A Phase 1/2 interventional study of Tauroursodeoxycholic Acid and Placebo oral capsule in Progressive Multiple Sclerosis, sponsored by Johns Hopkins University. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-05-03.
Sponsored by Johns Hopkins University · Phase 1/2, Interventional, and Treatment
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.
244 studies on the registry are indexed under Multiple Sclerosis, Chronic Progressive; 53 are open to participants now.
This study's enrollment of 59 is close to the median of 54 across 189 interventional studies indexed under Multiple Sclerosis, Chronic Progressive.
Browse Multiple Sclerosis, Chronic Progressive studies →Johns Hopkins University is the lead sponsor of 1,815 studies on the registry; 315 are open to participants now.
Of its 208 completed or terminated interventional studies of FDA-regulated products, 144 (69%) have results posted.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Tauroursodeoxycholic acid (Taurolite) 250 mg four capsules by mouth, twice daily for 16 weeks.
Drug: Tauroursodeoxycholic Acid
Placebo oral capsule four capsules by mouth, twice daily for 16 weeks.
Drug: Placebo oral capsule
Participants will be given 1 gram of Tauroursodeoxycholic acid twice daily in the form of four 250mg capsules.
Also known as: Taurolite
Participants will be given four capsules of the placebo twice daily.
Number of Participants With at Least One Treatment-related Adverse Event
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms.
Time frame: 16 weeks
Number of Total Treatment-related Adverse Events
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms.
Time frame: 16 weeks
Incidence of Treatment-related Adverse Events (AE)
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms. AE incidence will be measured as total number of events per 1000 exposure years.
Time frame: 16 weeks
Change in Fasting Bile Acid Levels in Plasma
The change of targeted bile acid levels over the course of 16 weeks (duration of the study) is reported. Bile acid levels (ng/mL) were log transformed before analysis to approximate normal distribution. Units are log(levels) per 16 weeks. Values are derived from linear mixed-effects models.
Time frame: Baseline to 16 weeks
Change in Microbiome Alpha-diversity Measured by Shannon Index at the End of the Study
Change in Shannon index of the gut microbiota between baseline and end of study (16 weeks). Shot-gun metagenomic sequencing in first morning stool specimen was utilized to derive the microbiome composition. Higher values of the index indicate more diversity in the microbial community. The minimum value the Shannon index can take is 0 (no diversity). There is no upper limit to the index.
Time frame: Baseline to 16 weeks
Change in Flow Cytometric Assessments of Peripheral Blood Mononuclear Cells (PBMCs)
Change in flow cytometric assessments over the course of 16 weeks (duration of the study). Cells are expressed as ratios of their parent types. Units reported as change in the ratio per 16 weeks. Values are derived from linear mixed-effects models.
Time frame: Baseline to 16 weeks
Change in Quality of Life Based on Multiple Sclerosis Quality of Life (MSQOL)-54 Instrument
Change in physical and mental health scores as assessed using the Multiple Sclerosis Quality of Life-54 (MSQOL-54) instrument over the course of 16 weeks (duration of the study). 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. Scores can range from 0 to 100.
Time frame: Baseline to 16 weeks
| Milestone | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| Started | 28 | 26 |
| Completed | 25 | 20 |
| Not completed | 3 | 6 |
| Withdrew: Lost to follow-up | 2 | 6 |
| Withdrew: Adverse event | 1 | 0 |
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms.
| Participants | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| Number of Participants With at Least One Treatment-related Adverse Event | 10 | 7 |
The change of targeted bile acid levels over the course of 16 weeks (duration of the study) is reported. Bile acid levels (ng/mL) were log transformed before analysis to approximate normal distribution. Units are log(levels) per 16 weeks. Values are derived from linear mixed-effects models.
| log(levels[ng/mL]) per 16 weeks | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| Chenodeoxycholic Acid | 0.44 (-0.14 to 1.02) | 0.48 (-0.13 to 1.09) |
| Cholic Acid | -0.4 (-1.04 to 0.23) | 0.58 (-0.21 to 1.38) |
| Deoxycholic Acid | 0.14 (-0.21 to 0.48) | 0.46 (0.13 to 0.8) |
| Glycochenodeoxycholic Acid | 0.3 (-0.14 to 0.74) | -0.09 (-0.63 to 0.44) |
| Glycocholic Acid | -0.02 (-0.46 to 0.42) | -0.32 (-0.88 to 0.24) |
| Glycodeoxycholic Acid | 0.1 (-0.36 to 0.56) | 0.14 (-0.36 to 0.63) |
| Glycolithocholic Acid | 0.9 (0.33 to 1.47) | 0.35 (-0.01 to 0.71) |
| Glycoursodeoxycholic Acid | 2.3 (1.32 to 3.29) | 0.14 (-0.35 to 0.64) |
| Lithocholic Acid | 0.75 (0.28 to 1.22) | 0.2 (-0.12 to 0.51) |
| Taurochenodeoxycholic Acid | 0.05 (-0.49 to 0.59) | -0.04 (-0.64 to 0.57) |
| Taurocholic Acid | -0.23 (-0.74 to 0.28) | -0.26 (-0.83 to 0.3) |
| Taurodeoxycholic Acid | -0.31 (-0.87 to 0.25) | 0.1 (-0.45 to 0.65) |
| Taurolithocholic Acid | 0.15 (-0.19 to 0.5) | 0.14 (-0.13 to 0.42) |
| Tauroursodeoxycholic Acid | 2.52 (1.3 to 3.73) | -0.22 (-0.52 to 0.07) |
| Ursodeoxycholic Acid | 1.89 (0.76 to 3.02) | 0.37 (-0.03 to 0.78) |
Change in Shannon index of the gut microbiota between baseline and end of study (16 weeks). Shot-gun metagenomic sequencing in first morning stool specimen was utilized to derive the microbiome composition. Higher values of the index indicate more diversity in the microbial community. The minimum value the Shannon index can take is 0 (no diversity). There is no upper limit to the index.
| score on a scale | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| Change in Microbiome Alpha-diversity Measured by Shannon Index at the End of the Study | -0.15 (-0.47 to 0.18) | 0.06 (-0.3 to 0.41) |
Change in flow cytometric assessments over the course of 16 weeks (duration of the study). Cells are expressed as ratios of their parent types. Units reported as change in the ratio per 16 weeks. Values are derived from linear mixed-effects models.
| parent cell ratio per 16 weeks | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| T helper memory cells | -1.34 (-4.63 to 1.96) | 3.05 (-1.88 to 7.98) |
| Cytotoxic T memory cells | -1.9 (-5.42 to 1.61) | 2.61 (-2.62 to 7.84) |
Change in physical and mental health scores as assessed using the Multiple Sclerosis Quality of Life-54 (MSQOL-54) instrument over the course of 16 weeks (duration of the study). 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. Scores can range from 0 to 100.
| score on a scale | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| MSQOL - Physical Component | -0.15 (-4.37 to 4.07) | 0.01 (-4.7 to 4.71) |
| MSQOL - Mental Component | -0.44 (-4.48 to 3.61) | 4.57 (-0.09 to 9.23) |
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms.
| total treatment-related adverse events | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| Number of Total Treatment-related Adverse Events | 11 | 8 |
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms. AE incidence will be measured as total number of events per 1000 exposure years.
| total events per 1000 exposure years | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| Incidence of Treatment-related Adverse Events (AE) | 1218 | 1061 |
Collected over 16 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| TUDCA Treatment | 0/26 (0%) | 1/26 (3.8%) | 9/26 (34.6%) |
| Placebo Oral Capsule | 0/21 (0%) | 0/21 (0%) | 7/21 (33.3%) |
| Event | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| CholecystitisHepatobiliary disorders | 1/26 | 0/21 |
| Event | TUDCA Treatment | Placebo Oral Capsule |
|---|---|---|
| DiarrheaGastrointestinal disorders | 3/26 | 0/21 |
| Urinary Tract InfectionRenal and urinary disorders | 0/26 | 2/21 |
| Abdominal crampsGastrointestinal disorders | 2/26 | 1/21 |
| Nausea/VomitingGastrointestinal disorders | 2/26 | 0/21 |
| Gastroesophageal RefluxGastrointestinal disorders | 2/26 | 1/21 |
| ConstipationGastrointestinal disorders | 0/26 | 1/21 |
| Increased flatulenceGastrointestinal disorders | 1/26 | 1/21 |
| BronchitisRespiratory, thoracic and mediastinal disorders | 0/26 | 1/21 |
| Deep Vein ThrombosisVascular disorders | 0/26 | 1/21 |
| Facial rash (left)Skin and subcutaneous tissue disorders | 1/26 | 0/21 |
| Age, Continuous(years) | TUDCA Treatment | Placebo Oral Capsule | Total |
|---|---|---|---|
| Mean | 56 ± 11 | 58 ± 10 | 57 ± 10 |
| Sex: Female, Male(Participants) | TUDCA Treatment | Placebo Oral Capsule | Total |
|---|---|---|---|
| Female | 14 | 16 | 30 |
| Male | 14 | 10 | 24 |
| Race (NIH/OMB)(Participants) | TUDCA Treatment | Placebo Oral Capsule | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 28 | 26 | 54 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Jun 2018 to primary completion in Apr 2022, 46 months.
Results were first posted in May 2023, 1 year after primary completion.
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Studies of the drug tested here: Ursodoxicoltaurine
Multiple Sclerosis, Chronic Progressive →