Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Study Identifier:
LAL-CL03
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete
Available Documents
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: Sebelipase alfa (SBC-102)
Date
May 2011 - Jan 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 24 Months
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Participant's parent or legal guardian provided written informed consent/permission prior to any study procedures.
- Male or female child with documented decreased LAL activity relative to the normal range of the laboratory performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis.
- Growth failure with onset before 6 months of age.
Exclusion Criteria
- Clinically important concurrent disease or comorbidities.
- Had received an investigational product other than sebelipase alfa within 14 days prior to the first dose.
- Participant was older than 24 months of age.
- Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplant.
- Previous hematopoietic stem cell or liver transplant.
- Known hypersensitivity to eggs.
Sex
Female & Male
Age
N/A - 24 Months
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: Sebelipase alfa (SBC-102)
Date
May 2011 - Jan 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 24 Months years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Participant's parent or legal guardian provided written informed consent/permission prior to any study procedures.
- Male or female child with documented decreased LAL activity relative to the normal range of the laboratory performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis.
- Growth failure with onset before 6 months of age.
Exclusion Criteria
- Clinically important concurrent disease or comorbidities.
- Had received an investigational product other than sebelipase alfa within 14 days prior to the first dose.
- Participant was older than 24 months of age.
- Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplant.
- Previous hematopoietic stem cell or liver transplant.
- Known hypersensitivity to eggs.
Protocol Summary
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.
Trial Locations
Location
Status
Location
Irvine, California, United States, 92697
Status
N/A
Location
Cairo, Egypt, 11771
Status
N/A
Location
Grenoble, France, 38700
Status
N/A
Location
Paris, France, 75015
Status
N/A
Location
Dublin, Ireland, 1
Status
N/A
Location
London, United Kingdom, SE1 7EH
Status
N/A
Go to page