Trial Outcomes & Findings for An Efficacy Trial of Low Dose All-trans Retinoic Acid in Patients With Primary Sclerosing Cholangitis (NCT NCT03359174)
NCT ID: NCT03359174
Last Updated: 2021-08-09
Results Overview
Blood will be drawn at each time point to compare pre- and post-treatment values for each individual.
TERMINATED
PHASE2
2 participants
Baseline to week 24.
2021-08-09
Participant Flow
Patients were recruited from physicians case load at Yale University, Digestive Disease's Liver clinic between May 2018 and December 2018. Both participants were enrolled in May of 2018.
Of the two patients who screened, both met inclusion and exclusion criterial and were enrolled
Participant milestones
| Measure |
All-trans Retinoic Acid (ATRA) Therapy
Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
All-trans retinoic acid: Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
All-trans Retinoic Acid (ATRA) Therapy
Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
All-trans retinoic acid: Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
An Efficacy Trial of Low Dose All-trans Retinoic Acid in Patients With Primary Sclerosing Cholangitis
Baseline characteristics by cohort
| Measure |
All-trans Retinoic Acid (ATRA) Therapy
n=2 Participants
Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
All-trans retinoic acid: Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to week 24.Population: Data were not collected
Blood will be drawn at each time point to compare pre- and post-treatment values for each individual.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to week 24.Population: Only 1 patient completed the study, and no trial end point was achieved so there was no data to record. .
Blood is drawn at each time point to assess the outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to week 24.Population: Only 1 patient completed the study, data not reported.
Blood is drawn at each time point to assess the outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to week 24.Population: Only 1 patient completed the study, data not reported.
Blood is drawn at each time point to assess the outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to week 24.Population: Only 1 patient completed the study, data not reported.
Blood is drawn at each time point to assess the outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to week 24.Population: Only 1 patient completed the study, data not reported.
Blood is drawn at each time point to assess the outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to week 24.Population: Only 1 patient completed the study, data not reported.
Transient Elastography will be performed at baseline and week 24 to assess the outcome.
Outcome measures
Outcome data not reported
Adverse Events
All-trans Retinoic Acid (ATRA) Therapy
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
All-trans Retinoic Acid (ATRA) Therapy
n=2 participants at risk
Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
All-trans retinoic acid: Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
|
|---|---|
|
Hepatobiliary disorders
alkaline phosphatase increase
|
50.0%
1/2 • Number of events 1 • Up to 28 weeks post treatment initiation.
|
|
Hepatobiliary disorders
Blood bilirubin increased
|
50.0%
1/2 • Number of events 1 • Up to 28 weeks post treatment initiation.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place