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Intercept made a business decision to terminate the study based on FDA's request for voluntary withdrawal of Ocaliva and the issuance of clinical hold on studies under US IND involving OCA.
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This study will evaluate the efficacy, safety and tolerability, as well as PK/PD of OCA in eligible pediatric participants with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterostomy). The double-blind period comprises of 2 phases: dose titration phase and age expansion treatment phase.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participants receiving OCA | Active Comparator | Participants will be randomized to receive OCA (starting at 1.5 milligrams [mg] adult equivalent dose [AED]) orally, with water, once daily. Dose will be titrated every 2 weeks in a stepwise manner for the first 6 weeks, starting at 1.5 mg AED and titrating through 3 mg AED to a maximum of 5 mg AED, as tolerated; a discussion with the Medical Monitor is encouraged when determining uptitration if considerable signs or symptoms have arisen. Following the 6-week dose titration phase, participants will continue at the tolerated dose for approximately 24 months in Age Expansion Treatment Phase. |
|
| Participants receiving Matching placebo | Placebo Comparator | Participants will be randomized to receive matching placebo orally, with water, once daily. Dose will be titrated every 2 weeks in a stepwise manner for the first 6 weeks, starting at 1.5 mg AED and titrating through 3 mg AED to a maximum of 5 mg AED, as tolerated; a discussion with the Medical Monitor is encouraged when determining uptitration if considerable signs or symptoms have arisen. Following the 6-week dose titration phase, participants will continue at the tolerated dose for approximately 24 months in Age Expansion Treatment Phase. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OCA | Drug | OCA will be administered. |
| |
| Matching Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Composite Liver-Related Clinical Events | Number of participants experiencing any of the following: All-cause death; Liver transplantation; Hospitalization (≥24 hours) for variceal bleeding, hepatic encephalopathy, or spontaneous bacterial peritonitis; Clinically relevant ascites requiring therapeutic paracentesis have been presented. No clinical outcome events were reported at the time of the study termination; hence It was not possible to conduct the planned primary efficacy analysis. | Up to Week 48 |
| Change From Baseline in Pediatric End-stage Liver Disease (PELD) Score | The Pediatric End-stage Liver Disease (PELD) score is a scale used to assess the severity of chronic liver disease in pediatric participants younger than 12 years of age. The total PELD score ranges from negative values to positive values, with no absolute minimum or maximum, with higher scores indicating more severe liver disease and greater urgency for liver transplantation. The score is calculated using total bilirubin, international normalized ratio (INR), albumin, age at listing, and growth failure. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening. | Baseline and up to Week 48 |
| Change From Baseline in Model of End-stage Liver Disease With Sodium (MELD-NA) Score | The Model of End-stage Liver Disease with Sodium (MELD-Na) score is a scale used to assess the severity of chronic liver disease in participants 12 years of age and older, ranging from 6 (less ill) to 40 (gravely ill). Higher scores indicate more severe liver disease and greater urgency for liver transplantation. The score is derived from total bilirubin, serum creatinine, INR, and serum sodium. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening. | Baseline and up to Week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Improvement in GGT and Direct Bilirubin (Composite Responder Endpoint) | Participants were considered responders if both of the following criteria were met at EOS: ≥40% reduction from baseline in Gamma Glutamyl Transferase (GGT), and ≥25% reduction from baseline in direct (conjugated) bilirubin. Participants with missing values were considered non-responders. | Up to Week 48 |
Not provided
Inclusion criteria:
Exclusion criteria:
Prior liver transplant or active status on transplant list.
Participants diagnosed with biliary atresia splenic malformation (BASM).
Conjugated (direct) bilirubin ≥ upper limit of normal (ULN) of site-specific reference range. If conjugated bilirubin is not available: total bilirubin ≥2 mg/dL (34.2 mol/L).
Platelets <120,000/μL
International normalized ratio (INR) ≥1.5.
Current or history of complications of decompensated chronic liver disease including:
Height and weight Z-score <-2 per site-specific reference ranges.
Acholic (pale) stools.
Aspartate aminotransferase (AST) >4x ULN.
Alanine aminotransferase >4x ULN
GGT >500 Units per Liter (U/L)
On anticoagulation therapy
Albumin <3.5 grams per deciliter (g/dL).
Inability to swallow tablets (i.e., tablet or mini-tablet formulations).
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| Name | Affiliation | Role |
|---|---|---|
| Lynda Szczech, MD | Intercept Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Queensland Childrens Hospital | South Brisbane | Queensland | 4101 | Australia | ||
| Women's and Children's Hospital |
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A total of 28 participants were enrolled.
This was a global, multicenter, double-blind, placebo-controlled study that evaluated efficacy, safety, and tolerability as well as pharmacokinetic/pharmacodynamic of Obeticholic Acid (OCA) in pediatric participants with biliary atresia with successful Hepatoportoenterostomy (HPE).
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| ID | Title | Description |
|---|---|---|
| FG000 | Obeticholic Acid | Participants were randomized to receive OCA 1.5 milligrams (mg) orally, once daily. The dose was titrated every 2 weeks to 3 mg, to a maximum of 5 mg, as tolerated. Following the 6-week dose titration phase, participants continued at the tolerated dose for approximately 24 months in the Age Expansion Treatment Phase. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Dose Titration Phase (Up to 6 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 | Sep 14, 2023 | Apr 7, 2026 |
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| Drug |
Matching Placebo will be administered. |
|
| Change From Baseline in GGT | Blood samples were collected to assess GGT levels. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Baseline and up to Week 48 |
| Change From Baseline in Total and Direct (Conjugated) Bilirubin | Blood samples were collected to assess direct bilirubin levels. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Baseline and up to Week 48 |
| Change From Baseline in Endogenous Bile Acids | Plasma samples were collected to assess endogenous bile acids. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Baseline and up to Week 48 |
| Change From Baseline in Liver Stiffness as Assessed by Transient Elastography | Liver stiffness was measured using ultrasound elastography. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Baseline and up to Week 48 |
| Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | TEAEs were defined as adverse events that were reported or worsened on or after the first dose of study treatment. Serious adverse events are adverse events resulting in death, are immediately life threatening, requires in-patient hospitalization or prolongation of existing hospitalization, or results in persistent or significant disability/incapacity. | Up to Week 48 |
| Change From Baseline in Plasma Levels of Fat-Soluble Vitamin D | Plasma samples were collected to assess levels of fat-soluble vitamins D. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Baseline and Week 48 |
| Change From Baseline in Plasma Levels of Fat-Soluble Vitamin K | Plasma samples were collected to assess levels of fat-soluble vitamin K. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Baseline and Week 48 |
| North Adelaide |
| South Australia |
| 5006 |
| Australia |
| Royal Childrens Hospital | Parkville | Victoria | 3104 | Australia |
| Alberta Childrens Hospital | Calgary | Alberta | T3B 6A8 | Canada |
| Stollery Children's Hospital | Edmonton | Alberta | Canada |
| Children's Hospital, Zhejiang University School of Medicine | Hangzhou | Zhejiang | 310006 | China |
| Guangzhou Women And Childrens Medical Center | Guangzhou | China |
| Children's Hospital of Fudan University | Shanghai | China |
| Childrens Hospital of Shanghai | Shanghai | China |
| Children's Hospital of Shanxi | Taiyuan | China |
| Queen Mary Hospital | Hong Kong | Hong Kong |
| Hadassah Medical Center | Jerusalem | Israel |
| Shaare-Zedek Medical Center | Jerusalem | Israel |
| Hospital Raja Perempuan Azinab II | Kota Bharu | Kelantan | 15586 | Malaysia |
| University Malaya Medical Center | Kuala Lumpur | 59100 | Malaysia |
| Starship Child Health | Auckland | 1142 | New Zealand |
| KK Women's and Children's Hospital | Singapore | Singapore |
| Taichung Veterans General Hospital | Taichung | Taiwan |
| National Chen Kung University Hospital | Tainan | Taiwan |
| National Taiwan University Hospital | Taipei | Taiwan |
| Linkou Chang Gung Memorial Hospital | Taoyuan | Taiwan |
| Akdeniz Üniversitesi Tıp Fakültesi Hastanesi Pediatrik Gastroenteroloji | Konyaalti | Antalya | 07050 | Turkey (Türkiye) |
| Ege Üniversitesi Hastanesi Pediatrik Gastroenteroloji Bölümü | Bornova | İzmir | 35100 | Turkey (Türkiye) |
| Hacettepe Universitesi ihsan Dogramaci Cocuk Hastansesi | Ankara | 06230 | Turkey (Türkiye) |
| FG001 |
| Placebo |
Participants were randomized to receive matching placebo orally, once daily. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Age Expansion Phase (Up to 24 Months) |
|
|
The intent-to-treat (ITT) Population included all randomized participants.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Obeticholic Acid (OCA) | Participants were randomized to receive OCA 1.5 milligrams (mg) orally, once daily. The dose was titrated every 2 weeks to 3 mg, to a maximum of 5 mg, as tolerated. Following the 6-week dose titration phase, participants continued at the tolerated dose for approximately 24 months in the Age Expansion Treatment Phase. |
| BG001 | Placebo | Participants were randomized to receive matching placebo orally, once daily. |
| BG002 | 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 |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | Number of Participants With Composite Liver-Related Clinical Events | Number of participants experiencing any of the following: All-cause death; Liver transplantation; Hospitalization (≥24 hours) for variceal bleeding, hepatic encephalopathy, or spontaneous bacterial peritonitis; Clinically relevant ascites requiring therapeutic paracentesis have been presented. No clinical outcome events were reported at the time of the study termination; hence It was not possible to conduct the planned primary efficacy analysis. | ITT Population | Posted | Count of Participants | Participants | Up to Week 48 |
|
|
| |||||||||||||||||||||||||||||
| Primary | Change From Baseline in Pediatric End-stage Liver Disease (PELD) Score | The Pediatric End-stage Liver Disease (PELD) score is a scale used to assess the severity of chronic liver disease in pediatric participants younger than 12 years of age. The total PELD score ranges from negative values to positive values, with no absolute minimum or maximum, with higher scores indicating more severe liver disease and greater urgency for liver transplantation. The score is calculated using total bilirubin, international normalized ratio (INR), albumin, age at listing, and growth failure. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening. | ITT Population. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | score on a scale | Baseline and up to Week 48 |
| ||||||||||||||||||||||||||||||
| Primary | Change From Baseline in Model of End-stage Liver Disease With Sodium (MELD-NA) Score | The Model of End-stage Liver Disease with Sodium (MELD-Na) score is a scale used to assess the severity of chronic liver disease in participants 12 years of age and older, ranging from 6 (less ill) to 40 (gravely ill). Higher scores indicate more severe liver disease and greater urgency for liver transplantation. The score is derived from total bilirubin, serum creatinine, INR, and serum sodium. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening. | ITT Population. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | score on a scale | Baseline and up to Week 48 |
| ||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Improvement in GGT and Direct Bilirubin (Composite Responder Endpoint) | Participants were considered responders if both of the following criteria were met at EOS: ≥40% reduction from baseline in Gamma Glutamyl Transferase (GGT), and ≥25% reduction from baseline in direct (conjugated) bilirubin. Participants with missing values were considered non-responders. | ITT Population | Posted | Number | percentage of participants | Up to Week 48 |
|
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in GGT | Blood samples were collected to assess GGT levels. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Safety Population, which included all participants who received at least one dose of study drug. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | Units/liter (U/L) | Baseline and up to Week 48 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Total and Direct (Conjugated) Bilirubin | Blood samples were collected to assess direct bilirubin levels. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Safety Population. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | micromol/liter (mcmol/L) | Baseline and up to Week 48 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Endogenous Bile Acids | Plasma samples were collected to assess endogenous bile acids. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | Safety Population. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | micromol/liter (mcmol/L) | Baseline and up to Week 48 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Liver Stiffness as Assessed by Transient Elastography | Liver stiffness was measured using ultrasound elastography. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | ITT Population. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | kilopascals (kPa) | Baseline and up to Week 48 |
|
| |||||||||||||||||||||||||||||
| Secondary | Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | TEAEs were defined as adverse events that were reported or worsened on or after the first dose of study treatment. Serious adverse events are adverse events resulting in death, are immediately life threatening, requires in-patient hospitalization or prolongation of existing hospitalization, or results in persistent or significant disability/incapacity. | Safety Population | Posted | Count of Participants | Participants | Up to Week 48 |
|
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Plasma Levels of Fat-Soluble Vitamin D | Plasma samples were collected to assess levels of fat-soluble vitamins D. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | ITT population. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | nanomoles/L (nmol/L) | Baseline and Week 48 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Plasma Levels of Fat-Soluble Vitamin K | Plasma samples were collected to assess levels of fat-soluble vitamin K. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement. | ITT population. Only those participants with data available at specified time points have been presented. | Posted | Mean | Standard Deviation | nanograms/millilitres (ng/mL) | Baseline and Week 48 |
|
|
Up to Week 48
Treatment emergent adverse events (TEAEs) and serious TEAEs were collected in Safety Population, which included all participants who received at least one dose of study drug.
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 | Obeticholic Acid (OCA) | Participants were randomized to receive OCA 1.5 milligrams (mg) orally, once daily. The dose was titrated every 2 weeks to 3 mg, to a maximum of 5 mg, as tolerated. Following the 6-week dose titration phase, participants continued at the tolerated dose for approximately 24 months in the Age Expansion Treatment Phase. | 0 | 14 | 3 | 14 | 13 | 14 |
| EG001 | Placebo | Participants were randomized to receive matching placebo orally, once daily. | 0 | 14 | 0 | 14 | 11 | 14 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infected aural fistula | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Body tinea | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Conjunctivitis bacterial | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Infected aural fistula | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Laryngopharyngitis | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Scarlet fever | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Vulval cellulitis | Infections and infestations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Frequent bowel movements | Gastrointestinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Liver function test increased | Investigations | MedDRA 28.1 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Dacryostenosis acquired | Eye disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Eyelids pruritus | Eye disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 28.1 | Non-systematic Assessment |
| |
| Incorrect dose administered | Injury, poisoning and procedural complications | MedDRA 28.1 | Non-systematic Assessment |
| |
| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA 28.1 | Non-systematic Assessment |
| |
| Traumatic pain | Injury, poisoning and procedural complications | MedDRA 28.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Suspected drug-induced liver injury | Hepatobiliary disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 28.1 | Non-systematic Assessment |
| |
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 28.1 | Non-systematic Assessment |
| |
| Aggression | Psychiatric disorders | MedDRA 28.1 | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Intercept Pharmaceuticals, Inc. | 844-782-4278 | medinfo@interceptpharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 21, 2025 | Apr 7, 2026 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D001656 | Biliary Atresia |
| ID | Term |
|---|---|
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D004065 | Digestive System Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
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