Trial · NCT01307098
Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
NCT01307098, Phase 1, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2018-12-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency. Trials, record NCT01307098. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01307098
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
- NCT number
- NCT01307098
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 9 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Interventions
- Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
- First posted
- 2011-03-02
- Last updated
- 2018-12-11
- Results first posted
- 2018-12-11
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Screening up to Day 52 | 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. |
Source ClinicalTrials.gov
Outcomes1
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 Infusion-Related Reactions (IRRs) | Screening up to Day 52 | Safety Analysis Set: All participants who received any full or partial dose of sebelipase alfa in this study. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group12
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 Infusion-Related Reactions (IRRs) | Sebelipase Alfa 0.35 mg/kg | TEAEs | 1 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 1 mg/kg | TEAEs | 3 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 3 mg/kg | TEAEs | 3 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 0.35 mg/kg | SAEs | 0 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 1 mg/kg | SAEs | 0 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 3 mg/kg | SAEs | 0 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 0.35 mg/kg | IRRs | 0 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 1 mg/kg | IRRs | 0 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 3 mg/kg | IRRs | 0 | participants | |
| Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) | Sebelipase Alfa 0.35 mg/kg | TEAEs Leading to Study Discontinuation | 0 | participants |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sebelipase Alfa 0.35 mg/kg | 3 | |
| Overall Study | STARTED | Sebelipase Alfa 1 mg/kg | 3 | |
| Overall Study | STARTED | Sebelipase Alfa 3 mg/kg | 3 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Sebelipase Alfa 0.35 mg/kg | 3 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Sebelipase Alfa 1 mg/kg | 3 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Sebelipase Alfa 3 mg/kg | 3 | |
| Overall Study | COMPLETED | Sebelipase Alfa 0.35 mg/kg | 3 | |
| Overall Study | COMPLETED | Sebelipase Alfa 1 mg/kg | 3 | |
| Overall Study | COMPLETED | Sebelipase Alfa 3 mg/kg | 3 | |
| Overall Study | NOT COMPLETED | Sebelipase Alfa 0.35 mg/kg | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events81
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 | Abdominal discomfort | Gastrointestinal disorders | Sebelipase Alfa 0.35 mg/kg | 0 | 3 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Sebelipase Alfa 1 mg/kg | 0 | 3 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Sebelipase Alfa 3 mg/kg | 1 | 3 | |
| other | Abdominal distension | Gastrointestinal disorders | Sebelipase Alfa 0.35 mg/kg | 0 | 3 | |
| other | Abdominal distension | Gastrointestinal disorders | Sebelipase Alfa 1 mg/kg | 0 | 3 | |
| other | Abdominal distension | Gastrointestinal disorders | Sebelipase Alfa 3 mg/kg | 1 | 3 | |
| other | Abdominal pain | Gastrointestinal disorders | Sebelipase Alfa 0.35 mg/kg | 0 | 3 | |
| other | Abdominal pain | Gastrointestinal disorders | Sebelipase Alfa 1 mg/kg | 1 | 3 | |
| other | Abdominal pain | Gastrointestinal disorders | Sebelipase Alfa 3 mg/kg | 0 | 3 | |
| other | Abdominal pain lower | Gastrointestinal disorders | Sebelipase Alfa 0.35 mg/kg | 0 | 3 |
1 to 10 of 81Source ClinicalTrials.gov
See all 81 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sebelipase Alfa 0.35 mg/kg | ||
| Total, all-cause mortality | Sebelipase Alfa 1 mg/kg | ||
| Total, all-cause mortality | Sebelipase Alfa 3 mg/kg | ||
| Total, other adverse events | Sebelipase Alfa 0.35 mg/kg | 1 | 3 |
| Total, other adverse events | Sebelipase Alfa 1 mg/kg | 3 | 3 |
| Total, other adverse events | Sebelipase Alfa 3 mg/kg | 3 | 3 |
| Total, serious adverse events | Sebelipase Alfa 0.35 mg/kg | 0 | 3 |
| Total, serious adverse events | Sebelipase Alfa 1 mg/kg | 0 | 3 |
| Total, serious adverse events | Sebelipase Alfa 3 mg/kg | 0 | 3 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 23348766 | RESULT |
Source PubMed
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/NCT01307098
GET https://conductscience.org/api/trials/v1/NCT01307098/events?page=2