Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
Study Identifier:
LAL-CL06
ClinicalTrials.gov Identifier:
EudraCT Identifier:
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
Date
Jun 2014 - Dec 2017
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 8+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Participant was \>8 months of age at the time of dosing.
- Confirmation of LAL-D diagnosis as determined by the central laboratory or, for participants with prior hematopoietic stem cell transplant or liver transplant, historical enzyme activity or molecular genetic testing confirming a diagnosis of LAL-D.
- Participants \>8 months but \<4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
- Dyslipidemia
- Elevated transaminases
- Impaired growth
- Suspected malabsorption
- Other clinical manifestation of LAL-D
- Participants ≥4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
- Evidence of advanced liver disease
- Histologically confirmed disease recurrence in participants with past liver or hematopoietic transplant
- Persistent dyslipidemia
- Suspected malabsorption
- Other clinical manifestation of LAL-D
Exclusion Criteria
- Participant had known causes of active liver disease other than LAL-D, which had not been adequately treated.
- Participant received a hematopoietic stem cell or liver transplant \<2 years from the time of dosing.
- Participant with co-morbidities other than complications due to LAL-D, which were irreversible or associated with a high mortality risk within 6 months or would interfere with study compliance or data interpretation.
Sex
Female & Male
Age
8+ years
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: Sebelipase Alfa
Date
Jun 2014 - Dec 2017
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 8+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Participant was \>8 months of age at the time of dosing.
- Confirmation of LAL-D diagnosis as determined by the central laboratory or, for participants with prior hematopoietic stem cell transplant or liver transplant, historical enzyme activity or molecular genetic testing confirming a diagnosis of LAL-D.
- Participants \>8 months but \<4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
- Dyslipidemia
- Elevated transaminases
- Impaired growth
- Suspected malabsorption
- Other clinical manifestation of LAL-D
- Participants ≥4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
- Evidence of advanced liver disease
- Histologically confirmed disease recurrence in participants with past liver or hematopoietic transplant
- Persistent dyslipidemia
- Suspected malabsorption
- Other clinical manifestation of LAL-D
Exclusion Criteria
- Participant had known causes of active liver disease other than LAL-D, which had not been adequately treated.
- Participant received a hematopoietic stem cell or liver transplant \<2 years from the time of dosing.
- Participant with co-morbidities other than complications due to LAL-D, which were irreversible or associated with a high mortality risk within 6 months or would interfere with study compliance or data interpretation.
Protocol Summary
This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).
Trial Locations
Location
Status
Location
Chicago, Illinois, United States, 60611
Status
N/A
Location
Shreveport, Louisiana, United States, 71103
Status
N/A
Location
Cincinnati, Ohio, United States, 45229
Status
N/A
Location
Westmead, Australia, NSW 2145
Status
N/A
Location
Brussels, Belgium, 1200
Status
N/A
Location
São Paulo, Brazil, 04024-002
Status
N/A
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