Trial Outcomes & Findings for Vidofludimus Calcium for Primary Sclerosing Cholangitis (NCT NCT03722576)
NCT ID: NCT03722576
Last Updated: 2022-10-17
Results Overview
The number of subjects who have both an ALP reduction from baseline to week 24 that is greater or equal to 25% and their AST increase from baseline is less than or equal to 33% at week 24. ALP measured as international units per liter (IU/L). AST measured as international units per liter (IU/L).
COMPLETED
PHASE2
18 participants
Baseline to 24 weeks
2022-10-17
Participant Flow
Participant milestones
| Measure |
Vidofludimus Calcium (VC)
Daily dosing of Vidofludimus Calcium (VC) over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Overall Study
STARTED
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18
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Overall Study
COMPLETED
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11
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Overall Study
NOT COMPLETED
|
7
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Reasons for withdrawal
| Measure |
Vidofludimus Calcium (VC)
Daily dosing of Vidofludimus Calcium (VC) over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Overall Study
Withdrawal by Subject
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3
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Overall Study
Adverse Event
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2
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Overall Study
Physician Decision
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1
|
|
Overall Study
Lost to Follow-up
|
1
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Baseline Characteristics
Vidofludimus Calcium for Primary Sclerosing Cholangitis
Baseline characteristics by cohort
| Measure |
Vidofludimus Calcium (VC)
n=18 Participants
Daily dosing of VC over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Age, Continuous
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45.7 years
STANDARD_DEVIATION 15.2 • n=5 Participants
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Sex: Female, Male
Female
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11 Participants
n=5 Participants
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Sex: Female, Male
Male
|
7 Participants
n=5 Participants
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|
Race/Ethnicity, Customized
White
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18 Participants
n=5 Participants
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Race/Ethnicity, Customized
Not Hispanic or Latino
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17 Participants
n=5 Participants
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Race/Ethnicity, Customized
Hispanic or Latino
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1 Participants
n=5 Participants
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Region of Enrollment
United States
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18 participants
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline to 24 weeksPopulation: Only subjects who completed treatment were analyzed
The number of subjects who have both an ALP reduction from baseline to week 24 that is greater or equal to 25% and their AST increase from baseline is less than or equal to 33% at week 24. ALP measured as international units per liter (IU/L). AST measured as international units per liter (IU/L).
Outcome measures
| Measure |
Vidofludimus Calcium (VC)
n=11 Participants
Daily dosing of VC over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels.
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3 Participants
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SECONDARY outcome
Timeframe: Baseline to 24 weeksPopulation: Only subjects who completed the lab tests at 24 weeks were analyzed
Number of subjects with abnormal (not within normal range) AST levels. AST is an enzyme found in high amounts in liver, heart, and muscle cells. This test is mainly done along with other tests such as alkaline phosphatase and bilirubin to diagnose and monitor liver disease. This test evaluates hepatocyte integrity, as serum levels of this enzyme rise in response to a variety of forms of injury to hepatic cells. The normal range is 5 to 40 U/L . Units: U/L
Outcome measures
| Measure |
Vidofludimus Calcium (VC)
n=10 Participants
Daily dosing of VC over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Abnormal Aspartate Aminotransferase (AST)
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8 Participants
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SECONDARY outcome
Timeframe: 24 weeksPopulation: Only subjects who completed the lab tests at 24 weeks were analyzed
Number of subjects with abnormal (not within normal range) ALT levels. An enzyme normally present in liver and heart cells that is released into the bloodstream when the liver or heart is damaged. The blood ALT levels are elevated with liver damage (for example, from viral hepatitis) or with an insult to the heart (for example, from a heart attack). The normal range is 7 to 56 U/L. Units: U/L
Outcome measures
| Measure |
Vidofludimus Calcium (VC)
n=10 Participants
Daily dosing of VC over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Abnormal Alanine Aminotransferase (ALT)
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8 Participants
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SECONDARY outcome
Timeframe: 24 weeksNumber of subjects with abnormal (not within normal range) Total Bilirubin levels. Bilirubin is a yellowish pigment found in bile, a fluid made by the liver. A small amount of older red blood cells are replaced by new blood cells every day. Bilirubin is left after these older blood cells are removed. The liver helps break down bilirubin so that it can be removed from the body in the stool. The normal range for total bilirubin is 0.3 to 1.2 mg/dL Units: mg/dL
Outcome measures
| Measure |
Vidofludimus Calcium (VC)
n=10 Participants
Daily dosing of VC over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Abnormal Total Bilirubin
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3 Participants
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SECONDARY outcome
Timeframe: 24 weeksPopulation: Only subjects who completed the lab tests at 24 weeks were analyzed
Number of subjects with abnormal (not within normal range) direct bilirubin levels. In the liver, bilirubin is changed into a form that your body can get rid of. This is called conjugated bilirubin or direct bilirubin. This bilirubin travels from the liver into the small intestine. A very small amount passes into your kidneys and is excreted in your urine. Normal range for direct bilirubin is 0.3 and 1.2 milligrams per deciliter (mg/dL). Units: mg/dL
Outcome measures
| Measure |
Vidofludimus Calcium (VC)
n=10 Participants
Daily dosing of VC over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Abnormal Direct Bilirubin
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4 Participants
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Adverse Events
Vidofludimus Calcium (VC)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Vidofludimus Calcium (VC)
n=18 participants at risk
Daily dosing of VC over 6 months
Vidofludimus calcium: During the 6-month treatment period, subjects received 30 mg VC orally once daily. This was preceded by a lead-in dosing period where subjects received 15 mg VC once daily for 1 week.
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|---|---|
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Gastrointestinal disorders
Abdominal Pain
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Investigations
Alkaline phosphatase increase
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Immune system disorders
Allergic reaction
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11.1%
2/18 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Gastrointestinal disorders
Diarrhea
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16.7%
3/18 • Number of events 3 • Adverse Events were collected from baseline to end of study, approximately 8 months
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General disorders
Fatigue
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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General disorders
Fever Pyrexia
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11.1%
2/18 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Nervous system disorders
Headache
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Renal and urinary disorders
Hematuria
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11.1%
2/18 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Respiratory, thoracic and mediastinal disorders
Nasal congestion
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
|
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Gastrointestinal disorders
Nausea
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16.7%
3/18 • Number of events 3 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Skin and subcutaneous tissue disorders
Pruritus
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22.2%
4/18 • Number of events 4 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Gastrointestinal disorders
Stomach pain
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Renal and urinary disorders
Urinary Tract Infection
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
|
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Musculoskeletal and connective tissue disorders
Achy neck and shoulders
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Gastrointestinal disorders
Bleeding from ileostomy
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
|
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Respiratory, thoracic and mediastinal disorders
Cold
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11.1%
2/18 • Number of events 2 • Adverse Events were collected from baseline to end of study, approximately 8 months
|
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Gastrointestinal disorders
Discolored Stool
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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General disorders
Flu
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Gastrointestinal disorders
Gatroenteritis
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Skin and subcutaneous tissue disorders
Lipoma on back
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Pregnancy, puerperium and perinatal conditions
Miscarriage
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Gastrointestinal disorders
Upper right abdominal pain
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Gastrointestinal disorders
Worsened loose stools
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Investigations
Worsening liver enzymes
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Gastrointestinal disorders
Cholangitis
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5.6%
1/18 • Number of events 1 • Adverse Events were collected from baseline to end of study, approximately 8 months
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place