Trial · NCT02193867
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
NCT02193867, Phase 2, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2019-11-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency. Trials, record NCT02193867. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02193867
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
- NCT number
- NCT02193867
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 10 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa
- First posted
- 2014-07-18
- Last updated
- 2019-11-18
- Results first posted
- 2019-11-18
Source ClinicalTrials.gov
Registered outcomes8
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 through Month 37 | The number of participants experiencing severe TEAEs is presented for participants who received sebelipase alfa in this open-label study. Adverse events were obtained through spontaneous reporting or elicited by specific questioning or examination of the participant's parent or legal guardian. An adverse event was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant, whether or not causally related to administration of study drug. Adverse event severity was graded by the Investigator as mild, moderate, or severe based on definitions developed from Clinical Data Interchange Standards Consortium Study Data Tabulation Model standard terminology v3.1.1. Adverse events reporting was from the date of informed consent until completion of the follow-up visit at approximately 30 days after the last dose of study drug. A summary of all serious and other nonserious AEs regardless of causality is located in the Reported AE module. |
| Secondary | Percentage Of Participants Surviving To 12, 18, 24, And 36 Months Of Age | Baseline through Month 12, Month 18, Month 24, and Month 36 | The percentage of participants in the FAS who survived to 12, 18, 24, and 36 months of age. The exact confidence interval was calculated using the Clopper-Pearson method. Participants with unknown survival status at the age specified in the analysis were excluded. At 36 months, there were 2 participants who were alive and still on study who had not yet reached the age specified in the analysis. As such, these participants were excluded from the calculation of percent surviving. |
| Secondary | Median Age At Death | Baseline through Month 36 | Age at death for participants who died during the study. All deaths were assessed by the Investigator as unrelated to study drug. |
| Secondary | Change From Baseline In Percentiles For Weight For Age (WFA) At 12, 24, And 36 Months | Baseline, Month 12, Month 24, and Month 36 | This outcome measure evaluated the effects of sebelipase alfa on growth by measuring the changes from baseline in percentiles for WFA. Percentiles for WFA were summarized as observed values by visit. Baseline was defined as the last available assessment prior to the first infusion of sebelipase alfa. |
| Secondary | Number Of Participants With Stunting, Wasting, Or Underweight At Baseline, 12, 24, And 36 Months | Baseline to Month 12, Month 24, and Month 36 | The number of participants who met criteria for the following 3 dichotomous indicators of under nutrition were reported. These indicators included the following: 1. Stunting was defined as at least 2 standard deviations below the median for length-for-age/height-for-age. 2. Wasting was defined as wasting at least 2 standard deviations below the median for weight-for-length/weight-for-height. 3. Underweight was defined as at least 2 standard deviations below the median for WFA. |
| Secondary | Change From Baseline In Serum Transaminases (ALT And AST) At Month 12, 24, And 36 | Baseline, Month 12, Month 24, and Month 36 | This outcome measure evaluated the effects of sebelipase alfa on liver function by measuring the change from baseline in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) at months 12, 24, and 36. Results are reported in units/liter (U/L). |
| Secondary | Change From Baseline In Serum Ferritin At Month 12, 24, And 36 | Baseline, Month 12, Month 24, and Month 36 | The median change in the inflammatory marker serum ferritin from Baseline to Months 12, 24, and 36 is presented. The number of participants analyzed reflects only those from the FAS who had both a baseline value and a value at the indicated timepoint (Months 12, 24, and 36). Results are reported in micrograms (ug)/L. |
| Secondary | Number Of Participants Achieving And Maintaining Transfusion-free Hemoglobin Normalization (TFHN) | Baseline through Month 36 | The number of participants achieving and maintaining TFHN are presented. For TFHN to be achieved, the participant had to meet the following criteria: 1. Two post baseline measurements of hemoglobin, at least 4 weeks apart, were above the age-adjusted lower limit of normal (LLN); 2. No known additional measurements of hemoglobin were below the age-adjusted LLN during the (minimum) 4 week period; 3. No transfusions were administered to the participant during the (minimum) 4 week period, or for 2 weeks prior to the first hemoglobin measurement in the (minimum) 4 week period. If all 3 criteria were met, a participant was considered to have achieved TFHN on the date of the first hemoglobin assessment in the 4 week period. A participant was considered to have maintained TFHN if he/she was transfusion free at Week 6 and had no abnormally low hemoglobin levels (levels below the age adjusted LLN) beginning at Week 8 of the study and continuing for at least 13 weeks (3 months). |
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 | Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) | Screening through Month 37 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage Of Participants Surviving To 12, 18, 24, And 36 Months Of Age | Baseline through Month 12, Month 18, Month 24, and Month 36 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | percentage of participants | NUMBER |
| SECONDARY | Median Age At Death | Baseline through Month 36 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | months | MEDIAN |
| SECONDARY | Change From Baseline In Percentiles For Weight For Age (WFA) At 12, 24, And 36 Months | Baseline, Month 12, Month 24, and Month 36 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | percentile | MEDIAN |
| SECONDARY | Number Of Participants With Stunting, Wasting, Or Underweight At Baseline, 12, 24, And 36 Months | Baseline to Month 12, Month 24, and Month 36 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline In Serum Transaminases (ALT And AST) At Month 12, 24, And 36 | Baseline, Month 12, Month 24, and Month 36 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | U/L | MEDIAN |
| SECONDARY | Change From Baseline In Serum Ferritin At Month 12, 24, And 36 | Baseline, Month 12, Month 24, and Month 36 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | ug/L | MEDIAN |
| SECONDARY | Number Of Participants Achieving And Maintaining Transfusion-free Hemoglobin Normalization (TFHN) | Baseline through Month 36 | FAS: All participants who received any amount of sebelipase alfa (1.0, 3.0, 5.0, or 7.5 mg/kg qw) during the study and where applicable, met end point criteria. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group31
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) | Open-Label Sebelipase Alfa | 7 | Participants | ||
| Percentage Of Participants Surviving To 12, 18, 24, And 36 Months Of Age | Open-Label Sebelipase Alfa | Month 12 | 90 | 95% Confidence Interval 55.5 to 99.7 | percentage of participants |
| Percentage Of Participants Surviving To 12, 18, 24, And 36 Months Of Age | Open-Label Sebelipase Alfa | Month 18 | 80 | 95% Confidence Interval 44.4 to 97.5 | percentage of participants |
| Percentage Of Participants Surviving To 12, 18, 24, And 36 Months Of Age | Open-Label Sebelipase Alfa | Month 24 | 80 | 95% Confidence Interval 44.4 to 97.5 | percentage of participants |
| Percentage Of Participants Surviving To 12, 18, 24, And 36 Months Of Age | Open-Label Sebelipase Alfa | Month 36 | 75 | 95% Confidence Interval 34.9 to 96.8 | percentage of participants |
| Median Age At Death | Open-Label Sebelipase Alfa | 9.33 | Full Range 4.9 to 13.8 | months | |
| Change From Baseline In Percentiles For Weight For Age (WFA) At 12, 24, And 36 Months | Open-Label Sebelipase Alfa | Month 12 | 27.760 | Full Range 1.34 to 67.58 | percentile |
| Change From Baseline In Percentiles For Weight For Age (WFA) At 12, 24, And 36 Months | Open-Label Sebelipase Alfa | Month 24 | 41.276 | Full Range 7.54 to 63.77 | percentile |
| Change From Baseline In Percentiles For Weight For Age (WFA) At 12, 24, And 36 Months | Open-Label Sebelipase Alfa | Month 36 | 59.310 | Full Range 36.39 to 72.51 | percentile |
| Number Of Participants With Stunting, Wasting, Or Underweight At Baseline, 12, 24, And 36 Months | Open-Label Sebelipase Alfa | Stunting, Baseline | 4 | Participants |
1 to 10 of 31Source ClinicalTrials.gov
See all 31 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Open-Label Sebelipase Alfa | 10 | |
| Overall Study | Received At Least 1 Dose Of Study Drug | Open-Label Sebelipase Alfa | 10 | |
| Overall Study | Completed 18 Months Of Treatment | Open-Label Sebelipase Alfa | 8 | |
| Overall Study | COMPLETED | Open-Label Sebelipase Alfa | 6 | |
| Overall Study | NOT COMPLETED | Open-Label Sebelipase Alfa | 4 |
Source ClinicalTrials.gov
Adverse events1,540
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 | Vomiting | Gastrointestinal disorders | Sebelipase Alfa: 1.0 mg/kg qw | 5 | 10 | 11 |
| other | Vomiting | Gastrointestinal disorders | Sebelipase Alfa: 3.0 mg/kg qw | 6 | 9 | 15 |
| other | Vomiting | Gastrointestinal disorders | Sebelipase Alfa: 5.0 mg/kg qw | 4 | 7 | 11 |
| other | Vomiting | Gastrointestinal disorders | Sebelipase Alfa: 7.5 mg/kg qw | 0 | 1 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Sebelipase Alfa: 1.0 mg/kg qw | 4 | 10 | 12 |
| other | Diarrhoea | Gastrointestinal disorders | Sebelipase Alfa: 3.0 mg/kg qw | 5 | 9 | 23 |
| other | Diarrhoea | Gastrointestinal disorders | Sebelipase Alfa: 5.0 mg/kg qw | 5 | 7 | 29 |
| other | Diarrhoea | Gastrointestinal disorders | Sebelipase Alfa: 7.5 mg/kg qw | 0 | 1 | 0 |
| other | Teething | Gastrointestinal disorders | Sebelipase Alfa: 1.0 mg/kg qw | 0 | 10 | 0 |
| other | Teething | Gastrointestinal disorders | Sebelipase Alfa: 3.0 mg/kg qw | 3 | 9 | 5 |
1 to 10 of 1,540Source ClinicalTrials.gov
See all 1,540 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sebelipase Alfa: 1.0 mg/kg qw | 1 | 10 |
| Total, all-cause mortality | Sebelipase Alfa: 3.0 mg/kg qw | 0 | 9 |
| Total, all-cause mortality | Sebelipase Alfa: 5.0 mg/kg qw | 1 | 7 |
| Total, all-cause mortality | Sebelipase Alfa: 7.5 mg/kg qw | 0 | 1 |
| Total, other adverse events | Sebelipase Alfa: 1.0 mg/kg qw | 9 | 10 |
| Total, other adverse events | Sebelipase Alfa: 3.0 mg/kg qw | 9 | 9 |
| Total, other adverse events | Sebelipase Alfa: 5.0 mg/kg qw | 7 | 7 |
| Total, other adverse events | Sebelipase Alfa: 7.5 mg/kg qw | 1 | 1 |
| Total, serious adverse events | Sebelipase Alfa: 1.0 mg/kg qw | 8 | 10 |
| Total, serious adverse events | Sebelipase Alfa: 3.0 mg/kg qw | 8 | 9 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02193867
GET https://conductscience.org/api/trials/v1/NCT02193867/results?page=2