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Phase 2 N=54 Randomized Double-blind Treatment

A Trial of Bile Acid Supplementation in Patients With Multiple Sclerosis

Progressive Multiple Sclerosis

Enrolled (actual)
54
Serious AEs
2.1%
Results posted
May 2023
Primary outcome: Primary: Number of Participants With at Least One Treatment-related Adverse Event — 10; 7 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Tauroursodeoxycholic Acid (Drug); Placebo oral capsule (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Johns Hopkins University
Primary completion
Apr 2022

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Participants With at Least One Treatment-related Adverse Event
10; 7
PRIMARY
Number of Total Treatment-related Adverse Events
11; 8
PRIMARY
Incidence of Treatment-related Adverse Events (AE)
1218; 1061
SECONDARY
Change in Fasting Bile Acid Levels in Plasma
0.44; 0.48; -0.4; 0.58; 0.14; 0.46
SECONDARY
Change in Microbiome Alpha-diversity Measured by Shannon Index at the End of the Study
-0.15; 0.06
SECONDARY
Change in Flow Cytometric Assessments of Peripheral Blood Mononuclear Cells (PBMCs)
-1.34; 3.05; -1.9; 2.61
SECONDARY
Change in Quality of Life Based on Multiple Sclerosis Quality of Life (MSQOL)-54 Instrument
-0.15; 0.01; -0.44; 4.57

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.

Eligibility Criteria

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 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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03423121). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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