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Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome (IMAGO)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01903460
First Posted: July 19, 2013
Last Update Posted: December 7, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Shire
Results First Submitted: November 2, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition: Alagille Syndrome
Interventions: Drug: LUM001
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
LUM001 140ug/kg/Day Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.
LUM001 280ug/kg/Day Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.
Placebo Cohort A Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.
Placebo Cohort B Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.

Participant Flow:   Overall Study
    LUM001 140ug/kg/Day   LUM001 280ug/kg/Day   Placebo Cohort A   Placebo Cohort B
STARTED   6   8   3   3 
COMPLETED   6   8   3   2 
NOT COMPLETED   0   0   0   1 
Adverse Event                0                0                0                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
All randomized participants

Reporting Groups
  Description
LUM001 140ug/kg/Day Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.
LUM001 280ug/kg/Day Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.
Placebo Cohort A Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.
Placebo Cohort B Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.
Total Total of all reporting groups

Baseline Measures
   LUM001 140ug/kg/Day   LUM001 280ug/kg/Day   Placebo Cohort A   Placebo Cohort B   Total 
Overall Participants Analyzed 
[Units: Participants]
 6   8   3   3   20 
Age 
[Units: Years]
Mean (Standard Deviation)
 5.8  (4.49)   6.8  (6.73)   5.0  (2.00)   4.3  (3.21)   5.9  (4.93) 
Age, Customized 
[Units: Participants]
         
< 2 years   0   3   0   0   3 
2 to 4 years   3   1   1   2   7 
5 to 8 years   1   1   2   1   5 
9 to 12 years   2   0   0   0   2 
13 to 18 years   0   3   0   0   3 
Gender 
[Units: Participants]
         
Female   2   3   3   2   10 
Male   4   5   0   1   10 
Region of Enrollment 
[Units: Participants]
         
United Kingdom   6   8   3   3   20 


  Outcome Measures
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1.  Primary:   Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level   [ Time Frame: Baseline to 13 weeks or end of treatment ]

2.  Secondary:   Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes   [ Time Frame: Baseline to 13 weeks or end of treatment ]

3.  Secondary:   Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores   [ Time Frame: Baseline to 13 weeks or end of treatment ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Physician
Organization: Shire Development LLC
phone: +1 866 842 5335



Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01903460     History of Changes
Other Study ID Numbers: LUM001-302
2012-005346-38 ( EudraCT Number )
SHP625-302 ( Other Identifier: Shire Development LLC )
First Submitted: July 16, 2013
First Posted: July 19, 2013
Results First Submitted: November 2, 2015
Results First Posted: December 7, 2015
Last Update Posted: December 7, 2015