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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01757184




Registration number
NCT01757184
Ethics application status
Date submitted
17/12/2012
Date registered
28/12/2012
Date last updated
29/12/2020

Titles & IDs
Public title
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
Scientific title
A Multicenter, Randomized, Placebo-controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency
Secondary ID [1] 0 0
LAL-CL02
Universal Trial Number (UTN)
Trial acronym
ARISE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Lysosomal Acid Lipase Deficiency 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sebelipase Alfa
Treatment: Drugs - Placebo

Experimental: Double-blind Sebelipase Alfa - Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow for 20 weeks.

Placebo comparator: Double-blind Placebo - Double-blind Period: IV infusions of matched placebo administered qow for 20 weeks.

Experimental: Open-label Sebelipase Alfa/Sebelipase Alfa - 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.

Experimental: 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. Dose modifications were permitted during the Open-label Period.


Treatment: Drugs: Sebelipase Alfa
IV infusions of sebelipase alfa

Treatment: Drugs: Placebo
IV infusions of matched placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage Of Participants Achieving Alanine Aminotransferase Normalization
Timepoint [1] 0 0
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)
Secondary outcome [1] 0 0
Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C)
Timepoint [1] 0 0
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).
Secondary outcome [2] 0 0
Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C)
Timepoint [2] 0 0
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).
Secondary outcome [3] 0 0
Percentage Of Participants Achieving Aspartate Aminotransferase Normalization
Timepoint [3] 0 0
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).
Secondary outcome [4] 0 0
Percent Change From Baseline In Triglycerides
Timepoint [4] 0 0
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).
Secondary outcome [5] 0 0
Percent Change From Baseline In High-density Lipoprotein Cholesterol (HDL-C)
Timepoint [5] 0 0
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).
Secondary outcome [6] 0 0
Percent Change From Baseline In Liver Fat Content
Timepoint [6] 0 0
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).
Secondary outcome [7] 0 0
Participants With Improvement In Liver Histopathology (Decrease Of > 5% In Hepatic Steatosis Score)
Timepoint [7] 0 0
Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline up to Week 52.
Secondary outcome [8] 0 0
Percent Change From Baseline In Liver Volume
Timepoint [8] 0 0
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).

Eligibility
Key inclusion criteria
* Participant and/or participant's parent or legal guardian provided informed consent.
* Participant was = 4 years of age on the date of informed consent.
* Deficiency of LAL enzyme activity confirmed by dried blood spot testing at screening.
* Alanine aminotransferase = 1.5x upper limit of normal on 2 consecutive screening measurements obtained at least 1 week apart.
* Female participants of childbearing potential must not have been pregnant or breastfeeding and must have agreed to use a medically acceptable method of preventing contraception from screening until 4 weeks after the last dose of study drug.
* Participant receiving lipid-lowering therapies must have been on a stable dose of the medication for at least 6 weeks prior to randomization and was willing to remain on a stable dose for at least the first 32 weeks of treatment in the study.
* Participant receiving medications for the treatment of nonalcoholic fatty liver disease must have been on a stable dose for at least 16 weeks prior to randomization and was willing to remain on a stable dose for at least the first 32 weeks of treatment in the study.
Minimum age
4 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Severe hepatic dysfunction (Child-Pugh Class C).
* Other medical conditions or comorbidities, which, in the opinion of the Investigator, would have interfered with study compliance or data interpretation.
* Previous hematopoietic or liver transplant procedure.
* Received treatment with high-dose corticosteroids (acute or chronic) within 26 weeks. (Note: Participants receiving maintenance therapy with low-dose oral, intranasal, topical, or inhaled corticosteroids were considered eligible for the study).
* Known hypersensitivity to eggs.
* Participated in a study employing an investigational medicinal product within 4 weeks prior to randomization.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/01/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
11/12/2018
Sample size
Target
Accrual to date
Final
66
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Brisbane
Recruitment hospital [2] 0 0
- New Lambton
Recruitment hospital [3] 0 0
- Parkville
Recruitment hospital [4] 0 0
- Perth
Recruitment postcode(s) [1] 0 0
- Brisbane
Recruitment postcode(s) [2] 0 0
- New Lambton
Recruitment postcode(s) [3] 0 0
- Parkville
Recruitment postcode(s) [4] 0 0
- Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Delaware
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
Argentina
State/province [9] 0 0
Córdoba
Country [10] 0 0
Croatia
State/province [10] 0 0
Zagreb
Country [11] 0 0
Czechia
State/province [11] 0 0
Olomouc
Country [12] 0 0
Czechia
State/province [12] 0 0
Prague
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
France
State/province [14] 0 0
Vandoeuvre les Nancy
Country [15] 0 0
Germany
State/province [15] 0 0
Freiburg
Country [16] 0 0
Germany
State/province [16] 0 0
Mainz
Country [17] 0 0
Germany
State/province [17] 0 0
Munich
Country [18] 0 0
Italy
State/province [18] 0 0
Bergamo
Country [19] 0 0
Italy
State/province [19] 0 0
Genoa
Country [20] 0 0
Italy
State/province [20] 0 0
Padova
Country [21] 0 0
Japan
State/province [21] 0 0
Tokyo
Country [22] 0 0
Japan
State/province [22] 0 0
Tottori
Country [23] 0 0
Mexico
State/province [23] 0 0
Mexico City
Country [24] 0 0
Poland
State/province [24] 0 0
Warsaw
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Moscow
Country [26] 0 0
Spain
State/province [26] 0 0
Elche
Country [27] 0 0
Spain
State/province [27] 0 0
Madrid
Country [28] 0 0
Spain
State/province [28] 0 0
Oviedo
Country [29] 0 0
Turkey
State/province [29] 0 0
Ankara
Country [30] 0 0
Turkey
State/province [30] 0 0
Izmir
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Cambridge
Country [32] 0 0
United Kingdom
State/province [32] 0 0
London
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Plymouth

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Florian Abel, MD
Address 0 0
Alexion Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01757184