A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 66

Inclusion Criteria

1. Subject and/or subject’s parent or legal guardian understand the full nature and purpose of the study, including possible risks and side effects, and is willing and able to comply with all compulsory study procedures (including liver biopsy and magnetic resonance imaging [MRI] assessments, as applicable) and provides informed consent/permission prior to any study procedures being performed. If the subject is <18 years of age, he/she is willing to provide assent where required per local regulations and if deemed able to do so.
2. Subject is =4 years of age on the date of informed consent.
3. Deficiency of lysosomal acid lipase (LAL) enzyme activity confirmed by dried blood spot (DBS) testing at screening, based on the definition of deficiency provided by the central laboratory performing the assay.
4. ALT =1.5x ULN (based on the age- and gender-specific normal ranges of the central laboratory performing the assay) on 2 consecutive screening ALT measurements obtained at least 1 week apart.
5. Female subjects of childbearing potential must (a) have negative serum pregnancy test at screening (b) cannot be breastfeeding, and (c) must agree to use a medically acceptable method of preventing conception from the screening visit until 4 weeks after the last dose of study drug administered under this protocol. A female of childbearing potential is defined as a menarchal female who has not had a hysterectomy, bilateral oophorectomy, or medically documented ovarian failure, and who is not postmenopausal.
6. Subjects receiving lipid-lowering therapies must be on a stable dose of the medication for at least 6 weeks prior to randomization and be willing to remain on a stable dose for at least the first 32 weeks of treatment in the study.
7. Subjects receiving medications for the treatment of non-alcoholic fatty liver disease (e.g., glitazones, high-dose vitamin E, metformin, ursodeoxycholic acid [UDCA]) must be on a stable dose for at least 16 weeks prior to randomization and be willing to remain on a stable dose for at least the first 32 weeks of treatment in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 26
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 29
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1. Severe hepatic dysfunction (Child-Pugh Class C).
2. Other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation, including but not restricted to severe intercurrent illness, known causes of active liver disease other than LALD (e.g., chronic viral hepatitis, autoimmune hepatitis, alcoholic liver disease, or physician concerns about excess alcohol consumption), human immunodeficiency virus (HIV), poorly-controlled diabetes, or cancers other than non-melanoma skin cancer.
3. Previous hematopoietic or liver transplant procedure.
4. Received treatment with high-dose corticosteroids (acute or chronic) within 26 weeks prior to randomization. (Note: Subjects receiving maintenance therapy with low-dose oral, intranasal, topical, or inhaled corticosteroids are considered eligible for the study.)
5. Participated in a study employing an investigational medicinal product within 30 days prior to randomization.
6. Known hypersensitivity to eggs.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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