New to MyHealth?
Manage Your Care From Anywhere.
Access your health information from any device with MyHealth. You can message your clinic, view lab results, schedule an appointment, and pay your bill.
ALREADY HAVE AN ACCESS CODE?
DON'T HAVE AN ACCESS CODE?
NEED MORE DETAILS?
MyHealth for Mobile
Get the iPhone MyHealth app »
Get the Android MyHealth app »
A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency/ ARISE (Acid Lipase Replacement Investigating Safety and Efficacy)
Trial ID or NCT#
Status
RECRUITING
Purpose
This Phase 3 study will evaluate the efficacy and safety of 1 mg/kg IV infusions of SBC-102 (sebelipase alfa) administered every other week in patients with late onset lysosomal acid lipase (LAL) deficiency (cholesteryl ester storage disease). Late onset LAL Deficiency is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL Deficiency other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for LAL Deficiency patients.
Official Title
A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency
Eligibility Criteria
- - Subject and/or subject's parent or legal guardian provides informed consent
- - Subject is ≥4 years of age
- - Deficiency of LAL enzyme activity confirmed by dried blood spot (DBS) testing at screening
- - ALT ≥1.5x ULN
- - Female subjects of childbearing potential must not be pregnant or breastfeeding
- - Subjects receiving lipid-lowering therapies must be on a stable dose of the medication
- - Subjects receiving medications for the treatment of non-alcoholic fatty liver disease must be on a stable dose
- - Severe hepatic dysfunction (Child-Pugh Class C)
- - Other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere 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: Subjects receiving maintenance therapy with low-dose oral, intranasal, topical, or inhaled corticosteroids are considered eligible for the study)
- - Known hypersensitivity to eggs
- - Participated in a study employing an investigational medicinal product within 4 weeks prior to randomization
Investigator(s)
Contact us to find out if this trial is right for you.
CONTACT
Greg Enns, MD
(650) 498-5798
View on ClinicalTrials.gov
About this Clinical Trial
Your Message Will Go To
Greg Enns, MD(650) 498-5798