Clinical Trials

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).

The purpose of this study is to determine the efficacy of frexalimab in delaying the disability progression and the safety up to approximately 51 months administration of study intervention compared to placebo in male and female participants with nrSPMS (aged 18 to 60 years at the time of enrollment).

Patients with progressive forms of multiple sclerosis (MS) have limited

therapeutic options and require new, effective treatments. Ocrelizumab, an

anti-CD20 monoclonal antibody (mAb) that depletes B cells, is currently

the only approved drug in the United States for primary progressive MS

(PPMS). Although ocrelizumab has been shown to deplete B cells in the

cerebrospinal fluid (CSF), a lack of reduction of oligoclonal bands (OCBs)

or immunoglobulin G (IgG) index indicates incomplete depletion and

continued persistence of pathogenic B cells in the brain (Yu 2013), which

could limit its potential efficacy. In addition to anti-CD20 antibodies, only

siponimod, a small molecule drug targeting sphingosine 1 phosphate (S1P)

receptor, and cladribine, a purine nucleoside analogue, are currently

approved for secondary progressive MS (SPMS). None of these medications

fully halt or reverse disease progression. Chimeric antigen receptor

(CAR)-T cells targeting CD19 have a wider spectrum of B-cell depletion

than anti-CD20 mAbs and can target central nervous system (CNS)-resident

B cells by crossing the blood-brain-barrier (BBB) (Richter 2024, Dunn 2025,

Seshadri 2024). These autologous CD19 CAR-T cell therapies have recently

shown early signs of efficacy in MS (Dunn 2025) and other autoimmune

conditions, although further studies are needed to confirm their

effectiveness and safety (Haghikia 2024).

TRX319 is a novel allogeneic, off-the-shelf CD19 CAR-Type 1 regulatory

T (Tr1) cell product capable of suppressing pathogenic T-cell and

inflammatory responses (by inhibiting the production of pro-inflammatory

cytokines IL-1, IL-6, and TNF-) in addition to robust CD19 CAR

mediated B-cell depletion. Because TRX319 has anti-inflammatory

properties, TRX319 cells are expected to have an improved safety profile

(due to IL-6 inhibition) compared to current effector CD19 CAR-T cell

therapies that can result in cytokine release syndrome (CRS) and immune

effector cell-associated neurotoxicity syndrome (ICANS). Therefore,

TRX319 may represent a novel treatment option for patients with

progressive forms of MS.

The purpose of this study is to independently measure the annualized relapse rate (ARR) with administration of frexalimab compared to a daily oral dose of teriflunomide in male and female participants with relapsing forms of multiple sclerosis. People diagnosed with relapsing forms of multiple sclerosis are eligible for enrollment as long as they meet all the inclusion criteria and none of the exclusion criteria.

The purpose of this study is to determine the prevalence, diagnostic accuracy and predictors of antinueronal antibody positivity in serum and cerebrospinal fluid (CSF) of patients iwth focal epilepsy of unknown cause (high) compared to patients with epilepsy of known cause (intermediate) and idiopathic genetic generallized epilepsy (low).  

The purpose of this study is to compare the effectiveness of 7T imaging to the current conventional imaging (3T) to improve MR sensitivity to the changes of multiple sclerosis.  Using FDA-cleared 7T imaging would have a substantial impact on patient management.

The purposes of this study are to explore the association between female sex, reproductive history and clinical phenotypic variability in multiple sclerosis (MS), to explore the association between female sex, reproductive history and radiologic phenotypic variability in MS, and to explore the association between female sex, genetic and environmental interactions in MS.

This study aims to gain a better understanding of the autonomic responses of subjects with multiple sclerosis compared to healthy controls. Utilizing lab-based testing to induce well-characterized autonomic responses, the integrity of the autonomic nervous system through the spinal cord will be assessed though baseline catecholamine/glucocorticoid levels, recordings of sympathetic outflow, and spectral analysis of blood pressure and heart rate variability. Armed with these results, we will generate cardiovascular autonomic phenotypes for individuals to demonstrate normal and abnormal autonomic responses.

The purpose of this study is to longitudinally measure cognitive performance in patients with multiple sclerosis and progressive immune-mediated neurodegenerative disorders in order to determine the rate of progression, and to establish quantitative functional biomarkers that can be used to measure treatment effectiveness in therapy trials directly targeting progressive immune-mediated neurodegeneration.

The purpose of this study is to develop a battery of balance tests for deployment in the clinical setting using the APDM wearable system and to establish a comparable battery for longitudinal in-home assessment using the single GENEActiv wearable device.