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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

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ClinicalTrials.gov Identifier: NCT01488097
Recruitment Status : Completed
First Posted : December 8, 2011
Results First Posted : May 9, 2016
Last Update Posted : July 20, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Cholesterol Ester Storage Disease (CESD)
Lysosomal Acid Lipase Deficiency
LAL-Deficiency
Intervention: Drug: sebelipase alfa

  Participant Flow

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
9 participants who completed Study LAL-CL01 (received all 4 doses of sebelipase alfa) were screened for eligibility for enrollment in this extension study (LAL-CL04). 8 participants met all enrollment criteria and were enrolled. 1 participant who required a liver transplant no longer met the entry criteria.

Reporting Groups
  Description
Open-Label Sebelipase Alfa Participants were administered sebelipase alfa once weekly (qw) as an intravenous (IV) infusion at the same dose received in Study LAL-CL01 (0.35, 1, or 3 milligrams per kilogram [mg/kg]) for 4 weeks. After the initial 4 qw doses, participants transitioned to dosing every other week (qow) at either 1 mg/kg (participants who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (participants who initiated dosing at 3 mg/kg qw). Subsequent modifications to the dose and dosing frequency were permitted for individual participants based on observed safety, tolerability, and clinical response to treatment. Participants could continue to receive treatment with sebelipase alfa for up to 5 years.

Participant Flow:   Overall Study
    Open-Label Sebelipase Alfa
STARTED   8 
Received Study Drug in Extension Study   8 
COMPLETED   7 
NOT COMPLETED   1 
Lost to Follow-up                1 



  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.
All participants who received any amount of study drug in the extension study.

Reporting Groups
  Description
Open-Label Sebelipase Alfa Participants were administered sebelipase alfa qw as an IV infusion at the same dose received in Study LAL-CL01 (0.35, 1, or 3 mg/kg) for 4 weeks. After the initial 4 qw doses, participants transitioned to dosing qow at either 1 mg/kg (participants who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (participants who initiated dosing at 3 mg/kg qw). Subsequent modifications to the dose and dosing frequency were permitted for individual participants based on observed safety, tolerability, and clinical response to treatment. Participants could continue to receive treatment with sebelipase alfa for up to 5 years.

Baseline Measures
   Open-Label Sebelipase Alfa 
Overall Participants Analyzed 
[Units: Participants]
 8 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      8 100.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 30.3  (10.69) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      2  25.0% 
Male      6  75.0% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      0   0.0% 
Not Hispanic or Latino      8 100.0% 
Unknown or Not Reported      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      0   0.0% 
White      8 100.0% 
More than one race      0   0.0% 
Unknown or Not Reported      0   0.0% 
Region of Enrollment 
[Units: Participants]
Count of Participants
 
Canada   1 
Czechia   1 
United States   3 
United Kingdom   2 
France   1 


  Outcome Measures

1.  Primary:   Number Of Participants Reporting TEAEs And IARs   [ Time Frame: From after first dose administration post-Baseline through EOS during study LAL-CL04 ]

2.  Secondary:   Changes From Baseline In ALT And AST   [ Time Frame: Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS ]

3.  Secondary:   Changes From Baseline In Liver Volume   [ Time Frame: Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS ]

4.  Secondary:   Changes From Baseline In Liver Fat Content   [ Time Frame: Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, and Week 260 ]

5.  Secondary:   Changes From Baseline In GGT And ALP   [ Time Frame: Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS ]

6.  Secondary:   Changes From Baseline In Serum Lipids   [ Time Frame: Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS ]

7.  Secondary:   Changes From Baseline In Serum Ferritin   [ Time Frame: Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS ]

8.  Secondary:   Changes From Baseline In Hs-CRP   [ Time Frame: Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS ]


  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.


Results Point of Contact:  
Name/Title: Alexion Pharmaceuticals Inc.
Organization: Alexion Pharmaceuticals Inc.
phone: 475-230-2596
e-mail: clinicaltrials@alexion.com


Publications of Results:

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01488097     History of Changes
Other Study ID Numbers: LAL-CL04
First Submitted: November 26, 2011
First Posted: December 8, 2011
Results First Submitted: January 14, 2016
Results First Posted: May 9, 2016
Last Update Posted: July 20, 2018