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This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease [CESD]).
Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL-D other than supportive care. Enzyme replacement therapy may be a potential new treatment option for LAL-D participants.
Lysosomal acid lipase deficiency (LAL-D) is a genetic disease characterized by abnormal lipid accumulation in many parts of the body due to a marked decrease in activity of the enzyme lysosomal acid lipase (LAL).
The LAL-D disease spectrum ranges from a presentation in infants that is rapidly progressive to a presentation that occurs in childhood, adolescence, or less frequently, in adulthood in which the rate of disease progression is more variable. Irrespective of where a patient is on the disease spectrum, LAL-D is associated with significant burden of disease and a shortened life expectancy in some patients.
The non-infantile onset form of the disease, also known as CESD, occurs in both children and adults and is an under-appreciated cause of fatty liver with prominent microvesicular steatosis, fibrosis, and cirrhosis. Although the natural history of the disease has not been well studied, serious complications are frequently described, including early death, liver transplantation, or cardiovascular accidents. Other complications include premature atherosclerosis (hardening of arteries) associated with high levels of total cholesterol and low-density lipoprotein (LDL) cholesterol, often called the "bad" cholesterol. The levels of triglycerides can also be high and the levels of high-density lipoprotein (HDL) cholesterol (the "good" cholesterol) are typically low.
In the past, treatments mainly focused on control of the lipid abnormalities through diet and the use of lipid-lowering medications, which only address some aspects of the disease, while progression to fibrosis and cirrhosis may still occur. In preclinical studies and clinical studies in participants with LAL-D, treatment with SBC-102 (sebelipase alfa, Kanuma®) has been shown to produce improvements in markers of liver damage and in the lipid abnormalities. The purpose of this study was to examine the effects of using SBC-102 to treat LAL-D through a placebo-controlled, randomized, double-blinded study in children and adults.
This multicenter, randomized, double-blind, placebo-controlled study involving 66 participants evaluated the safety and efficacy of enzyme-replacement therapy with sebelipase alfa (administered intravenously at a dose of 1 mg/kg of body weight qow). The study included a 20-week placebo-controlled period followed by open-label treatment periods for all participants. The primary end-point was normalization of the alanine aminotransferase level. Secondary end-points included additional disease-related efficacy and safety assessments.
Final study results have not been published in a peer-reviewed journal.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Double-blind Sebelipase Alfa | Experimental | Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow for 20 weeks. |
|
| Double-blind Placebo | Placebo Comparator | Double-blind Period: IV infusions of matched placebo administered qow for 20 weeks. |
|
| Open-label Sebelipase Alfa/Sebelipase Alfa | Experimental | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. Dose modifications were permitted during the Open-label Period. |
|
| Open-label Placebo/Sebelipase Alfa | Experimental | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. Dose modifications were permitted during the Open-label Period. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sebelipase Alfa | Drug | IV infusions of sebelipase alfa |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Of Participants Achieving Alanine Aminotransferase Normalization | 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. | 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Florian Abel, MD | Alexion Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tucson | Arizona | 85724 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29628368 | Background | Wilson DP, Friedman M, Marulkar S, Hamby T, Bruckert E. Sebelipase alfa improves atherogenic biomarkers in adults and children with lysosomal acid lipase deficiency. J Clin Lipidol. 2018 May-Jun;12(3):604-614. doi: 10.1016/j.jacl.2018.02.020. Epub 2018 Mar 9. | |
| 26352813 | Background | Burton BK, Balwani M, Feillet F, Baric I, Burrow TA, Camarena Grande C, Coker M, Consuelo-Sanchez A, Deegan P, Di Rocco M, Enns GM, Erbe R, Ezgu F, Ficicioglu C, Furuya KN, Kane J, Laukaitis C, Mengel E, Neilan EG, Nightingale S, Peters H, Scarpa M, Schwab KO, Smolka V, Valayannopoulos V, Wood M, Goodman Z, Yang Y, Eckert S, Rojas-Caro S, Quinn AG. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. N Engl J Med. 2015 Sep 10;373(11):1010-20. doi: 10.1056/NEJMoa1501365. |
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To assess eligibility, participants were screened for a period of up to 6 weeks prior to enrollment in the study. A total of 86 participants were screened. Six of these participants underwent rescreening (of which 2 were eligible for the study). In total, 66 participants were eligible for the study and 20 participants were screen failures.
A total of 56 study centers located in 17 countries were initiated in this study. Participants were enrolled and treated at 41 centers in 15 countries, including 35 primary centers where participants initiated treatment and 6 qualified local medical centers where participants who were medically stable were transferred for long-term treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Double-blind Sebelipase Alfa | Double-blind Period: Intravenous (IV) infusions of sebelipase alfa at a dose of 1 milligram/kilogram (mg/kg) administered every other week (qow). |
| FG001 | Double-blind Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double-blind Period |
|
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| Placebo | Drug | IV infusions of matched placebo |
|
| 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). |
| Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C) | 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. | 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). |
| Percentage Of Participants Achieving Aspartate Aminotransferase Normalization | 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. | 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). |
| Percent Change From Baseline In Triglycerides | 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. | 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). |
| Percent Change From Baseline In High-density Lipoprotein Cholesterol (HDL-C) | 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. | 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). |
| Percent Change From Baseline In Liver Fat Content | 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. | 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). |
| Participants With Improvement In Liver Histopathology (Decrease Of > 5% In Hepatic Steatosis Score) | 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. | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline up to Week 52. |
| Percent Change From Baseline In Liver Volume | 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. | 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). |
| Palo Alto |
| California |
| 94304 |
| United States |
| San Francisco | California | 94143-0214 | United States |
| Wilmington | Delaware | 19803 | United States |
| Chicago | Illinois | 60611-2605 | United States |
| Boston | Massachusetts | 02115 | United States |
| Buffalo | New York | 14222 | United States |
| Manhasset | New York | 11030 | United States |
| New York | New York | 10029 | United States |
| Cincinnati | Ohio | 45229 | United States |
| Philadelphia | Pennsylvania | 19104 | United States |
| Córdoba | Argentina |
| Brisbane | Australia |
| New Lambton | Australia |
| Parkville | Australia |
| Perth | Australia |
| Zagreb | Croatia |
| Olomouc | Czechia |
| Prague | Czechia |
| Paris | France |
| Vandœuvre-lès-Nancy | France |
| Freiburg im Breisgau | Germany |
| Mainz | Germany |
| Munich | Germany |
| Bergamo | Italy |
| Genoa | Italy |
| Padova | Italy |
| Tokyo | Japan |
| Tottori | Japan |
| Mexico City | Mexico |
| Warsaw | Poland |
| Moscow | Russia |
| Elche | Spain |
| Madrid | Spain |
| Oviedo | Spain |
| Ankara | Turkey (Türkiye) |
| Izmir | Turkey (Türkiye) |
| Cambridge | United Kingdom |
| London | United Kingdom |
| Plymouth | United Kingdom |
| 34774639 | Derived | Burton BK, Feillet F, Furuya KN, Marulkar S, Balwani M. Sebelipase alfa in children and adults with lysosomal acid lipase deficiency: Final results of the ARISE study. J Hepatol. 2022 Mar;76(3):577-587. doi: 10.1016/j.jhep.2021.10.026. Epub 2021 Nov 10. |
| 24993530 | Derived | Valayannopoulos V, Malinova V, Honzik T, Balwani M, Breen C, Deegan PB, Enns GM, Jones SA, Kane JP, Stock EO, Tripuraneni R, Eckert S, Schneider E, Hamilton G, Middleton MS, Sirlin C, Kessler B, Bourdon C, Boyadjiev SA, Sharma R, Twelves C, Whitley CB, Quinn AG. Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency. J Hepatol. 2014 Nov;61(5):1135-42. doi: 10.1016/j.jhep.2014.06.022. Epub 2014 Jun 30. |
Double-blind Period: IV infusions of placebo administered qow.
| FG002 | Open-label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| FG003 | Open-label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| Received at Least 1 Dose of Study Drug |
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| COMPLETED |
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| NOT COMPLETED |
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| Open-label Period |
|
|
All participants who received at least 1 dose (or any portion of a dose) of sebelipase alfa or placebo in the Double-blind Period.
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| ID | Title | Description |
|---|---|---|
| BG000 | Double-Blind Sebelipase Alfa | Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow. |
| BG001 | Double-Blind Placebo | Double-blind Period: IV infusions of placebo administered qow. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Double-blind Period | Mean | Standard Deviation | years |
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| Age, Continuous | Open-label Period | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Percentage Of Participants Achieving Alanine Aminotransferase Normalization | 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. | 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). | Posted | Number | percentage of participants | 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) |
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| Secondary | Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C) | 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. | 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). | Posted | Mean | Standard Deviation | percent change | 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). |
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| Secondary | Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C) | 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. | 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). | Posted | Mean | Standard Deviation | percent change | 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). |
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| Secondary | Percentage Of Participants Achieving Aspartate Aminotransferase Normalization | 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. | 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). | Posted | Number | percentage of participants | 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). |
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| Secondary | Percent Change From Baseline In Triglycerides | 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. | 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). | Posted | Mean | Standard Deviation | percent change | 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). |
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| Secondary | Percent Change From Baseline In High-density Lipoprotein Cholesterol (HDL-C) | 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. | 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). | Posted | Mean | Standard Deviation | percent change | 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). |
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| Secondary | Percent Change From Baseline In Liver Fat Content | 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. | 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). | Posted | Mean | Standard Deviation | percent change | 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). |
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| Secondary | Participants With Improvement In Liver Histopathology (Decrease Of > 5% In Hepatic Steatosis Score) | 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. | 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). | Posted | Number | participants | Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline up to Week 52. |
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| Secondary | Percent Change From Baseline In Liver Volume | 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. | 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). | Posted | Mean | Standard Deviation | percent change | 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). |
|
In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa).
Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Double-blind Sebelipase Alfa | Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow. | 0 | 36 | 2 | 36 | 31 | 36 |
| EG001 | Double-blind Placebo | Double-blind Period: IV infusions of placebo administered qow. | 0 | 30 | 1 | 30 | 28 | 30 |
| EG002 | Open-label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. | 0 | 36 | 6 | 36 | 35 | 36 |
| EG003 | Open-label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. | 0 | 30 | 5 | 30 | 29 | 30 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eyelid oedema | Eye disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Infusion-related reaction | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment | Procedural pain after liver biopsy unrelated to study drug. |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Patellofemoral pain syndrome | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Plantar fasciitis | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Hepatic cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (15.1) | Systematic Assessment |
| |
| Metastases to lung | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (15.1) | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Hepatic encephalopathy | Nervous system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Hyperaemia | Vascular disorders | MedDRA (15.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Procedural Pain | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment | Procedural pain after liver biopsy deemed by the Investigator to be unrelated to study drug. |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA (15.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Vulvovaginitis | Infections and infestations | MedDRA (15.1) | Systematic Assessment | Adverse event occurring in female participants only. |
|
| Otitis media | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | MedDRA (15.1) | Systematic Assessment |
| |
| Odynophagia | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Eructation | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA (15.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA (15.1) | Systematic Assessment | Adverse event occurring in female participants only. |
|
| Blood cholesterol increased | Investigations | MedDRA (15.1) | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA (15.1) | Systematic Assessment |
| |
| Cardiac murmur | Investigations | MedDRA (15.1) | Systematic Assessment |
| |
| Eosinophil count increased | Investigations | MedDRA (15.1) | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Autism spectrum disorder | Psychiatric disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (15.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (15.1) | Systematic Assessment |
|
Institution and/or Principal Investigator may publish or make an individual oral or written presentation of publication of study results either 1) after Alexion publishes the study data or 2) 18 months after the completion of the study, whichever comes first.
Alexion reserves the right to review and comment on a communication at least 30 days prior to submission with a provision to extend the review for an additional 60 days where information is relevant to a patent application.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 855-752-2356 | clinicaltrials@alexion.com |
| ID | Term |
|---|---|
| D015223 | Wolman Disease |
| D016464 | Lysosomal Storage Diseases |
| D015217 | Cholesterol Ester Storage Disease |
| ID | Term |
|---|---|
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000603932 | Sebelipase alfa |
Not provided
Not provided
Not provided
| Withdrawal by Subject |
|
| Discontinued by Sponsor |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
Double-blind Period: IV infusions of placebo administered qow.
| OG002 | Open-label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|
Double-blind Period: IV infusions of placebo administered qow.
| OG002 | Open-Label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-Label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|
| OG001 |
| Double-Blind Placebo |
Double-blind Period: IV infusions of placebo administered qow. |
| OG002 | Open-Label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-Label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|
| OG002 | Open-Label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-Label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|
| OG002 | Open-Label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-Label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|
| OG002 | Open-Label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-Label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|
| OG002 |
| Open-Label Sebelipase Alfa/Sebelipase Alfa |
Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-Label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|
| OG002 | Open-Label Sebelipase Alfa/Sebelipase Alfa | Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
| OG003 | Open-Label Placebo/Sebelipase Alfa | Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period. |
|
|
|