BEAT-MS Multiple Sclerosis Trial

BEAT-MS Multiple Sclerosis Trial
Recruiting
18 years - 55 years
All
Phase N/A
2 Locations

Brief description of study

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 of study

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 of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Multiple Sclerosis
  • Age: 18 years - 55 years
  • Gender: All

Inclusion Criteria:
  • Age 18 to 55 years, inclusive, at the time of the screening Visit -2.
  • Diagnosis of MS according to the 2017 McDonald Criteria139.
  • EDSS ≤ 6.0 at the time of randomization (Day 0).
  • 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.
  • 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
Exclusion Criteria:
  • Diagnosis of primary progressive MS according to the 2017 McDonald criteria.
  • History of neuromyelitis optica spectrum disorder or MOG antibody disease.
  • 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.
  • 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.
  • Initiation of any BAT DMT (see Section 5.2.1) between Visit -2 and randomization (Day 0).
  • Brain MRI or cerebrospinal fluid (CSF) examination indicating a diagnosis of progressive multifocal leukoencephalopathy (PML).
  • History of cytopenia consistent with the diagnosis of myelodysplastic syndrome (MDS).
  • Presence of unexplained cytopenia, polycythemia, thrombocythemia or leukocytosis.
  • History of sickle cell anemia or other hemoglobinopathy.
  • 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.
  • Presence or history of mild to severe cirrhosis.

Updated on 04 Aug 2024. Study ID: TX7616

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