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

• Conditions: Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).; MedDRA version: 15.1Level: HLTClassification code 10024579Term: Lysosomal storage disordersSystem Organ Class: 100000004850; MedDRA version: 15.1Level: HLGTClassification code 10021605Term: Inborn errors of metabolismSystem Organ Class: 10027433 - Metabolism and nutrition disorders; MedDRA version: 15.1Level: SOCClassification code 10027433Term: Metabolism and nutrition disordersSystem Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-06-28
• Last Update Posted: 10 June 2013

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

A subject must meet all of the following criteria to be eligible to participate in this study:
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:
weight decreasing across at least 2 of the 11 major centiles on a
standard World Health Organization (WHO) weight-for-age (WFA)
chart (1st, 3rd, 5th, 10th, 25th, 50th, 75th, 90th, 95th, 97th, 99th);
OR
body weight in kg below the 10th centile on a standard WHO WFA
chart AND no weight gain for the 2 weeks prior to screening;
OR
loss of >5% of birth weight in a child who is older than 2 weeks of
age.
*NOTE regarding Inclusion Criterion # 3: In the unusual circumstance where a child whose older (biological) sibling had a documented rapidly progressive course of LAL Deficiency with growth failure before 6 months of age, the child may be included in the study before meeting the growth failure criterion as defined above if the Investigator has substantial clinical concerns based on evidence of the rapid disease progression requiring urgent medical intervention. Inclusion under these exceptional circumstances requires submission of a written summary of the infant’s medical status for review by the Sponsor and must be approved by a written confirmation from the Sponsor after consultation with the Study Safety Committee. The subject must meet all other entry criteria as stated.
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

A subject who meets any of the following criteria will be ineligible to
participate in this study:
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.

Study & Design

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