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

This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease [CESD]).

Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL-D other than supportive care. Enzyme replacement therapy may be a potential new treatment option for LAL-D participants.

Stanford is currently accepting patients for this trial.

Stanford Investigator(s):

Intervention(s):

  • drug: Placebo
  • drug: Sebelipase Alfa

Eligibility


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.

Ages Eligible for Study

4 Years - N/A

Genders Eligible for Study

All

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Greg Enns, MD
(650) 498-5798
I'm interested

Our research team includes physicians, residents, medical students, research assistants, and volunteers. Our research topics include medical imaging, device validation,  mobile application development, and pharmaceutical trials.  

Some of the Neuro-Opthalmic concerns we investigate include Multiple Sclerosis, Optic Neuritis, IIH, and ICP.