Trial Outcomes & Findings for Safety and Tolerability of Cilofexor in Participants With Primary Sclerosing Cholangitis (PSC) and Compensated Cirrhosis (NCT NCT04060147)

Last Updated: 2023-07-27

Results Overview

Treatment-emergent adverse events (TEAEs) were either defined any AEs with an onset date on or after the study drug start date and no later than 30 days after permanent discontinuation of study drug or any AEs leading to premature discontinuation of study drug.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

11 participants

Primary outcome timeframe

First dose date up to 12 weeks plus 30 days

Results posted on

2023-07-27

Participant Flow

Participants were enrolled at study sites in the United States.

18 participants were screened.

Participant milestones

Participant milestones
Measure	Cilofexor Cilofexor 30 mg tablet orally once daily from Week 1 to Week 4, followed by cilofexor 60 mg (2 X 30 mg) tablets orally once daily from Week 5 to Week 8, followed by cilofexor 100 mg tablet orally once daily from Week 9 to Week 12. Participants received cilofexor for a total duration of 12 weeks.
Overall Study STARTED	11
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Cilofexor Cilofexor 30 mg tablet orally once daily from Week 1 to Week 4, followed by cilofexor 60 mg (2 X 30 mg) tablets orally once daily from Week 5 to Week 8, followed by cilofexor 100 mg tablet orally once daily from Week 9 to Week 12. Participants received cilofexor for a total duration of 12 weeks.
Overall Study Withdrew consent	1

Baseline Characteristics

Safety and Tolerability of Cilofexor in Participants With Primary Sclerosing Cholangitis (PSC) and Compensated Cirrhosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cilofexor n=11 Participants Cilofexor 30 mg tablet orally once daily from Week 1 to Week 4, followed by cilofexor 60 mg (2 X 30 mg) tablets orally once daily from Week 5 to Week 8, followed by cilofexor 100 mg tablet orally once daily from Week 9 to Week 12. Participants received cilofexor for a total duration of 12 weeks.
Age, Continuous	49 years STANDARD_DEVIATION 14.1 • n=99 Participants
Sex: Female, Male Female	5 Participants n=99 Participants
Sex: Female, Male Male	6 Participants n=99 Participants
Race/Ethnicity, Customized Race · Black or African American	1 Participants n=99 Participants
Race/Ethnicity, Customized Race · White	10 Participants n=99 Participants
Race/Ethnicity, Customized Ethnicity · Not Hispanic or Latino	10 Participants n=99 Participants
Race/Ethnicity, Customized Ethnicity · Hispanic or Latino	1 Participants n=99 Participants
Region of Enrollment United States	11 Participants n=99 Participants

PRIMARY outcome

Timeframe: First dose date up to 12 weeks plus 30 days

Population: Safety Analysis Set included all participants who took at least 1 dose of study drug.

Outcome measures

Outcome measures
Measure	Cilofexor n=11 Participants Cilofexor 30 mg tablet orally once daily from Week 1 to Week 4, followed by cilofexor 60 mg (2 X 30 mg) tablets orally once daily from Week 5 to Week 8, followed by cilofexor 100 mg tablet orally once daily from Week 9 to Week 12. Participants received cilofexor for a total duration of 12 weeks.
Percentage of Participants Who Experienced Treatment-emergent Adverse Events (TEAEs)	81.8 percentage of participants

PRIMARY outcome

Timeframe: First dose date up to 12 weeks plus 30 days

Population: Participants in the Safety Analysis Set were analyzed.

A treatment emergent SAE was defined as an event that, at any dose, resulted in the following: death ; life-threatening situation; in-patient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; a congenital anomaly/birth defect; a medically important event or reaction: such events may not be immediately life-threatening or result in death or hospitalization but may jeopardize the subject or may require intervention to prevent one of the other outcomes constituting SAEs.

Outcome measures

Outcome measures
Measure	Cilofexor n=11 Participants Cilofexor 30 mg tablet orally once daily from Week 1 to Week 4, followed by cilofexor 60 mg (2 X 30 mg) tablets orally once daily from Week 5 to Week 8, followed by cilofexor 100 mg tablet orally once daily from Week 9 to Week 12. Participants received cilofexor for a total duration of 12 weeks.
Percentage of Participants Who Experienced Treatment Emergent Serious Adverse Events (SAEs)	0 percentage of participants

PRIMARY outcome

Timeframe: First dose date up to 12 weeks plus 30 days

Population: Participants in the Safety Analysis Set were analyzed.

Treatment-emergent laboratory abnormalities are defined as values that increase at least 1 toxicity grade from baseline at any postbaseline time point, up to and including the date of last dose of study drug plus 30 days. Grade 1: mild; Grade 2: moderate; Grade 3: severe; Grade 4: life-threatening.

Outcome measures

Outcome measures
Measure	Cilofexor n=11 Participants Cilofexor 30 mg tablet orally once daily from Week 1 to Week 4, followed by cilofexor 60 mg (2 X 30 mg) tablets orally once daily from Week 5 to Week 8, followed by cilofexor 100 mg tablet orally once daily from Week 9 to Week 12. Participants received cilofexor for a total duration of 12 weeks.
Percentage of Participants Who Experienced Laboratory Abnormalities Grade 1	54.5 percentage of participants
Percentage of Participants Who Experienced Laboratory Abnormalities Grade 2	36.4 percentage of participants
Percentage of Participants Who Experienced Laboratory Abnormalities Grade 3	9.1 percentage of participants
Percentage of Participants Who Experienced Laboratory Abnormalities Grade 4	0 percentage of participants

Adverse Events

Cilofexor

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Cilofexor n=11 participants at risk Cilofexor 30 mg tablet orally once daily from Week 1 to Week 4, followed by cilofexor 60 mg (2 X 30 mg) tablets orally once daily from Week 5 to Week 8, followed by cilofexor 100 mg tablet orally once daily from Week 9 to Week 12. Participants received cilofexor for a total duration of 12 weeks.
Ear and labyrinth disorders Vertigo	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Gastrointestinal disorders Constipation	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Gastrointestinal disorders Defaecation urgency	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Gastrointestinal disorders Diarrhoea	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Gastrointestinal disorders Nausea	18.2% 2/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
General disorders Fatigue	18.2% 2/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
General disorders Ill-defined disorder	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
General disorders Oedema peripheral	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Infections and infestations Upper respiratory tract infection	18.2% 2/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Infections and infestations Urinary tract infection	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Injury, poisoning and procedural complications Jaw fracture	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Injury, poisoning and procedural complications Lip injury	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Injury, poisoning and procedural complications Skin laceration	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Muscle spasms	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Nervous system disorders Headache	18.2% 2/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Reproductive system and breast disorders Menopausal symptoms	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Reproductive system and breast disorders Oligomenorrhoea	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Cough	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Epistaxis	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Pruritus	72.7% 8/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Rash papular	9.1% 1/11 • All-Cause Mortality: Enrollment up to 17 weeks; Adverse Events: First dose date up to 12 weeks plus 30 days All-cause mortality: All enrolled analysis set included all participants who received a study participant identification number in the study after screening. Adverse events: Safety analysis set included all participants who took at least 1 dose of study drug.

Additional Information

Gilead Clinical Study Information Center

Gilead Sciences

Phone: 1-833-445-3230 (GILEAD-0)

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met: * The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or * The study has been completed at all study sites for at least 2 years
Publication restrictions are in place

Restriction type: OTHER