Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01757184
• Date of registration: 17/12/2012
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency ARISE
• Scientific title: A Multicenter, Randomized, Placebo-controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency
• Date of first enrolment: January 22, 2013
• Target sample size: 66
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01757184
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Brazil, Canada, Croatia, Czech Republic, Czechia, France, Germany, Greece, Israel, Italy, Japan, Mexico, Poland, Russian Federation, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Florian Abel, MD
• Affiliation: Alexion Pharmaceuticals

Key inclusion & exclusion criteria

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.

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.



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Lysosomal Acid Lipase Deficiency

Intervention(s)

Drug: Placebo

Drug: Sebelipase Alfa

Primary Outcome(s)

Percentage Of Participants Achieving Alanine Aminotransferase Normalization [Time Frame: 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(s)

Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C) [Time Frame: 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 [Time Frame: 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) [Time Frame: 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 Low-density Lipoprotein Cholesterol (LDL-C) [Time Frame: 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) [Time Frame: 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 Fat Content [Time Frame: 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 Volume [Time Frame: 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 [Time Frame: 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 ID(s)

LAL-CL02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 18/04/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01757184