Trial · NCT01371825
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
NCT01371825, Phase 2, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2016-04-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency. Trials, record NCT01371825. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01371825
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
- NCT number
- NCT01371825
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 9 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Lysosomal Acid Lipase Deficiency, Wolman Disease
- Interventions
- Sebelipase alfa (SBC-102)
- First posted
- 2011-06-13
- Last updated
- 2019-01-30
- Results first posted
- 2016-04-18
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Of Participants In The Primary Efficacy Analysis Set (PES) Surviving To 12 Months Of Age | Month 12 | The primary efficacy endpoint was the percentage of participants (%) in the PES who survived to at least 12 months of age. |
| Secondary | Percentage Of Participants Surviving Beyond 12 Months Of Age | 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. |
| Secondary | Median Age At Death | 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. |
| Secondary | Change From Baseline To Months 12, 24, 36, 48, And 60 In Weight For Age (WFA) Percentiles | 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. |
| Secondary | Number Of Participants With Stunting, Wasting, Or Underweight | 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. |
| Secondary | Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum Transaminases (ALT And AST) | 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). |
| Secondary | Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum Ferritin | 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. |
| Secondary | Number Of Participants Achieving And Maintaining Transfusion-free Hemoglobin Normalization [TFHN] | 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 be maintained, the participant must have been transfusion-free beginning at Week 6 and had all hemoglobin assessments above the lower limit of normal beginning in Week 8 and lasting at least 13 weeks. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Of Participants In The Primary Efficacy Analysis Set (PES) Surviving To 12 Months Of Age | Month 12 | The PES included participants who received any amount of sebelipase alfa and who were ≤8 months of age on the date of their first infusion of sebelipase alfa. All 9 participants were included in the PES. | Percentage of participants | NUMBER |
| SECONDARY | Percentage Of Participants Surviving Beyond 12 Months Of Age | Baseline to Month 18, Month 24, Month 36, Month 48, and Month 60 | Evaluable participants in the PES, which included participants who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa. There were 2 non-evaluable participants at Month 60, defined as participants who were alive, still in the study, and had not yet reached 60 months of age. | Percentage of participants | NUMBER |
| SECONDARY | Median Age At Death | Baseline to Week 260 | Participants in the PES who died during the study were included in this analysis. The PES included all 9 participants who received any amount of sebelipase alfa and who were ≤8 months of age on the date of their first infusion of sebelipase alfa. | months | MEDIAN |
| SECONDARY | Change From Baseline To Months 12, 24, 36, 48, And 60 In Weight For Age (WFA) Percentiles | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 | The PES included participants who received any amount of sebelipase alfa and who were ≤8 months of age on the date of their first infusion of sebelipase alfa. All 9 participants were included in the PES. | WFA Percentile | MEDIAN |
| SECONDARY | Number Of Participants With Stunting, Wasting, Or Underweight | Baseline to Month 12, Month 24, Month 36, Month 48, and Month 60 | The PES included participants who received any amount of sebelipase alfa and who were ≤8 months of age on the date of their first infusion of sebelipase alfa. All 9 participants were included in the PES. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum Transaminases (ALT And AST) | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 | The PES included participants who received any amount of sebelipase alfa and who were ≤8 months of age on the date of their first infusion of sebelipase alfa. All 9 participants were included in the PES. | units/Liter (U/L) | MEDIAN |
| SECONDARY | Change From Baseline To Months 12, 24, 36, 48, And 60 In Serum Ferritin | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 | The PES included participants who received any amount of sebelipase alfa and who were ≤8 months of age on the date of their first infusion of sebelipase alfa. All 9 participants were included in the PES. | micrograms/Liter (µg/L) | MEDIAN |
| SECONDARY | Number Of Participants Achieving And Maintaining Transfusion-free Hemoglobin Normalization [TFHN] | Baseline to Month 60 | Participants in the PES who received treatment with sebelipase alfa for at least 4 weeks (and could therefore be assessed for short-term TFHN). The PES included participants who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group53
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Of Participants In The Primary Efficacy Analysis Set (PES) Surviving To 12 Months Of Age | Open-Label Sebelipase Alfa | 67 | 95% Confidence Interval 29.9 to 92.5 | Percentage of participants | |
| Percentage Of Participants Surviving Beyond 12 Months Of Age | Open-Label Sebelipase Alfa | Survival Through 18 Months of Age | 56 | 95% Confidence Interval 21.2 to 86.3 | Percentage of participants |
| Percentage Of Participants Surviving Beyond 12 Months Of Age | Open-Label Sebelipase Alfa | Survival Through 24 Months of Age | 56 | 95% Confidence Interval 21.2 to 86.3 | Percentage of participants |
| Percentage Of Participants Surviving Beyond 12 Months Of Age | Open-Label Sebelipase Alfa | Survival Through 36 Months of Age | 56 | 95% Confidence Interval 21.2 to 86.3 | Percentage of participants |
| Percentage Of Participants Surviving Beyond 12 Months Of Age | Open-Label Sebelipase Alfa | Survival Through 48 Months of Age | 56 | 95% Confidence Interval 21.2 to 86.3 | Percentage of participants |
| Percentage Of Participants Surviving Beyond 12 Months Of Age | Open-Label Sebelipase Alfa | Survival Through 60 Months of Age | 43 | 95% Confidence Interval 9.9 to 81.6 | Percentage of participants |
| Median Age At Death | Open-Label Sebelipase Alfa | 3.63 | Full Range 2.8 to 15.0 | months | |
| Change From Baseline To Months 12, 24, 36, 48, And 60 In Weight For Age (WFA) Percentiles | Open-Label Sebelipase Alfa | Month 12 | 7.469 | Full Range 5.35 to 13.77 | WFA Percentile |
| Change From Baseline To Months 12, 24, 36, 48, And 60 In Weight For Age (WFA) Percentiles | Open-Label Sebelipase Alfa | Month 24 | 21.787 | Full Range 0.91 to 30.37 | WFA Percentile |
| Change From Baseline To Months 12, 24, 36, 48, And 60 In Weight For Age (WFA) Percentiles | Open-Label Sebelipase Alfa | Month 36 | 14.037 | Full Range -0.35 to 89.00 | WFA Percentile |
1 to 10 of 53Source ClinicalTrials.gov
See all 53 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Open-Label Sebelipase Alfa | 9 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Open-Label Sebelipase Alfa | 9 | |
| Overall Study | COMPLETED | Open-Label Sebelipase Alfa | 5 | |
| Overall Study | NOT COMPLETED | Open-Label Sebelipase Alfa | 4 |
Source ClinicalTrials.gov
Adverse events221
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Open-Label Sebelipase Alfa | 4 | 9 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Open-Label Sebelipase Alfa | 1 | 9 | |
| other | Tachycardia | Cardiac disorders | Open-Label Sebelipase Alfa | 2 | 9 | |
| other | Bradycardia | Cardiac disorders | Open-Label Sebelipase Alfa | 2 | 9 | |
| other | Cardiovascular disorder | Cardiac disorders | Open-Label Sebelipase Alfa | 1 | 9 | |
| other | Hydrocele | Congenital, familial and genetic disorders | Open-Label Sebelipase Alfa | 2 | 9 | |
| other | Ear pain | Ear and labyrinth disorders | Open-Label Sebelipase Alfa | 2 | 9 | |
| other | Middle ear inflammation | Ear and labyrinth disorders | Open-Label Sebelipase Alfa | 1 | 9 | |
| other | Adrenal insufficiency | Endocrine disorders | Open-Label Sebelipase Alfa | 1 | 9 | |
| other | Conjunctivitis | Eye disorders | Open-Label Sebelipase Alfa | 1 | 9 |
1 to 10 of 221Source ClinicalTrials.gov
See all 221 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Open-Label Sebelipase Alfa | 4 | 9 |
| Total, other adverse events | Open-Label Sebelipase Alfa | 9 | 9 |
| Total, serious adverse events | Open-Label Sebelipase Alfa | 9 | 9 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28179030 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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