A research study to look at the long-term safety, effectiveness and the effects on the body of a new drug, SBC-102, in adults with liver problems caused by a deficiency in the enzyme that breaks down fats. This is an extension study for those subjects who previously received treatment in study LAL-CL01.

AN OPEN LABEL MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF SBC-102 IN ADULT SUBJECTS WITH LIVER DYSFUNCTION DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY WHO PREVIOUSLY RECEIVED TREATMENT IN STUDY LAL-CL01 - Extension to LAL-CL01 in patients with liver dysfunction due to LAL deficiency

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-001513-13-GB

Enrollment

Registered

2011-08-31

Start date

2011-10-24

Completion date

Unknown

Last updated

2019-04-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare disorder, with an estimated prevalence of less than 0.2 lives per 100,000. Although a single disease, LAL Deficiency has two phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease (WD). Both forms of the disease lead to the accumulation of fats, in various tissues and cel

Interventions

Product Name: recombinant human lysosomal acid lipase Product Code: SBC-102 Pharmaceutical Form: Solution for infusion INN or Proposed INN: NA

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subject understands the full nature and purpose of the study, including possible risks and side effects, and is willing and able to comply with all study procedures and provide informed consent. 2. Subject received all 4 scheduled doses of SBC-102 in study LAL-CL01 with no life-threatening or unmanageable study drug toxicity. 3. Female subjects have a negative serum pregnancy test at screening, and are not breast-feeding. 4. Female subjects of childbearing potential are willing and able to use a highly effective and approved contraceptive method(s) from the date of informed consent until 30 days after last dose of IMP. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 9 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: 1. Clinically significant concurrent disease, serious inter-current illness, concomitant medications, or other extenuating circumstances that, in the opinion of the Investigator, would interfere with study participation or the interpretation of the effects of SBC-102. 2. Clinically significant abnormal values on screening laboratory tests, other than liver function or lipid panel tests.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective of the study is to evaluate the long-term safety and tolerability of SBC-102 in subjects with liver dysfunction due to LAL Deficiency; Secondary Objective: The secondary objectives are (1) to evaluate the long-term efficacy of SBC-102 in subjects with liver dysfunction due to LAL Deficiency (2) to characterize repeat-dose pharmacokinetics of SBC-102 delivered by intravenous (IV) infusion; and (3) to determine the effect of SBC-102 on pharmacodynamic biomarkers. ; Primary end point(s): Primary safety endpoints: adverse events ; changes in vital signs , physical examination findings, 12-lead electrocardiogram (ECG) parameters, and clinical laboratory tests ; anti-SBC-102 anti-drug antibodies (ADAs), and use of concomitant therapies. ;Timepoint(s) of evaluation of this end point: Ongoing, please see study protocol.	—

Measure

Time frame

Main Objective: The primary objective of the study is to evaluate the long-term safety and tolerability of SBC-102 in subjects with liver dysfunction due to LAL Deficiency; Secondary Objective: The secondary objectives are (1) to evaluate the long-term efficacy of SBC-102 in subjects with liver dysfunction due to LAL Deficiency (2) to characterize repeat-dose pharmacokinetics of SBC-102 delivered by intravenous (IV) infusion; and (3) to determine the effect of SBC-102 on pharmacodynamic biomarkers. ; Primary end point(s): Primary safety endpoints: adverse events ; changes in vital signs , physical examination findings, 12-lead electrocardiogram (ECG) parameters, and clinical laboratory tests ; anti-SBC-102 anti-drug antibodies (ADAs), and use of concomitant therapies. ;Timepoint(s) of evaluation of this end point: Ongoing, please see study protocol.

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Secondary

Measure	Time frame
Secondary end point(s): Efficacy and Pharmacodynamic endpoints Patient health outcome measures Pharmacokinetic parameters ;Timepoint(s) of evaluation of this end point: Ongoing, see study protocol	—

Countries

Czech Republic, France, United Kingdom, United States

Contacts

Public ContactDirector of Clinical Operations

Synageva Biopharma Corp

clinicaltrials@synageva.com+1 781 35799

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026