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

Phase 1

• Conditions: Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).; MedDRA version: 13.1Level: SOCClassification code 10027433Term: Metabolism and nutrition disordersSystem Organ Class: 10027433 - Metabolism and nutrition disorders; MedDRA version: 13.1Level: HLGTClassification code 10021605Term: Inborn errors of metabolismSystem Organ Class: 10027433 - Metabolism and nutrition disorders; MedDRA version: 13.1Level: HLTClassification code 10024579Term: Lysosomal storage disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2011-000032-28-FR
• Lead Sponsor: Synageva Biopharma Corp.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-04-11
• Last Update Posted: 12 March 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 8

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 by:
- crossing 2 major centile lines on standard weight for age curves over time
OR
- less than 10th percentile weight for age and falling from the curve
OR
- loss of = 5% of birth weight
Are the trial subjects under 18? yes
Number of subjects for this age range: 8
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

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 that in the opinion of the investigator would interfere with study participation

2. Extreme prematurity (<36 weeks gestational age at birth)

3. Subject is older than 24 months of age

4. Has received an investigational medicinal product within 14 days prior to the first dose

5. Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation

6. Previous hematopoietic stem cell transplant

7. Anticipated life expectancy < 2 weeks due to progression of LAL Deficiency

8. Known hypersensitivity to eggs

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety and tolerability of SBC-102 in children with growth failure due to LAL Deficiency.;Secondary Objective: To determine the effect of SBC-102 on growth;Primary end point(s): Treatment-emergent AEs and changes in vital signs, physical examination findings, clinical laboratory tests, use of concomitant medications, and anti-drug antibodies between Day 0 and Day 105;Timepoint(s) of evaluation of this end point: All study visits.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Change in weight and length from Day 0 to Day 135;Timepoint(s) of evaluation of this end point: Every week between Day 0 and Day 135.