Trial Outcomes & Findings for Ursodeoxycholic Acid (UDCA) for Hepatic Sarcoidosis (NCT NCT03602976)

NCT ID: NCT03602976

Last Updated: 2023-06-07

Results Overview

Alkaline Phosphatase (U/L) or Gamma Glutamyl Transferase (U/L) 6 months after initiation of ursodeoxycholic acid

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 7 participants
• Primary outcome timeframe: 6 months after initiaiton of UDCA
• Results posted on: 2023-06-07

Participant Flow

Participant milestones

Measure	UDCA at Month 6 Ursodeoxycholic Acid: weight-based dosing
Overall Study STARTED	7
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	UDCA at Month 6 Ursodeoxycholic Acid: weight-based dosing
Overall Study Withdrawal by Subject	1

Baseline Characteristics

One baseline subject did not undergo fibroscan at baseline assessment due to COVID restrictions

Baseline characteristics by cohort

Measure	Subjects That Completed 6-month Observation Period n=6 Participants Single-Arm UDCA
Age, Categorical <=18 years	0 Participants n=6 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=6 Participants
Age, Categorical >=65 years	2 Participants n=6 Participants
Age, Continuous	58 years n=6 Participants
Sex: Female, Male Female	4 Participants n=6 Participants
Sex: Female, Male Male	2 Participants n=6 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=6 Participants
Race (NIH/OMB) Asian	0 Participants n=6 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=6 Participants
Race (NIH/OMB) Black or African American	4 Participants n=6 Participants
Race (NIH/OMB) White	2 Participants n=6 Participants
Race (NIH/OMB) More than one race	0 Participants n=6 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=6 Participants
Region of Enrollment United States	6 participants n=6 Participants
Alkaline Phosphatase (U/L)	257.7 U/L n=6 Participants
Gamma Glutamyl Transferase (U/L)	302.5 U/L n=6 Participants
Liver Stiffness by Transient Elastography	8.3 kPa n=5 Participants • One baseline subject did not undergo fibroscan at baseline assessment due to COVID restrictions
Bilirubin	0.72 mg/dL n=6 Participants
Alamine Aminotransferase (ALT)	50.8 U/L n=6 Participants
Aspartate Aminotransferase (AST)	43.3 U/L n=6 Participants

PRIMARY outcome

Timeframe: 6 months after initiaiton of UDCA

Alkaline Phosphatase (U/L) or Gamma Glutamyl Transferase (U/L) 6 months after initiation of ursodeoxycholic acid

Outcome measures

Measure	UDCA at Month 6 n=6 Participants Ursodeoxycholic Acid: weight-based dosing	UDCA at Month 6 Ursodeoxycholic Acid: weight-based dosing
Alkaline Phosphatase (U/L) or Gamma Glutamyl Transferase (U/L) 6 Months After Initiation of Ursodeoxycholic Acid Alkaline Phosphatase	202.2 U/L Interval 128.0 to 321.0	—
Alkaline Phosphatase (U/L) or Gamma Glutamyl Transferase (U/L) 6 Months After Initiation of Ursodeoxycholic Acid Gamma Glutamyl Transferase	111.8 U/L Interval 25.0 to 304.0	—

SECONDARY outcome

Timeframe: Baseline and 6 months after initiation of UDCA

kPa as assessed by Fibroscan

Outcome measures

Measure	UDCA at Month 6 n=5 Participants Ursodeoxycholic Acid: weight-based dosing	UDCA at Month 6 n=5 Participants Ursodeoxycholic Acid: weight-based dosing
Liver Stiffness	8.3 kPa Interval 4.6 to 14.3	6.3 kPa Interval 3.7 to 13.2

SECONDARY outcome

Timeframe: Baseline and 6 months after initiation of UDCA

Outcome measures

Measure	UDCA at Month 6 n=6 Participants Ursodeoxycholic Acid: weight-based dosing	UDCA at Month 6 n=6 Participants Ursodeoxycholic Acid: weight-based dosing
Bilirubin	0.72 mg/dL Interval 0.4 to 1.2	0.77 mg/dL Interval 0.3 to 1.9

SECONDARY outcome

Timeframe: Baseline and 6 months after initiation of UDCA

Outcome measures

Measure	UDCA at Month 6 n=6 Participants Ursodeoxycholic Acid: weight-based dosing	UDCA at Month 6 n=6 Participants Ursodeoxycholic Acid: weight-based dosing
AST	43.3 U/L Interval 21.0 to 66.0	31.2 U/L Interval 18.0 to 47.0

SECONDARY outcome

Timeframe: Baseline and 6 months after initiation of UDCA

Outcome measures

Measure	UDCA at Month 6 n=6 Participants Ursodeoxycholic Acid: weight-based dosing	UDCA at Month 6 n=6 Participants Ursodeoxycholic Acid: weight-based dosing
ALT	50.8 U/L Interval 24.0 to 103.0	29.8 U/L Interval 15.0 to 62.0

Adverse Events

UDCA at Month 6

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	UDCA at Month 6 n=7 participants at risk Ursodeoxycholic Acid: weight-based dosing
Gastrointestinal disorders Nausea	14.3% 1/7 • Entirety of the study - up to 2 years
Gastrointestinal disorders Abdominal discomfort	14.3% 1/7 • Entirety of the study - up to 2 years

Additional Information

Dr. Ethan Weinberg

University of Pennsylvania

Phone: 215-349-8222

Email: ethan.weinberg@pennmedicine.upenn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place