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.

• Register: EUCTR
• Last refreshed on: 30 April 2019
• Main ID: EUCTR2011-001513-13-GB
• Date of registration: 31/08/2011
• Prospective Registration: Yes
• Primary sponsor: Synageva Biopharma Corp.
• Public title: 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.
• Scientific title: 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
• Date of first enrolment: 24/10/2011
• Target sample size: 9
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-001513-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Czech Republic, France, United Kingdom, United States

Contacts

• Name: Director of Clinical Operations
• Affiliation: Synageva Biopharma Corp

• Name: Director of Clinical Operations
• Affiliation: Synageva Biopharma Corp

Key inclusion & exclusion 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:
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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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 cell types.
MedDRA version: 14.1 Level: SOC Classification code 10027433 Term: Metabolism and nutrition disorders System Organ Class: 10027433 - Metabolism and nutrition disorders
MedDRA version: 14.1 Level: HLGT Classification code 10021605 Term: Inborn errors of metabolism System Organ Class: 10027433 - Metabolism and nutrition disorders
MedDRA version: 14.1 Level: HLT Classification code 10024579 Term: Lysosomal storage disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: recombinant human lysosomal acid lipase
Product Code: SBC-102
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: NA
CAS Number: 1276027-63-4
Current Sponsor code: SBC-102
Other descriptive name: recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

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.

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

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.

Secondary Outcome(s)

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

Secondary ID(s)

LAL-CL04

Source(s) of Monetary Support

Synageva Biopharma Corp

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 08/07/2018
• Date Completed: 21/06/2017
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-001513-13/results