A Research Study to Evaluate Safety and Efficacy of DUR-928 in Subjects With Primary Sclerosing Cholangitis (PSC)

• ClinicalTrials.gov Identifier: NCT03394781
• Recruitment Status: Terminated
• First Posted: January 9, 2018
• Results First Posted: October 17, 2022
• Last Update Posted: October 17, 2022
• Sponsor: Durect
• Information provided by (Responsible Party): Durect

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Primary Sclerosing Cholangitis
• Intervention: Drug: DUR-928
• Enrollment: 5

Participant Flow

Recruitment Details	Study was terminated early due to lack of enrollment.
Pre-assignment Details

Arm/Group Title	DUR-928 10 mg	DUR-928 50 mg
Arm/Group Description	10 mg oral suspension DUR-928: oral suspension daily for 28 days	50 mg oral suspension DUR-928: oral suspension daily for 28 days
Period Title: Overall Study
Started	3	2
Completed	3	2
Not Completed	0	0

Baseline Characteristics

Arm/Group Title		DUR-928 10 mg	DUR-928 50 mg	Total
Arm/Group Description		10 mg oral suspension DUR-928: oral suspension daily for 28 days	50 mg oral suspension DUR-928: oral suspension daily for 28 days	Total of all reporting groups
Overall Number of Baseline Participants		3	2	5
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	3 participants	2 participants	5 participants
	<=18 years	0 0.0%	0 0.0%	0 0.0%
	Between 18 and 65 years	3 100.0%	1 50.0%	4 80.0%
	>=65 years	0 0.0%	1 50.0%	1 20.0%
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	3 participants	2 participants	5 participants
	Female	1 33.3%	1 50.0%	2 40.0%
	Male	2 66.7%	1 50.0%	3 60.0%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	3 participants	2 participants	5 participants
	Hispanic or Latino	0 0.0%	0 0.0%	0 0.0%
	Not Hispanic or Latino	3 100.0%	2 100.0%	5 100.0%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	3 participants	2 participants	5 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%
	Asian	0 0.0%	0 0.0%	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%
	Black or African American	0 0.0%	0 0.0%	0 0.0%
	White	3 100.0%	1 50.0%	4 80.0%
	More than one race	0 0.0%	0 0.0%	0 0.0%
	Unknown or Not Reported	0 0.0%	1 50.0%	1 20.0%
Region of Enrollment; Measure Type: Count of Participants; Unit of measure: Participants
United States	Number Analyzed	3 participants	2 participants	5 participants
		3 100.0%	2 100.0%	5 100.0%

Outcome Measures

1. Primary Outcome

Title	Percent Change of Alkaline Phosphatase (ALP) From Baseline
Description	[Not Specified]
Time Frame	Day 28 (end of treatment) and Day 56 (end of study/early termination)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	DUR-928 10 mg	DUR-928 50 mg
Arm/Group Description:	10 mg oral suspension DUR-928: oral suspension daily for 28 days	50 mg oral suspension DUR-928: oral suspension daily for 28 days
Overall Number of Participants Analyzed	3	2
Mean (Standard Deviation); Unit of Measure: percent change
Percent change of ALP from baseline to Day 28	23.7 (10.7)	16.8 (2.97)
Percent change of ALP from baseline to Day 56	10.7 (17.04)	11.05 (8.68)

2. Secondary Outcome

Title	Percent Change of Liver Enzymes and Serum Bile Acids (sBA)
Description	Liver enzymes include alanine transaminase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), bilirubin. Percent change from baseline through the end of study treatment (Day 28) and throughout the follow-up period (Day 56).
Time Frame	Day 28 and Day 56

Outcome Measure Data

Analysis Population Description

Study terminated early due to lack of enrollment.

Arm/Group Title		DUR-928 10 mg	DUR-928 50 mg
Arm/Group Description:		10 mg oral suspension DUR-928: oral suspension daily for 28 days	50 mg oral suspension DUR-928: oral suspension daily for 28 days
Overall Number of Participants Analyzed		3	2
Mean (Standard Deviation); Unit of Measure: percent change
percent ALT change from baseline to Day 28	Number Analyzed	3 participants	2 participants
		32.87 (48.87)	3.0 (29.56)
percent AST change from baseline to Day 28	Number Analyzed	3 participants	2 participants
		24.10 (35.03)	-20.25 (3.23)
percent GGT change from baseline to Day 28	Number Analyzed	3 participants	2 participants
		7.3 (4.16)	8.15 (1.49)
percent direct bilirubin change from baseline to Day 28	Number Analyzed	2 participants	1 participants
		14.2 (24.61)	34.6 [1] (NA)
percent total bilirubin change from baseline to Day 28	Number Analyzed	3 participants	2 participants
		12.03 (23.09)	23.85 (33.73)
percent serum bile acids change from baseline to Day 28	Number Analyzed	3 participants	2 participants
		-13.93 (15.00)	-44.10 (46.95)
percent ALT change from baseline to Day 56	Number Analyzed	3 participants	2 participants
		-9.77 (15.30)	-12.7 (5.66)
percent AST change from baseline to Day 56	Number Analyzed	3 participants	2 participants
		-16.23 (9.17)	-9.70 (19.09)
percent GGT change from baseline to Day 56	Number Analyzed	3 participants	2 participants
		-7.10 (11.87)	-3.2 (2.26)
percent direct bilirubin change from baseline to Day 56	Number Analyzed	1 participants	1 participants
		-12.90 [1] (NA)	19.20 [1] (NA)
percent total bilirubin change from baseline to Day 56	Number Analyzed	3 participants	2 participants
		-24.60 (20.46)	19.14 (15.61)
percent serum bile acids change from baseline to Day 56	Number Analyzed	3 participants	2 participants
		35.23 (79.79)	-35.75 (72.90)

[1]

Standard Deviation not calculable because data were only collected for 1 participant.

3. Secondary Outcome

Title	Percent of Subjects With Reduction of Serum Alkaline Phosphatase (ALP) From Baseline
Description	[Not Specified]
Time Frame	Day 28 (end of treatment) and Day 56 (end of follow-up)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	DUR-928 10 mg	DUR-928 50 mg
Arm/Group Description:	10 mg oral suspension DUR-928: oral suspension daily for 28 days	50 mg oral suspension DUR-928: oral suspension daily for 28 days
Overall Number of Participants Analyzed	3	2
Measure Type: Count of Participants; Unit of Measure: Participants
subjects with ≥ 40% reduction of serum ALP from baseline at day 28/end of treatment	0 0.0%	0 0.0%
subjects with ≥ 40% reduction of serum ALP from baseline at day 56/end of follow-up	0 0.0%	0 0.0%

4. Secondary Outcome

Title	Percent Change of Selected Biomarkers From Baseline Through the End of Study Treatment and Throughout the Follow-up Period.
Description	[Not Specified]
Time Frame	Day 28 and Day 56

Outcome Measure Data

Analysis Population Description

The samples were not assayed to generate data for this Outcome Measure due to early study termination.

Arm/Group Title	DUR-928 10 mg	DUR-928 50 mg
Arm/Group Description:	10 mg oral suspension DUR-928: oral suspension daily for 28 days	50 mg oral suspension DUR-928: oral suspension daily for 28 days
Overall Number of Participants Analyzed	0	0

No data displayed because Outcome Measure has zero total analyzed.

Adverse Events

Time Frame	56 days
Adverse Event Reporting Description	Treatment Emergent Adverse Events
Arm/Group Title	DUR-928 10 mg		DUR-928 50 mg
Arm/Group Description	10 mg oral suspension DUR-928: oral suspension daily for 28 days		50 mg oral suspension DUR-928: oral suspension daily for 28 days
All-Cause Mortality
	DUR-928 10 mg		DUR-928 50 mg
	Affected / at Risk (%)		Affected / at Risk (%)
Total	0/3 (0.00%)		0/2 (0.00%)
Serious Adverse Events
	DUR-928 10 mg		DUR-928 50 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/3 (0.00%)		0/2 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	DUR-928 10 mg		DUR-928 50 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/3 (66.67%)		1/2 (50.00%)
Cardiac disorders
WORSENING SINUS BRADYCARDIA *	1/3 (33.33%)	1	0/2 (0.00%)	0
Gastrointestinal disorders
EXCESSIVE GAS (FLATUS) *	1/3 (33.33%)	1	0/2 (0.00%)	0
Hepatobiliary disorders
DRUG INDUCED LIVER INJURY *	1/3 (33.33%)	1	0/2 (0.00%)	0
Investigations
ELEVATED ALKALINE PHOSPHATASE *	0/3 (0.00%)	0	1/2 (50.00%)	1
ELEVATED ALT *	0/3 (0.00%)	0	1/2 (50.00%)	1
ELEVATED AST *	0/3 (0.00%)	0	1/2 (50.00%)	1
ELEVATED GGT *	0/3 (0.00%)	0	1/2 (50.00%)	1
ELEVATED SERUM BILE ACIDS *	0/3 (0.00%)	0	1/2 (50.00%)	1
Skin and subcutaneous tissue disorders
LEFT EAR ERYTHEMA PRESENT *	0/3 (0.00%)	0	1/2 (50.00%)	1
* Indicates events were collected by non-systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

If a joint publication is not submitted within 18-24 months after study completion, PI shall have the right to submit to sponsor a proposed results communication based on results at their institution. Sponsor can review proposed results communications prior to public release, can request removal of confidential information, and can embargo communications regarding trial results for up to 105-120 days after submission to sponsor.

Results Point of Contact

• Name/Title: Executive Director, Regulatory Affairs
• Organization: DURECT Corporation
• Phone: 408-777-1829
• EMail: jill.burns@durect.com

• Responsible Party: Durect
• ClinicalTrials.gov Identifier: NCT03394781
• Other Study ID Numbers: C928-008
• First Submitted: January 3, 2018
• First Posted: January 9, 2018
• Results First Submitted: August 10, 2022
• Results First Posted: October 17, 2022
• Last Update Posted: October 17, 2022