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Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01488097
First received: November 26, 2011
Last updated: April 5, 2016
Last verified: April 2016
Results First Received: January 14, 2016  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Cholesterol Ester Storage Disease(CESD)
Lysosomal Acid Lipase Deficiency
Intervention: Drug: SBC-102 (sebelipase alfa)

  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
The study was conducted at a total of 11 sites in the United States, United Kingdom, France, Canada and the Czech Republic

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
9 subjects who completed treatment in study LAL-CL01 were eligible to be and were screened for potential enrollment in this extension study (LAL-CL04). 8 subjects met all enrollment criteria and were enrolled, treated, and analyzed. 1 subject who required a liver transplant prior to deciding to enter the study no longer met the entry criteria.

Reporting Groups
  Description
Open-Label Sebelipase Alfa Subjects received IV infusions of sebelipase alfa. Each subject initiated treatment in this extension study at the once weekly (qw) dose of sebelipase alfa that he/she received in study LAL-CL01 (0.35, 1, or 3 mg/kg qw). After 4 initial weekly doses, subjects transitioned to every other week (qow) dosing at either 1 mg/kg (subjects who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (subjects who initiated dosing at 3 mg/kg qw). In the event of disease progression (based on protocol defined criteria) after at least 12 weeks of treatment in this study, subjects could be considered for a dose increase to 3 mg/kg qow (from 1 mg/kg qow) or to 3 mg/kg qw (from 3 mg/kg qow).

Participant Flow for 2 periods

Period 1:   Long-term Treatment (up to Week 104)
    Open-Label Sebelipase Alfa  
STARTED     8  
COMPLETED     8  
NOT COMPLETED     0  

Period 2:   Long-term Treatment (Beyond Week 104)
    Open-Label Sebelipase Alfa  
STARTED     8  
COMPLETED     8 [1]
NOT COMPLETED     0  
[1] This period is ongoing.



  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.
No text entered.

Reporting Groups
  Description
Open-Label Sebelipase Alfa Subjects received IV infusions of sebelipase alfa. Each subject initiated treatment in this extension study at the once weekly (qw) dose of sebelipase alfa that he/she received in study LAL-CL01 (0.35, 1, or 3 mg/kg qw). After 4 initial weekly doses, subjects transitioned to every other week (qow) dosing at either 1 mg/kg (subjects who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (subjects who initiated dosing at 3 mg/kg qw). In the event of disease progression (based on protocol defined criteria) after at least 12 weeks of treatment in this study, subjects could be considered for a dose increase to 3 mg/kg qow (from 1 mg/kg qow) or to 3 mg/kg qw (from 3 mg/kg qow).

Baseline Measures
    Open-Label Sebelipase Alfa  
Number of Participants  
[units: participants]
  8  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     8  
>=65 years     0  
Age  
[units: years]
Mean (Standard Deviation)
  30.3  (10.69)  
Gender  
[units: participants]
 
Female     2  
Male     6  
Ethnicity (NIH/OMB)  
[units: participants]
 
Hispanic or Latino     0  
Not Hispanic or Latino     8  
Unknown or Not Reported     0  
Race (NIH/OMB)  
[units: participants]
 
American Indian or Alaska Native     0  
Asian     0  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     0  
White     8  
More than one race     0  
Unknown or Not Reported     0  
Region of Enrollment  
[units: participants]
 
Canada     1  
Czech Republic     1  
United States     3  
United Kingdom     2  
France     1  



  Outcome Measures
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1.  Primary:   ALT and AST Changes From Baseline   [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]

2.  Secondary:   Change From Baseline in Liver Volume   [ Time Frame: From baseline (study LAL-CL04) to week 10 or 12, week 24, week 52, week 104 ]

3.  Secondary:   Change From Baseline in Liver Fat Content   [ Time Frame: From baseline (study LAL-CL04) to week 10 or 12, week 24, week 52, week 104 ]

4.  Secondary:   GGT and ALP Changes From Baseline   [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]

5.  Secondary:   Lipid Changes From Baseline   [ Time Frame: From baseline (study LAL-CL01) to week 10 or 12, week 24, week 52, week 104 ]

6.  Secondary:   Serum Ferritin Change From Baseline   [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]

7.  Secondary:   High Sensitivity C-reactive Protein (Hs-CRP) Change From Baseline   [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]


  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 NOT 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.


Results Point of Contact:  
Name/Title: Mark Friedman, Medical Director
Organization: Alexion Pharmaceuticals
phone: 781-357-9953
e-mail: mark.friedman@alxn.com


Publications of Results:

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01488097     History of Changes
Other Study ID Numbers: LAL-CL04
Study First Received: November 26, 2011
Results First Received: January 14, 2016
Last Updated: April 5, 2016
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Czech Republic: State Institute for Drug Control
Canada: Health Canada