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This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysfunction due to lysosomal acid lipase (LAL) deficiency.
Participants who successfully received all 4 doses of sebelipase alfa in Study LAL-CL01 and opted to continue treatment in the extension study underwent screening assessments to determine study eligibility. Eligible participants initiated treatment in the extension study at least 4 weeks after their last dose of sebelipase alfa in Study LAL-CL01. This extension study consisted of a treatment period of up to 5 years, and a follow-up period of approximately 30 days after the last dose of sebelipase alfa.
Cholesteryl ester storage disease (CESD) is the late onset phenotype for LAL deficiency, a lysosomal storage disorder, which also has an early onset phenotype that primarily affects infants. CESD can present in childhood but often goes unrecognized until adulthood when the underlying pathology is advanced. Many of the signs and symptoms are common to patients with other liver conditions.
CESD is an autosomal recessive genetic condition and is characterized by hepatomegaly, persistently abnormal liver function tests (LFTs) and type II hyperlipidemia. Splenomegaly and evidence of mild hypersplenism may affect some patients. Untreated, CESD may lead to fibrosis, cirrhosis, liver failure and death.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open-Label Sebelipase Alfa | Experimental | Participants were administered sebelipase alfa once weekly (qw) as an intravenous (IV) infusion at the same dose received in Study LAL-CL01 (0.35, 1, or 3 milligrams per kilogram [mg/kg]) for 4 weeks. After the initial 4 qw doses, participants transitioned to dosing every other week (qow) at either 1 mg/kg (participants who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (participants who initiated dosing at 3 mg/kg qw). Subsequent modifications to the dose and dosing frequency were permitted for individual participants based on observed safety, tolerability, and clinical response to treatment. Participants could continue to receive treatment with sebelipase alfa for up to 5 years. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| sebelipase alfa | Drug | Sebelipase alfa is a recombinant human lysosomal acid lipase. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number Of Participants Reporting TEAEs And IARs | 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). | From after first dose administration post-Baseline through EOS during study LAL-CL04 |
| Measure | Description | Time Frame |
|---|---|---|
| Changes From Baseline In ALT And AST | 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. | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Eureka | California | 95501-320 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23348766 | Result | Balwani M, Breen C, Enns GM, Deegan PB, Honzik T, Jones S, Kane JP, Malinova V, Sharma R, Stock EO, Valayannopoulos V, Wraith JE, Burg J, Eckert S, Schneider E, Quinn AG. Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease. Hepatology. 2013 Sep;58(3):950-7. doi: 10.1002/hep.26289. Epub 2013 Mar 28. | |
| 32657505 |
| Label | URL |
|---|---|
| Alexion website | View source |
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9 participants who completed Study LAL-CL01 (received all 4 doses of sebelipase alfa) were screened for eligibility for enrollment in this extension study (LAL-CL04). 8 participants met all enrollment criteria and were enrolled. 1 participant who required a liver transplant no longer met the entry criteria.
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| ID | Title | Description |
|---|---|---|
| FG000 | Open-Label Sebelipase Alfa | Participants were administered sebelipase alfa once weekly (qw) as an intravenous (IV) infusion at the same dose received in Study LAL-CL01 (0.35, 1, or 3 milligrams per kilogram [mg/kg]) for 4 weeks. After the initial 4 qw doses, participants transitioned to dosing every other week (qow) at either 1 mg/kg (participants who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (participants who initiated dosing at 3 mg/kg qw). Subsequent modifications to the dose and dosing frequency were permitted for individual participants based on observed safety, tolerability, and clinical response to treatment. Participants could continue to receive treatment with sebelipase alfa for up to 5 years. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 5, 2016 | Jun 18, 2018 |
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| Changes From Baseline In Liver Volume | 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. | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| Changes From Baseline In Liver Fat Content | 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. | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, and Week 260 |
| Changes From Baseline In GGT And ALP | 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. | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| Changes From Baseline In Serum Lipids | 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. | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| Changes From Baseline In Serum Ferritin | 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. | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| Changes From Baseline In Hs-CRP | 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. | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| Sacramento |
| California |
| 95817 |
| United States |
| San Francisco | California | 94143 | United States |
| Minneapolis | Minnesota | 55455 | United States |
| New York | New York | 10029 | United States |
| Greater Sudbury | Ontario | Canada |
| Prague | Czechia |
| Paris | France |
| Leeds | United Kingdom |
| Salford | United Kingdom |
| Derived |
| Malinova V, Balwani M, Sharma R, Arnoux JB, Kane J, Whitley CB, Marulkar S, Abel F. Sebelipase alfa for lysosomal acid lipase deficiency: 5-year treatment experience from a phase 2 open-label extension study. Liver Int. 2020 Sep;40(9):2203-2214. doi: 10.1111/liv.14603. Epub 2020 Aug 9. |
| Received Study Drug in Extension Study |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
All participants who received any amount of study drug in the extension study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Open-Label Sebelipase Alfa | Participants were administered sebelipase alfa qw as an IV infusion at the same dose received in Study LAL-CL01 (0.35, 1, or 3 mg/kg) for 4 weeks. After the initial 4 qw doses, participants transitioned to dosing qow at either 1 mg/kg (participants who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (participants who initiated dosing at 3 mg/kg qw). Subsequent modifications to the dose and dosing frequency were permitted for individual participants based on observed safety, tolerability, and clinical response to treatment. Participants could continue to receive treatment with sebelipase alfa for up to 5 years. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| |||||||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
|
| 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 | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number Of Participants Reporting TEAEs And IARs | 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). | Safety Analysis Set: All participants who received any amount of study drug in the extension study. | Posted | Count of Participants | Participants | From after first dose administration post-Baseline through EOS during study LAL-CL04 |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline In ALT And AST | 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. | 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. | Posted | Mean | Standard Deviation | units (U)/liter (L) | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline In Liver Volume | 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. | 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. | Posted | Mean | Standard Deviation | Multiples of Normal (MN) | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline In Liver Fat Content | 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. | 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. | Posted | Mean | Standard Deviation | percentage fat fraction | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, and Week 260 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline In GGT And ALP | 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. | 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. | Posted | Mean | Standard Deviation | U/L | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline In Serum Lipids | 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. | 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. | Posted | Mean | Standard Deviation | mg/dL | Baseline, Week 10 or 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline In Serum Ferritin | 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. | 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. | Posted | Mean | Standard Deviation | microgram (µg)/L | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Changes From Baseline In Hs-CRP | 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. | 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. | Posted | Mean | Standard Deviation | mg/L | Baseline, Week 12, Week 24, Week 52, Week 104, Week 156, Week 208, Week 260, and EOS |
|
|
From after first dose administration post-Baseline through EOS during study LAL-CL04
Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
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 | Open-Label Sebelipase Alfa | Participants were administered sebelipase alfa qw as an IV infusion at the same dose received in Study LAL-CL01 (0.35, 1, or 3 mg/kg) for 4 weeks. After the initial 4 qw doses, participants transitioned to qow dosing at either 1 mg/kg (participants who initiated treatment at 0.35 or 1 mg/kg qw) or 3 mg/kg (participants who initiated dosing at 3 mg/kg qw). Subsequent modifications to the dose and dosing frequency were permitted for individual participants based on observed safety, tolerability, and clinical response to treatment. Participants could continue to receive treatment with sebelipase alfa for up to 5 years. | 0 | 8 | 1 | 8 | 8 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cholecystitis | Hepatobiliary disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (20.0) | Systematic Assessment | Cholecystectomy for cholelithiasis |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bradycardia | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Blepharitis | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Conjunctivitis | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Keratitis | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Occular hyperaemia | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Faeces soft | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Malocclusion | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Chills | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Granulomatous liver disease | Hepatobiliary disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Hepatic pain | Hepatobiliary disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Bacterial vaginosis | Infections and infestations | MedDRA (20.0) | Systematic Assessment | Affects only female participants |
|
| Cellulitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Ear lobe infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Infected dermal cyst | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Rash pustular | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Subcutaneous abscess | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Cartilage injury | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Iliotibial band syndrome | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Procedural dizziness | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA (20.0) | Systematic Assessment |
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| Blood creatinine increased | Investigations | MedDRA (20.0) | Systematic Assessment |
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| C-reactive protein increased | Investigations | MedDRA (20.0) | Systematic Assessment |
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| Eosinophil count increased | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| Low density lipoprotein increased | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| Lymphocyte count increased | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| Mean cell volume increased | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| Prothrombin time abnormal | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| Vitamin B12 decreased | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Limb mass | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Neck mass | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Spinal pain | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Lipoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
| |
| Oral neoplasm benign | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
| |
| Amnesia | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Clonic convulsion | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dyskinesia | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Laryngeal oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dermal cyst | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Ephelides | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pigmentation disorder | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Skin mass | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Hyperaemia | Vascular disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA (20.0) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | Alexion Pharmaceuticals Inc. | 475-230-2596 | clinicaltrials@alexion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 16, 2017 | Jun 18, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| D015217 | Cholesterol Ester Storage Disease |
| D015223 | Wolman Disease |
| D016464 | Lysosomal Storage Diseases |
| 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 |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000603932 | Sebelipase alfa |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| United States |
|
| United Kingdom |
|
| France |
|
| Title | Measurements |
|---|
|
| TEAEs Leading to Study Discontinuation |
|
| Units |
|---|
| Counts |
|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
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| Participants |
|
|
| Participants |
|
|