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Ongoing clinical trials for lysosomal acid lipase deficiency (LALD): Actively recruiting


Lysosomal acid lipase deficiency (LALD)

Study title:

A Phase III Multicenter Study of SBC-102 (sebelipase alfa) in patients with Lysosomal Acid Lipase (LAL) Deficiency

Description of study:

Lysosomal acid lipase deficiency (LALD) is a genetic disease characterized by abnormal fat accumulation in many parts of the body due to a decrease in activity of the enzyme lysosomal acid lipase (LAL). The late onset form of the disease occurs in children and adults and can cause cirrhosis and dyslipidemia. Late onset LALD is characterized by an enlarged liver, abnormal liver function tests (LFTs) and elevated cholesterol levels.

The ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) clinical study will evaluate the safety and efficacy of sebelipase alfa (SBC-102, recombinant human LAL) administered every other week as an intravenous infusion in patients with late onset LALD.

Details of the study are available at or (search term “sebelipase alfa”).

Basic eligibility criteria:

Patients diagnosed with suspected LALD can be screened at the MGH Fatty Liver Clinic. Those with LALD may be eligible for enrollment.

Principal Investigator:

Kathleen Corey, MD, MPH


Kathleen Corey; 617-724-1685

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