Clinical Trial Results:
A Randomized, Double-Blind, Placebo-Controlled, Proof-of-Concept Study Evaluating the Efficacy and Safety of Baricitinib (LY3009104) in Patients with Primary Biliary Cholangitis Who Have an Inadequate Response or are Intolerant to UDCA
Summary
|
|
EudraCT number |
2018-003365-34 |
Trial protocol |
GB IT |
Global end of trial date |
26 Sep 2019
|
Results information
|
|
Results version number |
v1(current) |
This version publication date |
04 Oct 2020
|
First version publication date |
04 Oct 2020
|
Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
|
|||
Trial identification
|
|||
Sponsor protocol code |
I4V-MC-JAIV
|
||
Additional study identifiers
|
|||
ISRCTN number |
- | ||
US NCT number |
NCT03742973 | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
Trial number: 17039 | ||
Sponsors
|
|||
Sponsor organisation name |
Eli Lilly and Company
|
||
Sponsor organisation address |
Lilly Corporate Center, Indianapolis, IN, United States, 46285
|
||
Public contact |
Available Mon - Fri 9 AM - 5 PM EST, Eli Lilly and Company, 1 877-CTLilly,
|
||
Scientific contact |
Available Mon - Fri 9 AM - 5 PM EST, Eli Lilly and Company, 1 877-285-4559,
|
||
Paediatric regulatory details
|
|||
Is trial part of an agreed paediatric investigation plan (PIP) |
No
|
||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Results analysis stage
|
|||
Analysis stage |
Final
|
||
Date of interim/final analysis |
26 Sep 2019
|
||
Is this the analysis of the primary completion data? |
No
|
||
Global end of trial reached? |
Yes
|
||
Global end of trial date |
26 Sep 2019
|
||
Was the trial ended prematurely? |
Yes
|
||
General information about the trial
|
|||
Main objective of the trial |
To evaluate the effect of baricitinib 4-mg QD compared to placebo on PBC disease
|
||
Protection of trial subjects |
This study was conducted in accordance with International Conference on Harmonization (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
|
||
Background therapy |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility and cohort B is not reported due to early study termination based on enrollment futility. | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
28 Mar 2019
|
||
Long term follow-up planned |
No
|
||
Independent data monitoring committee (IDMC) involvement? |
Yes
|
||
Population of trial subjects
|
|||
Number of subjects enrolled per country |
|||
Country: Number of subjects enrolled |
United States: 2
|
||
Worldwide total number of subjects |
2
|
||
EEA total number of subjects |
0
|
||
Number of subjects enrolled per age group |
|||
In utero |
0
|
||
Preterm newborn - gestational age < 37 wk |
0
|
||
Newborns (0-27 days) |
0
|
||
Infants and toddlers (28 days-23 months) |
0
|
||
Children (2-11 years) |
0
|
||
Adolescents (12-17 years) |
0
|
||
Adults (18-64 years) |
0
|
||
From 65 to 84 years |
2
|
||
85 years and over |
0
|
|
||||||||||
Recruitment
|
||||||||||
Recruitment details |
- | |||||||||
Pre-assignment
|
||||||||||
Screening details |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility and cohort B is not reported due to early study termination based on enrollment futility. | |||||||||
Period 1
|
||||||||||
Period 1 title |
Overall Study (overall period)
|
|||||||||
Is this the baseline period? |
Yes | |||||||||
Allocation method |
Randomised - controlled
|
|||||||||
Blinding used |
Double blind | |||||||||
Roles blinded |
Subject, Investigator | |||||||||
Blinding implementation details |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility and cohort B is not reported due to early study termination based on enrollment futility.
|
|||||||||
Arms
|
||||||||||
Are arms mutually exclusive |
Yes
|
|||||||||
Arm title
|
Baricitinib Cohort A | |||||||||
Arm description |
Participants received 2 milligram (mg) of Baricitinib tablet orally once a day for 12 weeks. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
Baricitinib
|
|||||||||
Investigational medicinal product code |
||||||||||
Other name |
||||||||||
Pharmaceutical forms |
Tablet
|
|||||||||
Routes of administration |
Oral use
|
|||||||||
Dosage and administration details |
Participants received 2 milligram (mg) of Baricitinib tablet orally once a day for 12 weeks.
|
|||||||||
Arm title
|
Placebo Cohort A | |||||||||
Arm description |
Participants received placebo orally once daily (QD) for 12 weeks. | |||||||||
Arm type |
Placebo | |||||||||
Investigational medicinal product name |
Placebo
|
|||||||||
Investigational medicinal product code |
||||||||||
Other name |
||||||||||
Pharmaceutical forms |
Tablet
|
|||||||||
Routes of administration |
Oral use
|
|||||||||
Dosage and administration details |
Participants received placebo orally once daily (QD) for 12 weeks.
|
|||||||||
|
|
||||||||||||||||||||||||||||||||||||||||||||||||||||
Baseline characteristics reporting groups
|
||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Overall Study
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility. | |||||||||||||||||||||||||||||||||||||||||||||||||||
|
|
|||
End points reporting groups
|
|||
Reporting group title |
Baricitinib Cohort A
|
||
Reporting group description |
Participants received 2 milligram (mg) of Baricitinib tablet orally once a day for 12 weeks. | ||
Reporting group title |
Placebo Cohort A
|
||
Reporting group description |
Participants received placebo orally once daily (QD) for 12 weeks. |
|
|||||||||||||
End point title |
Change From Baseline in Alkaline Phosphatase (ALP) | ||||||||||||
End point description |
9999 is reported and represents no data available (NA). Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, Week 12.
|
||||||||||||
|
|||||||||||||
Notes [1] - Zero participants reported in cohort A due to protection of personal identifiable information. [2] - Zero participants reported in cohort A due to protection of personal identifiable information. |
|||||||||||||
Statistical analysis title |
Statistical Analysis Cohort A | ||||||||||||
Statistical analysis description |
Zero participants reported in cohort A due to protection of personal identifiable information.
|
||||||||||||
Comparison groups |
Baricitinib Cohort A v Placebo Cohort A
|
||||||||||||
Number of subjects included in analysis |
2
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other [3] | ||||||||||||
P-value |
= 9999 [4] | ||||||||||||
Method |
Other | ||||||||||||
Parameter type |
9999 | ||||||||||||
Confidence interval |
|||||||||||||
Notes [3] - Zero participants reported in cohort A due to protection of personal identifiable information. [4] - Zero participants reported in cohort A due to protection of personal identifiable information. |
|
|||||||||||||
End point title |
Percentage of Participants With Alkaline Phosphatase (ALP) <1.67 x Upper Limit of Normal (ULN) (and at Least 15% Decrease From Baseline) and Total Bilirubin Level Less Than ULN | ||||||||||||
End point description |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Week 12
|
||||||||||||
|
|||||||||||||
Notes [5] - Zero participants reported in cohort A due to protection of personal identifiable information. [6] - Zero participants reported in cohort A due to protection of personal identifiable information. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Change From Baseline in Itch Numeric Rating Scale (NRS) | ||||||||||||
End point description |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Baseline, Week 12
|
||||||||||||
|
|||||||||||||
Notes [7] - Zero participants reported in cohort A due to protection of personal identifiable information. [8] - Zero participants reported in cohort A due to protection of personal identifiable information. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Change From Baseline in Fatigue NRS | ||||||||||||
End point description |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Baseline, Week 12
|
||||||||||||
|
|||||||||||||
Notes [9] - Zero participants reported in cohort A due to protection of personal identifiable information. [10] - Zero participants reported in cohort A due to protection of personal identifiable information. |
|||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||
Adverse events information [1]
|
||||||||||||||||
Timeframe for reporting adverse events |
Up to 6 months
|
|||||||||||||||
Adverse event reporting additional description |
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility and cohort B is not reported due to early study termination based on enrollment futility.
|
|||||||||||||||
Assessment type |
Systematic | |||||||||||||||
Dictionary used for adverse event reporting
|
||||||||||||||||
Dictionary name |
MedDRA | |||||||||||||||
Dictionary version |
18.0
|
|||||||||||||||
Reporting groups
|
||||||||||||||||
Reporting group title |
Baricitinib Cohort A
|
|||||||||||||||
Reporting group description |
Participants received 2 mg of Baricitinib tablet orally once a day for 12 weeks. | |||||||||||||||
Reporting group title |
Placebo Cohort A
|
|||||||||||||||
Reporting group description |
Participants received placebo orally once daily (QD) for 12 weeks. | |||||||||||||||
|
||||||||||||||||
Frequency threshold for reporting non-serious adverse events: 5% | ||||||||||||||||
|
||||||||||||||||
Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility and cohort B is not reported due to early study termination based on enrollment futility. |
|
|||
Substantial protocol amendments (globally) |
|||
Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
||
28 Sep 2018 |
The following changes were made to the protocol:
Study population: A note was added on number system used for inclusion and exclusion criteria to avoid confusion to the numbering.
Exclusion Criteria: Added clarifying language that cirrhosis should include complications.
Method of Treatment Assignment: Changed method from stratification to dynamic minimization.
Permanent discontinuation from study treatment: added discontinuation criterion of eFGR ≤40mL/min/1.73m2.
Minor editorial changes for clarity were made. |
||
Interruptions (globally) |
|||
Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
Zero participants reported in cohort A due to protection of personal identifiable information based on enrollment futility and cohort B is not reported due to study termination based on enrollment futility. |