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Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

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ClinicalTrials.gov Identifier: NCT01307098
Recruitment Status : Completed
First Posted : March 2, 2011
Results First Posted : December 11, 2018
Last Update Posted : December 11, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Cholesterol Ester Storage Disease(CESD)
Lysosomal Acid Lipase Deficiency
LAL-Deficiency
Interventions Drug: Sebelipase alfa 0.35 mg/kg
Drug: Sebelipase alfa 1 mg/kg
Drug: Sebelipase alfa 3 mg/kg
Enrollment 9
Recruitment Details  
Pre-assignment Details Participants were screened for eligibility for enrollment in this study (LAL-CL01). Nine participants met all enrollment criteria and were enrolled and allocated to 1 of 3 cohorts.
Arm/Group Title Sebelipase Alfa 0.35 mg/kg Sebelipase Alfa 1 mg/kg Sebelipase Alfa 3 mg/kg
Hide Arm/Group Description Cohort 1: Participants were administered once weekly (qw) infusions of 0.35 milligrams/kilogram (mg/kg) sebelipase alfa. Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa. Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.
Period Title: Overall Study
Started 3 3 3
Received at Least 1 Dose of Study Drug 3 3 3
Completed 3 3 3
Not Completed 0 0 0
Arm/Group Title Sebelipase Alfa 0.35 mg/kg Sebelipase Alfa 1 mg/kg Sebelipase Alfa 3 mg/kg Total
Hide Arm/Group Description Cohort 1: Participants were administered qw infusions of 0.35 mg/kg sebelipase alfa. Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa. Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa. Total of all reporting groups
Overall Number of Baseline Participants 3 3 3 9
Hide Baseline Analysis Population Description
All participants who received any full or partial dose of sebelipase alfa in this study.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
3
 100.0%
3
 100.0%
3
 100.0%
9
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 3 participants 3 participants 9 participants
32.3  (7.57) 35.3  (14.22) 27.0  (12.17) 31.6  (10.74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
Female
1
  33.3%
1
  33.3%
1
  33.3%
3
  33.3%
Male
2
  66.7%
2
  66.7%
2
  66.7%
6
  66.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
3
 100.0%
3
 100.0%
3
 100.0%
9
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
3
 100.0%
3
 100.0%
3
 100.0%
9
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs)
Hide Description Safety and tolerability of sebelipase alfa was primarily assessed by monitoring the number of participants reporting treatment-emergent adverse events (TEAEs), including serious adverse events, and infusion-related reactions (IRRs). The number of participants who discontinued from the study due to a TEAE is also presented. An IRR was defined as any adverse event that occurred between the start of the infusion and 4 hours after completion of the infusion and was assessed by the Investigator as at least possibly related to study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Time Frame Screening up to Day 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set: All participants who received any full or partial dose of sebelipase alfa in this study.
Arm/Group Title Sebelipase Alfa 0.35 mg/kg Sebelipase Alfa 1 mg/kg Sebelipase Alfa 3 mg/kg
Hide Arm/Group Description:
Cohort 1: Participants were administered qw infusions of 0.35 mg/kg sebelipase alfa.
Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa.
Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.
Overall Number of Participants Analyzed 3 3 3
Measure Type: Number
Unit of Measure: participants
TEAEs 1 3 3
SAEs 0 0 0
IRRs 0 0 0
TEAEs Leading to Study Discontinuation 0 0 0
Time Frame Screening up to Day 52
Adverse Event Reporting Description Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
 
Arm/Group Title Sebelipase Alfa 0.35 mg/kg Sebelipase Alfa 1 mg/kg Sebelipase Alfa 3 mg/kg
Hide Arm/Group Description Cohort 1: Participants were administered qw infusions of 0.35 mg/kg sebelipase alfa. Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa. Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.
All-Cause Mortality
Sebelipase Alfa 0.35 mg/kg Sebelipase Alfa 1 mg/kg Sebelipase Alfa 3 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Hide Serious Adverse Events
Sebelipase Alfa 0.35 mg/kg Sebelipase Alfa 1 mg/kg Sebelipase Alfa 3 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/3 (0.00%)   0/3 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Sebelipase Alfa 0.35 mg/kg Sebelipase Alfa 1 mg/kg Sebelipase Alfa 3 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/3 (33.33%)   3/3 (100.00%)   3/3 (100.00%) 
Gastrointestinal disorders       
Abdominal discomfort  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Abdominal distension  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Abdominal pain  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Abdominal pain lower  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Diarrhoea  1  1/3 (33.33%)  2/3 (66.67%)  0/3 (0.00%) 
Dyspepsia  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Flatulence  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Nausea  1  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%) 
Rectal haemorrhage  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
General disorders       
Catheter site related reaction  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Feeling hot  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Oedema peripheral  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Infections and infestations       
Upper respiratory tract infection  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Viral upper respiratory tract infection  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Metabolism and nutrition disorders       
Hypercholesterolaemia  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Hypertriglyceridaemia  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Musculoskeletal and connective tissue disorders       
Bone pain  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Musculoskeletal pain  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Myalgia  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Nervous system disorders       
Headache  1  0/3 (0.00%)  1/3 (33.33%)  2/3 (66.67%) 
Presyncope  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Syncope  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Renal and urinary disorders       
Urine odour abnormal  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Reproductive system and breast disorders       
Breast swelling  1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Vaginal haemorrhage  1 [1]  0/1 (0.00%)  1/1 (100.00%)  0/1 (0.00%) 
Skin and subcutaneous tissue disorders       
Dermatitis contact  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Night sweats  1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (13.1)
[1]
Affects only female participants
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
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals Inc.
Organization: Alexion Pharmaceuticals Inc.
Phone: 855-752-2356
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01307098    
Other Study ID Numbers: LAL-CL01
First Submitted: March 1, 2011
First Posted: March 2, 2011
Results First Submitted: October 30, 2018
Results First Posted: December 11, 2018
Last Update Posted: December 11, 2018