Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
CompletedPhase 2, PHASE3Results postedLast updated 30 January 2019
What this trial tests
Phase 2, PHASE3 trial testing Sebelipase alfa (SBC-102) in Lysosomal Acid Lipase Deficiency in 9 participants. Completed in 3 January 2018.
Under 24 Months, any sex, with Lysosomal Acid Lipase Deficiency or Wolman Disease. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage Of Participants In The Primary Efficacy Analysis Set (PES) Surviving To 12 Months Of AgePrimary· Month 12
The primary efficacy endpoint was the percentage of participants (%) in the PES who survived to at least 12 months of age.
Group
Value
95% CI
Open-Label Sebelipase Alfa
67
29.9 – 92.5
Percentage Of Participants Surviving Beyond 12 Months Of AgeSecondary· Baseline to Month 18, Month 24, Month 36, Month 48, and Month 60
The percentage of participants in the PES who survived to at least 18 months of age.
Survival Through 18 Months of Age
Group
Value
95% CI
Open-Label Sebelipase Alfa
56
21.2 – 86.3
Survival Through 24 Months of Age
Group
Value
95% CI
Open-Label Sebelipase Alfa
56
21.2 – 86.3
Survival Through 36 Months of Age
Group
Value
95% CI
Open-Label Sebelipase Alfa
56
21.2 – 86.3
Survival Through 48 Months of Age
Group
Value
95% CI
Open-Label Sebelipase Alfa
56
21.2 – 86.3
Survival Through 60 Months of Age
Group
Value
95% CI
Open-Label Sebelipase Alfa
43
9.9 – 81.6
Median Age At DeathSecondary· Baseline to Week 260
Participants in the PES who died during the study, including 3 participants who died after having received between 1 and 4 infusions of sebelipase alfa and 1 participant who died after approximately 40 weeks on treatment.
Group
Value
95% CI
Open-Label Sebelipase Alfa
3.63
2.8 – 15.0
Change From Baseline To Months 12, 24, 36, 48, And 60 In Weight For Age (WFA) PercentilesSecondary· Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
Baseline is defined as the last measurement prior to the first infusion of sebelipase alfa.
Month 12
Group
Value
95% CI
Open-Label Sebelipase Alfa
7.469
5.35 – 13.77
Month 24
Group
Value
95% CI
Open-Label Sebelipase Alfa
21.787
0.91 – 30.37
Month 36
Group
Value
95% CI
Open-Label Sebelipase Alfa
14.037
-0.35 – 89.00
Month 48
Group
Value
95% CI
Open-Label Sebelipase Alfa
15.770
4.06 – 86.50
Month 60
Group
Value
95% CI
Open-Label Sebelipase Alfa
19.869
7.36 – 71.39
Number Of Participants With Stunting, Wasting, Or UnderweightSecondary· Baseline to Month 12, Month 24, Month 36, Month 48, and Month 60
The number of participants who met criteria for the following dichotomous indicators of under nutrition were reported. These indicators included the following:
* Stunting was defined as at least 2 standard deviations below the median for length-for-age/height-for-age;
* Wasting was defined as wasting at least 2 standard deviations below the median for weight-for-length/weight-for-height; and
* Underweight was defined as at least 2 standard deviations below the median for WFA.
Stunting, Baseline
Group
Value
95% CI
Open-Label Sebelipase Alfa
4
Stunting, Month 12
Group
Value
95% CI
Open-Label Sebelipase Alfa
1
Stunting, Month 24
Group
Value
95% CI
Open-Label Sebelipase Alfa
0
Stunting, Month 36
Group
Value
95% CI
Open-Label Sebelipase Alfa
0
Stunting, Month 48
Group
Value
95% CI
Open-Label Sebelipase Alfa
0
Stunting, Month 60
Group
Value
95% CI
Open-Label Sebelipase Alfa
0
Wasting, Baseline
Group
Value
95% CI
Open-Label Sebelipase Alfa
2
Wasting, Month 12
Group
Value
95% CI
Open-Label Sebelipase Alfa
0
Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum Transaminases (ALT And AST)Secondary· Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
Change from Baseline to Months 12, 24, 36, 48, and 60 for alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
ALT, Month 12
Group
Value
95% CI
Open-Label Sebelipase Alfa
-13.50
-121.00 – 12.00
ALT, Month 24
Group
Value
95% CI
Open-Label Sebelipase Alfa
-5.00
-111.00 – 228.00
ALT, Month 36
Group
Value
95% CI
Open-Label Sebelipase Alfa
-4.00
-100.00 – 107.0
ALT, Month 48
Group
Value
95% CI
Open-Label Sebelipase Alfa
-27.50
-129.00 – -2.00
ALT, Month 60
Group
Value
95% CI
Open-Label Sebelipase Alfa
-27.00
-122.00 – 2.00
AST, Month 12
Group
Value
95% CI
Open-Label Sebelipase Alfa
-43.50
-62.00 – -29.00
AST, Month 24
Group
Value
95% CI
Open-Label Sebelipase Alfa
-30.00
-49.00 – 67.00
AST, Month 36
Group
Value
95% CI
Open-Label Sebelipase Alfa
-33.00
-49.00 – 86.00
Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum FerritinSecondary· Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
The median change in serum ferritin from Baseline to Months 12, 24, 36, 48, and 60 is presented.
Month 12
Group
Value
95% CI
Open-Label Sebelipase Alfa
-294.40
-562.2 – -271.0
Month 24
Group
Value
95% CI
Open-Label Sebelipase Alfa
-239.00
-298.0 – -235.0
Month 36
Group
Value
95% CI
Open-Label Sebelipase Alfa
-262.95
-566.6 – -166.0
Month 48
Group
Value
95% CI
Open-Label Sebelipase Alfa
-268.00
-278.0 – -179.0
Month 60
Group
Value
95% CI
Open-Label Sebelipase Alfa
-213.00
-543.9 – -155.0
Number Of Participants Achieving And Maintaining Transfusion-free Hemoglobin Normalization [TFHN]Secondary· Baseline to Month 60
The number of participants achieving and maintaining TFHN are presented.
For TFHN to be achieved, the participant must a) have had 2 post-baseline measurements of hemoglobin at least 4 weeks apart that were both above the age-adjusted lower limit of normal; b) have had no known additional measurements of hemoglobin that were below the age-adjusted lower limit of normal during the (minimum) 4-week period; and c) have had no transfusions during the (minimum) 4-week period, and also no transfusions for 2 weeks prior to the first hemoglobin measurement in the (minimum) 4-week period.
For TFHN to
Achieved TFHN
Group
Value
95% CI
Open-Label Sebelipase Alfa
6
Maintained TFHN
Group
Value
95% CI
Open-Label Sebelipase Alfa
2
Adverse events — posted to ClinicalTrials.gov
Time frame: Week 260.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Open-Label Sebelipase Alfa
Serious: 9/9 (100%)
Deaths: 4/9
Serious adverse events (34 terms)
Reaction
System
Open-Label Sebelipase Alfa
Pyrexia
General disorders
—
Catheter site infection
Infections and infestations
—
Device related infection
Infections and infestations
—
Viral infection
Infections and infestations
—
Pallor
Vascular disorders
—
Chills
General disorders
—
Lymphadenopathy
Blood and lymphatic system disorders
—
Cardiac arrest
Cardiac disorders
—
Diarrhoea
Gastrointestinal disorders
—
Peritoneal haemorrhage
Gastrointestinal disorders
—
Hyperthermia
General disorders
—
Hepatic failure
Hepatobiliary disorders
—
Bacterial pyelonephritis
Infections and infestations
—
Device related sepsis
Infections and infestations
—
Respiratory tract infection
Infections and infestations
—
Roseola
Infections and infestations
—
Staphylococcal bacteraemia
Infections and infestations
—
Staphylococcal sepsis
Infections and infestations
—
Upper respiratory tract infection
Infections and infestations
—
Weight decreased
Investigations
—
Dehydration
Metabolism and nutrition disorders
—
Failure to thrive
Metabolism and nutrition disorders
—
Food intolerance
Metabolism and nutrition disorders
—
Metabolic acidosis
Metabolism and nutrition disorders
—
Poor venous access
Vascular disorders
—
Other adverse events (187 terms — click to expand)
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc.
Last refreshed: 30 January 2019
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01371825.