Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Study Identifier:
LAL-CL08
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other
Available Documents
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: Sebelipase Alfa
Date
Jun 2014 - Oct 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 8 Months
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Participant's parent or legal guardian (if applicable) consent to participation in the study
- Confirmation of documented decreased LAL activity relative to the normal range of the lab performing the assay or confirmation of LAL-D diagnosis as determined by a Sponsor-approved central laboratory
- Substantial clinical concerns, in the opinion of Investigator and Sponsor, of rapid disease progression requiring urgent medical intervention including, but not restricted to the following:
- Marked abdominal distension and hepatomegaly
- Failure to thrive
- Disturbance of coagulation
- Severe anemia
- Sibling with rapidly progressive course of LAL-D
Exclusion Criteria
- Clinically important concurrent disease
- Participant was \> 8 months of age at the time of first dosing
- Participant received an investigational medicinal product other than sebelipase alfa within 14 days prior to the first dose of sebelipase alfa in this study
- Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation
- Previous hematopoietic stem cell or liver transplant
- Known hypersensitivity to eggs
Sex
Female & Male
Age
N/A - 8 Months
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: Sebelipase Alfa
Date
Jun 2014 - Oct 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 8 Months years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Participant's parent or legal guardian (if applicable) consent to participation in the study
- Confirmation of documented decreased LAL activity relative to the normal range of the lab performing the assay or confirmation of LAL-D diagnosis as determined by a Sponsor-approved central laboratory
- Substantial clinical concerns, in the opinion of Investigator and Sponsor, of rapid disease progression requiring urgent medical intervention including, but not restricted to the following:
- Marked abdominal distension and hepatomegaly
- Failure to thrive
- Disturbance of coagulation
- Severe anemia
- Sibling with rapidly progressive course of LAL-D
Exclusion Criteria
- Clinically important concurrent disease
- Participant was \> 8 months of age at the time of first dosing
- Participant received an investigational medicinal product other than sebelipase alfa within 14 days prior to the first dose of sebelipase alfa in this study
- Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation
- Previous hematopoietic stem cell or liver transplant
- Known hypersensitivity to eggs
Protocol Summary
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
Trial Locations
Location
Status
Location
Phoenix, Arizona, United States, 85016
Status
N/A
Location
Kuopio, Finland
Status
N/A
Location
Naples, Italy
Status
N/A
Location
Birmingham, United Kingdom
Status
N/A
Location
Manchester, United Kingdom
Status
N/A