Trial Outcomes & Findings for Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (NCT NCT04674761)

NCT ID: NCT04674761

Last Updated: 2023-11-02

Results Overview

Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 52 participants
• Primary outcome timeframe: Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.
• Results posted on: 2023-11-02

Participant Flow

Participant milestones

Measure	Odevixibat (A4250) Capsules for oral administration once daily for 24 weeks (120 μg/kg/day). Odevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.	Placebo Capsules for oral administration (to match active) once daily for 24 weeks. Placebo: Placebo identical in appearance to experimental drug (odevixibat).
Overall Study STARTED	35	17
Overall Study COMPLETED	35	17
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Baseline characteristics by cohort

Measure	Odevixibat (A4250) n=35 Participants Capsules for oral administration once daily for 24 weeks (120 μg/kg/day). Odevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.	Placebo n=17 Participants Capsules for oral administration (to match active) once daily for 24 weeks. Placebo: Placebo identical in appearance to experimental drug (odevixibat).	Total n=52 Participants Total of all reporting groups
Age, Categorical <=18 years	35 Participants n=5 Participants	17 Participants n=7 Participants	52 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	6.73 years STANDARD_DEVIATION 3.780 • n=5 Participants	5.40 years STANDARD_DEVIATION 4.411 • n=7 Participants	6.29 years STANDARD_DEVIATION 4.003 • n=5 Participants
Age, Customized < 10 years	29 Participants n=5 Participants	13 Participants n=7 Participants	42 Participants n=5 Participants
Age, Customized > = 10 years and < 18 years	6 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	11 Participants n=7 Participants	25 Participants n=5 Participants
Sex: Female, Male Male	21 Participants n=5 Participants	6 Participants n=7 Participants	27 Participants n=5 Participants
Race/Ethnicity, Customized White	30 Participants n=5 Participants	13 Participants n=7 Participants	43 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized Asian	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants	4 participants n=7 Participants	11 participants n=5 Participants
Region of Enrollment United Kingdom	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Malaysia	2 participants n=5 Participants	1 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Netherlands	3 participants n=5 Participants	3 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment Turkey	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Belgium	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Poland	7 participants n=5 Participants	3 participants n=7 Participants	10 participants n=5 Participants
Region of Enrollment Italy	4 participants n=5 Participants	2 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment France	3 participants n=5 Participants	1 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment Germany	6 participants n=5 Participants	2 participants n=7 Participants	8 participants n=5 Participants
Genetic Mutation JAG1	32 Participants n=5 Participants	16 Participants n=7 Participants	48 Participants n=5 Participants
Genetic Mutation NOTCH2	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.

Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.

Outcome measures

Measure	Odevixibat (A4250) n=35 Participants Capsules for oral administration once daily for 24 weeks. Odevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.	Placebo n=17 Participants Capsules for oral administration (to match active) once daily for 24 weeks. Placebo: Placebo identical in appearance to experimental drug (odevixibat).
Change From Baseline in Scratching Score	-1.69 score on a scale Standard Error 0.174	-0.8 score on a scale Standard Error 0.233

SECONDARY outcome

Timeframe: Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.

Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24

Outcome measures

Measure	Odevixibat (A4250) n=35 Participants Capsules for oral administration once daily for 24 weeks. Odevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.	Placebo n=17 Participants Capsules for oral administration (to match active) once daily for 24 weeks. Placebo: Placebo identical in appearance to experimental drug (odevixibat).
Serum Bile Acid Levels	-90.35 μmol/L Standard Error 21.336	22.39 μmol/L Standard Error 28.463

Adverse Events

Odevixibat (A4250)

Serious events: 5 serious events

Other events: 26 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Measure	Odevixibat (A4250) n=35 participants at risk Capsules for oral administration once daily for 24 weeks. Odevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.	Placebo n=17 participants at risk Capsules for oral administration (to match active) once daily for 24 weeks. Placebo: Placebo identical in appearance to experimental drug (odevixibat).
Investigations International normalised ratio increased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
General disorders Pyrexia	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Ear and labyrinth disorders Cerumen impaction	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Abdominal pain	2.9% 1/35 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Constipation	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Haematemesis	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Otitis media chronic	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Pneumonia	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Rhinovirus infection	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Subcutaneous abscess	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Tonsillitis	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Adenoidal hypertrophy	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.

Other adverse events

Measure	Odevixibat (A4250) n=35 participants at risk Capsules for oral administration once daily for 24 weeks. Odevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.	Placebo n=17 participants at risk Capsules for oral administration (to match active) once daily for 24 weeks. Placebo: Placebo identical in appearance to experimental drug (odevixibat).
Injury, poisoning and procedural complications Contusion	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Injury, poisoning and procedural complications Ligament sprain	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Injury, poisoning and procedural complications Vaccination complication	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Alanine aminotransferase increased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Blood pressure diastolic increased	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Blood triglycerides increased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Gamma-glutamyltransferase increased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Hepatic enzyme increased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Blood and lymphatic system disorders Anaemia macrocytic	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Blood and lymphatic system disorders Coagulopathy	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Blood and lymphatic system disorders Lymphadenopathy	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Eye disorders Cataract cortical	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Abdominal pain	11.4% 4/35 • Number of events 4 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Abdominal pain upper	2.9% 1/35 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Aphthous ulcer	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Diarrhoea	28.6% 10/35 • Number of events 11 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Faeces discoloured	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Faeces soft	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Frequent bowel movements	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Nausea	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Gastrointestinal disorders Vomiting	5.7% 2/35 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
General disorders Asthenia	5.7% 2/35 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
General disorders Fatigue	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
General disorders Pyrexia	22.9% 8/35 • Number of events 8 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	23.5% 4/17 • Number of events 7 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Hepatobiliary disorders Jaundice	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Immune system disorders Hypersensitivity	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Immune system disorders Seasonal allergy	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Bronchitis	8.6% 3/35 • Number of events 4 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations COVID-19	14.3% 5/35 • Number of events 5 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	23.5% 4/17 • Number of events 4 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Conjunctivitis	5.7% 2/35 • Number of events 3 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Gastroenteritis	5.7% 2/35 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Influenza	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Nasopharyngitis	5.7% 2/35 • Number of events 4 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Otitis externa	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Otitis media	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Pharyngitis	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Respiratory tract infection	8.6% 3/35 • Number of events 3 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Subcutaneous abscess	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Tonsillitis	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Upper respiratory tract infection	8.6% 3/35 • Number of events 3 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	11.8% 2/17 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Viral infection	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Viral rash	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Infections and infestations Viral upper respiratory tract infection	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations International normalised ratio increased	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	11.8% 2/17 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Platelet count decreased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Vitamin A decreased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Vitamin E decreased	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Investigations Weight decreased	5.7% 2/35 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Metabolism and nutrition disorders Hypophagia	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Metabolism and nutrition disorders Vitamin D deficiency	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Musculoskeletal and connective tissue disorders Pain in extremity	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Nervous system disorders Bell's palsy	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Nervous system disorders Headache	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Renal and urinary disorders Proteinuria	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Asthma	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Cough	8.6% 3/35 • Number of events 4 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	11.8% 2/17 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Nasal turbinate hypertrophy	0.00% 0/35 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Pharyngeal inflammation	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	2.9% 1/35 • Number of events 2 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	5.9% 1/17 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Skin and subcutaneous tissue disorders Skin lesion	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Skin and subcutaneous tissue disorders Urticaria	2.9% 1/35 • Number of events 1 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.
Vascular disorders Haematoma	8.6% 3/35 • Number of events 3 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.	0.00% 0/17 • The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.

Additional Information

Albireo AB

Albireo AB

Phone: medinfo@albireopharma.com

Email: medinfo@albireopharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee Albireo will retain the ownership of all data. All proposed publications based on this study must be subject to the sponsor's approval requirements.
• Publication restrictions are in place

Restriction type: OTHER