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Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy) (ODYSSEY FH II)

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ClinicalTrials.gov Identifier: NCT01709500
Recruitment Status : Completed
First Posted : October 18, 2012
Results First Posted : October 28, 2015
Last Update Posted : October 28, 2015
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Heterozygous Familial Hypercholesterolemia
Interventions: Drug: LMT (atorvastatin, simvastatin, or rosuvastatin)
Drug: alirocumab
Drug: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The study was conducted at 26 sites in 4 countries. Overall, 322 participants were screened between 28 Nov 2012 and 26 Apr 2013, 73 of whom were screen failures.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Randomization was stratified according to prior history of myocardial infarction or ischemic stroke, and intensity of statin treatment. Assignment to treatment arms was done centrally using an Interactive Voice/Web Response System in a 2:1 (alirocumab: placebo) ratio after confirmation of selection criteria.

Reporting Groups
  Description
Alirocumab 75 mg/up to 150 mg Alirocumab 75 mg SC injection every two weeks (Q2W) added to stable dose of statin with or without LMT for 78 weeks. Alirocumab dose up-titrated to 150 mg Q2W from Week 12 when LDL-C levels ≥70 mg/dL at Week 8.
Placebo Placebo matched to alirocumab SC injection for 78-week treatment duration.

Participant Flow:   Overall Study
    Alirocumab 75 mg/up to 150 mg   Placebo
STARTED   167   82 
Treated   167   81 
COMPLETED   0   0 
NOT COMPLETED   167   82 
Randomized but not treated                0                1 
Adverse Event                5                1 
Poor compliance to protocol                2                1 
Other                3                1 
Related to IMP administration                1                0 
Treatment ongoing                156                78 



  Baseline Characteristics

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.
No text entered.

Reporting Groups
  Description
Alirocumab 75 mg/up to 150 mg Alirocumab 75 mg SC injection Q2W added to stable dose of statin with or without LMT for 78 weeks. Alirocumab dose up-titrated to 150 mg Q2W from Week 12 when LDL-C levels ≥70 mg/dL at Week 8.
Placebo Placebo matched to alirocumab SC injection for 78­-week treatment duration.
Total Total of all reporting groups

Baseline Measures
   Alirocumab 75 mg/up to 150 mg   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 167   82   249 
Age 
[Units: Years]
Mean (Standard Deviation)
 53.2  (12.93)   53.2  (12.55)   53.2  (12.8) 
Gender 
[Units: Participants]
     
Female   81   37   118 
Male   86   45   131 
Calculated LDL-C in mmol/L [1] 
[Units: mmol/L]
Mean (Standard Deviation)
 3.485  (1.065)   3.471  (1.071)   3.480  (1.065) 
[1] Calculated LDL-C values were obtained using Friedewald formula.
Calculated LDL-C in mg/dL [1] 
[Units: mg/dL]
Mean (Standard Deviation)
 134.6  (41.1)   134.0  (41.4)   134.4  (41.1) 
[1] Calculated LDL-C values were obtained using Friedewald formula.


  Outcome Measures

1.  Primary:   Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent­-to­-Treat (ITT) Analysis   [ Time Frame: From Baseline to Week 52 ]

2.  Secondary:   Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis   [ Time Frame: From Baseline to Week 52 ]

3.  Secondary:   Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

4.  Secondary:   Percent Change From Baseline in Calculated LDL-C at Week 12 - On- Treatment Analysis   [ Time Frame: From Baseline to Week 52 ]

5.  Secondary:   Percent Change From Baseline in Apolipoprotein (Apo) B at Week 24 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

6.  Secondary:   Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis   [ Time Frame: From Baseline to Week 52 ]

7.  Secondary:   Percent Change From Baseline in Non-High ­Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

8.  Secondary:   Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis   [ Time Frame: From Baseline to Week 52 ]

9.  Secondary:   Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

10.  Secondary:   Percent Change From Baseline in Apo B at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

11.  Secondary:   Percent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

12.  Secondary:   Percent Change From Baseline in Total-C at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

13.  Secondary:   Percent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

14.  Secondary:   Percentage of Very High CV Risk Participants Reaching Calculated LDL­C <70 mg/dL (1.81 mmol/L) or High CV Risk Participants Reaching Calculated LDL­C <100 mg/dL (2.59 mmol/L) at Week 24 - ITT Analysis   [ Time Frame: Up to Week 52 ]

15.  Secondary:   Percentage of Very High CV Risk Participants Reaching Calculated LDL­-C <70 mg/dL (1.81 mmol/L) or High CV Risk Participants Reaching Calculated LDL­-C <100 mg/dL (2.59 mmol/L) at Week 24 - On-Treatment Analysis   [ Time Frame: Up to week 52 ]

16.  Secondary:   Percentage of Participants Reaching Calculated LDL­C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis   [ Time Frame: Up to Week 52 ]

17.  Secondary:   Percentage of Participants Reaching Calculated LDL­-C <70 mg/dL (1.81 mmol/L) at Week 52 - On-Treatment Analysis   [ Time Frame: Up to Week 52 ]

18.  Secondary:   Percent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

19.  Secondary:   Percent Change From Baseline in HDL-C at Week 24 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

20.  Secondary:   Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

21.  Secondary:   Percent Change From Baseline in Apo A-1 at Week 24 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

22.  Secondary:   Percent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

23.  Secondary:   Percent Change From Baseline in HDL-C at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

24.  Secondary:   Percent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]

25.  Secondary:   Percent Change From Baseline in Apo A­1 at Week 12 - ITT Analysis   [ Time Frame: From Baseline to Week 52 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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
No text entered.


  More Information

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: Clinical Trial Management
Organization: Regeneron Pharmaceuticals, Inc
e-mail: clinicaltrials@regeneron.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01709500     History of Changes
Other Study ID Numbers: R727-CL-1112
First Submitted: October 8, 2012
First Posted: October 18, 2012
Results First Submitted: July 29, 2015
Results First Posted: October 28, 2015
Last Update Posted: October 28, 2015