Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02193867
• Date of registration: 07/07/2014
• Prospective Registration: No
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
• Scientific title: A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
• Date of first enrolment: June 6, 2014
• Target sample size: 10
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02193867
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Finland, Italy, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Participant's parent or legal guardian (if applicable) consent to participation in the
study

2. 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

3. 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:

1. Clinically important concurrent disease

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

3. Participant received an investigational medicinal product other than sebelipase alfa
within 14 days prior to the first dose of sebelipase alfa 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



Age minimum: N/A
Age maximum: 8 Months
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 through Month 37]

Secondary Outcome(s)

Change From Baseline In Serum Ferritin At Month 12, 24, And 36 [Time Frame: Baseline, Month 12, Month 24, and Month 36]

Number Of Participants With Stunting, Wasting, Or Underweight At Baseline, 12, 24, And 36 Months [Time Frame: Baseline to Month 12, Month 24, and Month 36]

Percentage Of Participants Surviving To 12, 18, 24, And 36 Months Of Age [Time Frame: Baseline through Month 12, Month 18, Month 24, and Month 36]

Number Of Participants Achieving And Maintaining Transfusion-free Hemoglobin Normalization (TFHN) [Time Frame: Baseline through Month 36]

Change From Baseline In Percentiles For Weight For Age (WFA) At 12, 24, And 36 Months [Time Frame: Baseline, Month 12, Month 24, and Month 36]

Change From Baseline In Serum Transaminases (ALT And AST) At Month 12, 24, And 36 [Time Frame: Baseline, Month 12, Month 24, and Month 36]

Median Age At Death [Time Frame: Baseline through Month 36]

Secondary ID(s)

LAL-CL08

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 18/11/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02193867