Phase 3
Completed N=9
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Lysosomal Acid Lipase Deficiency · Wolman Disease
Source: ClinicalTrials.gov NCT01371825 ↗
Enrolled (actual)
9
Serious AEs
100.0%
Results posted
Apr 2016
Primary outcomePrimary: Percentage Of Participants In The Primary Efficacy Analysis Set (PES) Surviving To 12 Months Of Age — 67 Percentage of participants
◆ Published Evidence
Established
88citations · ~10 / year
Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study.
Summary
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.
Linked Publications
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Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Of Participants In The Primary Efficacy Analysis Set (PES) Surviving To 12 Months Of Age |
67 | — |
| SECONDARY Percentage Of Participants Surviving Beyond 12 Months Of Age |
56; 56; 56; 56; 43 | — |
| SECONDARY Median Age At Death |
3.63 | — |
| SECONDARY Change From Baseline To Months 12, 24, 36, 48, And 60 In Weight For Age (WFA) Percentiles |
7.469; 21.787; 14.037; 15.770; 19.869 | — |
| SECONDARY Number Of Participants With Stunting, Wasting, Or Underweight |
4; 1; 0; 0; 0; 0 | — |
| SECONDARY Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum Transaminases (ALT And AST) |
-13.50; -5.00; -4.00; -27.50; -27.00; -43.50 | — |
| SECONDARY Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum Ferritin |
-294.40; -239.00; -262.95; -268.00; -213.00 | — |
| SECONDARY Number Of Participants Achieving And Maintaining Transfusion-free Hemoglobin Normalization [TFHN] |
6; 2 | — |
Eligibility Criteria
Inclusion Criteria
- Participant's parent or legal guardian provided written informed consent/permission prior to any study procedures.
- Male or female child with documented decreased LAL activity relative to the normal range of the laboratory performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis.
- Growth failure with onset before 6 months of age.
Exclusion Criteria
- Clinically important concurrent disease or comorbidities.
- Had received an investigational product other than sebelipase alfa within 14 days prior to the first dose.
- Participant was older than 24 months of age.
- Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplant.
- Previous hematopoietic stem cell or liver transplant.
- Known hypersensitivity to eggs.
Data sourced from ClinicalTrials.gov (NCT01371825) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.