Trial · NCT02112994

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

NCT02112994, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2019-01-15

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency. Trials, record NCT02112994. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02112994

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
NCT number: NCT02112994
Status: Completed
Phases: Phase 2
Enrollment: 31 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Lysosomal Acid Lipase Deficiency
Interventions: Sebelipase Alfa
First posted: 2014-04-14
Last updated: 2019-12-04
Other registries' IDs: eudract:2011-004287-30
Results first posted: 2019-01-15

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)	Screening, Week 144	The number of participants experiencing severe TEAEs is presented for participants who received sebelipase alfa in this open-label study. Adverse events (AEs) information was obtained at each scheduled contact with the participant (or participant's parent or legal guardian). An AE was defined as any untoward medical occurrence that did not require a causal relationship with study drug administration. An AE could have been any unfavorable and unintended sign, symptom, or disease temporally associated with the use of the study drug, whether or not considered related to the study drug. Pre-existing conditions that worsened in severity during the study were reported as AEs. A summary of all serious and other non-serious AEs regardless of causality is located in the Reported AE module. Severity assessed using Clinical Data Interchange Standards Consortium Study Data Tabulation Model standard terminology v3.1.1. Data presented only according to age group, not dose of study drug received.
Secondary	Percent Change In Serum Lipids From Baseline To Week 144	Baseline, Week 144	The effect of sebelipase alfa on lipid metabolism was evaluated by measuring the change from baseline to Week 144 in 4 serum lipids: low-density lipoprotein cholesterol (LDL-C); high-density lipoprotein cholesterol (HDL-C); non-HDL-C; triglycerides. Blood samples for these clinical laboratory tests were collected at scheduled time points and analyzed by a central laboratory.
Secondary	Participants Testing Positive For Anti-drug Antibodies (ADAs)	Week 144	The impact of ADAs on the safety and immunogenicity of sebelipase alfa was evaluated by testing for ADAs in participants who received sebelipase alfa in this open-label study. Blood samples for assessment were collected prior to study infusions at Week 2, Week 4, Week 8, Week 12, and every 12 weeks thereafter. Participants testing positive for ADAs were also tested for the presence of neutralizing antibodies that inhibited sebelipase alfa enzyme activity and/or cellular uptake. Any participant experiencing a moderate or severe infusion-associated reaction (IAR) was to have an additional assessment of ADAs at the next study visit (prior to study drug infusion); these participants were to also have serum samples collected at 1 to 2 hours after IAR onset and at the next study visit (prior to study drug infusion) for analysis of serum tryptase. The count of participants who became ADA positive and who tested positive for neutralizing antibodies are presented.
Secondary	Percent Change In Body Mass Index (BMI)-For-Age Percentile From Baseline To Week 144 In Pediatric Participants	Baseline, Week 144	To evaluate the effects of sebelipase alfa on growth parameters in pediatric participants (≤18 years old) presenting with evidence of growth delay, the percent change in the anthropometric parameter of BMI-for-age percentile from Baseline to Week 144 is reported. Anthropometric parameters were plotted on standard growth curves. When possible, historical data on growth parameters was also incorporated into the analyses. Percentiles and Z-scores for BMI-for-age were determined using standard growth charts appropriate to a participant's age on the date of the assessment: the World Health Organization standard growth chart for participants ≤2 years of age and the Centers for Disease Control standard growth chart for participants \>2 years of age.
Secondary	Shift In Child-Pugh Status From Baseline To Week 144	Baseline, Week 144	In order to evaluate the effects of sebelipase alfa on liver function, the number of participants with a shift in Child-Pugh status from Baseline to Week 144 is reported. The status is based on the Child-Pugh score, which is used in clinical practice to assess prognosis in individuals with chronic liver disease. Laboratory data were used in derivation of the score by summing individual scores (scored 1-3, with 3 indicating most severe) from clinical laboratory test results and physical examinations, including total serum bilirubin, serum albumin, prothrombin time, ascites, and hepatic encephalopathy. The total score was used to determine the Child-Pugh status, reported as Class A (score of 5 or 6), Class B (score of 7 to 9), or Class C (score of 10 to 15). Higher scores and higher categories represented a worse outcome. Data reported as 1 of 2 types of shifts in class: No Change from Baseline; Decline from Baseline.

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)	Screening, Week 144	Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Percent Change In Serum Lipids From Baseline To Week 144	Baseline, Week 144	Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.	Percent Change	MEDIAN
SECONDARY	Participants Testing Positive For Anti-drug Antibodies (ADAs)	Week 144	Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Percent Change In Body Mass Index (BMI)-For-Age Percentile From Baseline To Week 144 In Pediatric Participants	Baseline, Week 144	Full Analysis Set: All pediatric participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.	Percent Change	MEAN
SECONDARY	Shift In Child-Pugh Status From Baseline To Week 144	Baseline, Week 144	Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group21

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)	2-<4 Years		1		Participants
Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)	4-18 Years		1		Participants
Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)	>18 Years		2		Participants
Percent Change In Serum Lipids From Baseline To Week 144	2-<4 Years	LDL-C	-37.5	Full Range -52 to -25	Percent Change
Percent Change In Serum Lipids From Baseline To Week 144	4-18 Years	LDL-C	-29.2	Full Range -59 to 23	Percent Change
Percent Change In Serum Lipids From Baseline To Week 144	>18 Years	LDL-C	-22.5	Full Range -37 to -8	Percent Change
Percent Change In Serum Lipids From Baseline To Week 144	2-<4 Years	HDL-C	76.5	Full Range 30 to 132	Percent Change
Percent Change In Serum Lipids From Baseline To Week 144	4-18 Years	HDL-C	24.2	Full Range -4 to 90	Percent Change
Percent Change In Serum Lipids From Baseline To Week 144	>18 Years	HDL-C	6.1	Full Range -10 to 22	Percent Change
Percent Change In Serum Lipids From Baseline To Week 144	2-<4 Years	Non-HDL-C	-39.1	Full Range -53 to -29	Percent Change

1 to 10 of 21Source ClinicalTrials.gov

See all 21 results by group

Participant flow30

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	2-<4 Years	6
Overall Study	STARTED	4-18 Years	16
Overall Study	STARTED	>18 Years	9
Overall Study	Received at Least 1 Dose of Study Drug	2-<4 Years	6
Overall Study	Received at Least 1 Dose of Study Drug	4-18 Years	16
Overall Study	Received at Least 1 Dose of Study Drug	>18 Years	9
Overall Study	Received 0.35 mg/kg QOW	2-<4 Years	0
Overall Study	Received 0.35 mg/kg QOW	4-18 Years	0
Overall Study	Received 0.35 mg/kg QOW	>18 Years	1
Overall Study	Received 1 mg/kg QOW	2-<4 Years	6

1 to 10 of 30Source ClinicalTrials.gov

See all 30 participant flow

Adverse events595

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
other	Iron deficiency anaemia	Blood and lymphatic system disorders	0.35 mg/kg QOW	0	1
other	Iron deficiency anaemia	Blood and lymphatic system disorders	1.0 mg/kg QOW	0	31
other	Iron deficiency anaemia	Blood and lymphatic system disorders	1.0 mg/kg QW	0	2
other	Iron deficiency anaemia	Blood and lymphatic system disorders	3.0 mg/kg QOW	1	11
other	Iron deficiency anaemia	Blood and lymphatic system disorders	3.0 mg/kg QW	0	4
other	Macrocytosis	Blood and lymphatic system disorders	0.35 mg/kg QOW	0	1
other	Macrocytosis	Blood and lymphatic system disorders	1.0 mg/kg QOW	0	31
other	Macrocytosis	Blood and lymphatic system disorders	1.0 mg/kg QW	0	2
other	Macrocytosis	Blood and lymphatic system disorders	3.0 mg/kg QOW	1	11
other	Macrocytosis	Blood and lymphatic system disorders	3.0 mg/kg QW	0	4

1 to 10 of 595Source ClinicalTrials.gov

See all 595 adverse events

Adverse event totals15

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	0.35 mg/kg QOW	0	1
Total, all-cause mortality	1.0 mg/kg QOW	0	31
Total, all-cause mortality	1.0 mg/kg QW	0	2
Total, all-cause mortality	3.0 mg/kg QOW	0	11
Total, all-cause mortality	3.0 mg/kg QW	0	4
Total, other adverse events	0.35 mg/kg QOW	0	1
Total, other adverse events	1.0 mg/kg QOW	30	31
Total, other adverse events	1.0 mg/kg QW	0	2
Total, other adverse events	3.0 mg/kg QOW	10	11
Total, other adverse events	3.0 mg/kg QW	4	4

1 to 10 of 15Source ClinicalTrials.gov

See all 15 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
35442238	DERIVED

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

The record and each of its lists are JSON, twenty rows a page. No key.

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