A research study to look at the safety, effectiveness and the 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

An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency - SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency

Status

Active, not recruiting

Phases

Phase 1Phase 2Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-000032-28-GB

Enrollment

Registered

2011-01-17

Start date

2011-04-18

Completion date

Unknown

Last updated

2018-09-10

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

Conditions

Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). 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 stora

Interventions

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 Spons

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, and provides written informed consent/permission prior to any study procedures being performed 2. Male or female child with a documented decreased LAL activity relative to the normal range of the lab performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis of LAL Deficiency 3. Growth failure with onset before 6 months of age, as defined in the protocol 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 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Clinically important concurrent disease or co-morbidities which, in the opinion of the Investigator and Sponsor, would interfere with study participation, including, but not restricted to, congestive heart failure, ongoing circulatory collapse requiring inotropic support, acute or chronic renal failure, additional severe congenital abnormality, or other extenuating circumstances such as life-threatening under nutrition or rapidly progressive liver disease. 2. Subject is >24 months of age. (Note: Subjects >8 months of age on the date of first infusion will not be eligible for the primary efficacy analysis.) 3. Has received an investigational medicinal product other than SBC-102 within 14 days prior to the first dose of SBC-102 in this study. 4. Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation. 5. Previous hematopoietic stem cell or liver transplant. 6. Known hypersensitivity to eggs.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective of the study is to evaluate the effect of SBC-102 therapy on survival at 12 months of age in children with growth failure due to LAL Deficiency.;Secondary Objective: (1) to evaluate the safety and tolerability of SBC-102 in children with growth failure due to LAL Deficiency; (2) to evaluate the effect of SBC-102 therapy on survival beyond 12 months of age in children with growth failure due to LAL Deficiency; (3) to evaluate the effects of SBC-102 on growth parameters in children with growth failure due to LAL Deficiency; (4) to evaluate the effects of SBC-102 on hepatomegaly, splenomegaly, and liver function in children with growth failure due to LAL Deficiency; (5) to determine the effects of SBC 102 on hematological parameters in children with growth failure due to LAL Deficiency; and (6) to characterize the PK of SBC 102 delivered by intravenous (IV) infusion.;Primary end point(s): Efficacy: proportion of subjects surviving to 12 months of age;Timepoint(s) of evaluation of this end point: continuously, see protocol	—

Measure

Time frame

Main Objective: The primary objective of the study is to evaluate the effect of SBC-102 therapy on survival at 12 months of age in children with growth failure due to LAL Deficiency.;Secondary Objective: (1) to evaluate the safety and tolerability of SBC-102 in children with growth failure due to LAL Deficiency; (2) to evaluate the effect of SBC-102 therapy on survival beyond 12 months of age in children with growth failure due to LAL Deficiency; (3) to evaluate the effects of SBC-102 on growth parameters in children with growth failure due to LAL Deficiency; (4) to evaluate the effects of SBC-102 on hepatomegaly, splenomegaly, and liver function in children with growth failure due to LAL Deficiency; (5) to determine the effects of SBC 102 on hematological parameters in children with growth failure due to LAL Deficiency; and (6) to characterize the PK of SBC 102 delivered by intravenous (IV) infusion.;Primary end point(s): Efficacy: proportion of subjects surviving to 12 months of age;Timepoint(s) of evaluation of this end point: continuously, see protocol

—

Secondary

Measure	Time frame
Secondary end point(s): Efficacy: (1) changes from baseline in percentiles and/or z-scores for WFA, WFL/WFH, and LFA/HFA and the corresponding growth status indicators of underweight, wasting, and stunting, as well as changes from baseline in z scores for head circumference-for-age (HCFA) and mid-upper arm circumference-for-age (MUACFA); (2) changes from baseline in aspartate aminotransferase (AST) and ALT; (3) normalization of hemoglobin levels without requirement for blood transfusion; and (4) change from baseline in serum ferritin. Safety: incidence of AEs, SAEs, and IRRs; changes from baseline clinical laboratory tests; changes in vital signs during and post-infusion relative to pre-infusion values; physical examination findings; use of concomitant medications/therapies; and characterization of ADAs Pharmacokinetics: maximum observed serum concentration (Cmax) and apparent serum clearance (CL), as data permit. The impact of ADAs on SBC-102 PK will also be explored.;Timepoint(s) of evaluation of this end point: continuously, see protocol	—

Measure

Time frame

Secondary end point(s): Efficacy: (1) changes from baseline in percentiles and/or z-scores for WFA, WFL/WFH, and LFA/HFA and the corresponding growth status indicators of underweight, wasting, and stunting, as well as changes from baseline in z scores for head circumference-for-age (HCFA) and mid-upper arm circumference-for-age (MUACFA); (2) changes from baseline in aspartate aminotransferase (AST) and ALT; (3) normalization of hemoglobin levels without requirement for blood transfusion; and (4) change from baseline in serum ferritin. Safety: incidence of AEs, SAEs, and IRRs; changes from baseline clinical laboratory tests; changes in vital signs during and post-infusion relative to pre-infusion values; physical examination findings; use of concomitant medications/therapies; and characterization of ADAs Pharmacokinetics: maximum observed serum concentration (Cmax) and apparent serum clearance (CL), as data permit. The impact of ADAs on SBC-102 PK will also be explored.;Timepoint(s) of evaluation of this end point: continuously, see protocol

—

Countries

France, Germany, Ireland, Italy, Taiwan, United Kingdom, United States

Contacts

Public ContactAmy Simonds

Synageva BioPharma Corp.

clinicaltrials@synageva.com0017813579900

Outcome results

None listed

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