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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-000482-36 | EudraCT Number |
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The study is a randomized, double-blind, placebo-controlled, multicenter study. It is a 13-week Phase 2 study in adults with primary biliary cirrhosis designed to compare the effect of daily dosing with UDCA in combination with LUM001 or placebo.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LUM001 and Ursodeoxycholic Acid (UDCA) | Experimental | Administered orally once daily |
|
| Placebo and Ursodeoxycholic Acid (UDCA) | Placebo Comparator | Administered orally once daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LUM001 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET) | Pruritus was assessed using ItchRO measure, administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). (ItchRO) scores ranged from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit. | Baseline and Week 13/ET |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13 | ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the product. A serious adverse event (SAE) was defined as an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly or birth defect; an important medical event that did not meet any of the above criteria but jeopardized the participant or required medical or surgical intervention to prevent one of the outcomes listed above. A TEAE was defined as any AE that occurred during the study, from the start of investigational product dosing through the end of the study (13 weeks of treatment period (or ET) + 14 days ]), or that worsened since the start of dosing. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Mirum | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Scripps Clinic | La Jolla | California | 92037 | United States | ||
| University of California at Davis |
Not provided
| Label | URL |
|---|---|
| Related Info | View source |
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A total of 87 participants were screened out of which 66 participants were randomized into the study and the remaining 21 were screen failures.
The study was conducted in 24 centers in the United Kingdom, Canada, and the United States between 19 August 2013 and 09 April 2015.
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| ID | Title | Description |
|---|---|---|
| FG000 | LUM001 10 mg + UDCA (Cohort A) | In Cohort A, participants received LUM001 tablet in combination with ursodeoxycholic acid (UDCA) orally once daily at a dosage of 2.5 up to a maximum of 10 milligram (mg) during the dose-escalation period over a 3 week period. Thereafter, participants received LUM001 10 mg tablet along with one placebo matched to LUM001 orally once daily for another 10 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
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Not provided
|
| Ursodeoxycholic Acid | Drug |
|
|
| Baseline, Weeks 4, 8 and 13 |
| Change From Baseline in Pruritus Using Adult ItchRO Average Daily Scores at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) | ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. Adult ItchRO average daily score was the sum of daily scores divided by the number of days adult ItchRO was completed, using the 7 days prior to the reported visit date. | Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) |
| Change From Baseline in Alkaline Phosphatase (ALP) at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) | Laboratory serum ALP enzyme levels were evaluated using blood samples collected. | Baseline, Weeks 4, 8, 13 and Last Post-baseline (Week 13/ET) |
| Change From Baseline in 5-D Itch Score at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) | The 5-D itch (validated instrument to measure pruritus) scale was developed for the multidimensional quantification of pruritus that is sensitive to change over time. The 5-D itch scale included 5 domains (duration, degree, direction, disability, and distribution of pruritus). The total 5-D score was obtained by scoring each of the domains separately and then summing them together. 5-D total scores ranged between 5 (no pruritus) and 25 (most severe pruritus). | Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) |
| Change From Baseline in Fasting Serum Bile Acid Level at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) | Laboratory serum bile acid level levels were evaluated using blood samples collected. | Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) |
| Change From Baseline in Bile Acid Synthesis as Measured by Serum 7 Alpha-Hydroxy-4-Cholesten-3-One C4 Level [7 Alpha C4]) at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) | C4 7 alpha-hydroxy-4-cholesten-3-one is an intermediate in the biochemical synthesis of bile acids from cholesterol and its concentrations reflect the activity of the bile acid synthetic pathway. Elevated levels of C4 indicate bile acid malabsorption. Laboratory C4 levels were evaluated using blood samples collected. | Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET) |
| From the first dose of study drug until the 13 weeks of treatment period (or ET) + 14 days (approximately 15 weeks) |
| Sacramento |
| California |
| 95817 |
| United States |
| University of Miami | Miami | Florida | 33136 | United States |
| University of Chicago Medical Center | Chicago | Illinois | 60637 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| University of Louisville | Louisville | Kentucky | 40202 | United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Minnesota Gastroenterology | Saint Paul | Minnesota | 55114 | United States |
| St. Louis University | St Louis | Missouri | 63104 | United States |
| Weill Cornell Medical College | New York | New York | 10021 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Advanced Liver Therapies at St. Lukes Episcopal Hospital | Houston | Texas | 77030 | United States |
| University of Utah Health Science Center | Salt Lake City | Utah | 84132 | United States |
| Liver Institute of Virginia | Newport News | Virginia | 23602 | United States |
| Hunter Holmes McGuire VA Medical Center | Richmond | Virginia | 23249 | United States |
| University of Washington Harborview Medical Center | Seattle | Washington | 98104 | United States |
| University Health Network, Toronto Western Hospital | Toronto | Ontario | M5T 2S8 | Canada |
| University of Birmingham | Birmingham | England | B15 2TT | United Kingdom |
| Royal Liverpool & Broadgreen University Hospital | Liverpool | England | L7 8XP | United Kingdom |
| Newcastle University | Newcastle upon Tyne | England | NE1 4LP | United Kingdom |
| Oxford University Hospitals (John Radcliffe) | Oxford | England | OX3 9DU | United Kingdom |
| Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Imperial College London St Mary's Hospital | London | W2 1NY | United Kingdom |
| FG001 | LUM001 20 mg + UDCA (Cohort B) | In Cohort B, participants received LUM001 tablets in combination with UDCA orally once daily at a dosage of 2.5 mg up to a maximum of 20 mg during the dose-escalation period over a 4 week period. Thereafter, participants received LUM001 20 mg (2x10 mg) tablet orally once daily for another 9 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| FG002 | Placebo + UDCA (Cohort A) | In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| FG003 | Placebo + UDCA (Cohort B) | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | LUM001 10 mg + UDCA (Cohort A) | In Cohort A, participants received LUM001 tablet in combination with ursodeoxycholic acid (UDCA) orally once daily at a dosage of 2.5 up to a maximum of 10 milligram (mg) during the dose-escalation period over a 3 week period. Thereafter, participants received LUM001 10 mg tablet along with one placebo matched to LUM001 orally once daily for another 10 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| BG001 | LUM001 20 mg + UDCA (Cohort B) | In Cohort B, participants received LUM001 tablets in combination with UDCA orally once daily at a dosage of 2.5 mg up to a maximum of 20 mg during the dose-escalation period over a 4 week period. Thereafter, participants received LUM001 20 mg (2x10 mg) tablet orally once daily for another 9 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| BG002 | Placebo + UDCA (Cohort A) | In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| BG003 | Placebo + UDCA (Cohort B) | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| BG004 | 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 |
|
| 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 Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET) | Pruritus was assessed using ItchRO measure, administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). (ItchRO) scores ranged from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit. | The mITT population included all participants who were randomized, received at least 1 dose of treatment, and had at least 1 post-baseline ItchRO assessment. | Posted | Mean | Standard Deviation | units on a scale | Baseline and Week 13/ET |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13 | ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit. | mITT Population. Here, "n" signifies the number of participants evaluable for the respective time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 4, 8 and 13 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pruritus Using Adult ItchRO Average Daily Scores at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) | ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. Adult ItchRO average daily score was the sum of daily scores divided by the number of days adult ItchRO was completed, using the 7 days prior to the reported visit date. | mITT Population. Here, "n" signifies the number of participants evaluable for the respective time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Alkaline Phosphatase (ALP) at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) | Laboratory serum ALP enzyme levels were evaluated using blood samples collected. | mITT Population. Here, "n" signifies the number of participants evaluable for the respective time points. | Posted | Mean | Standard Deviation | units per liter (U/L) | Baseline, Weeks 4, 8, 13 and Last Post-baseline (Week 13/ET) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in 5-D Itch Score at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) | The 5-D itch (validated instrument to measure pruritus) scale was developed for the multidimensional quantification of pruritus that is sensitive to change over time. The 5-D itch scale included 5 domains (duration, degree, direction, disability, and distribution of pruritus). The total 5-D score was obtained by scoring each of the domains separately and then summing them together. 5-D total scores ranged between 5 (no pruritus) and 25 (most severe pruritus). | mITT Population. Here, "n" signifies the number of participants evaluable for the respective time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Fasting Serum Bile Acid Level at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) | Laboratory serum bile acid level levels were evaluated using blood samples collected. | mITT Population. Here, "n" signifies the number of participants evaluable for the respective time points. | Posted | Mean | Standard Deviation | micromoles per liter | Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Bile Acid Synthesis as Measured by Serum 7 Alpha-Hydroxy-4-Cholesten-3-One C4 Level [7 Alpha C4]) at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) | C4 7 alpha-hydroxy-4-cholesten-3-one is an intermediate in the biochemical synthesis of bile acids from cholesterol and its concentrations reflect the activity of the bile acid synthetic pathway. Elevated levels of C4 indicate bile acid malabsorption. Laboratory C4 levels were evaluated using blood samples collected. | mITT Population. Here, "n" signifies the number of participants evaluable for the respective time points. | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the product. A serious adverse event (SAE) was defined as an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly or birth defect; an important medical event that did not meet any of the above criteria but jeopardized the participant or required medical or surgical intervention to prevent one of the outcomes listed above. A TEAE was defined as any AE that occurred during the study, from the start of investigational product dosing through the end of the study (13 weeks of treatment period (or ET) + 14 days ]), or that worsened since the start of dosing. | The Safety Population included all participants who were randomized and received at least 1 dose of the study drug. One participant was randomized to LUM001 10 mg, but was down-titrated to 5 mg dose due to tolerability issues. The safety data has been summarized based on the study dose actually received by the participant. | Posted | Number | participants | From the first dose of study drug until the 13 weeks of treatment period (or ET) + 14 days (approximately 15 weeks) |
|
From the first dose of study drug until the 13 weeks of treatment period (or ET) + 14 days (approximately 15 weeks)
One subject was randomized to LUM001 10 mg, but was down-titrated to 5mg dose due to tolerability issues. One participant was randomized to LUM001 10 mg, but was down-titrated to 5 mg dose due to tolerability issues. The safety data has been summarized based on the study dose actually received by the participant.
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 | LUM001 5 mg + UDCA | Participants received LUM001 5 mg tablet orally once daily for a period of 13 weeks in combination with UDCA. | 0 | 1 | 1 | 1 | ||
| EG001 | LUM001 10 mg + UDCA | Participants received LUM001 10 mg tablet orally once daily for a period of 13 weeks in combination with UDCA. | 2 | 20 | 18 | 20 | ||
| EG002 | LUM001 20 mg + UDCA | Participants received LUM001 20 mg (2x 10 mg) tablet for 20 mg daily dose in combination with UDCA orally once daily for a period of 13 weeks. | 1 | 21 | 20 | 21 | ||
| EG003 | Placebo + UDCA | Participants received placebo matched to LUM001 tablet orally once daily for a period of 13 weeks in combination with UDCA. | 0 | 24 | 16 | 24 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Myocardial Infarction | Cardiac disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Gastrointestinal Haemorrhage | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pancytopenia | Blood and lymphatic system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Dry Eye | Eye disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Abdominal Distension | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Dry Mouth | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Faeces Discoloured | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Gastrooesophageal Reflux Disease | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Gingival Pain | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Mouth Ulceration | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Chills | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Influenza Like Illness | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Anal Injury | Injury, poisoning and procedural complications | MedDRA (16.0) | Non-systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA (16.0) | Non-systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Muscular Weakness | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Musculoskeletal Chest Pain | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Vaginal Discharge | Reproductive system and breast disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Laryngeal Inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pharyngeal Erythema | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Sinus Operation | Surgical and medical procedures | MedDRA (16.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
|
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Physician | Mirum | 1-650-667-4085 | medinfo@mirumpharma.com |
| ID | Term |
|---|---|
| D008105 | Liver Cirrhosis, Biliary |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D008107 | Liver Diseases |
| D008103 | Liver Cirrhosis |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D014580 | Ursodeoxycholic Acid |
| ID | Term |
|---|---|
| D003840 | Deoxycholic Acid |
| D002793 | Cholic Acids |
| D001647 | Bile Acids and Salts |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D002757 | Cholanes |
Not provided
Not provided
| Male |
|
| Change at Week 13/ET |
|
| The difference between treatment groups in change from Baseline to Week 13/ET in ItchRO weekly sum score was evaluated by ANCOVA using a GLM. The model included terms for treatment group, ALP level (strata), treatment group by ALP level interaction, and Baseline ItchRO weekly sum score as a covariate. Least squares mean change from Baseline to Week 13/ET, along with 95% confidence interval for the mean, were presented. | ANCOVA | 0.4380 | p-value (LUM001 LS Mean = Placebo LS Mean). | Least squares mean difference | -3.99 | 2-Sided | 95 | -14.20 | 6.23 | Superiority or Other |
| OG002 | Placebo + UDCA (Cohort A) | In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| OG003 | Placebo + UDCA (Cohort B) | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
|
|
| OG002 | Placebo + UDCA (Cohort A) | In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| OG003 | Placebo + UDCA (Cohort B) | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
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| OG002 |
| Placebo + UDCA (Cohort A) |
In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| OG003 | Placebo + UDCA (Cohort B) | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
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|
| OG002 | Placebo + UDCA (Cohort A) | In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| OG003 | Placebo + UDCA (Cohort B | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
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|
| OG002 |
| Placebo + UDCA (Cohort A) |
In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| OG003 | Placebo + UDCA (Cohort B) | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
|
|
| OG002 | Placebo + UDCA (Cohort A) | In Cohort A, participants received placebo (matched to LUM001) once daily for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
| OG003 | Placebo + UDCA (Cohort B) | In Cohort B, participants received placebo (matched to LUM001) for a period of 13 weeks, in combination with UDCA. Participants continued UDCA alone for an additional period of 4 weeks. |
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| OG001 | LUM001 10 mg + UDCA | Participants received LUM001 10 mg tablet orally once daily for a period of 13 weeks in combination with UDCA. |
| OG002 | LUM001 20 mg + UDCA | Participants received LUM001 20 mg (2x 10 mg) tablet for 20 mg daily dose in combination with UDCA orally once daily for a period of 13 weeks. |
| OG003 | Placebo + UDCA | Participants received placebo matched to LUM001 tablet orally once daily for a period of 13 weeks in combination with UDCA. |
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