A Phase 3 interventional study of A4250 (odevixibat) and Placebo in PFIC1 and PFIC2, sponsored by Albireo. Completed at 45 sites in 15 countries. Open to participants aged 6 Months to 18 Years. Per ClinicalTrials.gov, last updated 2025-11-28.
Sponsored by Albireo (part of Ipsen) · Phase 3, Interventional, and Treatment
Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2.
Up to 50 sites in the following countries will take part in this study:
Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Sweden, Turkey, United Kingdom, United States, and Saudi Arabia
150 studies on the registry are indexed under Cholestasis; 18 are open to participants now.
This study's enrollment of 62 is above the median of 53 across 90 interventional studies indexed under Cholestasis.
Browse Cholestasis studies →Albireo is the lead sponsor of 11 studies on the registry; none are open to participants now.
First submitted before Albireo became part of Ipsen.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Participant with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Capsules for oral administration (40 ug/kg) once daily for 24 weeks
Drug: A4250 (odevixibat)
Capsules for oral administration (120 ug/kg) once daily for 24 weeks
Drug: A4250 (odevixibat)
Capsules for oral administration (to match active) once daily for 24 weeks
Drug: Placebo
A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT).
Placebo identical in appearance to active drug (A4250).
Percentage of Participants With at Least a 70% Reduction in Fasting Serum Bile Acid (s-BA) Concentration From Baseline to the End of Treatment or Reaching a Level <=70 Micromoles Per Liter (Mcmol/L) After 24 Weeks of Treatment
Fasting s-BA baseline was calculated as the average of the last 2 values prior to the first dose. The end value was the average of the values at Weeks 22 and 24 after the start of double-blind treatment. Participants who had at least 70% reduction in fasting s-BA from baseline to the end of treatment or reached \<=70 mcmol/L after 24 weeks of treatment were considered as responder. Participants with missing average at the end of treatment were classified as non-responder. Percentages are rounded to hundredth decimal.
Time frame: From Baseline (Day 1) up to Week 24
Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100.
Time frame: From Baseline (Day 1) up to Week 24
Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24
Blood samples for analysis of s-BA were drawn at all visits. Participants were to fast (water intake only was permissible) for at least 4 hours prior to the collection of samples. Exceptions could be made for infants \<12 months of age if they were unable to fast for the full 4 hours. Baseline was the average of the last 2 non-missing values of fasting s-BA concentration prior to the first dose of study treatment.
Time frame: Baseline (Day 1) and Weeks 12 and 24
Change From Baseline in Serum Alanine Aminotransferase (ALT) Concentration at Weeks 12 and 24
Blood samples were collected to determine the ALT concentration. Baseline was the last available assessment before the first dose of study treatment.
Time frame: Baseline (Day 1) and Weeks 12 and 24
Change From Baseline in Growth Parameters at Weeks 12 and 24
The change in the growth parameters was assessed using linear growth deficit \[height (centimeter), weight (kilogram) and body mass index (BMI) (kg/meter square)\] compared to standard growth curve (Z-score) calculated by using the software or methods from the centers for disease control (CDC) website for participants with age \>=2 years old and from the world health organization website for participants with age \<2 years old. Participants whose accurate age was not available, Z-score was not calculated. A Z-score indicated how many standard deviation's (SD) a participant's measurement (like height, weight and BMI), was from the average for their age and sex. A Z-score of 0 represents the median or 50th percentile, while positive or negative values show how far above or below average a measurement was. Baseline was the last available assessment before the first dose of study treatment.
Time frame: Baseline (Day 1) and Weeks 12 and 24
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
The responder for pruritus scores was defined as a participant who achieved at least a 1-point reduction in the ObsRO pruritus score. Percentages are rounded to hundredth decimal.
Time frame: Weeks 12 and 24
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
The sleep disturbance were recorded twice daily via the electronic diary (eDiary). Participants and/or caregivers completed the eDiary every day in the morning and in the evening. The morning diary was completed shortly after the participant woke up and was used to record nighttime itching and scratching severity, aspects of sleep disturbance, and tiredness upon waking (AM scores). The evening/bedtime diary was completed just before the participant went to bed and recorded participant's itching and scratching severity, and tiredness during the day (PM scores). Both morning and bedtime diaries included Albireo ObsRO and PRO items. Baseline was the average of 14-day scores before the first dose of study treatment.
Time frame: Baseline (Day 1) and Weeks 1 to 4, Weeks 5 to 8, Weeks 9 to 12, Weeks 13 to 16, Weeks 17 to 20, and Weeks 21 to 24
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
The ObsRO and electronic diary (eDiary) PRO scale called PRUCISION was used to assess pruritus and sleep outcomes. Participants and/or caregivers completed the eDiary every day in the morning (AM scores) and in the evening (PM scores). Specifically, for difficulty falling or staying asleep, the following 2 respective questions were asked in the Morning Daily eDiary: 1) How hard was it to fall asleep last night because of your itching. and 2) How hard was it to stay asleep last night because of your itching. The PRO was a 5-point scale, and scores ranged from 0 (no itching) to 28 (the worst itching), where higher scores indicated a greater amount of itching, sleep disturbance, and tiredness. No subscales were used. Change in sleep parameters measured with the Albireo PRO and ObsRO instruments by each 4-week interval over the 24-week treatment period was assessed. Baseline was the average of 14-day scores before the first dose of study treatment.
Time frame: Baseline (Day 1) and Weeks 1 to 4, Weeks 5 to 8, Weeks 9 to 12, Weeks 13 to 16, Weeks 17 to 20, and Weeks 21 to 24
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
Time frame: From Baseline (Day 1) up to Week 24
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo PRO instrument, only participants \>=8 years of age completed the Albireo PRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
Time frame: From Baseline (Day 1) up to Week 24
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
Time frame: Baseline (Day 1) and Weeks 1 to 4, Weeks 5 to 8, Weeks 9 to 12, Weeks 13 to 16, Weeks 17 to 20, and Weeks 21 to 24
Number of Participants Underwent Biliary Diversion Surgery and Liver Transplantation
The number of participants underwent biliary diversion surgery and liver transplantation was determined.
Time frame: From Baseline (Day 1) up to Week 24
Number of Participants Achieved Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO and PRO Instruments Over the 24-Week Treatment Period
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO and PRO instruments. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100.
Time frame: From Baseline (Day 1) up to Week 24
This Phase 3, double-blind, placebo-controlled study was conducted in children with progressive familial intrahepatic cholestasis (PFIC) at 33 study centers in 12 countries between 16 May 2018 and 28 July 2020.
| Milestone | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Started | 20 | 23 | 19 |
| Received treatment | 20 | 23 | 19 |
| Completed | 15 | 18 | 16 |
| Not completed | 5 | 5 | 3 |
| Withdrew: Adverse event | 0 | 0 | 1 |
| Withdrew: Lack of efficacy | 5 | 4 | 2 |
| Withdrew: Other | 0 | 1 | 0 |
Fasting s-BA baseline was calculated as the average of the last 2 values prior to the first dose. The end value was the average of the values at Weeks 22 and 24 after the start of double-blind treatment. Participants who had at least 70% reduction in fasting s-BA from baseline to the end of treatment or reached \<=70 mcmol/L after 24 weeks of treatment were considered as responder. Participants with missing average at the end of treatment were classified as non-responder. Percentages are rounded to hundredth decimal.
| percentage of participants | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Percentage of Participants With at Least a 70% Reduction in Fasting Serum Bile Acid (s-BA) Concentration From Baseline to the End of Treatment or Reaching a Level <=70 Micromoles Per Liter (Mcmol/L) After 24 Weeks of Treatment | 0 (0.00 to 16.84) | 43.5 (23.19 to 65.51) | 21.1 (6.05 to 45.57) |
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100.
| percentage of assessments | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period | 28.74 ± 5.209 | 58.31 ± 6.205 | 47.69 ± 8.110 |
Blood samples for analysis of s-BA were drawn at all visits. Participants were to fast (water intake only was permissible) for at least 4 hours prior to the collection of samples. Exceptions could be made for infants \<12 months of age if they were unable to fast for the full 4 hours. Baseline was the average of the last 2 non-missing values of fasting s-BA concentration prior to the first dose of study treatment.
| mcmol/L | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Week 12 | 7.44 ± 24.588 | -113.70 ± 38.011 | -106.44 ± 41.201 |
| Week 24 | 18.64 ± 31.559 | -145.03 ± 41.951 | -72.90 ± 52.617 |
Blood samples were collected to determine the ALT concentration. Baseline was the last available assessment before the first dose of study treatment.
| units/L | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Week 12 | 1.7 ± 10.50 | -25.9 ± 23.36 | -13.8 ± 19.42 |
| Week 24 | 3.7 ± 4.95 | -27.9 ± 17.97 | -25.3 ± 22.47 |
The change in the growth parameters was assessed using linear growth deficit \[height (centimeter), weight (kilogram) and body mass index (BMI) (kg/meter square)\] compared to standard growth curve (Z-score) calculated by using the software or methods from the centers for disease control (CDC) website for participants with age \>=2 years old and from the world health organization website for participants with age \<2 years old. Participants whose accurate age was not available, Z-score was not calculated. A Z-score indicated how many standard deviation's (SD) a participant's measurement (like height, weight and BMI), was from the average for their age and sex. A Z-score of 0 represents the median or 50th percentile, while positive or negative values show how far above or below average a measurement was. Baseline was the last available assessment before the first dose of study treatment.
| z-score | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Height deficit: Week 12 | -0.03 ± 0.127 | 0.01 ± 0.108 | -0.06 ± 0.100 |
| Height deficit: Week 24 | -0.16 ± 0.104 | 0.05 ± 0.105 | 0.00 ± 0.163 |
| Weight deficit: Week 12 | 0.13 ± 0.066 | 0.20 ± 0.078 | 0.00 ± 0.100 |
| Weight deficit: Week 24 | 0.10 ± 0.102 | 0.29 ± 0.106 | 0.15 ± 0.124 |
| BMI deficit: Week 12 | 0.18 ± 0.148 | 0.23 ± 0.114 | 0.08 ± 0.147 |
| BMI deficit: Week 24 | 0.26 ± 0.156 | 0.36 ± 0.113 | 0.20 ± 0.203 |
The responder for pruritus scores was defined as a participant who achieved at least a 1-point reduction in the ObsRO pruritus score. Percentages are rounded to hundredth decimal.
| percentage of participants | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| AM and PM scores combined: Week 12 (Bi-weekly score) | 5.0 (0.13 to 24.87) | 60.9 (38.54 to 80.29) | 42.1 (20.25 to 66.50) |
| AM and PM scores combined: Week 12 (Monthly score) | 10.0 (1.23 to 31.70) | 52.2 (30.59 to 73.18) | 42.1 (20.25 to 66.50) |
| AM and PM scores combined: Week 24 (Bi-weekly score) | 11.8 (1.46 to 36.44) | 43.5 (23.19 to 65.51) | 33.3 (13.34 to 59.01) |
| AM and PM scores combined: Week 24 (Monthly score) | 10.5 (1.30 to 33.14) | 52.2 (30.59 to 73.18) | 31.6 (12.58 to 56.55) |
| AM score: Week 12 (Bi-weekly score) | 10.0 (1.23 to 31.70) | 60.9 (38.54 to 80.29) | 42.1 (20.25 to 66.50) |
| AM score: Week 12 (Monthly score) | 10.0 (1.23 to 31.70) | 56.5 (34.49 to 76.81) | 42.1 (20.25 to 66.50) |
| AM score: Week 24 (Bi-weekly score) | 11.8 (1.46 to 36.44) | 39.1 (19.71 to 61.46) | 33.3 (13.34 to 59.01) |
| AM score: Week 24 (Monthly score) | 15.8 (3.38 to 39.58) | 52.2 (30.59 to 73.18) | 42.1 (20.25 to 66.50) |
| PM score: Week 12 (Bi-weekly score) | 10.0 (1.23 to 31.70) | 65.2 (42.73 to 83.62) | 36.8 (16.29 to 61.64) |
| PM score: Week 12 (Monthly score) | 10.0 (1.23 to 31.70) | 60.9 (38.54 to 80.29) | 36.8 (16.29 to 61.64) |
| PM score: Week 24 (Bi-weekly score) | 11.8 (1.46 to 36.44) | 56.5 (34.49 to 76.81) | 33.3 (13.34 to 59.01) |
| PM score: Week 24 (Monthly score) | 10.5 (1.30 to 33.14) | 52.2 (30.59 to 73.18) | 31.6 (12.58 to 56.55) |
The sleep disturbance were recorded twice daily via the electronic diary (eDiary). Participants and/or caregivers completed the eDiary every day in the morning and in the evening. The morning diary was completed shortly after the participant woke up and was used to record nighttime itching and scratching severity, aspects of sleep disturbance, and tiredness upon waking (AM scores). The evening/bedtime diary was completed just before the participant went to bed and recorded participant's itching and scratching severity, and tiredness during the day (PM scores). Both morning and bedtime diaries included Albireo ObsRO and PRO items. Baseline was the average of 14-day scores before the first dose of study treatment.
| percentage of days | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Seeing blood due to scratching: Weeks 1 to 4 | -15.79 ± 4.700 | -14.42 ± 5.854 | -12.97 ± 6.036 |
| Seeing blood due to scratching: Weeks 5 to 8 | -12.10 ± 6.353 | -26.22 ± 7.427 | -13.99 ± 7.770 |
| Seeing blood due to scratching: Weeks 9 to 12 | -13.49 ± 6.320 | -29.22 ± 7.593 | -16.89 ± 9.258 |
| Seeing blood due to scratching: Weeks 13 to 16 | -15.75 ± 6.264 | -33.38 ± 8.219 | -18.16 ± 9.403 |
| Seeing blood due to scratching: Weeks 17 to 20 | -22.02 ± 7.999 | -32.14 ± 8.531 | -18.02 ± 9.967 |
| Seeing blood due to scratching: Weeks 21 to 24 | -23.72 ± 8.563 | -30.82 ± 8.184 | -15.52 ± 10.465 |
| Help falling asleep: Weeks 1 to 4 | -0.91 ± 1.596 | -14.73 ± 5.683 | -20.37 ± 6.390 |
| Help falling asleep: Weeks 5 to 8 | -2.35 ± 2.397 | -25.03 ± 7.527 | -31.49 ± 10.209 |
| Help falling asleep: Weeks 9 to 12 | -0.90 ± 1.152 | -31.03 ± 8.061 | -36.72 ± 11.154 |
| Help falling asleep: Weeks 13 to 16 | -0.86 ± 1.392 | -46.10 ± 9.677 | -37.76 ± 12.013 |
| Help falling asleep: Weeks 17 to 20 | -6.27 ± 3.862 | -45.65 ± 9.524 | -40.51 ± 12.566 |
| Help falling asleep: Weeks 21 to 24 | -3.19 ± 2.890 | -51.75 ± 9.857 | -32.58 ± 14.573 |
| Soothing: Weeks 1 to 4 | -1.15 ± 1.338 | -12.92 ± 5.911 | -21.88 ± 6.228 |
| Soothing: Weeks 5 to 8 | -3.76 ± 2.773 | -17.89 ± 7.562 | -35.97 ± 9.795 |
| Soothing: Weeks 9 to 12 | -5.00 ± 2.819 | -29.79 ± 8.570 | -41.21 ± 10.796 |
| Soothing: Weeks 13 to 16 | -2.12 ± 3.006 | -42.79 ± 10.364 | -40.23 ± 11.611 |
| Soothing: Weeks 17 to 20 | -4.73 ± 3.742 | -44.96 ± 10.485 | -40.13 ± 12.324 |
| Soothing: Weeks 21 to 24 | -7.64 ± 6.182 | -51.48 ± 10.323 | -34.87 ± 13.369 |
| Sleeping with the caregiver: Weeks 1 to 4 | -4.49 ± 1.961 | -15.30 ± 6.396 | -24.57 ± 7.024 |
| Sleeping with the caregiver: Weeks 5 to 8 | -5.93 ± 3.649 | -19.73 ± 7.061 | -35.58 ± 10.053 |
| Sleeping with the caregiver: Weeks 9 to 12 | -5.74 ± 3.585 | -26.12 ± 9.543 | -36.00 ± 10.361 |
| Sleeping with the caregiver: Weeks 13 to 16 | -4.96 ± 3.481 | -40.55 ± 10.501 | -37.52 ± 11.083 |
| Sleeping with the caregiver: Weeks 17 to 20 | -2.59 ± 3.688 | -42.20 ± 10.247 | -35.14 ± 11.563 |
| Sleeping with the caregiver: Weeks 21 to 24 | -5.45 ± 4.844 | -49.35 ± 10.466 | -33.14 ± 11.801 |
| Taking medications to induce sleep: Weeks 1 to 4 | -1.78 ± 1.921 | -0.61 ± 1.913 | 0.98 ± 2.320 |
| Taking medications to induce sleep: Weeks 5 to 8 | 1.44 ± 4.292 | 0.18 ± 3.018 | -2.43 ± 3.036 |
| Taking medications to induce sleep: Weeks 9 to 12 | 2.40 ± 4.271 | 1.31 ± 3.656 | -5.74 ± 4.967 |
| Taking medications to induce sleep: Weeks 13 to 16 | 0.16 ± 4.647 | -1.61 ± 2.556 | -4.58 ± 4.681 |
| Taking medications to induce sleep: Weeks 17 to 20 | 3.53 ± 5.384 | -1.99 ± 2.141 | -3.26 ± 6.403 |
| Taking medications to induce sleep: Weeks 21 to 24 | 5.50 ± 5.307 | -0.03 ± 2.251 | 2.04 ± 8.413 |
The ObsRO and electronic diary (eDiary) PRO scale called PRUCISION was used to assess pruritus and sleep outcomes. Participants and/or caregivers completed the eDiary every day in the morning (AM scores) and in the evening (PM scores). Specifically, for difficulty falling or staying asleep, the following 2 respective questions were asked in the Morning Daily eDiary: 1) How hard was it to fall asleep last night because of your itching. and 2) How hard was it to stay asleep last night because of your itching. The PRO was a 5-point scale, and scores ranged from 0 (no itching) to 28 (the worst itching), where higher scores indicated a greater amount of itching, sleep disturbance, and tiredness. No subscales were used. Change in sleep parameters measured with the Albireo PRO and ObsRO instruments by each 4-week interval over the 24-week treatment period was assessed. Baseline was the average of 14-day scores before the first dose of study treatment.
| scores on a scale | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Difficulty falling asleep: Weeks 1 to 4 | -0.46 ± 0.143 | -0.72 ± 0.494 | -0.59 ± 0.394 |
| Difficulty falling asleep: Weeks 5 to 8 | -0.34 ± 0.018 | -1.40 ± 1.009 | -0.65 ± 0.571 |
| Difficulty falling asleep: Weeks 9 to 12 | -0.38 ± 0.339 | -2.31 ± 0.499 | -0.95 ± 0.650 |
| Difficulty falling asleep: Weeks 13 to 16 | -0.16 ± 0.268 | -2.07 ± 0.863 | -1.01 ± 0.665 |
| Difficulty falling asleep: Weeks 17 to 20 | -0.52 ± 0.228 | -2.12 ± 0.974 | -1.02 ± 0.710 |
| Difficulty falling asleep: Weeks 21 to 24 | -0.25 ± 0.179 | -2.22 ± 0.823 | -0.82 ± 0.658 |
| Difficulty staying asleep: Weeks 1 to 4 | -0.13 ± 0.411 | -0.70 ± 0.548 | -0.43 ± 0.440 |
| Difficulty staying asleep: Weeks 5 to 8 | 0.09 ± 0.518 | -1.48 ± 0.932 | -0.50 ± 0.553 |
| Difficulty staying asleep: Weeks 9 to 12 | 0.02 ± 0.661 | -2.51 ± 0.352 | -0.77 ± 0.670 |
| Difficulty staying asleep: Weeks 13 to 16 | 0.13 ± 0.625 | -2.46 ± 0.464 | -0.89 ± 0.626 |
| Difficulty staying asleep: Weeks 17 to 20 | -0.15 ± 0.147 | -2.36 ± 0.637 | -0.95 ± 0.644 |
| Difficulty staying asleep: Weeks 21 to 24 | 0.25 ± 0.429 | -2.55 ± 0.448 | -0.78 ± 0.633 |
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
| percentage of assessments | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| AM and PM scores combined: Weeks 0 to 4 | 33.48 ± 5.881 | 51.40 ± 5.818 | 42.48 ± 7.508 |
| AM and PM scores combined: Weeks 0 to 8 | 32.72 ± 5.871 | 54.23 ± 5.887 | 48.21 ± 7.479 |
| AM and PM scores combined: Weeks 0 to 12 | 32.35 ± 5.945 | 58.85 ± 5.738 | 49.15 ± 7.428 |
| AM and PM scores combined: Weeks 0 to 16 | 30.67 ± 5.522 | 58.83 ± 5.925 | 48.31 ± 7.624 |
| AM and PM scores combined: Weeks 0 to 18 | 29.88 ± 5.462 | 58.80 ± 6.004 | 48.25 ± 7.809 |
| AM and PM scores combined: Weeks 0 to 20 | 29.45 ± 5.376 | 58.68 ± 6.079 | 48.10 ± 7.975 |
| AM score: Weeks 0 to 4 | 31.07 ± 5.436 | 48.14 ± 6.161 | 47.37 ± 8.439 |
| AM score: Weeks 0 to 8 | 31.07 ± 5.213 | 51.16 ± 6.129 | 51.79 ± 8.001 |
| AM score: Weeks 0 to 12 | 31.07 ± 5.374 | 56.06 ± 5.922 | 52.26 ± 7.693 |
| AM score: Weeks 0 to 16 | 29.91 ± 5.246 | 56.41 ± 6.183 | 50.47 ± 7.660 |
| AM score: Weeks 0 to 18 | 28.93 ± 5.224 | 56.35 ± 6.249 | 49.92 ± 7.800 |
| AM score: Weeks 0 to 20 | 28.50 ± 5.206 | 56.27 ± 6.293 | 49.74 ± 7.993 |
| AM score: Weeks 0 to 24 | 28.17 ± 5.276 | 55.73 ± 6.396 | 48.92 ± 8.197 |
| PM score: Weeks 0 to 4 | 35.89 ± 7.055 | 54.66 ± 6.594 | 37.59 ± 7.629 |
| PM score: Weeks 0 to 8 | 34.38 ± 7.290 | 57.30 ± 6.478 | 44.64 ± 7.804 |
| PM score: Weeks 0 to 12 | 33.63 ± 7.404 | 61.65 ± 6.330 | 46.05 ± 7.884 |
| PM score: Weeks 0 to 16 | 31.43 ± 7.006 | 61.26 ± 6.205 | 46.15 ± 8.178 |
| PM score: Weeks 0 to 18 | 30.83 ± 6.898 | 61.25 ± 6.225 | 46.57 ± 8.363 |
| PM score: Weeks 0 to 20 | 30.39 ± 6.812 | 61.09 ± 6.284 | 46.47 ± 8.434 |
| PM score: Weeks 0 to 24 | 29.31 ± 6.592 | 60.89 ± 6.385 | 46.47 ± 8.456 |
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo PRO instrument, only participants \>=8 years of age completed the Albireo PRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
| percentage of assessments | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| AM and PM scores combined: Weeks 0 to 4 | 64.29 ± 8.929 | 43.75 ± 25.893 | 38.93 ± 15.272 |
| AM and PM scores combined: Weeks 0 to 8 | 46.88 ± 12.946 | 52.68 ± 25.893 | 41.96 ± 16.178 |
| AM and PM scores combined: Weeks 0 to 12 | 41.96 ± 10.417 | 63.39 ± 18.155 | 45.00 ± 16.939 |
| AM and PM scores combined: Weeks 0 to 16 | 38.62 ± 3.348 | 66.74 ± 18.080 | 46.96 ± 17.477 |
| AM and PM scores combined: Weeks 0 to 18 | 37.50 ± 0.198 | 67.66 ± 18.849 | 47.14 ± 17.558 |
| AM and PM scores combined: Weeks 0 to 20 | 34.82 ± 0.179 | 67.32 ± 20.179 | 48.07 ± 18.010 |
| AM and PM scores combined: Weeks 0 to 24 | 31.70 ± 1.637 | 68.88 ± 19.772 | 47.65 ± 17.997 |
| AM score: Weeks 0 to 4 | 55.36 ± 8.929 | 41.07 ± 23.214 | 37.86 ± 16.115 |
| AM score: Weeks 0 to 8 | 39.29 ± 14.286 | 50.00 ± 21.429 | 38.93 ± 16.243 |
| AM score: Weeks 0 to 12 | 33.93 ± 10.119 | 61.31 ± 11.310 | 41.43 ± 16.562 |
| AM score: Weeks 0 to 16 | 31.70 ± 1.339 | 65.18 ± 12.500 | 42.68 ± 16.554 |
| AM score: Weeks 0 to 18 | 31.35 ± 1.190 | 66.67 ± 13.492 | 42.22 ± 16.343 |
| AM score: Weeks 0 to 20 | 28.93 ± 0.357 | 66.07 ± 15.357 | 43.71 ± 16.860 |
| AM score: Weeks 0 to 24 | 26.19 ± 1.190 | 66.72 ± 16.718 | 43.24 ± 16.835 |
| PM score: Weeks 0 to 4 | 73.21 ± 8.929 | 46.43 ± 28.571 | 40.00 ± 15.042 |
| PM score: Weeks 0 to 8 | 54.46 ± 11.607 | 55.36 ± 30.357 | 45.00 ± 16.545 |
| PM score: Weeks 0 to 12 | 50.00 ± 10.714 | 65.48 ± 25.000 | 48.57 ± 17.715 |
| PM score: Weeks 0 to 16 | 45.54 ± 5.357 | 68.30 ± 23.661 | 51.25 ± 18.751 |
| PM score: Weeks 0 to 18 | 43.65 ± 1.587 | 68.65 ± 24.206 | 52.06 ± 19.230 |
| PM score: Weeks 0 to 20 | 40.71 ± 0.000 | 68.57 ± 25.000 | 52.43 ± 19.568 |
| PM score: Weeks 0 to 24 | 37.20 ± 2.083 | 71.04 ± 22.825 | 52.06 ± 19.595 |
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
| percentage of assessments | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| AM and PM scores combined: Weeks 1 to 4 | 33.48 ± 5.881 | 51.40 ± 5.818 | 42.48 ± 7.508 |
| AM and PM scores combined: Weeks 5 to 8 | 31.96 ± 6.515 | 57.07 ± 6.714 | 53.95 ± 8.423 |
| AM and PM scores combined: Weeks 9 to 12 | 31.61 ± 6.794 | 68.09 ± 6.357 | 53.67 ± 9.404 |
| AM and PM scores combined: Weeks 13 to 16 | 28.36 ± 7.534 | 67.59 ± 7.779 | 54.35 ± 9.802 |
| AM and PM scores combined: Weeks 17 to 20 | 32.62 ± 8.583 | 66.79 ± 7.805 | 56.14 ± 10.655 |
| AM and PM scores combined: Weeks 21 to 24 | 35.42 ± 8.783 | 68.57 ± 6.926 | 55.12 ± 10.047 |
| AM score: Weeks 1 to 4 | 31.07 ± 5.436 | 48.14 ± 6.161 | 47.37 ± 8.439 |
| AM score: Weeks 5 to 8 | 31.07 ± 5.812 | 54.19 ± 6.783 | 56.20 ± 8.483 |
| AM score: Weeks 9 to 12 | 31.07 ± 6.747 | 65.84 ± 6.854 | 55.95 ± 9.383 |
| AM score: Weeks 13 to 16 | 29.62 ± 8.502 | 66.07 ± 8.042 | 53.57 ± 9.550 |
| AM score: Weeks 17 to 20 | 30.24 ± 8.733 | 64.11 ± 8.121 | 55.58 ± 11.171 |
| AM score: Weeks 21 to 24 | 37.30 ± 10.909 | 64.37 ± 7.324 | 54.35 ± 10.801 |
| PM score: Weeks 1 to 4 | 35.89 ± 7.055 | 54.66 ± 6.594 | 37.59 ± 7.629 |
| PM score: Weeks 5 to 8 | 32.86 ± 8.138 | 59.94 ± 7.355 | 51.69 ± 9.051 |
| PM score: Weeks 9 to 12 | 32.14 ± 8.078 | 70.34 ± 6.816 | 51.39 ± 10.015 |
| PM score: Weeks 13 to 16 | 27.10 ± 8.997 | 69.11 ± 7.783 | 55.13 ± 10.664 |
| PM score: Weeks 17 to 20 | 35.00 ± 10.323 | 69.46 ± 7.848 | 56.70 ± 10.554 |
| PM score: Weeks 21 to 24 | 33.54 ± 9.699 | 72.76 ± 7.185 | 55.90 ± 9.976 |
The number of participants underwent biliary diversion surgery and liver transplantation was determined.
| Participants | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Biliary diversion surgery | 0 | 0 | 0 |
| Liver transplantation | 0 | 0 | 0 |
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO and PRO instruments. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100.
| Participants | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Albireo ObsRO Instrument: AM and PM scores combined | 4 | 17 | 9 |
| Albireo ObsRO Instrument: AM score | 3 | 13 | 10 |
| Albireo ObsRO Instrument: PM score | 5 | 16 | 9 |
| Albireo PRO Instrument: AM and PM scores combined | 0 | 1 | 3 |
| Albireo PRO Instrument: AM score | 0 | 1 | 3 |
| Albireo PRO Instrument: PM score | 0 | 1 | 3 |
Collected over All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | 0/20 (0%) | 5/20 (25%) | 17/20 (85%) |
| A4250 Low Dose | 0/23 (0%) | 0/23 (0%) | 19/23 (82.6%) |
| A4250 High Dose | 0/19 (0%) | 3/19 (15.8%) | 16/19 (84.2%) |
| Event | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| Supraventricular tachycardiaCardiac disorders | 0/20 | 0/23 | 1/19 |
| Urinary tract infectionInfections and infestations | 1/20 | 0/23 | 1/19 |
| Liver function test increasedInvestigations | 0/20 | 0/23 | 1/19 |
| DehydrationMetabolism and nutrition disorders | 0/20 | 0/23 | 1/19 |
| PyrexiaGeneral disorders | 1/20 | 0/23 | 0/19 |
| Gastroenteritis adenovirusInfections and infestations | 1/20 | 0/23 | 0/19 |
| H1N1 influenzaInfections and infestations | 1/20 | 0/23 | 0/19 |
| InfluenzaInfections and infestations | 1/20 | 0/23 | 0/19 |
| Viral infectionInfections and infestations | 1/20 | 0/23 | 0/19 |
| Viral upper respiratory tract infectionInfections and infestations | 1/20 | 0/23 | 0/19 |
| Event | Placebo | A4250 Low Dose | A4250 High Dose |
|---|---|---|---|
| DiarrhoeaGastrointestinal disorders | 1/20 | 9/23 | 4/19 |
| PyrexiaGeneral disorders | 5/20 | 7/23 | 5/19 |
| Upper respiratory tract infectionInfections and infestations | 3/20 | 3/23 | 5/19 |
| ConstipationGastrointestinal disorders | 4/20 | 0/23 | 0/19 |
| VomitingGastrointestinal disorders | 0/20 | 4/23 | 3/19 |
| Alanine aminotransferase increasedInvestigations | 1/20 | 3/23 | 3/19 |
| CoughRespiratory, thoracic and mediastinal disorders | 3/20 | 0/23 | 2/19 |
| RashSkin and subcutaneous tissue disorders | 3/20 | 0/23 | 0/19 |
| Blood bilirubin increasedInvestigations | 2/20 | 3/23 | 2/19 |
| SplenomegalyBlood and lymphatic system disorders | 0/20 | 0/23 | 2/19 |
Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of study treatment.
| Age, Categorical(Participants) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| <=18 years | 20 | 23 | 19 | 62 |
| Between 18 and 65 years | 0 | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 | 0 |
| Age, Continuous(years) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| Mean | 3.75 ± 3.853 | 3.86 ± 3.660 | 5.24 ± 4.188 | 4.25 ± 3.883 |
| Age, Customized(Participants) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| Age Category 1 — 6 months to 5 years | 16 | 17 | 14 | 47 |
| Age Category 1 — 6 to 12 years | 3 | 5 | 4 | 12 |
| Age Category 1 — 13 to 18 years | 1 | 1 | 1 | 3 |
| Sex: Female, Male(Participants) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| Female | 8 | 12 | 11 | 31 |
| Male | 12 | 11 | 8 | 31 |
| Race/Ethnicity, Customized(Participants) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| Race — White | 17 | 18 | 17 | 52 |
| Race — Black or African American | 0 | 2 | 0 | 2 |
| Race — Asian | 1 | 0 | 1 | 2 |
| Race — American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Race — Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Race — Other | 2 | 3 | 1 | 6 |
| Race/Ethnicity, Customized(Participants) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| Ethnicity — Hispanic or Latino | 1 | 0 | 0 | 1 |
| Ethnicity — Not Hispanic or Latino | 19 | 23 | 19 | 61 |
| Region of Enrollment(participants) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| United States | 3 | 2 | 3 | 8 |
| United Kingdom | 5 | 6 | 2 | 13 |
| Saudi Arabia | 1 | 0 | 2 | 3 |
| Canada | 1 | 1 | 0 | 2 |
| Netherlands | 1 | 0 | 2 | 3 |
| Turkey | 1 | 7 | 3 | 11 |
| Poland | 2 | 0 | 1 | 3 |
| Italy | 1 | 1 | 0 | 2 |
| Israel | 1 | 0 | 1 | 2 |
| France | 1 | 3 | 1 | 5 |
| Australia | 1 | 0 | 0 | 1 |
| Germany | 2 | 3 | 4 | 9 |
| Type of PFIC(Participants) | Placebo | A4250 Low Dose | A4250 High Dose | Total |
|---|---|---|---|---|
| Type 1 | 5 | 7 | 5 | 17 |
| Type 2 | 15 | 16 | 14 | 45 |
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Studies of the drug tested here: Odevixibat