Vancomycin Study in Multiple Sclerosis (MS)

NCT05539729 | Recruiting Phase 1 FDA Drug

• 1 other identifier: GCO-22-0462
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The overall goal of this study is to elucidate a mechanism by which vancomycin modulates the gut-brain axis in multiple sclerosis (MS). The gut microbiome plays an important role in autoimmunity, including MS. However, the identity of gut microbes modulating neuroinflammation in MS and their mechanisms of action remain obscure. Hence, here the research team proposes to investigate the effects of vancomycin on the gut microbiota composition, peripheral immune function, and brain MRI lesions in MS patients.

Conditions

Multiple Sclerosis

Keywords

gut microbiome; peripheral immune function; neuroinflammation; gut-brain axis

Outcome Measures

Primary Outcomes (3)

• Changes in abundance of butyrate producing bacteria

  Changes in abundance of butyrate producing bacteria from baseline treatment up to 6 weeks

  Baseline up to 6 weeks

• Changes in Serum Butyrate levels

  Changes in serum butyrate level from baseline treatment up to 6 weeks Butyrate is a substance that is produce when gut bacteria breaks down food. Butyrate can get into our blood circulation and regulate how our immune cells function.

  Baseline up to 6 weeks

• Changes in number of peripheral T cells

  Change in frequency of peripheral regulatory T cells baseline treatment up to 6 weeks. T cells are a type of lymphocyte. Lymphocytes are a type of white blood cell. They make up part of the immune system. T cells help the body fight diseases or harmful substances, such as bacteria or viruses.

  Baseline up to 6 weeks

Secondary Outcomes (14)

• Changes in abundance of short chain fatty acids (SCFAs)-producing bacteria

  Baseline and 12 months

• Change in stool SCFAs levels

  Baseline and 12 months

• Change in serum SCFAs levels

  Baseline and 12 months

• Change in number of gadolium enhancing brain lesions

  Baseline and 12 months

• Change in volume of gadolium enhancing brain lesions

  Baseline and 12 months

Study Arms (2)

Vancomycin

EXPERIMENTAL

125mg antibiotic taken 4 times daily by mouth

Drug: Vancomycin

Placebo

PLACEBO COMPARATOR

Matching placebo taken 4 times daily by mouth

Drug: Placebo

Interventions

Vancomycin DRUG

A marketed antibiotic (Study Drug) supplied by Amerisource Bergen, by the Mount Sinai Investigational Drug Services (IDS), and encapsulated in red coating to match the placebo.

Vancomycin

Placebo DRUG

Placebo created by the IDS and encapsulated in red coating to match the Study Drug.

Placebo

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• aged 18 - 50
• newly diagnosed MS (2017 McDonald criteria), CIS or RIS patients, who have experienced symptoms no earlier than the past year
• treatment naive
• able to understand the risks, benefits, and alternatives of participation and give meaningful consent

You may not qualify if:

• antibiotic use within the past 90 days;
• pre- or probiotic use within past month or corticosteroids use within the past month;
• use of tobacco products within the past 1 month;
• history of treatment with immunosuppressants;
• history of gastroenteritis within the past month or diagnosis with a chronic infectious disease, i.e. hepatitis B, C or HIV;
• pregnancy or less than 6 months postpartum;
• irritable bowel syndrome and other bowel dysfunction such as constipation;
• history of bowel surgery;
• inflammatory bowel disease, rheumatoid arthritis, systemic lupus erythematosus, diabetes and any other auto-immune illness;
• diagnosis with another neurological disease, behavioral or psychiatric conditions that would be incompatible with a safe and successful participation in the study (such as severe major depression, schizophrenia and presence of psychotic symptoms);
• eating disorders such as anorexia nervosa, bulimia, or binge eating syndrome;
• travel outside of the country within the past month;
• contraindication to vancomycin including estimated glomerular filtration rate of \<60ml/min, impaired hearing or known allergy.
• Contraindication to MRI such as implanted metallic objects

Sponsors & Collaborators

• Icahn School of Medicine at Mount Sinai: lead
• Doris Duke Charitable Foundation: collaborator

Study Sites (1)

Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai

New York, New York, 10029, United States

RECRUITING

MeSH Terms

Conditions

Multiple Sclerosis; Neuroinflammatory Diseases

Interventions

Vancomycin

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Inflammation; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Glycopeptides; Glycoconjugates; Carbohydrates; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Stephanie K Tankou, MD

  Icahn School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Susan E Filomena, BA

CONTACT

212-2413841; susan.filomena@mssm.edu

Gena Persad

CONTACT

212-241-6604; gena.persad@mssm.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: The research team will be blinded to the treatment group (placebo/vancomycin).
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor, Neurology

Model Details: Placebo-controlled blinded trial

Study Record Dates

• First Submitted: September 12, 2022
• First Posted: September 14, 2022
• Study Start: January 31, 2023
• Primary Completion: March 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: September 10, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)