treatment mortality. 39. Presence of any other concomitant medical condition that the investigator deems incompatible with trial participation.
Study sites (23)
CA
- Stanford Multiple Sclerosis Center · Palo Alto, CA 94304Emma Martinez · Study contact · (650) 387-5907 · eam1@stanford.eduJeffrey Dunn, MD, FAAN · Principal investigatorMore trials in Palo Alto
CO
- Rocky Mountain Multiple Sclerosis Center, University of Colorado School of Medicine · Aurora, CO 80045Timber Bourassa, BS · Study contact · 303-724-8305 · NeurologyResearchPartners@cuanschutz.eduJohn R. Corboy, MD · Principal investigatorMore trials in Aurora
IL
- Northwestern University · Evanston, IL 60208Matthew Selle · Study contact · autoimmunesct@nm.orgGeorge Georges, MD · Principal investigatorBruce Cohen, MD · Principal investigatorMore trials in Evanston
MA
- University of Massachusetts Memorial Medical Center · Worcester, MA 01655Irina Radu, MD, MHA · Study contact · 774-441-7754 · irina.radu@umassmed.eduCarolina Ionete, MD, PhD · Principal investigatorMore trials in Worcester
MI
- Henry Ford Neuroscience Institute · Detroit, MI 48202Kelly Tundo · Study contact · kciach1@hfhs.orgMirela Cerghet, MD · Principal investigatorMore trials in Detroit
MN
- University of Minnesota Multiple Sclerosis Center · Minneapolis, MN 55455More trials in Minneapolis
- Mayo Clinic · Rochester, MN 55905Lisa Roemer · Study contact · 507-293-9754 · Roemer.Lisa@mayo.eduB. Mark Keegan, MD,FRCPC · Principal investigatorMore trials in Rochester
MO
- John L. Trotter Multiple Sclerosis Center, Washington University School of Medicine in St. Louis · St Louis, MO 63110Laura Teeter · Study contact · 314-747-6247 · lteeter@wustl.eduGregory Wu, MD, PhD · Principal investigatorMore trials in St Louis
NC
- Duke University Medical Center · Durham, NC 27710More trials in Durham
NY
- Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Siinai · New York, NY 10029Sarah Myoung · Study contact · 212-241-3391 · sarah.myoung@mssm.eduGena Persad · Study contact · 212-241-6604Aaron Miller, MD · Principal investigatorMore trials in New York
- Rochester Multiple Sclerosis Center, University of Rochester · Rochester, NY 14620Andrew D. Goodman, MD · Principal investigatorMore trials in Rochester
OH
- University of Cincinnati (UC) Waddell Center for Multiple Sclerosis · Cincinnati, OH 45219Tiffany Rupert, CCRC · Study contact · 513-558-0269 · BEATMSResearch@UCHealth.comAram Zabeti, MD · Principal investigatorMore trials in Cincinnati
- Mellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic · Cleveland, OH 44195Alecia Chase · Study contact · 216-780-4935 · chasea@ccf.orgJeffrey A. Cohen, MD · Principal investigatorMore trials in Cleveland
OR
- Multiple Sclerosis Center, Oregon Health & Science University · Portland, OR 97239Debbie Guess, RN · Study contact · 503-494-7651 · griffide@ohsu.eduYadav Vijayshree, MD,MCR,FANA,FAAN · Principal investigatorMore trials in Portland
PA
- Penn Comprehensive MS Center, Hospital of the University of Pennsylvania · Philadelphia, PA 19104MS MS Clinical Research Team · Study contact · 215-906-4778 · msresearch@pennmedicine.upenn.eduAmit Bar-Or, MD,FRCP,FAAN,FANA · Principal investigatorMore trials in Philadelphia
TX
- University of Texas Southwestern Medical Center: Division of Multiple Sclerosis and Neuroimmunology · Dallas, TX 75390Manual Huichapa · Study contact · 214-645-8216 · manuel.huichapa@utsoutwestern.eduBenjamin Greenberg, MD · Principal investigatorMore trials in Dallas
- Maxine Mesigner Multiple Sclerosis Comprehensive Care Center, Baylor College of Medicine Medical Center · Houston, TX 77030Tahari Griffin · Study contact · 713-798-6097 · tgriffin@bcm.eduGeorge J. Hutton, MD · Principal investigatorMore trials in Houston
VA
- University of Virginia · Charlottesville, VA 22903Matthew Baron · Study contact · 434-297-4102 · ukx3sv@uvahealth.orgMini Singh, MD · Principal investigatorMore trials in Charlottesville
- Virginia Commonwealth University Multiple Sclerosis Treatment and Research Center · Richmond, VA 23219Unsong Oh, MD · Study contact · 804-828-3067 · unsong.oh@vcuhealth.orgUnsong Oh, MD · Principal investigatorMore trials in Richmond
WA
- Clinical Research Division, Fred Hutchinson Cancer Research Center · Seattle, WA 98109More trials in Seattle
- Multiple Sclerosis Center, Swedish Neuroscience Institute · Seattle, WA 98122Izzy Bueno · Study contact · 206-320-4120 · Isabella.Bueno1@providence.orgJames D. Bowen, MD · Principal investigatorMore trials in Seattle
- Multiple Sclerosis Center at Northwest Hospital · Seattle, WA 98133Elisa McGee · Study contact · 206-598-9260 · emcgee@uw.eduAnnette Wundes, MD · Principal investigatorMore trials in Seattle
WI
- Medical College of Wisconsin · Milwaukee, WI 53226Pam Dailey · Study contact · 262-689-1846 · pdailey@mcw.eduAhmed Obeidat, M.D. · Principal investigatorMore trials in Milwaukee