Trial Outcomes & Findings for Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (NCT NCT04524390)
NCT ID: NCT04524390
Last Updated: 2025-03-19
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
75 participants
Primary outcome timeframe
From baseline to Week 26
Results posted on
2025-03-19
Participant Flow
A total of 75 participants were enrolled at 19 sites across 8 countries (China, Germany, Poland, Singapore, Taiwan, United Kingdom, United States, and Vietnam).
The screening period starts when informed consent (by the legally authorized representative) is signed. The duration of the screening period is up to 3 weeks, during which all procedures listed for the screening visit in the schedule of assessment must be completed. A total of 77 patients were screened.
Participant milestones
| Measure |
Double Blind - Maralixibat
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Open Label - Maralixibat
The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat.
|
|---|---|---|---|
|
Double Blind
STARTED
|
40
|
35
|
0
|
|
Double Blind
COMPLETED
|
28
|
24
|
0
|
|
Double Blind
NOT COMPLETED
|
12
|
11
|
0
|
|
Open-Label
STARTED
|
0
|
0
|
52
|
|
Open-Label
COMPLETED
|
0
|
0
|
1
|
|
Open-Label
NOT COMPLETED
|
0
|
0
|
51
|
Reasons for withdrawal
| Measure |
Double Blind - Maralixibat
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Open Label - Maralixibat
The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat.
|
|---|---|---|---|
|
Double Blind
Consent Withdrawal by subject
|
2
|
1
|
0
|
|
Double Blind
Adverse Event
|
5
|
3
|
0
|
|
Double Blind
Liver Transplant
|
5
|
6
|
0
|
|
Double Blind
Disease Progression
|
0
|
1
|
0
|
|
Open-Label
Drug interruption criteria
|
0
|
0
|
1
|
|
Open-Label
Liver Transplant
|
0
|
0
|
1
|
|
Open-Label
Disease Progression
|
0
|
0
|
1
|
|
Open-Label
Adverse Event
|
0
|
0
|
3
|
|
Open-Label
Study Termination by sponsor
|
0
|
0
|
45
|
Baseline Characteristics
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Baseline characteristics by cohort
| Measure |
Double Blind Period - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment.
|
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment.
|
Total
n=75 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
Infants and toddlers (28 days-23 months)
|
35 participants
n=5 Participants
|
40 participants
n=7 Participants
|
75 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
28 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
59 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
5 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Vietnam
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Region of Enrollment
Singapore
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
China
|
18 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Region of Enrollment
Taiwan
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From baseline to Week 26Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Mean Change in Total Serum Bilirubin Levels
|
-3.5 mg/dl
Standard Error 0.853
|
-3.11 mg/dl
Standard Error 0.947
|
SECONDARY outcome
Timeframe: From baseline to Week 26Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Mean Change in Total Serum Bile Acids
|
-51.19 umol/L
Standard Error 24.436
|
-5.29 umol/L
Standard Error 28.440
|
SECONDARY outcome
Timeframe: From baseline to Week 26Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26
|
24 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: From Baseline to Week 26Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites)
Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.
|
8 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: From Baseline to Week 26Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Proportion of Participants Undergoing Liver Transplantation or Death
|
5 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: From Baseline to Week 26Splenomegaly =\> (spleen size \>2 cm below the costal margin palpated on physical examination)
Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices.
|
3 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: From Baseline to Week 26Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL
|
23 Participants
|
18 Participants
|
SECONDARY outcome
Timeframe: From Baseline to Week 26Outcome measures
| Measure |
Double Blind - Maralixibat
n=40 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
Double Blind - Placebo
n=35 Participants
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
|
|---|---|---|
|
Proportion of Participants With Mean sBA Levels ≤40 mmol/L
|
10 Participants
|
7 Participants
|
Adverse Events
Double Blind - Maralixibat
Serious events: 26 serious events
Other events: 38 other events
Deaths: 0 deaths
Double Blind - Placebo
Serious events: 25 serious events
Other events: 33 other events
Deaths: 0 deaths
Open Label - Maralixibat
Serious events: 21 serious events
Other events: 47 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Double Blind - Maralixibat
n=40 participants at risk
All participants who receive at least 1 dose of study medication during the double-blinded Maralixibat Period.
|
Double Blind - Placebo
n=35 participants at risk
All participants who receive at least 1 dose of study medication during the double-blinded Placebo Period.
|
Open Label - Maralixibat
n=52 participants at risk
All participants who receive at least 1 dose of study medication during the Open-Label Maralixibat Period.
|
|---|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Investigations
Hepatic enzyme increased
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Injury, poisoning and procedural complications
Accidental exposure to product by child
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Injury, poisoning and procedural complications
Procedural complication
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Blood and lymphatic system disorders
Coagulopathy
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
General disorders
Pyrexia
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
General disorders
Systemic inflammatory response syndrome
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Immune system disorders
Drug hypersensitivity
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Faeces discoloured
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Ileus
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Mouth ulceration
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Stomatitis
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Cholangitis
|
37.5%
15/40 • Number of events 23 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
48.6%
17/35 • Number of events 25 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
15.4%
8/52 • Number of events 10 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Cholangitis acute
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Drug-induced liver injury
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Jaundice
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Liver injury
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Adenovirus infection
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Bronchiolitis
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
COVID-19
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
8.6%
3/35 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Cytomegalovirus infection
|
2.5%
1/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Gastrointestinal infection
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Otitis media
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Parainfluenzae virus infection
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Pertussis
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Pneumonia
|
20.0%
8/40 • Number of events 13 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
11.4%
4/35 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Respiratory syncytial virus bronchiolitis
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Respiratory syncytial virus infection
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Rhinovirus infection
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Rotavirus infection
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Sepsis
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Viral infection
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Metabolism and nutrition disorders
Hyperinsulinism
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
Other adverse events
| Measure |
Double Blind - Maralixibat
n=40 participants at risk
All participants who receive at least 1 dose of study medication during the double-blinded Maralixibat Period.
|
Double Blind - Placebo
n=35 participants at risk
All participants who receive at least 1 dose of study medication during the double-blinded Placebo Period.
|
Open Label - Maralixibat
n=52 participants at risk
All participants who receive at least 1 dose of study medication during the Open-Label Maralixibat Period.
|
|---|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
12.5%
5/40 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
14.3%
5/35 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
7.7%
4/52 • Number of events 6 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Investigations
Blood bilirubin increased
|
2.5%
1/40 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Investigations
Hepatic enzyme increased
|
7.5%
3/40 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Investigations
Spleen palpable
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Investigations
Vitamin A decreased
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Injury, poisoning and procedural complications
Scratch
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Blood and lymphatic system disorders
Anaemia
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
8.6%
3/35 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Blood and lymphatic system disorders
Coagulopathy
|
5.0%
2/40 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
8.6%
3/35 • Number of events 4 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Blood and lymphatic system disorders
Splenomegaly
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
General disorders
Pyrexia
|
35.0%
14/40 • Number of events 19 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
22.9%
8/35 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
23.1%
12/52 • Number of events 18 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
32.5%
13/40 • Number of events 19 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
22.9%
8/35 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
25.0%
13/52 • Number of events 16 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
4/40 • Number of events 4 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
17.1%
6/35 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Dyspepsia
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Gastrointestinal disorders
Haematochezia
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 4 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Cholangitis
|
15.0%
6/40 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
17.1%
6/35 • Number of events 6 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Hepatobiliary disorders
Hepatomegaly
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
8/40 • Number of events 9 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
14.3%
5/35 • Number of events 6 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
15.4%
8/52 • Number of events 9 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
7.5%
3/40 • Number of events 6 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
9.6%
5/52 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Skin and subcutaneous tissue disorders
Eczema
|
15.0%
6/40 • Number of events 9 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
11.4%
4/35 • Number of events 4 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.5%
3/40 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
8.6%
3/35 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 4 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Bronchitis
|
7.5%
3/40 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
8.6%
3/35 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
7.7%
4/52 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
COVID-19
|
7.5%
3/40 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
14.3%
5/35 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Nasopharyngitis
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 7 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Otitis media
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Pharyngitis
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
1.9%
1/52 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Pneumonia
|
15.0%
6/40 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
14.3%
5/35 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
9.6%
5/52 • Number of events 6 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Respiratory tract infection
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.7%
2/35 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
17.5%
7/40 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
8.6%
3/35 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
23.1%
12/52 • Number of events 23 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Viral upper respiratory tract infection
|
2.5%
1/40 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
11.4%
4/35 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
7.7%
4/52 • Number of events 6 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Cytomegalovirus infection
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
2.9%
1/35 • Number of events 1 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Influenza
|
0.00%
0/40 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Infections and infestations
Parainfluenzae virus infection
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/35 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
0.00%
0/52 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Metabolism and nutrition disorders
Vitamin A deficiency
|
7.5%
3/40 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
20.0%
7/35 • Number of events 7 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
5.8%
3/52 • Number of events 3 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
17.5%
7/40 • Number of events 7 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
25.7%
9/35 • Number of events 9 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
15.4%
8/52 • Number of events 8 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Metabolism and nutrition disorders
Vitamin E deficiency
|
5.0%
2/40 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
11.4%
4/35 • Number of events 4 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
7.7%
4/52 • Number of events 4 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
|
Metabolism and nutrition disorders
Zinc deficiency
|
12.5%
5/40 • Number of events 5 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
14.3%
5/35 • Number of events 6 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
3.8%
2/52 • Number of events 2 • Baseline to EOT. In this study the longest time frame was 102.4 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60