Clinical Trial Results:
An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)
Summary
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EudraCT number |
2015-000906-20 |
Trial protocol |
GB |
Global end of trial date |
24 Jul 2015
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Results information
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Results version number |
v1(current) |
This version publication date |
27 Nov 2019
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First version publication date |
27 Nov 2019
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
LUM001-601
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Mirum Pharmaceuticals, Inc. (Transferred from Shire to Mirum)
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Sponsor organisation address |
950 Tower Lane, Foster City, United States, CA 94404
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Public contact |
Chief Scientific Officer, Mirum Pharmaceuticals, Inc., medinfo@mirumpharma.com
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Scientific contact |
Chief Scientific Officer, Mirum Pharmaceuticals, Inc., medinfo@mirumpharma.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
24 Jul 2015
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
24 Jul 2015
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
The main objective of this trial is to evaluate the long-term safety of LUM001 in patients with ALGS or PFIC.
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered in Population of Trial Subjects section since ‘0’ could not be entered due to EudraCT system constraints.
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Protection of trial subjects |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered in Population of Trial Subjects section since ‘0’ could not be entered due to EudraCT system constraints.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
24 Jul 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
United Kingdom: 999999
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Worldwide total number of subjects |
999999
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EEA total number of subjects |
999999
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
999999
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||
Pre-assignment
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Screening details |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. | ||||||||||
Period 1
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Period 1 title |
Period 1 (overall period)
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||||||
Arms
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Arm title
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LUM001 | ||||||||||
Arm description |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints. | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
LUM001
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Oral solution
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Routes of administration |
Oral use
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Dosage and administration details |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints.
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Baseline characteristics reporting groups
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Reporting group title |
LUM001
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Reporting group description |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints. | ||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
LUM001
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Reporting group description |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints. |
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End point title |
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [1] | ||||||
End point description |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
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End point type |
Primary
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End point timeframe |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. |
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Notes [2] - Study was withdrawn with 0 participants. |
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No statistical analyses for this end point |
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Adverse events information [1]
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Timeframe for reporting adverse events |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
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Adverse event reporting additional description |
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
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Assessment type |
Non-systematic | ||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
NA
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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: Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. |
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. |