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Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

This study has been terminated.
(Interim analysis suggested that size of benefit anticipated from continued participation of patients in Part B no longer supported trial extension beyond Part A)
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01589237
First received: April 27, 2012
Last updated: September 26, 2016
Last verified: September 2016
Results First Received: June 17, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Familial Chylomicronemia Syndrome (FCS) (HLP Type I)
Intervention: Drug: LCQ908

  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
100% patients who completed the screening phase were enrolled in the study.

Reporting Groups
  Description
Placebo of Pradigastat (LCQ908) Regimen

Part A: Patients who were randomized to placebo in study CLCQ908B2302/NCT01514461. In current study, patients initiated at 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.

20 mg Pradigastat (LCQ908) Regimen

Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.

40 mg Pradigastat (LCQ908) Regimen

Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.

Pradigastat (LCQ908) Regimen- From Study A2212

Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.


Participant Flow for 2 periods

Period 1:   Part A (52 Weeks)
    Placebo of Pradigastat (LCQ908) Regimen   20 mg Pradigastat (LCQ908) Regimen   40 mg Pradigastat (LCQ908) Regimen   Pradigastat (LCQ908) Regimen- From Study A2212
STARTED   11 [1]   12   10   5 
COMPLETED   9   9   8   5 
NOT COMPLETED   2   3   2   0 
Subject/guardian decision                2                3                2                0 
[1] "Started" = Full analysis set/safety set

Period 2:   Part B (Planned for 78 Week-terminated)
    Placebo of Pradigastat (LCQ908) Regimen   20 mg Pradigastat (LCQ908) Regimen   40 mg Pradigastat (LCQ908) Regimen   Pradigastat (LCQ908) Regimen- From Study A2212
STARTED   5   6   4   4 
COMPLETED   0   0   0   0 
NOT COMPLETED   5   6   4   4 
Subject/guardian decision                0                1                0                0 
Study terminated by sponsor                5                5                4                3 
Physician Decision                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.
Full analysis set (FAS) – All subjects in the extension study who were in full analysis set in either study LCQ908A2212/NCT01146522 or LCQ908B2302/NCT01514461.

Reporting Groups
  Description
Placebo of Pradigastat (LCQ908) Regimen

Part A: Patients who were randomized to placebo in study CLCQ908B2302/NCT01514461. In current study, patients initiated at 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.

20 mg Pradigastat (LCQ908) Regimen

Part A: Patients who were randomized to LCQ908 20 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.

40 mg Pradigastat (LCQ908) Regimen

Part A: Patients who were randomized to LCQ908 40 mg in study CLCQ908B2302/NCT01514461. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.

Pradigastat (LCQ908) Regimen- From Study A2212

Part A: Patients who were randomized to LCQ908 in study CLCQ908A2212/NCT01146522. In current study, patients initiated at LCQ908 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose was allowed. One down titration allowed from the highest dose attained.

Part B: Patients who consented to take part in the Part B continued their treatment at the same dose as they took when they completed Part A. LCQ908 treatment could be titrated (within the dose range 10 mg to 40 mg) at the investigators discretion and based on individual patients’ tolerance and safety profile.

Total Total of all reporting groups

Baseline Measures
   Placebo of Pradigastat (LCQ908) Regimen   20 mg Pradigastat (LCQ908) Regimen   40 mg Pradigastat (LCQ908) Regimen   Pradigastat (LCQ908) Regimen- From Study A2212   Total 
Overall Participants Analyzed 
[Units: Participants]
 11   12   10   5   38 
Age 
[Units: Years]
Mean (Standard Deviation)
 52.9  (10.22)   44.1  (14.26)   43.6  (84.53)   52.2  (12.72)   47.6  (11.43) 
Gender 
[Units: Participants]
         
Female   5   6   2   3   16 
Male   6   6   8   2   22 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Patients With Any Adverse Events, Serious Adverse Events and Death   [ Time Frame: 52 weeks ]

2.  Secondary:   Changes From Baseline in Triglyceride Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]

3.  Secondary:   Changes From Baseline in Cholesterol Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]

4.  Secondary:   Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]

5.  Secondary:   Changes From Baseline in Glycerol Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]

6.  Secondary:   Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]

7.  Secondary:   Changes From Baseline in Apolipoprotein A1 Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]

8.  Secondary:   Changes From Baseline in Apolipoprotein B-48 Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]

9.  Secondary:   Changes From Baseline in Apolipoprotein B-100 Levels up to 52 Weeks   [ Time Frame: Baseline, Week 12, 24 and 52 ]


  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
As the anticipated benefit from the continued participation of patients in 18 month extension (Part B) was not supported by results of the December 2014 interim analysis, Novartis decided to terminate the Part B to be effective as of May 31, 2015.


  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 Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300
e-mail: trialandresults.registries@novartis.com



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01589237     History of Changes
Other Study ID Numbers: CLCQ908B2305
2012-000802-32 ( EudraCT Number )
Study First Received: April 27, 2012
Results First Received: June 17, 2016
Last Updated: September 26, 2016
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Netherlands: European Medicines Agency
France : European Medicines Agency
Germany: European Medicines Agency
Spain:European Medicines Agency
United Kingdom:European Medicines Agency
South Africa: Medicines Control Council