Trial · NCT02292654
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
NCT02292654, Phase 1, Phase 2, Completed, Genzyme, a Sanofi Company; results first posted 2021-02-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency. Trials, record NCT02292654. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02292654
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
- NCT number
- NCT02292654
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- Olipudase alfa
- First posted
- 2014-11-17
- Last updated
- 2022-03-23
- Results first posted
- 2021-02-01
Source ClinicalTrials.gov
Registered outcomes35
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From Baseline up to End of study (64 weeks) | TEAEs were defined as adverse events (AEs) that occurred or worsened during the on-treatment period (time from the start of investigational medicinal product \[IMP\]) administration until end of study (i.e. up to 64 weeks). |
| Primary | Number of Participants With Infusion-Associated Reactions (IARs) | Within up to 24 hours after start of any infusion (during the treatment period i.e. from Baseline up to 64 weeks) | IARs were defined as AEs that occurred during the infusion or within up to 24 hours after the start of infusion and were considered as related or possibly related to the study treatment by the investigator or the sponsor. Protocol-defined IAR: all AEs that were identified as an IAR by the investigator. Events occurring greater than or equal to (\>=) 24 hours after the start of an infusion might had been judged an IAR at the discretion of the investigator or sponsor. |
| Primary | Number of Participants With Change in Physical Examination | Baseline, Week 52 (last complete assessment) | Change from Normal assessment (at Baseline) to Abnormal assessment (at Week 52) was reported. Physical examinations included following observations/measurements: examination of the skin, head, eyes, ears, nose, and throat; lymph nodes; heart, lungs, and abdomen; extremities and joints. Abnormality in physical examinations was based on investigator's discretion. |
| Primary | Number of Participants With Change in Neurological Examination | Baseline, Week 52 (last assessment) | Change from Normal assessment (at Baseline) to Abnormal assessment (at Week 52) was reported. Neurological examination included: coordination examination, cranial nerve examination, extrapyramidal features, fundoscopy, gait and coordination examination, motor examination, tone peripheral nervous system, reflexes examination, sensory examination, strength examination, mental status. |
| Primary | Number of Participants With Abnormal Liver Function Laboratory Values at the End of Study | At End of Study (Week 64) | Abnormal values in alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, and alkaline phosphatase were reported. |
| Primary | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | From Baseline up to End of Study (64 weeks) | * Heart Rate (HR) High: \>=120 beats per minute (bpm) (adolescents), \>=120 bpm (children), \>=140 bpm (early children), \>=175 bpm (infants) \& increase from baseline (IFB) \>=20 bpm for all age categories. * HR Low: \<=50 bpm (adolescents), \<=50 bpm (children), \<=75 bpm (early children), \<=80 bpm (infants) \& decrease from baseline (DFB) \>=20 bpm for all age categories. * Systolic BP (SBP) High: \>=119 mmHg (adolescents), 108 mmHg (children), 101 mmHg (in early children), 98 mmHg (infants) \& IFB \>=20 mmHg for all age categories. * SBP Low: \<=90 mmHg (adolescents), \<= 80mm Hg (children), \<=70 mmHg (early children), \<=70 mmHg (infants) \& DFB \>=20 mmHg for all age categories. * Diastolic BP (DBP) High:\>=78 mmHg (adolescents), \>=72 mmHg (children), \>=59 mmHg (in early children), \>=54 mmHg (infants) \& IFB \>=10 mmHg for all age categories. * DBP Low:\<=54 mmHg (adolescents), \<=48 mmHg (children), \<=34 mmHg (early children), \<=34 mmHg (infants) \& DFB \>=10 mmHg for all age categories. |
| Primary | Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities | From Baseline up to End of Study (64 weeks) | Criteria for potentially clinically significant ECG abnormalities: * High PR Interval: \>=180 milliseconds (ms) in adolescents, 170 ms in children, 160 ms in early children, and 140 ms in infants; * High QRS Interval: \>=110 ms in adolescents, 100 ms in children, 95 ms in early children and 85 ms in infants; * Prolonged QTc Fridericia (QTc F): \>450 ms in male adolescents, children, early children and infants or 470 ms in female adolescents, * QTc F \>500 ms; * QTc F increase from baseline \>60 ms. |
| Primary | Change From Baseline in Safety Biomarker Level: High Sensitivity C Reactive Protein (hsCRP) at Week 64 | Baseline, Week 64 (pre-infusion) | |
| Primary | Change From Baseline in Safety Biomarker: Ceramide Level at Week 64 | Baseline, Week 64 (pre-infusion) | |
| Primary | Change From Baseline in Safety Biomarker: Iron at Week 64 | Baseline, Week 64 (pre-infusion) |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 registered outcomesOutcomes35
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From Baseline up to End of study (64 weeks) | Safety population included all participants who received at least 1 infusion (partial or total) of olipudase alfa. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Infusion-Associated Reactions (IARs) | Within up to 24 hours after start of any infusion (during the treatment period i.e. from Baseline up to 64 weeks) | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Change in Physical Examination | Baseline, Week 52 (last complete assessment) | Analysis was performed on safety population. One participant may be counted in multiple categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Change in Neurological Examination | Baseline, Week 52 (last assessment) | Analysis was performed on safety population. One participant may be counted in multiple categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Abnormal Liver Function Laboratory Values at the End of Study | At End of Study (Week 64) | Analysis was performed on safety population. One participant may be counted in multiple categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | From Baseline up to End of Study (64 weeks) | Analysis was performed on safety population. One participant may be counted in multiple (more than 1) categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities | From Baseline up to End of Study (64 weeks) | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in Safety Biomarker Level: High Sensitivity C Reactive Protein (hsCRP) at Week 64 | Baseline, Week 64 (pre-infusion) | Analysis was performed on safety population. Here, overall number of participants analyzed = participants with available data for this outcome measure. | mg/L | MEAN |
| PRIMARY | Change From Baseline in Safety Biomarker: Ceramide Level at Week 64 | Baseline, Week 64 (pre-infusion) | Analysis was performed on safety population. | mg/L | MEAN |
| PRIMARY | Change From Baseline in Safety Biomarker: Iron at Week 64 | Baseline, Week 64 (pre-infusion) | Analysis was performed on safety population. Here, overall number of participants analyzed = participants with available data for this outcome measure. | umol/L | MEAN |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 outcomesResults by group342
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Adolescent Cohort | Any TEAEs | 4 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Child Cohort | Any TEAEs | 9 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Infant/Early Child Cohort | Any TEAEs | 7 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Total Participants | Any TEAEs | 20 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Adolescent Cohort | Any TEAEs leading to death | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Child Cohort | Any TEAEs leading to death | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Infant/Early Child Cohort | Any TEAEs leading to death | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Total Participants | Any TEAEs leading to death | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Adolescent Cohort | Any TEAEs leading to dose reduction | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Olipudase Alfa: Child Cohort | Any TEAEs leading to dose reduction | 4 | Participants |
1 to 10 of 342Source ClinicalTrials.gov
See all 342 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Olipudase Alfa: Adolescent Cohort | 4 | |
| Overall Study | STARTED | Olipudase Alfa: Child Cohort | 9 | |
| Overall Study | STARTED | Olipudase Alfa: Infant/Early Child Cohort | 7 | |
| Overall Study | COMPLETED | Olipudase Alfa: Adolescent Cohort | 4 | |
| Overall Study | COMPLETED | Olipudase Alfa: Child Cohort | 9 | |
| Overall Study | COMPLETED | Olipudase Alfa: Infant/Early Child Cohort | 7 | |
| Overall Study | NOT COMPLETED | Olipudase Alfa: Adolescent Cohort | 0 | |
| Overall Study | NOT COMPLETED | Olipudase Alfa: Child Cohort | 0 | |
| Overall Study | NOT COMPLETED | Olipudase Alfa: Infant/Early Child Cohort | 0 |
Source ClinicalTrials.gov
Adverse events92
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 | Abdominal Pain | Gastrointestinal disorders | Olipudase Alfa: Adolescent Cohort | 0 | 4 | 0 |
| other | Abdominal Pain | Gastrointestinal disorders | Olipudase Alfa: Child Cohort | 5 | 9 | 19 |
| other | Abdominal Pain | Gastrointestinal disorders | Olipudase Alfa: Infant/Early Child Cohort | 1 | 7 | 1 |
| other | Abdominal Pain | Gastrointestinal disorders | Total Participants | 6 | 20 | 20 |
| other | Diarrhoea | Gastrointestinal disorders | Olipudase Alfa: Adolescent Cohort | 2 | 4 | 5 |
| other | Diarrhoea | Gastrointestinal disorders | Olipudase Alfa: Child Cohort | 5 | 9 | 10 |
| other | Diarrhoea | Gastrointestinal disorders | Olipudase Alfa: Infant/Early Child Cohort | 4 | 7 | 7 |
| other | Diarrhoea | Gastrointestinal disorders | Total Participants | 11 | 20 | 22 |
| other | Vomiting | Gastrointestinal disorders | Olipudase Alfa: Adolescent Cohort | 2 | 4 | 3 |
| other | Vomiting | Gastrointestinal disorders | Olipudase Alfa: Child Cohort | 6 | 9 | 21 |
1 to 10 of 92Source ClinicalTrials.gov
See all 92 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Olipudase Alfa: Adolescent Cohort | 0 | 4 |
| Total, all-cause mortality | Olipudase Alfa: Child Cohort | 0 | 9 |
| Total, all-cause mortality | Olipudase Alfa: Infant/Early Child Cohort | 0 | 7 |
| Total, all-cause mortality | Total Participants | 0 | 20 |
| Total, other adverse events | Olipudase Alfa: Adolescent Cohort | 4 | 4 |
| Total, other adverse events | Olipudase Alfa: Child Cohort | 9 | 9 |
| Total, other adverse events | Olipudase Alfa: Infant/Early Child Cohort | 7 | 7 |
| Total, other adverse events | Total Participants | 20 | 20 |
| Total, serious adverse events | Olipudase Alfa: Adolescent Cohort | 0 | 4 |
| Total, serious adverse events | Olipudase Alfa: Child Cohort | 1 | 9 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 33875845 | 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.
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.
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