NCT04047628 · National Institute of Allergy and Infectious Diseases (NIAID)
Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)
(BEAT-MS)
What this study is about
This is a multi-center forward-looking rater-masked (blinded) randomly assigned 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 randomly assigned at a 1 to 1 (1:1) ratio.
View original scientific description
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).
Interventions
PROCEDURE
Autologous Hematopoietic Stem Cell Transplantation
1. PBSC mobilization \& collection regimen per protocol/ institutional standards includes: intravenous cyclophosphamide (Cytoxan®), 4 grams/m\^2); intravenous mesna (Mesnex®),a total delivery of 4 grams/m\^2); oral dexamethasone, 10 mg dose, four times daily); subcutaneous filgrastim,10 mcg/kg/day until leukapheresis goal is completed; and CD34+ peripheral blood stem cells collection by leukapheresis. 2. Conditioning per protocol\& institutional standards: * 6-day BEAM (e.g. Carmustine (BCNU), Etoposide (VP-16), Cytarbine (Ara-C), and Melphalan) chemotherapy protocol and, * rabbit anti-thymocyte globulin (rATG) 2.5 mg/kg/day x2 3. Autologous cryopreserved graft infusion: The target Cluster of Differentiation (CD)34+ cell dose for infusion is 5 x 10\^6 CD34+ cells/kg (minimum 4 x 10\^6 CD34+ cells/kg; maximum 7.5 x 10\^6 CD34+ cells/kg). For 1\&2 above: Ideal body weight (IBW) versus Actual Body Weight (ABW) are applicable.
BIOLOGICAL
Best Available Therapy (BAT)
Disease-modifying therapy (DMT) selected by the Site Investigator from the below: * cladribine * natalizumab * alemtuzumab * ocrelizumab, * rituximab, * ofatumumab, or * ublituximab
Primary outcome measures
Multiple Sclerosis (MS) Relapse-Free Survival
Time frame: From Day 0 (Randomization to Treatment) Up to 36 Months (3 Years)
MS relapse-free survival, analyzed as time from treatment randomization until MS relapse or death from any cause.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
Where
- Palo Alto, California
- Aurora, Colorado
- Evanston, Illinois
- Worcester, Massachusetts
- Minneapolis, Minnesota
- Rochester, Minnesota
- St Louis, Missouri
- New York, New York
- Rochester, New York
- Durham, North Carolina
- Cincinnati, Ohio
- Cleveland, Ohio
And 8 more locations — see the full list below.
Collaborators
Immune Tolerance Network (ITN), Blood and Marrow Transplant Clinical Trials Network, PPD Development, LP, Rho Federal Systems Division, Inc.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jan 6, 2026 · Source of record for eligibility and locations