Trial · NCT03566238
This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
NCT03566238, Phase 3, Completed, Albireo; results first posted 2021-09-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2. Trials, record NCT03566238. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03566238
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
- NCT number
- NCT03566238
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 62 participants, actual
- Lead sponsor
- Albireo
- Conditions
- PFIC1, PFIC2
- Interventions
- A4250 (odevixibat), Placebo
- First posted
- 2018-06-25
- Last updated
- 2025-11-28
- Results first posted
- 2021-09-05
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | From Baseline (Day 1) up to Week 24 | 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. |
| Primary | Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period | From Baseline (Day 1) up to Week 24 | 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. |
| Secondary | Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24 | Baseline (Day 1) and 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. |
| Secondary | Change From Baseline in Serum Alanine Aminotransferase (ALT) Concentration at Weeks 12 and 24 | Baseline (Day 1) and 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. |
| Secondary | Change From Baseline in Growth Parameters at Weeks 12 and 24 | Baseline (Day 1) and 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. |
| Secondary | 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 | 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. |
| Secondary | Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period | 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 | 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. |
| Secondary | Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period | 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 | 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. |
| Secondary | 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 | From Baseline (Day 1) up to Week 24 | 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. |
| Secondary | 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 | From Baseline (Day 1) up to Week 24 | 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. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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 | From Baseline (Day 1) up to Week 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period | From Baseline (Day 1) up to Week 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. | percentage of assessments | MEAN |
| SECONDARY | Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24 | Baseline (Day 1) and Weeks 12 and 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at Baseline and Weeks 12 and 24 are reported. | mcmol/L | MEAN |
| SECONDARY | Change From Baseline in Serum Alanine Aminotransferase (ALT) Concentration at Weeks 12 and 24 | Baseline (Day 1) and Weeks 12 and 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at Baseline and Weeks 12 and 24 are reported. | units/L | MEAN |
| SECONDARY | Change From Baseline in Growth Parameters at Weeks 12 and 24 | Baseline (Day 1) and Weeks 12 and 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at Baseline and Weeks 12 and 24 are reported. | z-score | MEAN |
| SECONDARY | 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 | Weeks 12 and 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at specific timepoints are reported. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period | 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 | The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at specific timepoints are reported. | percentage of days | MEAN |
| SECONDARY | Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period | 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 | The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at specific timepoints are reported. | scores on a scale | MEAN |
| SECONDARY | 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 | From Baseline (Day 1) up to Week 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. | percentage of assessments | MEAN |
| SECONDARY | 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 | From Baseline (Day 1) up to Week 24 | The FAS included all randomized participants who received at least 1 dose of study treatment. | percentage of assessments | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group399
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
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 | Placebo | 0 | 95% Confidence Interval 0.00 to 16.84 | percentage of participants | |
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 | A4250 Low Dose | 43.5 | 95% Confidence Interval 23.19 to 65.51 | percentage of participants | |
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 | A4250 High Dose | 21.1 | 95% Confidence Interval 6.05 to 45.57 | percentage of participants | |
| Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period | Placebo | 28.74 | Standard Error 5.209 | percentage of assessments | |
| Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period | A4250 Low Dose | 58.31 | Standard Error 6.205 | percentage of assessments | |
| Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period | A4250 High Dose | 47.69 | Standard Error 8.110 | percentage of assessments | |
| Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24 | Placebo | Week 12 | 7.44 | Standard Error 24.588 | mcmol/L |
| Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24 | A4250 Low Dose | Week 12 | -113.70 | Standard Error 38.011 | mcmol/L |
| Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24 | A4250 High Dose | Week 12 | -106.44 | Standard Error 41.201 | mcmol/L |
| Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24 | Placebo | Week 24 | 18.64 | Standard Error 31.559 | mcmol/L |
1 to 10 of 399Source ClinicalTrials.gov
See all 399 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 20 | |
| Overall Study | STARTED | A4250 Low Dose | 23 | |
| Overall Study | STARTED | A4250 High Dose | 19 | |
| Overall Study | Received Treatment | Placebo | 20 | |
| Overall Study | Received Treatment | A4250 Low Dose | 23 | |
| Overall Study | Received Treatment | A4250 High Dose | 19 | |
| Overall Study | COMPLETED | Placebo | 15 | |
| Overall Study | COMPLETED | A4250 Low Dose | 18 | |
| Overall Study | COMPLETED | A4250 High Dose | 16 | |
| Overall Study | NOT COMPLETED | Placebo | 5 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events339
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Placebo | 1 | 20 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | A4250 Low Dose | 0 | 23 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | A4250 High Dose | 0 | 19 | |
| other | Splenomegaly | Blood and lymphatic system disorders | Placebo | 0 | 20 | |
| other | Splenomegaly | Blood and lymphatic system disorders | A4250 Low Dose | 0 | 23 | |
| other | Splenomegaly | Blood and lymphatic system disorders | A4250 High Dose | 2 | 19 | |
| other | Ear pain | Ear and labyrinth disorders | Placebo | 1 | 20 | |
| other | Ear pain | Ear and labyrinth disorders | A4250 Low Dose | 0 | 23 | |
| other | Ear pain | Ear and labyrinth disorders | A4250 High Dose | 1 | 19 | |
| other | Otorrhoea | Ear and labyrinth disorders | Placebo | 0 | 20 |
1 to 10 of 339Source ClinicalTrials.gov
See all 339 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 20 |
| Total, all-cause mortality | A4250 Low Dose | 0 | 23 |
| Total, all-cause mortality | A4250 High Dose | 0 | 19 |
| Total, other adverse events | Placebo | 17 | 20 |
| Total, other adverse events | A4250 Low Dose | 19 | 23 |
| Total, other adverse events | A4250 High Dose | 16 | 19 |
| Total, serious adverse events | Placebo | 5 | 20 |
| Total, serious adverse events | A4250 Low Dose | 0 | 23 |
| Total, serious adverse events | A4250 High Dose | 3 | 19 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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