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 stora
Conditions
Sponsors
Synageva Biopharma Corp.
Eligibility
Sex/Gender
All
Inclusion 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 =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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.;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;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.;Timepoint(s) of evaluation of this end point: continuously | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Survival rates at 18 months, 24 months, and other timepoints, as data permit, and median survival time.;Timepoint(s) of evaluation of this end point: continuously | — |
Countries
France, Germany, United Kingdom, United States
Contacts
Public ContactAmy Simonds
Synageva BioPharma Corp.
Outcome results
None listed