Trial Outcomes & Findings for A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA) (NCT NCT04718961)

NCT ID: NCT04718961

Last Updated: 2024-08-06

Results Overview

To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints: Proportion of participants experiencing one or more of the following: Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs. Clinically significant laboratory abnormalities

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: Through to end of treatment, up to 21 weeks
• Results posted on: 2024-08-06

Participant Flow

A total of 26 participants were screened at 25 sites in 3 countries (New Zealand, United Kingdom, and United States). Of them, 4 were enrolled in the study.

After a screening period of up to 10 days during which all procedures listed for the screening visit are completed, eligible patients diagnosed with ICP with screening sBA ≥10 μmol/L during screening or at any time during the current pregnancy were randomized with stratification in a 2-arm (1:1), open-label fashion to receive volixibat 20 mg BID or volixibat 80 mg BID. A total of 26 ICP patients were screened for the study. 22 of these patients were screen failures.

Participant milestones

Measure	Part 1 Arm 1 - Volixibat 20mg (Experimental) Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 1 Arm 2 - Volixibat 80mg (Experimental) Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 2 - Placebo (Placebo Comparator) Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.
Overall Study STARTED	2	2	0	0
Overall Study COMPLETED	2	2	0	0
Overall Study NOT COMPLETED	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)

Baseline characteristics by cohort

Measure	Part 1 Arm 1 - Volixibat 20mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 1 Arm 2 - Volixibat 80mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 2 - Placebo (Placebo Comparator) Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.	Total n=4 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	2 Participants n=7 Participants	—	—	4 Participants n=21 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	—	—	4 Participants n=21 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	—	—	1 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	2 Participants n=7 Participants	—	—	3 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants	2 Participants n=7 Participants	—	—	3 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	—	—	1 Participants n=21 Participants
Region of Enrollment United States	1 participants n=5 Participants	0 participants n=7 Participants	—	—	1 participants n=21 Participants
Region of Enrollment United Kingdom	1 participants n=5 Participants	2 participants n=7 Participants	—	—	3 participants n=21 Participants

PRIMARY outcome

Timeframe: Through to end of treatment, up to 21 weeks

Population: Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.

To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints:

Proportion of participants experiencing one or more of the following:

Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs.

Clinically significant laboratory abnormalities

Outcome measures

Measure	Part 1 Arm 1 - Volixibat 20mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 1 Arm 2 - Volixibat 80mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 2 - Placebo (Placebo Comparator) Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.
Assess the Safety and Tolerability of Volixibat in Participants With ICP	2 participants	2 participants	—	—

SECONDARY outcome

Timeframe: Through to end of treatment, up to 21 weeks

Population: Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.

Adult Itch Reported Outcome (ItchRO) is a 0 to 10 scale with 0 being "no itch" and 10 being "worst possible itch" where participants are responding to the following question "How would you rate the worst itch you experienced over the last 24hrs?"

Outcome measures

Measure	Part 1 Arm 1 - Volixibat 20mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 1 Arm 2 - Volixibat 80mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 2 - Placebo (Placebo Comparator) Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.
Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)	-2.5 units on a scale Standard Deviation 2.40	1.3 units on a scale Standard Deviation 2.31	—	—

SECONDARY outcome

Timeframe: At least one month after delivery.

Population: Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.

Outcome measures

Measure	Part 1 Arm 1 - Volixibat 20mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 1 Arm 2 - Volixibat 80mg (Experimental) n=2 Participants Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 2 - Placebo (Placebo Comparator) Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.
Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes	0 Participants	2 Participants	—	—

Adverse Events

Part 1 Arm 1 - Volixibat 20mg (Experimental)

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Part 1 Arm 2 - Volixibat 80mg (Experimental)

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Part 2 Arm 2 - Placebo (Placebo Comparator)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Part 1 Arm 1 - Volixibat 20mg (Experimental) n=2 participants at risk Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 1 Arm 2 - Volixibat 80mg (Experimental) n=2 participants at risk Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 2 - Placebo (Placebo Comparator) Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.
Gastrointestinal disorders Diarrhoea	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.

Other adverse events

Measure	Part 1 Arm 1 - Volixibat 20mg (Experimental) n=2 participants at risk Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 1 Arm 2 - Volixibat 80mg (Experimental) n=2 participants at risk Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.	Part 2 Arm 2 - Placebo (Placebo Comparator) Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.
Blood and lymphatic system disorders Anemia	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Psychiatric disorders Anxiety	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Respiratory, thoracic and mediastinal disorders Apnoic Episodes	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Gastrointestinal disorders Diarrhoea	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	100.0% 2/2 • Number of events 3 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Infections and infestations Gastroenteritis	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Nervous system disorders Headache	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Gastrointestinal disorders Heart Burn	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Gastrointestinal disorders Intermittent Abdominal Pain	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Gastrointestinal disorders Intermittent Diarrhoea	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Gastrointestinal disorders Intermittent Vomiting	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Gastrointestinal disorders Mild Diarrhoea	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Psychiatric disorders Postnatal Depression	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Pregnancy, puerperium and perinatal conditions Premature Labor	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Investigations Raised Blood Pressure	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Pregnancy, puerperium and perinatal conditions Threated Pre-Term Labor	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
Gastrointestinal disorders Vomiting	50.0% 1/2 • Number of events 1 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	0.00% 0/2 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	— 0/0 • All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks** Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.

Additional Information

Mirum Clinical Trials

Mirum Pharmaceuticals

Phone: 650-667-4085

Email: medinfo@mirumpharma.com

Results disclosure agreements

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

Restriction type: LTE60