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A Trial of Bile Acid Supplementation in Patients With Multiple Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03423121
Recruitment Status : Active, not recruiting
First Posted : February 6, 2018
Last Update Posted : May 6, 2022
Information provided by (Responsible Party):
Johns Hopkins University

Brief Summary:
This study aims to identify the safety and tolerability of bile acid supplementation in patients with progressive Multiple Sclerosis (MS). Participants will also be assessed for an impact of the bile acid on their immune system and gut microbiome. Half of the participants will receive the bile acid tauroursodeoxycholic acid (TUDCA) and half will receive placebo. The investigators believe participants who take TUDCA will have normalization of blood bile acid levels, a normalization of abnormal immune response and a normalization of the gut microbiome.

Condition or disease Intervention/treatment Phase
Progressive Multiple Sclerosis Drug: Tauroursodeoxycholic Acid Drug: Placebo oral capsule Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 58 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Care Provider)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Trial of Tauroursodeoxycholic Acid Supplementation in Progressive MS Patients
Actual Study Start Date : June 19, 2018
Actual Primary Completion Date : April 28, 2022
Estimated Study Completion Date : July 2022

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: TUDCA Treatment
Tauroursodeoxycholic acid (Taurolite) 250 mg four capsules by mouth, twice daily for 16 weeks.
Drug: Tauroursodeoxycholic Acid
Participants will be given 1 gram of Tauroursodeoxycholic acid twice daily in the form of four 250mg capsules.
Other Name: Taurolite

Placebo Comparator: Placebo oral capsule
Placebo oral capsule four capsules by mouth, twice daily for 16 weeks.
Drug: Placebo oral capsule
Participants will be given four capsules of the placebo twice daily.

Primary Outcome Measures :
  1. Incidence of Treatment-related adverse events [ Time Frame: 16 weeks ]
    Safety and tolerability will be assessed based on treatment-related adverse events in the two arms

Secondary Outcome Measures :
  1. Metabolomics [ Time Frame: Baseline to 16 weeks ]
    Change in fasting bile acid levels in plasma.

  2. Gut microbiota [ Time Frame: Baseline to 16 weeks ]
    Change in composition of the gut microbiota assessed using shot-gun metagenomic sequencing in first morning stool specimen.

  3. Immunophenotyping [ Time Frame: Baseline to 16 weeks ]
    Change in flow cytometric assessments of peripheral blood mononuclear cells.

  4. Quality of Life [ Time Frame: Baseline to 16 weeks ]
    Change in physical and mental health scores as assessed using the Multiple Sclerosis Quality of Life-54 (MSQOL-54) instrument. This 54-item instrument generates 12 subscales along with two summary scores, and two additional single-item measures. Two summary scores - physical health and mental health - are derived from a weighted combination of scale scores. Higher scores suggest a better quality of life.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of Progressive MS based on Lublin Criteria
  • Low bile acid levels identified using targeted metabolomics analysis
  • On the same therapy for the past 6 months and not expected to switch therapy in the next 6 months
  • No relapse in the past 3 months

Exclusion Criteria:

  • No previous history of liver disease or cholecystectomy
  • No stage IV/V chronic kidney disease or other severe metabolic derangements (e.g. poorly controlled thyroid disease or diabetes)
  • BMI < 15 kg/m2 and BMI > 40 kg/m2
  • Female patients who are pregnant or nursing, or not willing to use contraception
  • Chronic antibiotic use
  • Corticosteroid treatment within the past 30 days
  • Known history of other neuroinflammatory, neurodegenerative or systemic autoimmune disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03423121

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United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Sponsors and Collaborators
Johns Hopkins University
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Principal Investigator: Pavan Bhargava, MBBS, MD Johns Hopkins University
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Responsible Party: Johns Hopkins University Identifier: NCT03423121    
Other Study ID Numbers: IRB00144766
First Posted: February 6, 2018    Key Record Dates
Last Update Posted: May 6, 2022
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Multiple Sclerosis
Multiple Sclerosis, Chronic Progressive
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Antiviral Agents
Anti-Infective Agents
Cholagogues and Choleretics
Gastrointestinal Agents