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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002325-38 | EudraCT Number |
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Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in children with PFIC.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| A4250 | Experimental | Capsules for oral administration (40 or 120 µg/kg) once daily for 72 weeks, or 40 µg/kg/day for the first 12 weeks followed by 120 µg/kg/day for the remaining 60 weeks" |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| A4250 (odevixibat) | Drug | A4250 is a small molecule and selective inhibitor of IBAT |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Serum Bile Acids | Blood samples for analysis of fasting total serum bile acids were drawn at specified timepoints. Participants were to fast (water intake only) for at least 4 hours prior to the collection of samples for serum bile acids. Exceptions were made for infants <12 months of age if unable to fast for the full 4 hours. Baseline for Cohort 1 placebo/odevixibat and Cohort 2 groups was defined as the average of last 2 values before the first dose of study treatment in the study. Baseline for Cohort 1 odevixibat/odevixibat group was defined as average of last 2 values before the first dose of study treatment in study A4250-005 (NCT03566238). | Baseline and Week 72 |
| Proportion of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. | Baseline and Week 72 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. |
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Inclusion Criteria Cohort 1:
Inclusion Criteria Cohort 2:
Exclusion Criteria Cohort 1:
Exclusion Criteria Cohort 2:
Known pathologic variations of the ABCB11 gene that have been demonstrated to result in complete absence of the BSEP protein
Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to,inflammatory bowel disease.
Patient with past medical history or ongoing chronic (i.e., >3 months) diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae.
Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant, acute, or chronic infection, or past medical history of any major episode of infection requiring hospitalization or treatment with parenteral anti-infective treatment within 4 weeks of treatment start (Study Day 1) or completion of oral anti-infective treatment within 2 weeks prior to start of Screening Period.
Any patient with suspected or confirmed cancers except for basal cell carcinoma, and non-liver cancers treated at least 5 years prior to Screening with no evidence of recurrence.
Patient has had a liver transplant, or a liver transplant is planned within 6 months of the Screening/Inclusion Visit.
Decompensated liver disease, coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
INR >1.4 (the patient may be treated with Vitamin K intravenously, and if INR is ≤1.4 at resampling the patient may be included).
Serum ALT >10 × upper limit of normal (ULN) at Screening.
Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation.
Total bilirubin >10 × ULN at Screening.
Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC.
Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases.
Any patient who is pregnant or lactating or who is planning to become pregnant within 72 weeks of the Screening/Inclusion Visit.
Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intrauterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter (from signed informed consent through 90 days after last dose of study drug).
Patient with a past medical history of alcohol or substance abuse will be excluded. Patient must agree to refrain from illicit drug and alcohol use during the study.
Administration of bile acid or lipid binding resins and medications that slow GI motility.
Patient has had investigational exposure to a drug, biologic agent, or medical device within 30 days prior to Screening, or 5 half-lives of the study agent, whichever is longer.
Any other conditions or abnormalities which, in the opinion of the investigator or Medical Monitor, may compromise the safety of the patient, or interfere with the patient participating in or completing the study.
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| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States | ||
| Children's Hospital Colorado |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37456676 | Derived | Thompson RJ, Artan R, Baumann U, Calvo PL, Czubkowski P, Dalgic B, D'Antiga L, Di Giorgio A, Durmaz O, Gonzales E, Grammatikopoulos T, Gupte G, Hardikar W, Houwen RHJ, Kamath BM, Karpen SJ, Lacaille F, Lachaux A, Lainka E, Loomes KM, Mack CL, Mattsson JP, McKiernan P, Ni Q, Ozen H, Rajwal SR, Roquelaure B, Shteyer E, Sokal E, Sokol RJ, Soufi N, Sturm E, Tessier ME, van der Woerd WL, Verkade HJ, Vittorio JM, Wallefors T, Warholic N, Yu Q, Horn P, Kjems L. Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis. JHEP Rep. 2023 Apr 29;5(8):100782. doi: 10.1016/j.jhepr.2023.100782. eCollection 2023 Aug. |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.
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Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.
Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).
This study consisted of a 72-week treatment period and a 4-week follow-up period. An optional extension period for continued treatment until commercial availability of odevixibat followed the 72-week treatment period. A total of 116 participants were enrolled in the study. Results are presented up to DCO of 15 February 2024.
This Phase III, open-label extension was conducted in participants with progressive familial intrahepatic cholestasis (PFIC) at 43 centers in 13 countries (Belgium, France, Germany, Italy, Netherlands, Poland, United Kingdom, United States [US], Australia, Canada, Israel, Saudi Arabia, and Turkey). The first participant was enrolled on 28 September 2018 and data cut-off (DCO) date was 15 February 2024.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1: Placebo/Odevixibat | Participants who previously received placebo in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 microgram/kilogram/day (mcg/kg/day) for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period (72 Weeks) |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 11, 2022 | Jan 23, 2025 |
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| Weeks 0-4, Weeks 0-12, Weeks 0-22, Weeks 0-24, Weeks 0-36, Weeks 0-46, Weeks 0-48, Weeks 0-60, and Weeks 0-70 |
| Change From Baseline in Serum Bile Acids at Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, and 72 | Blood samples for analysis of fasting total serum bile acids were drawn at specified timepoints. Participants were to fast (water intake only) for at least 4 hours prior to the collection of samples for serum bile acids. Exceptions were made for infants <12 months of age if unable to fast for the full 4 hours. Baseline for Cohort 1 placebo/odevixibat, and Cohort 2 groups was defined as the average of last 2 values before the first dose of study treatment in the study. Baseline for Cohort 1 odevixibat/odevixibat group was defined as average of last 2 values before the first dose of study treatment in study A4250-005 (NCT03566238). | Baseline and Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, and 72 |
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, 73-76 | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, 73-76 |
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (AM Score) | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. AM score represents night-time itching/scratching and sleep disturbance. | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 |
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (PM Score) | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. PM score represents daytime itching/scratching and tiredness. | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 |
| Percentage of Responders for Pruritus Assessments Bi-Weekly (AM and PM) | A responder is defined as a participant who reports a decrease in pruritus score from unrounded baseline equivalent to or greater than the threshold of meaningful change estimated from the blinded psychometric analysis. The averaged pruritus score was used to calculate the percentage of participants achieving meaningful reduction at specified Week against the thresholds value of 1.00 based on bi-weekly scores at specified Week obtained from blinded psychometric analysis across all anchors support a threshold of 1.0 point for AM, PM and AM and PM scratching scores. ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with score from 0 to 4 where 0 is no scratching and 4 is worst possible scratching. | Weeks 1-2, 3-4, 5-6, 7-8, 9-10, 11-12, 13-14, 15-16, 17-18, 19-20, 21-22, 23-24, 35-36, 47-48, 59-60, and 71-72 |
| Percentage of Responders for Pruritus Assessments Monthly (AM and PM) | A responder is defined as a participant who reports a decrease in pruritus score from unrounded baseline equivalent to or greater than the threshold of meaningful change estimated from the blinded psychometric analysis. The averaged pruritus score was used to calculate the percentage of participants achieving meaningful reduction at specified Week against the thresholds value of 1.00 based on monthly scores at specified Week obtained from blinded psychometric analysis across all anchors support a threshold of 1.0 point for AM, PM and AM and PM scratching scores. ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with score from 0 to 4 where 0 is no scratching and 4 is worst possible scratching. | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 45-48, 58-60, and 68-72 |
| Percentage of Participants Achieving a Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO (AM and PM) | The percentage of participants who achieved positive pruritus assessment for more than 50% of the time for Weeks 0-72 is reported. A positive pruritus assessment is defined as a scratching score of <=1 or at least a 1-point decrease from baseline on the Albireo ObsRO instrument based on rounded baseline and was calculated based on reported eDiary data. At each assessment, the AM score was compared to the baseline AM average, and the PM score was compared to the baseline PM average. All assessments after intercurrent events (premature treatment discontinuation, death, or initiation of rescue treatments such as biliary diversion surgery or liver transplantation) or follow-up assessments (>= last dose day + 15 days) were excluded from analysis. | Week 72 |
| Number of Participants Who Underwent Biliary Diversion Surgery and Liver Transplantation | Participants who underwent biliary diversion surgery and or liver transplantation data has been reported. | Baseline and Weeks 24, 48, and 72 |
| Change From Baseline in Height Z-Scores | Growth factors like height was measured by the standardized assessments outlined in the US food and drug administration (FDA) guidance document. Height was measured using certified stadiometer. Change in growth parameters was assessed using linear growth (height) 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 (WHO) website for participants with age <2 years old. Participants whose accurate age was not available, Z-score was not calculated. Baseline is the last available assessment prior to first dose of study treatment. A Z-score indicates how many standard deviation's (SD) a participant's measurement (like height), 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 and Weeks 24, 48, 70 and 72 |
| Change From Baseline in Weight Z-Scores | Growth factors like weight was measured by the standardized assessments outlined in the US FDA guidance document. Weight was measured using certified weight scale. Change in growth parameters was assessed using linear growth (weight) compared to standard growth curve (Z-score), calculated by using the software or methods from the CDC website for participants with age >=2 years old and from the WHO website for participants with age <2 years old. Participants whose accurate age was not available, Z-score was not calculated. Baseline is the last available assessment prior to the first dose of study treatment. The Z-score indicates how many SDs a participant's measurement (like weight), 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 the average a measurement was. | Baseline and Weeks 24, 48, 70 and 72 |
| Change From Baseline in Body Mass Index (BMI) Z-Scores | Growth factors like BMI was measured by the standardized assessments outlined in the US FDA guidance document. BMI was calculated by weight (kg) / height (m)^2. Change in growth parameters was assessed using linear growth (BMI) compared to standard growth curve (Z-score), calculated by using the software or methods from the CDC website for participants with age >=2 years old and from the WHO website for participants with age <2 years old. Participants whose accurate age was not available, Z-score was not calculated. Baseline is the last available assessment prior to the first dose of study treatment. The Z-score indicates how many SDs a participant's measurement (like 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 the average a measurement was. | Baseline and Weeks 24, 48, 70 and 72 |
| Number of Participants With Use of Ursodeoxycholic Acid (UDCA) and/or Rifampicin at Weeks 24, 48, and 72 | Data for the number of participants with use of UDCA and rifampicin are reported. | Weeks 24, 48, and 72 |
| Change From Baseline to Week 72 in Pediatric End-Stage Liver Disease (PELD) Score | The PELD score was calculated for children under 12 years of age, ranged across negative to positive values. The calculation of the PELD score was done by converting the laboratory parameters: total bilirubin in milligram/deciliter (mg/dL), albumin in gram (g)/dL, and creatinine in mg/dL laboratory parameters were converted to units. PELD score was calculated as 4.80*ln (total bilirubin)+18.57*ln [international normalized ratio (INR)] - 6.87*ln (albumin) + 4.36 (if participant <1 year: scores for participants listed for liver transplantation before the participant's first birthday continued to include the value assigned for age (<1 year) until the participant reached the age of 24 months) + 6.67 (if the participant has growth failure [<-2 standard deviation]). The laboratory values <1.0 were set to 1.0 for the calculation of the PELD score. Lower scores represent less severe hepatic disease. Baseline is the last available assessment prior to the first dose of study treatment. | Baseline and Week 72 |
| Change From Baseline to Week 72 in Model for End-stage Liver Disease (MELD) Score for Children 12 Years of Age or Older | The MELD score was calculated for children 12 years of age or older ranges from 6 to 40. The calculation of the MELD score was done by converting the laboratory parameters in the following units: total bilirubin in mg/dL, albumin in g/dL, and creatinine in mg/dL laboratory parameters were converted to units. MELD score for children 12 years of age or older ranges from 6 to 40 was calculated as 9.57*ln (creatinine) + 3.78*ln (total bilirubin) + 11.2 *ln (INR) + 6.43. Laboratory values <1.0 were set to 1.0 and serum creatinine values >4.0 mg/dL were set to 4.0 for calculation of the MELD score. Lower scores represent less severe hepatic disease. Baseline is the last available assessment prior to the first dose of study treatment. | Baseline and Week 72 |
| Change From Baseline to Week 72 in Aspartate Aminotransferase (AST) to Platelet Ratio Index (APRI) Score | AST to APRI score was calculated as [(AST in units per liter {U/L})/ (AST upper limit of normal {ULN} in U/L)] * 100/ (platelets in 10^9/L). The APRI score is a way to assess fibrosis of the liver. The lower the APRI score (< 0.5), the greater the negative predictive value and ability to rule out cirrhosis; the higher the value (> 1.5) the greater the positive predictive value and ability to rule in cirrhosis. Lower values indicate less severe hepatic fibrosis. Baseline is the last available assessment prior to the first dose of study treatment. | Baseline and Week 72 |
| Change From Baseline to Week 72 in Fibrosis-4 (Fib-4) Score | Fib-4 score was calculated as (age * AST in U/L)/ (platelets in 10^9/L *√ ( alanine aminotransferase [ALT] in U/L). The FIB-4 score estimates the amount of scarring in the liver. A FIB-4 score <1.45 has a negative predictive value of 90% for advanced fibrosis (Ishak fibrosis score 4-6 which includes early bridging fibrosis to cirrhosis). In contrast, a FIB-4 > 3.25 would have a 97% specificity and a positive predictive value of 65% for advanced fibrosis. Lower values indicate less severe hepatic fibrosis. Baseline is the last available assessment prior to the first dose of study treatment. | Baseline and Week 72 |
| Denver |
| Colorado |
| 80045 |
| United States |
| Emory University School of Medicine | Atlanta | Georgia | 30329 | United States |
| Riley Hospital for Children - Riley Children's Specialists | Indianapolis | Indiana | 46202 | United States |
| Johns Hopkins School of Medicine | Baltimore | Maryland | 21287 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| Columbia University Medical Center - Presbyterian Hospital Building | New York | New York | 10032 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Baylor College of Medicine - Texas Children's Liver Center | Houston | Texas | 77030 | United States |
| The Royal Children's Hospital | Melbourne | Australia |
| Cliniques Universitaires Saint-Luc | Woluwe-Saint-Lambert | Belgium |
| The Hospital for Sick Children | Toronto | Canada |
| University and Pediatric Hospital of Lyon | Bron | France |
| Universite Paris SUD - Hpitaux Universitaires Paris-Sud - Hopital Bicetre | Le Kremlin-Bicêtre | France |
| Hospital De La Timone | Marseille | France |
| Hospital Necker-Enfants Maladies | Paris | France |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Kinderklinik Tubingen, Universitatsklinikum Tubingen | Tübingen | Germany |
| Univesitatsklinikum Tubingen Klinik fur Kinder und Jugendmedizin | Tübingen | Germany |
| Shaare-Zedek Mc | Jerusalem | Israel |
| Schneider Children's Medical Center Of Israel | Petah Tikva | Israel |
| Azienda Ospedaliera Papa Giovanni XXIII | Bergamo | Italy |
| University Hospital Of Padova | Padova | Italy |
| Ospedale Regina Margherita | Torino | Italy |
| University Medical Center Groningen | Groningen | Netherlands |
| Universitair Medisch Centrum (UMC) Utrecht | Utrecht | Netherlands |
| Instytut Pomnik - Centrum Zarowia Dziecka | Warsaw | Poland |
| King Faisal Specialist Hospital & Research Centre | Riyadh | Saudi Arabia |
| Hospital Universitari Vall d'Hebron | Barcelona | Spain |
| Hospital Universitario La Paz | Madrid | Spain |
| Astrid Lindgren Children's Hospital, Karolinska University Hospital | Solna | Sweden |
| Gazi University | Ankara | Turkey (Türkiye) |
| Hacettepe University Faculty of Medicine | Ankara | Turkey (Türkiye) |
| Akdeniz University | Antalya | Turkey (Türkiye) |
| Istanbul University Medical Faculty | Istanbul | Turkey (Türkiye) |
| Inonu University Medical Faculty | Malatya | Turkey (Türkiye) |
| Birmingham Women's and Children's NHS Foundation Trust | Birmingham | United Kingdom |
| Leeds General Infirmary | Leeds | United Kingdom |
| Institute of Liver Studies - Kings College Hospital | London | United Kingdom |
| Cohort 1: Odevixibat/Odevixibat |
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| FG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
| COMPLETED |
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| NOT COMPLETED |
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| Optional Extension Treatment (176 Weeks) |
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The Full Analysis Set (FAS) consisted of all participants who received at least 1 dose of study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1: Placebo/Odevixibat | Participants who previously received placebo in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| BG001 | Cohort 1: Odevixibat/Odevixibat | Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| BG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants | No |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | Count of Participants | Participants | No |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Serum Bile Acids | Blood samples for analysis of fasting total serum bile acids were drawn at specified timepoints. Participants were to fast (water intake only) for at least 4 hours prior to the collection of samples for serum bile acids. Exceptions were made for infants <12 months of age if unable to fast for the full 4 hours. Baseline for Cohort 1 placebo/odevixibat and Cohort 2 groups was defined as the average of last 2 values before the first dose of study treatment in the study. Baseline for Cohort 1 odevixibat/odevixibat group was defined as average of last 2 values before the first dose of study treatment in study A4250-005 (NCT03566238). | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | micromole per liter (µmol/L) | Baseline and Week 72 |
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| Primary | Proportion of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at Baseline and Week 72 are reported. | Posted | Mean | Standard Deviation | proportion of pruritus-participant-level | Baseline and Week 72 |
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| Secondary | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | proportion of pruritus-participant-level | Weeks 0-4, Weeks 0-12, Weeks 0-22, Weeks 0-24, Weeks 0-36, Weeks 0-46, Weeks 0-48, Weeks 0-60, and Weeks 0-70 |
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| Secondary | Change From Baseline in Serum Bile Acids at Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, and 72 | Blood samples for analysis of fasting total serum bile acids were drawn at specified timepoints. Participants were to fast (water intake only) for at least 4 hours prior to the collection of samples for serum bile acids. Exceptions were made for infants <12 months of age if unable to fast for the full 4 hours. Baseline for Cohort 1 placebo/odevixibat, and Cohort 2 groups was defined as the average of last 2 values before the first dose of study treatment in the study. Baseline for Cohort 1 odevixibat/odevixibat group was defined as average of last 2 values before the first dose of study treatment in study A4250-005 (NCT03566238). | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | µmol/L | Baseline and Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, and 72 |
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| Secondary | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, 73-76 | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | proportion of pruritus-participant-level | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, 73-76 |
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| Secondary | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (AM Score) | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. AM score represents night-time itching/scratching and sleep disturbance. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | proportion of pruritus-participant-level | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 |
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| Secondary | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (PM Score) | A positive pruritus assessment was defined as a scratching score of <=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The proportion 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. PM score represents daytime itching/scratching and tiredness. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | proportion of pruritus-participant-level | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 |
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| Secondary | Percentage of Responders for Pruritus Assessments Bi-Weekly (AM and PM) | A responder is defined as a participant who reports a decrease in pruritus score from unrounded baseline equivalent to or greater than the threshold of meaningful change estimated from the blinded psychometric analysis. The averaged pruritus score was used to calculate the percentage of participants achieving meaningful reduction at specified Week against the thresholds value of 1.00 based on bi-weekly scores at specified Week obtained from blinded psychometric analysis across all anchors support a threshold of 1.0 point for AM, PM and AM and PM scratching scores. ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with score from 0 to 4 where 0 is no scratching and 4 is worst possible scratching. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Number | 95% Confidence Interval | percentage of participants | Weeks 1-2, 3-4, 5-6, 7-8, 9-10, 11-12, 13-14, 15-16, 17-18, 19-20, 21-22, 23-24, 35-36, 47-48, 59-60, and 71-72 |
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| Secondary | Percentage of Responders for Pruritus Assessments Monthly (AM and PM) | A responder is defined as a participant who reports a decrease in pruritus score from unrounded baseline equivalent to or greater than the threshold of meaningful change estimated from the blinded psychometric analysis. The averaged pruritus score was used to calculate the percentage of participants achieving meaningful reduction at specified Week against the thresholds value of 1.00 based on monthly scores at specified Week obtained from blinded psychometric analysis across all anchors support a threshold of 1.0 point for AM, PM and AM and PM scratching scores. ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with score from 0 to 4 where 0 is no scratching and 4 is worst possible scratching. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Number | 95% Confidence Interval | percentage of participants | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 45-48, 58-60, and 68-72 |
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| Secondary | Percentage of Participants Achieving a Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO (AM and PM) | The percentage of participants who achieved positive pruritus assessment for more than 50% of the time for Weeks 0-72 is reported. A positive pruritus assessment is defined as a scratching score of <=1 or at least a 1-point decrease from baseline on the Albireo ObsRO instrument based on rounded baseline and was calculated based on reported eDiary data. At each assessment, the AM score was compared to the baseline AM average, and the PM score was compared to the baseline PM average. All assessments after intercurrent events (premature treatment discontinuation, death, or initiation of rescue treatments such as biliary diversion surgery or liver transplantation) or follow-up assessments (>= last dose day + 15 days) were excluded from analysis. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with non-missing value when >50% were included. | Posted | Number | 95% Confidence Interval | percentage of participants | Week 72 |
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| Secondary | Number of Participants Who Underwent Biliary Diversion Surgery and Liver Transplantation | Participants who underwent biliary diversion surgery and or liver transplantation data has been reported. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. | Posted | Count of Participants | Participants | No | Baseline and Weeks 24, 48, and 72 |
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| Secondary | Change From Baseline in Height Z-Scores | Growth factors like height was measured by the standardized assessments outlined in the US food and drug administration (FDA) guidance document. Height was measured using certified stadiometer. Change in growth parameters was assessed using linear growth (height) 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 (WHO) website for participants with age <2 years old. Participants whose accurate age was not available, Z-score was not calculated. Baseline is the last available assessment prior to first dose of study treatment. A Z-score indicates how many standard deviation's (SD) a participant's measurement (like height), 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. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Median | Full Range | Z-score | Baseline and Weeks 24, 48, 70 and 72 |
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| Secondary | Change From Baseline in Weight Z-Scores | Growth factors like weight was measured by the standardized assessments outlined in the US FDA guidance document. Weight was measured using certified weight scale. Change in growth parameters was assessed using linear growth (weight) compared to standard growth curve (Z-score), calculated by using the software or methods from the CDC website for participants with age >=2 years old and from the WHO website for participants with age <2 years old. Participants whose accurate age was not available, Z-score was not calculated. Baseline is the last available assessment prior to the first dose of study treatment. The Z-score indicates how many SDs a participant's measurement (like weight), 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 the average a measurement was. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Median | Full Range | Z-score | Baseline and Weeks 24, 48, 70 and 72 |
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| Secondary | Change From Baseline in Body Mass Index (BMI) Z-Scores | Growth factors like BMI was measured by the standardized assessments outlined in the US FDA guidance document. BMI was calculated by weight (kg) / height (m)^2. Change in growth parameters was assessed using linear growth (BMI) compared to standard growth curve (Z-score), calculated by using the software or methods from the CDC website for participants with age >=2 years old and from the WHO website for participants with age <2 years old. Participants whose accurate age was not available, Z-score was not calculated. Baseline is the last available assessment prior to the first dose of study treatment. The Z-score indicates how many SDs a participant's measurement (like 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 the average a measurement was. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Median | Full Range | Z-score | Baseline and Weeks 24, 48, 70 and 72 |
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| Secondary | Number of Participants With Use of Ursodeoxycholic Acid (UDCA) and/or Rifampicin at Weeks 24, 48, and 72 | Data for the number of participants with use of UDCA and rifampicin are reported. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | Posted | Count of Participants | Participants | No | Weeks 24, 48, and 72 |
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| Secondary | Change From Baseline to Week 72 in Pediatric End-Stage Liver Disease (PELD) Score | The PELD score was calculated for children under 12 years of age, ranged across negative to positive values. The calculation of the PELD score was done by converting the laboratory parameters: total bilirubin in milligram/deciliter (mg/dL), albumin in gram (g)/dL, and creatinine in mg/dL laboratory parameters were converted to units. PELD score was calculated as 4.80*ln (total bilirubin)+18.57*ln [international normalized ratio (INR)] - 6.87*ln (albumin) + 4.36 (if participant <1 year: scores for participants listed for liver transplantation before the participant's first birthday continued to include the value assigned for age (<1 year) until the participant reached the age of 24 months) + 6.67 (if the participant has growth failure [<-2 standard deviation]). The laboratory values <1.0 were set to 1.0 for the calculation of the PELD score. Lower scores represent less severe hepatic disease. Baseline is the last available assessment prior to the first dose of study treatment. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at Baseline and Week 72 are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 72 |
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| Secondary | Change From Baseline to Week 72 in Model for End-stage Liver Disease (MELD) Score for Children 12 Years of Age or Older | The MELD score was calculated for children 12 years of age or older ranges from 6 to 40. The calculation of the MELD score was done by converting the laboratory parameters in the following units: total bilirubin in mg/dL, albumin in g/dL, and creatinine in mg/dL laboratory parameters were converted to units. MELD score for children 12 years of age or older ranges from 6 to 40 was calculated as 9.57*ln (creatinine) + 3.78*ln (total bilirubin) + 11.2 *ln (INR) + 6.43. Laboratory values <1.0 were set to 1.0 and serum creatinine values >4.0 mg/dL were set to 4.0 for calculation of the MELD score. Lower scores represent less severe hepatic disease. Baseline is the last available assessment prior to the first dose of study treatment. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants 12 years of age or older are included in this analysis and reported. For Cohort 1: Placebo/Odevixibat change from baseline data was not collected as participant was under 12 years old, that aligns with MELD score calculation. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 72 |
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| Secondary | Change From Baseline to Week 72 in Aspartate Aminotransferase (AST) to Platelet Ratio Index (APRI) Score | AST to APRI score was calculated as [(AST in units per liter {U/L})/ (AST upper limit of normal {ULN} in U/L)] * 100/ (platelets in 10^9/L). The APRI score is a way to assess fibrosis of the liver. The lower the APRI score (< 0.5), the greater the negative predictive value and ability to rule out cirrhosis; the higher the value (> 1.5) the greater the positive predictive value and ability to rule in cirrhosis. Lower values indicate less severe hepatic fibrosis. Baseline is the last available assessment prior to the first dose of study treatment. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at Baseline and Week 72 are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 72 |
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| Secondary | Change From Baseline to Week 72 in Fibrosis-4 (Fib-4) Score | Fib-4 score was calculated as (age * AST in U/L)/ (platelets in 10^9/L *√ ( alanine aminotransferase [ALT] in U/L). The FIB-4 score estimates the amount of scarring in the liver. A FIB-4 score <1.45 has a negative predictive value of 90% for advanced fibrosis (Ishak fibrosis score 4-6 which includes early bridging fibrosis to cirrhosis). In contrast, a FIB-4 > 3.25 would have a 97% specificity and a positive predictive value of 65% for advanced fibrosis. Lower values indicate less severe hepatic fibrosis. Baseline is the last available assessment prior to the first dose of study treatment. | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at Baseline and Week 72 are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 72 |
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Treatment emergent serious adverse event (TESAEs) and non-serious treatment emergent adverse event (TEAEs) were collected from the start of study treatment administration (Day 1) up to 28 days after the last dose of study treatment, approximately 248 weeks. All-cause mortality (death) was assessed from signing of the informed consent form till DCO, approximately 281 weeks.
The FAS consisted of all participants who had received at least 1 dose of the study treatment.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1: Placebo/Odevixibat | Participants who previously received placebo in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. | 0 | 19 | 5 | 19 | 18 | 19 |
| EG001 | Cohort 1: Odevixibat/Odevixibat | Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. | 0 | 37 | 7 | 37 | 35 | 37 |
| EG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. | 0 | 60 | 23 | 60 | 57 | 60 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Coagulopathy | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Progressive familial intrahepatic cholestasis | Congenital, familial and genetic disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Colitis ulcerative | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Enterocutaneous fistula | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Varices oesophageal | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cholestasis | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Chronic hepatic failure | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Pseudomonal sepsis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Septic arthritis streptococcal | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Fracture displacement | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Alpha 1 foetoprotein increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood sodium decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Device dislocation | Product Issues | MedDRA (26.0) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Coagulopathy | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Ear discomfort | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Ear haemorrhage | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Otorrhoea | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Keratoconus | Eye disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Diarrhoea haemorrhagic | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Faeces soft | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Tooth impacted | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Hepatosplenomegaly | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Boston exanthema | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Gastrointestinal candidiasis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Postoperative wound infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Accidental overdose | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Immunisation reaction | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Post vaccination fever | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Scar | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Bile acids increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Bilirubin conjugated increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood sodium increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Lymph node palpable | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Platelet count increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Vitamin A increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Vitamin D decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Vitamin E decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vitamin E deficiency | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Trigger finger | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dyslexia | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Autism spectrum disorder | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Tic | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Bladder dysfunction | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Albireo, An Ipsen Company | see email | clinical.trials@ipsen.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 4, 2024 | Jan 17, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| C535933 | Cholestasis, progressive familial intrahepatic 1 |
| D002779 | Cholestasis |
| ID | Term |
|---|---|
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000713258 | odevixibat |
Not provided
Not provided
Not provided
| Physician Decision |
|
| Transition to Commercial Drug |
|
| Other |
|
| Ongoing Optional Extension Period |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black/African American |
|
| Asian |
|
| Other |
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues.
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues.
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
|
|
| Cohort 1: Odevixibat/Odevixibat |
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
|
|
| OG001 |
| Cohort 1: Odevixibat/Odevixibat |
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| Cohort 1: Odevixibat/Odevixibat |
Participants who previously received odevixibat capsule orally at a dose of 40 or 120 mcg/kg/day in study A4250-005 (NCT03566238) for 24 weeks received odevixibat capsule orally at a dose of 120 mcg/kg/day for 72 weeks, unless they required down-titration to the 40 mcg/kg/day due to tolerability issues. |
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|
| OG002 | Cohort 2: Odevixibat | Participants any age with any type of PFIC who either did not meet eligibility criteria for study A4250-005 (NCT03566238) or were eligible for enrolment in A4250-005 (NCT03566238) after recruitment was complete received odevixibat at a dose of 120 μg/kg/day or following protocol amendment 6.0, initiated at a dose of 40 mcg/kg/day with the possibility to dose escalate to 120 mcg/kg/day after 12 weeks if there was no improvement in pruritus based on investigator judgement, up to 72 weeks. |
|
|