Trial · NCT01488097
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
NCT01488097, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2016-05-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency. Trials, record NCT01488097. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01488097
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
- NCT number
- NCT01488097
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 8 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Interventions
- sebelipase alfa
- First posted
- 2011-12-08
- Last updated
- 2018-07-20
- Results first posted
- 2016-05-09
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number Of Participants Reporting TEAEs And IARs | From after first dose administration post-Baseline through EOS during study LAL-CL04 | 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-associated reactions (IARs). The number of participants who discontinued from the study due to a TEAE is also presented. An IAR was defined as any adverse event that occurred during the 2-hour infusion or within 4 hours after the end 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. TEAEs that occurred after the first dose administration at Week 1 through the End of Study (EOS) are presented. End of study was 30 days (+ 7 days) after the last dose of study drug (at Week 260). |
| Secondary | Changes From Baseline In ALT And AST | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Changes from Baseline to Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS for alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Baseline values were defined as the last measurement prior to the first infusion of sebelipase alfa in Study LAL-CL04. |
| Secondary | Changes From Baseline In Liver Volume | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Changes in liver volume from Baseline to Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS was assessed by magnetic resonance imaging (MRI). Baseline values were defined as the last measurement prior to the first infusion of sebelipase alfa in Study LAL-CL04. Liver volume was expressed as multiples of normal (MN), where normal is defined as 2.5% of body weight. |
| Secondary | Changes From Baseline In Liver Fat Content | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, and Week 260 | Changes in liver fat content from Baseline to Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, and Week 260, as assessed by multi-echo gradient-echo MRI. Baseline values were defined as the last measurement prior to the first infusion of sebelipase alfa in Study LAL-CL04. |
| Secondary | Changes From Baseline In GGT And ALP | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Changes from Baseline to Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS for gamma glutamyltransferase (GGT) and alkaline phosphatase (ALP). Baseline values were defined as the last measurement prior to the first infusion of sebelipase alfa in Study LAL-CL04. |
| Secondary | Changes From Baseline In Serum Lipids | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Lipid changes from Baseline to Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS were measured in serum for total cholesterol (Total-C), high density lipoprotein cholesterol (HDL-C), low density lipoprotein cholesterol (LDL-C), and triglycerides (TG). Baseline values were defined as the last measurement prior to the first infusion of sebelipase alfa in Study LAL-CL04. |
| Secondary | Changes From Baseline In Serum Ferritin | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Changes from Baseline to Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS for serum ferritin. Baseline values were defined as the last measurement prior to the first infusion of sebelipase alfa in Study LAL-CL04. |
| Secondary | Changes From Baseline In Hs-CRP | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Changes from Baseline to Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS for high sensitivity C-reactive protein (hs-CRP). Baseline values were defined as the last measurement prior to the first infusion of sebelipase alfa in Study LAL-CL04. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number Of Participants Reporting TEAEs And IARs | From after first dose administration post-Baseline through EOS during study LAL-CL04 | Safety Analysis Set: All participants who received any amount of study drug in the extension study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Changes From Baseline In ALT And AST | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Participants in the Full Analysis Set (FAS) for whom ALT and AST data were available at both Baseline and the indicated post-treatment time point. The FAS included all participants who received at least 1 complete infusion of sebelipase alfa in this study and who had at least 1 post-treatment measurement in this study. | units (U)/liter (L) | MEAN |
| SECONDARY | Changes From Baseline In Liver Volume | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Participants in the FAS for whom liver volume data were available at both Baseline and the indicated post-treatment time point. The FAS included all participants who received at least 1 complete infusion of sebelipase alfa in this study and who had at least 1 post-treatment measurement in this study. | Multiples of Normal (MN) | MEAN |
| SECONDARY | Changes From Baseline In Liver Fat Content | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, and Week 260 | Participants in the FAS for whom liver fat content data were available at both Baseline and the indicated post-treatment time point. The FAS included all participants who received at least 1 complete infusion of sebelipase alfa in this study and who had at least 1 post-treatment measurement in this study. | percentage fat fraction | MEAN |
| SECONDARY | Changes From Baseline In GGT And ALP | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Participants in the FAS for whom GGT and ALP data were available at both Baseline and the indicated post-treatment time point. The FAS included all participants who received at least 1 complete infusion of sebelipase alfa in this study and who had at least 1 post-treatment measurement in this study. | U/L | MEAN |
| SECONDARY | Changes From Baseline In Serum Lipids | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Participants in the FAS for whom lipid data were available at both Baseline and the indicated post-treatment time point. The FAS included all participants who received at least 1 complete infusion of sebelipase alfa in this study and who had at least 1 post-treatment measurement in this study. | mg/dL | MEAN |
| SECONDARY | Changes From Baseline In Serum Ferritin | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Participants in the FAS for whom serum ferritin data were available at both Baseline and the indicated post-treatment time point. The FAS included all participants who received at least 1 complete infusion of sebelipase alfa in this study and who had at least 1 post-treatment measurement in this study. | microgram (µg)/L | MEAN |
| SECONDARY | Changes From Baseline In Hs-CRP | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS | Participants in the FAS for whom hs-CRP data were available at both Baseline and the indicated post-treatment time point. The FAS included all participants who received at least 1 complete infusion of sebelipase alfa in this study and who had at least 1 post-treatment measurement in this study. | mg/L | MEAN |
Source ClinicalTrials.gov
Results by group99
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number Of Participants Reporting TEAEs And IARs | Open-Label Sebelipase Alfa | TEAEs | 8 | Participants | |
| Number Of Participants Reporting TEAEs And IARs | Open-Label Sebelipase Alfa | Serious TEAEs | 1 | Participants | |
| Number Of Participants Reporting TEAEs And IARs | Open-Label Sebelipase Alfa | IARs | 2 | Participants | |
| Number Of Participants Reporting TEAEs And IARs | Open-Label Sebelipase Alfa | TEAEs Leading to Study Discontinuation | 0 | Participants | |
| Changes From Baseline In ALT And AST | Open-Label Sebelipase Alfa | ALT Week 12 | -35.9 | Standard Deviation 19.99 | units (U)/liter (L) |
| Changes From Baseline In ALT And AST | Open-Label Sebelipase Alfa | ALT Week 24 | -38.1 | Standard Deviation 24.00 | units (U)/liter (L) |
| Changes From Baseline In ALT And AST | Open-Label Sebelipase Alfa | ALT Week 52 | -40.6 | Standard Deviation 20.50 | units (U)/liter (L) |
| Changes From Baseline In ALT And AST | Open-Label Sebelipase Alfa | ALT Week 104 | -42.5 | Standard Deviation 19.65 | units (U)/liter (L) |
| Changes From Baseline In ALT And AST | Open-Label Sebelipase Alfa | ALT Week 156 | -29.5 | Standard Deviation 20.38 | units (U)/liter (L) |
| Changes From Baseline In ALT And AST | Open-Label Sebelipase Alfa | ALT Week 208 | -26.7 | Standard Deviation 30.58 | units (U)/liter (L) |
1 to 10 of 99Source ClinicalTrials.gov
See all 99 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Open-Label Sebelipase Alfa | 8 | |
| Overall Study | Received Study Drug in Extension Study | Open-Label Sebelipase Alfa | 8 | |
| Overall Study | COMPLETED | Open-Label Sebelipase Alfa | 7 | |
| Overall Study | NOT COMPLETED | Open-Label Sebelipase Alfa | 1 |
Source ClinicalTrials.gov
Adverse events124
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Bradycardia | Cardiac disorders | Open-Label Sebelipase Alfa | 1 | 8 | |
| other | Palpitations | Cardiac disorders | Open-Label Sebelipase Alfa | 2 | 8 | |
| other | Tachycardia | Cardiac disorders | Open-Label Sebelipase Alfa | 2 | 8 | |
| other | Ear pain | Ear and labyrinth disorders | Open-Label Sebelipase Alfa | 3 | 8 | |
| other | Blepharitis | Eye disorders | Open-Label Sebelipase Alfa | 1 | 8 | |
| other | Conjunctivitis | Eye disorders | Open-Label Sebelipase Alfa | 1 | 8 | |
| other | Dry eye | Eye disorders | Open-Label Sebelipase Alfa | 1 | 8 | |
| other | Eye pain | Eye disorders | Open-Label Sebelipase Alfa | 1 | 8 | |
| other | Keratitis | Eye disorders | Open-Label Sebelipase Alfa | 1 | 8 | |
| other | Occular hyperaemia | Eye disorders | Open-Label Sebelipase Alfa | 1 | 8 |
1 to 10 of 124Source ClinicalTrials.gov
See all 124 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Open-Label Sebelipase Alfa | 0 | 8 |
| Total, other adverse events | Open-Label Sebelipase Alfa | 8 | 8 |
| Total, serious adverse events | Open-Label Sebelipase Alfa | 1 | 8 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01488097
GET https://conductscience.org/api/trials/v1/NCT01488097/results?page=2