Trial · NCT01757184
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
NCT01757184, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2016-04-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency. Trials, record NCT01757184. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01757184
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
- NCT number
- NCT01757184
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 66 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa, Placebo
- First posted
- 2012-12-28
- Last updated
- 2020-12-29
- Results first posted
- 2016-04-18
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Of Participants Achieving Alanine Aminotransferase Normalization | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256) | Alanine aminotransferase (ALT) normalization was defined as an abnormal baseline value (ALT \> the age- and gender-specific upper limit of normal \[ULN\] provided by the central laboratory performing the assay) that becomes normal (\< ULN). Alanine aminotransferase normalization was evaluated at the end of the Double-blind Period (the last double-blind assessment) and at the end of the Open-label Period (last open-label assessment). Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
| Secondary | Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Relative reduction (percentage change from baseline) in LDL-C, as assessed by laboratory measurements was evaluated at the end of the Double-blind Period and at the end of the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
| Secondary | Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Relative reduction (percent change from baseline) in non-HDL-C, as assessed by laboratory measurements, was evaluated at the end of the Double-blind Period and the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
| Secondary | Percentage Of Participants Achieving Aspartate Aminotransferase Normalization | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Aspartate aminotransferase (AST) normalization was defined as an abnormal baseline value (AST \> the age- and gender-specific ULN provided by the central laboratory performing the assay) that becomes normal (\< ULN). AST normalization was evaluated at the end of the Double-blind Period (the last Double-blind assessment) and at the end of the Open-label Period (last open-label assessment). Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
| Secondary | Percent Change From Baseline In Triglycerides | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Relative reduction (percent change from baseline) in triglycerides, as assessed by laboratory measurements, was evaluated at the end of the Double-blind Period and the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
| Secondary | Percent Change From Baseline In High-density Lipoprotein Cholesterol (HDL-C) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Relative increase (percent change from baseline) in HDL-C, assessed by laboratory measurements, was evaluated at the end of the Double-blind Period and the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
| Secondary | Percent Change From Baseline In Liver Fat Content | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Decrease in liver fat content, as assessed by magnetic resonance imaging (MRI), was evaluated in participants for whom imaging was performed. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
| Secondary | Participants With Improvement In Liver Histopathology (Decrease Of > 5% In Hepatic Steatosis Score) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline up to Week 52. | The number of participants who had an improvement in hepatic histopathology (defined as a decrease of \> 5% in hepatic steatosis score, assessed by morphometry), as determined by blinded central pathologist review, in the participants for whom liver biopsy was performed. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. |
| Secondary | Percent Change From Baseline In Liver Volume | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Relative reduction (percent change from baseline) in liver volume, as assessed by MRI, was evaluated in participants for whom imaging was performed. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Of Participants Achieving Alanine Aminotransferase Normalization | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256) | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percentage of participants | NUMBER |
| SECONDARY | Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percent change | MEAN |
| SECONDARY | Percentage Of Participants Achieving Aspartate Aminotransferase Normalization | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percentage of participants | NUMBER |
| SECONDARY | Percent Change From Baseline In Triglycerides | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline In High-density Lipoprotein Cholesterol (HDL-C) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline In Liver Fat Content | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percent change | MEAN |
| SECONDARY | Participants With Improvement In Liver Histopathology (Decrease Of > 5% In Hepatic Steatosis Score) | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline up to Week 52. | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | participants | NUMBER |
| SECONDARY | Percent Change From Baseline In Liver Volume | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256). | Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo).~Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa). | percent change | MEAN |
Source ClinicalTrials.gov
Results by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Of Participants Achieving Alanine Aminotransferase Normalization | Double-blind Sebelipase Alfa | 31 | percentage of participants | ||
| Percentage Of Participants Achieving Alanine Aminotransferase Normalization | Double-blind Placebo | 7 | percentage of participants | ||
| Percentage Of Participants Achieving Alanine Aminotransferase Normalization | Open-label Sebelipase Alfa/Sebelipase Alfa | 56 | percentage of participants | ||
| Percentage Of Participants Achieving Alanine Aminotransferase Normalization | Open-label Placebo/Sebelipase Alfa | 37 | percentage of participants | ||
| Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | Double-blind Sebelipase Alfa | -28.42 | Standard Deviation 22.304 | percent change | |
| Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | Double-blind Placebo | -6.25 | Standard Deviation 13.015 | percent change | |
| Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | Open-label Sebelipase Alfa/Sebelipase Alfa | -19.74 | Standard Deviation 33.262 | percent change | |
| Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | Open-label Placebo/Sebelipase Alfa | -18.09 | Standard Deviation 33.685 | percent change | |
| Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C) | Double-Blind Sebelipase Alfa | -27.97 | Standard Deviation 18.612 | percent change | |
| Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C) | Double-Blind Placebo | -6.94 | Standard Deviation 10.922 | percent change |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow32
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-blind Period | STARTED | Double-blind Sebelipase Alfa | 36 | |
| Double-blind Period | STARTED | Double-blind Placebo | 30 | |
| Double-blind Period | STARTED | Open-label Sebelipase Alfa/Sebelipase Alfa | 0 | |
| Double-blind Period | STARTED | Open-label Placebo/Sebelipase Alfa | 0 | |
| Double-blind Period | Received at Least 1 Dose of Study Drug | Double-blind Sebelipase Alfa | 36 | |
| Double-blind Period | Received at Least 1 Dose of Study Drug | Double-blind Placebo | 30 | |
| Double-blind Period | Received at Least 1 Dose of Study Drug | Open-label Sebelipase Alfa/Sebelipase Alfa | 0 | |
| Double-blind Period | Received at Least 1 Dose of Study Drug | Open-label Placebo/Sebelipase Alfa | 0 | |
| Double-blind Period | COMPLETED | Double-blind Sebelipase Alfa | 35 | |
| Double-blind Period | COMPLETED | Double-blind Placebo | 30 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 participant flowAdverse events476
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 | Diarrhoea | Gastrointestinal disorders | Double-blind Sebelipase Alfa | 6 | 36 | |
| other | Diarrhoea | Gastrointestinal disorders | Double-blind Placebo | 5 | 30 | |
| other | Diarrhoea | Gastrointestinal disorders | Open-label Sebelipase Alfa/Sebelipase Alfa | 7 | 36 | |
| other | Diarrhoea | Gastrointestinal disorders | Open-label Placebo/Sebelipase Alfa | 11 | 30 | |
| other | Abdominal pain | Gastrointestinal disorders | Double-blind Sebelipase Alfa | 3 | 36 | |
| other | Abdominal pain | Gastrointestinal disorders | Double-blind Placebo | 1 | 30 | |
| other | Abdominal pain | Gastrointestinal disorders | Open-label Sebelipase Alfa/Sebelipase Alfa | 7 | 36 | |
| other | Abdominal pain | Gastrointestinal disorders | Open-label Placebo/Sebelipase Alfa | 9 | 30 | |
| other | Constipation | Gastrointestinal disorders | Double-blind Sebelipase Alfa | 3 | 36 | |
| other | Constipation | Gastrointestinal disorders | Double-blind Placebo | 1 | 30 |
1 to 10 of 476Source ClinicalTrials.gov
See all 476 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Double-blind Sebelipase Alfa | 0 | 36 |
| Total, all-cause mortality | Double-blind Placebo | 0 | 30 |
| Total, all-cause mortality | Open-label Sebelipase Alfa/Sebelipase Alfa | 0 | 36 |
| Total, all-cause mortality | Open-label Placebo/Sebelipase Alfa | 0 | 30 |
| Total, other adverse events | Double-blind Sebelipase Alfa | 31 | 36 |
| Total, other adverse events | Double-blind Placebo | 28 | 30 |
| Total, other adverse events | Open-label Sebelipase Alfa/Sebelipase Alfa | 35 | 36 |
| Total, other adverse events | Open-label Placebo/Sebelipase Alfa | 29 | 30 |
| Total, serious adverse events | Double-blind Sebelipase Alfa | 2 | 36 |
| Total, serious adverse events | Double-blind Placebo | 1 | 30 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers4
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/NCT01757184
GET https://conductscience.org/api/trials/v1/NCT01757184/results?page=2