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Trial record 32 of 92 for:    Primary Sclerosing Cholangitis

Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis (CAMEO)

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ClinicalTrials.gov Identifier: NCT02061540
Recruitment Status : Completed
First Posted : February 13, 2014
Results First Posted : April 12, 2017
Last Update Posted : March 29, 2019
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Primary Sclerosing Cholangitis (PSC)
Intervention Drug: LUM001
Enrollment 27
Recruitment Details The study was conducted in 8 centers in Great Britain, Canada, and the United States between 22 April 2014 and 12 February 2016.
Pre-assignment Details A total of 37 participants were screened, of them 27 participants were enrolled in the study.
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
Period Title: Overall Study
Started 27
Down-Titrated on LUM001 During the Study 5
Completed 23
Not Completed 4
Reason Not Completed
Adverse Event             2
Withdrawal by Subject             2
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
Overall Number of Baseline Participants 27
Hide Baseline Analysis Population Description
Safety population included all participants who received at least 1 dose of the investigational product.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 27 participants
43.7  (11.35)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
Female
9
  33.3%
Male
18
  66.7%
1.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Hide Description An Adverse Event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered to be related to the investigational drug product. TEAEs were AEs with a start date on or after the first dose of investigational product and started prior to the last dose of investigational product plus 14 days.
Time Frame From start of study drug administration until Week 18
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included all participants who received at least 1 dose of the investigational product.
Arm/Group Title Maralixibat (LUM001) 1 mg Maralixibat (LUM001) 2.5 mg Maralixibat (LUM001) 5 mg Maralixibat (LUM001) 7.5 mg Maralixibat (LUM001) 10 mg
Hide Arm/Group Description:
Participants received LUM001 tablet orally once daily at a dose of 1 mg during Week 2 of the treatment period.
Participants received LUM001 tablet orally once daily at a dose of 2.5 mg during Week 3 of the treatment period.
Participants received LUM001 tablet orally once daily at a dose of 5 mg during Week 4 of the treatment period.
Participants received LUM001 tablet orally once daily at a dose of 7.5 mg during Week 5 of the treatment period.
Participants received LUM001 tablet orally once daily at a dose of 10 mg during Week 6 of the treatment period followed by stable dosing of 10 mg for 8 weeks.
Overall Number of Participants Analyzed 1 2 2 1 21
Measure Type: Number
Unit of Measure: participant
1 2 2 1 19
2.Primary Outcome
Title Change From Baseline in Fasting Serum Bile Acid Level at Week 14
Hide Description Serum bile acid levels were evaluated using blood samples collected.
Time Frame Baseline, Week 14
Hide Outcome Measure Data
Hide Analysis Population Description
Modified intent-to-treat (mITT) population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment.
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description:
Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: micromoles per liter
Baseline 38.941  (38.6614)
Change From Baseline -14.841  (31.3709)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Maralixibat (LUM001)
Comments Analysis was performed to compare baseline and endpoint data.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0043
Comments [Not Specified]
Method Wilcoxon's signed rank test
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -14.841
Confidence Interval (2-Sided) 95%
-27.251 to -2.432
Parameter Dispersion
Type: Standard Error of the Mean
Value: 6.0373
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in Liver Enzyme Levels in Serum
Hide Description Levels of liver enzymes such as Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (ALP) in serum were evaluated.
Time Frame Baseline, Week 14
Hide Outcome Measure Data
Hide Analysis Population Description
mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment.
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description:
Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: units per liter (U/L)
ALT: Baseline 108.5  (78.95)
ALT: Change From Baseline 10.5  (63.07)
AST: Baseline 88.3  (43.70)
AST: Change From Baseline 11.7  (34.14)
ALP: Baseline 471.6  (316.93)
ALP: Change From Baseline 36.7  (170.87)
4.Secondary Outcome
Title Change From Baseline in Bilirubin Levels at Week 14
Hide Description Total Bilirubin and Direct (Conjugated) Bilirubin levels were evaluated.
Time Frame Baseline, Week 14
Hide Outcome Measure Data
Hide Analysis Population Description
mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment.
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description:
Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: milligram per deciliter (mg/dL)
Total Bilirubin Level: Baseline 1.22  (0.775)
Total Bilirubin Level: Change From Baseline 0.24  (0.666)
Conjugated Bilirubin Level: Baseline 0.60  (0.510)
Conjugated Bilirubin Level: Change From Baseline 0.19  (0.450)
5.Secondary Outcome
Title Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score
Hide Description The Adult ItchRO instrument was completed twice daily using an electronic diary (eDiary). Each morning and evening score had a range from 0-10, with the higher score indicating increasing itch severity. The following was used for assessing the Adult ItchRO daily score: The score which represented the most severe itching for the day (morning or evening) was taken for each day as the daily score (maximum daily score of 10); If only 1 of the 2 scores was available for the day, the score that was available was used as the daily score; If both the morning and the evening scores were missing, the score was considered missing for the day.
Time Frame Baseline, Week 14
Hide Outcome Measure Data
Hide Analysis Population Description
mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment.
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description:
Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: units on scale
Baseline 15.00  (18.735)
Change From Baseline -7.67  (16.326)
6.Other Pre-specified Outcome
Title Change From Baseline for Other Biochemical Markers of Cholestasis: Total Cholesterol, Low Density Lipoprotein Cholesterol
Hide Description Total cholesterol (TC) level and low density lipoprotein cholesterol (LDLC) level were considered as biochemical markers of cholestasis.
Time Frame Baseline, Week 14
Hide Outcome Measure Data
Hide Analysis Population Description
mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment.
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description:
Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: mg/dL
TC Level: Baseline 213.0  (50.62)
TC Level: Change From Baseline -21.2  (25.46)
LDLC Level: Baseline 121.4  (40.52)
LDLC Level: Change From Baseline -16.3  (17.64)
Time Frame From start of study drug administration up to follow up (Week 18)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Maralixibat (LUM001)
Hide Arm/Group Description Participants received LUM001 tablet orally once daily at a dose of 0.5 milligram (mg) during Week 1; 1 mg during Week 2; 2.5 mg during Week 3; 5 mg during Week 4; 7.5 mg during Week 5; 10 mg during Week 6 followed by stable dosing of 10 mg for 8 weeks.
All-Cause Mortality
Maralixibat (LUM001)
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Maralixibat (LUM001)
Affected / at Risk (%) # Events
Total   4/27 (14.81%)    
Gastrointestinal disorders   
Melaena * 1  1/27 (3.70%)  1
Upper gastrointestinal haemorrhage * 1  1/27 (3.70%)  1
Hepatobiliary disorders   
Cholangitis * 1  1/27 (3.70%)  1
Infections and infestations   
Appendicitis * 1  1/27 (3.70%)  1
Injury, poisoning and procedural complications   
Joint dislocation * 1  1/27 (3.70%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 16.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Maralixibat (LUM001)
Affected / at Risk (%) # Events
Total   25/27 (92.59%)    
Gastrointestinal disorders   
Abdominal discomfort * 1  3/27 (11.11%)  4
Abdominal distension * 1  4/27 (14.81%)  5
Abdominal pain * 1  8/27 (29.63%)  8
Abdominal pain upper * 1  2/27 (7.41%)  4
Diarrhoea * 1  14/27 (51.85%)  27
Frequent bowel movements * 1  2/27 (7.41%)  2
Nausea * 1  9/27 (33.33%)  12
General disorders   
Asthenia * 1  2/27 (7.41%)  2
Fatigue * 1  4/27 (14.81%)  4
Pyrexia * 1  2/27 (7.41%)  2
Hepatobiliary disorders   
Cholangitis * 1  2/27 (7.41%)  2
Hepatomegaly * 1  2/27 (7.41%)  2
Infections and infestations   
Nasopharyngitis * 1  2/27 (7.41%)  2
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  3/27 (11.11%)  9
Muscle spasms * 1  2/27 (7.41%)  2
Neck pain * 1  2/27 (7.41%)  2
Nervous system disorders   
Headache * 1  6/27 (22.22%)  7
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 16.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Physician
Organization: Mirum
Phone: 1 650-667-4085
EMail: medinfo@mirumpharma.com
Layout table for additonal information
Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02061540     History of Changes
Other Study ID Numbers: LUM001-401
2014-005558-21 ( EudraCT Number )
First Submitted: February 11, 2014
First Posted: February 13, 2014
Results First Submitted: December 21, 2016
Results First Posted: April 12, 2017
Last Update Posted: March 29, 2019