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The mission of our clinical research is to foster discoveries in the novel therapies of inflammatory bowel diseases, gastrointestinal and pancreatic cancer, hepatitis, and other gastrointestinal disorders. Our doctors are committed to providing the best care and management of diseases while testing cutting-edge therapies.
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Ongoing clinical trials for lysosomal acid lipase deficiency (LALD): Actively recruiting
Lysosomal acid lipase deficiency (LALD)
A Phase III Multicenter Study of SBC-102 (sebelipase alfa) in patients with Lysosomal Acid Lipase (LAL) Deficiency
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 http://www.clinicaltrials.gov/ct2/show/NCT01757184 or http://www.clinicaltrials.gov (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.
Kathleen Corey, MD, MPH
Kathleen Corey; 617-724-1685
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