Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02112994
• Date of registration: 20/03/2014
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
• Scientific title: A Multi-Center, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency
• Date of first enrolment: June 24, 2014
• Target sample size: 31
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02112994
• Study type: Interventional
• Study design: Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Brazil, Canada, Croatia, Denmark, Germany, Italy, Mexico, Netherlands, Russian Federation, Spain, Turkey, United Kingdom, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

1. Participant was >8 months of age at the time of dosing.

2. 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.

3. 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

4. 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

Key Exclusion Criteria:

1. Participant had known causes of active liver disease other than LAL-D, which had not
been adequately treated.

2. Participant received a hematopoietic stem cell or liver transplant <2 years from the
time of dosing.

3. 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.



Age minimum: 8 Months
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Lysosomal Acid Lipase Deficiency

Intervention(s)

Drug: Sebelipase Alfa

Primary Outcome(s)

Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) [Time Frame: Screening, Week 144]

Secondary Outcome(s)

Participants Testing Positive For Anti-drug Antibodies (ADAs) [Time Frame: Week 144]

Shift In Child-Pugh Status From Baseline To Week 144 [Time Frame: Baseline, Week 144]

Percent Change In Body Mass Index (BMI)-For-Age Percentile From Baseline To Week 144 In Pediatric Participants [Time Frame: Baseline, Week 144]

Percent Change In Serum Lipids From Baseline To Week 144 [Time Frame: Baseline, Week 144]

Secondary ID(s)

2011-004287-30

LAL-CL06

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 15/01/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02112994