A research study to look at the safety, effectiveness and the long-term effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats who were previously treated with SBC-102

• Register: EUCTR
• Last refreshed on: 21 January 2013
• Main ID: EUCTR2011-002880-42-GB
• Date of registration: 12/09/2011
• Prospective Registration: Yes
• Primary sponsor: Synageva Biopharma Corp.
• Public title: A research study to look at the safety, effectiveness and the long-term effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats who were previously treated with SBC-102
• Scientific title: An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102 - Extension to LAL-CL03 in children with LAL deficiency
• Date of first enrolment: 05/12/2011
• Target sample size: 10
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-002880-42
• 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: no Placebo: no Other: no

Countries of recruitment

France, Germany, United Kingdom, United States

Contacts

• Name: Amy Simonds
• Affiliation: Synageva BioPharma Corp.

• Name: Amy Simonds
• Affiliation: Synageva BioPharma Corp.

Key inclusion & exclusion criteria

Inclusion criteria:
1. Subject’s parent or legal guardian understands the full nature and purpose of the study, including possible risks and side effects of study treatment and procedures, and provides written informed consent/permission prior to any study procedures being performed.
2. Subject completed treatment in study LAL-CL03.
OR
Subject received treatment with SBC-102 for at least 4 months under an expanded access treatment regimen,
3. Subject had no life-threatening or unmanageable study drug toxicity during treatment with SBC-102 under study LAL-CL03 or an expanded access treatment regimen.
4. Subjects must be <12 months of age unless previously enrolled in study LAL-CL03.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
1. Clinically important concurrent disease including, but not restricted to, congestive heart failure, acute or chronic renal failure, additional severe congenital abnormality, or other extenuating circumstances, including life threatening undernutrition or rapidly progressing liver disease, that in the opinion of the investigator would interfere with study participation.
2. Myeloablative preparation, or other systemic pre-transplant conditioning, for haematopoietic stem cell or liver transplantation.
3. Previous haematopoietic stem cell transplant.


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

Health Condition(s) or Problem(s) studied

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

Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).
MedDRA version: 14.0 Level: SOC Classification code 10027433 Term: Metabolism and nutrition disorders System Organ Class: 10027433 - Metabolism and nutrition disorders
MedDRA version: 14.0 Level: HLGT Classification code 10021605 Term: Inborn errors of metabolism System Organ Class: 10027433 - Metabolism and nutrition disorders
MedDRA version: 14.0 Level: HLT Classification code 10024579 Term: Lysosomal storage disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: recombinant human lysosomal acid lipase (rhLAL)
Product Code: SBC-102
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: not available
CAS Number: 1276027-63-4
Current Sponsor code: SBC-102
Other descriptive name: recombinant human lysosomal acid lipase (rhLAL)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Primary end point(s): The primary efficacy endpoint, the proportion of subjects surviving to 12 months of age, will be derived from the survivor function estimated by the product-limit method. The Kaplan-Meier survival curve will be presented and assumptions for the analysis method will be checked. An exact, two-sided CI will be calculated around the survival rate estimated by the product-limit method. All subjects enrolled in LAL-CL03 who received at least one complete infusion of IMP, regardless of whether they enter the extension study or not, will be considered in the analysis. Subjects who are alive at the time they prematurely end their participation in study LAL-CL03 or LAL-CL05, or who are alive at the end of study LAL-CL03 but do not enroll in study LAL-CL05 will be censored in the survival analysis; age of the subject on the date of the last study visit will be used in the analysis. Any deaths occurring while a subject is treated under study LAL-CL03 or LAL-CL05 will be counted. The survival estimate derived from this study will be compared to the near 0% survival rate reported in the literature for untreated subjects by 12 months of age. The product-limit analysis will be conducted in the Full Analysis Set for all subjects and, as subject numbers permit, for each dose level and other subgroups of interest.

Secondary Objective: To evaluate the long-term safety of SBC-102
To determine the effect of SBC-102 on growth
To characterize the repeat-dose pharmacokinetics of SBC 102

Main Objective: To evaluate the effect of SBC-102 therapy on overall survival at 12 months of age in children with growth failure due to LAL Deficiency.

Timepoint(s) of evaluation of this end point: continuously

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: continuously

Secondary end point(s): Survival rates at 18 months, 24 months, and other timepoints, as data permit, and median survival time.

Secondary ID(s)

LAL-CL05

Source(s) of Monetary Support

Synageva BioPharma Corp.