Trial · NCT02004704
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
NCT02004704, Phase 2, Completed, Genzyme, a Sanofi Company; results first posted 2024-07-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency. Trials, record NCT02004704. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02004704
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
- NCT number
- NCT02004704
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 25 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- GZ402665
- First posted
- 2013-12-09
- Last updated
- 2024-07-03
- Other registries' IDs
- eudract:2013-000051-40
- Results first posted
- 2024-07-03
Source ClinicalTrials.gov
Registered outcomes27
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), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first | An AE: Any untoward medical occurrence in participant or clinical investigation participant administered with pharmaceutical product, which did not necessarily have to have causal relationship with study treatment. SAEs: Any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect, was medically important event. TEAEs: AEs that started during on-treatment period-either in this study or in original study which were ongoing at the time the participant signed the written informed consent. An AESI: AE (serious or nonserious) of scientific and medical concern specific to sponsor's product or program, for which ongoing monitoring and rapid communication by investigator to sponsor was appropriate. |
| Primary | Number of Participants With IARs | From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first | Protocol-defined IARs were 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. Events occurring greater than or equal to (\>=) 24 hours after the start of an infusion might have been judged an IAR at the discretion of the investigator or sponsor. Algorithm-defined IARs were all AEs that started between the start of infusion and the end of infusion plus 24 hours, irrespective of the perceived relation with study treatment. |
| Primary | For Adults: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam | Baseline (Day 1 of original study), Month 78 | A complete physical examination included assessment of the participant's general appearance, general neurological status, skin, head, eyes, ears, nose, throat, lymph nodes, heart, lungs, abdomen, and extremities/joints. Shift from Baseline was monitored. An abbreviated physical exam (general appearance only) was only performed pre- and post-infusion for those who did not do complete exam. |
| Primary | For Pediatrics: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam | Baseline (Day 1 of original study), Month 84 | A complete physical examination included assessment of the participant's general appearance, skin, head, eyes, ears, nose, throat, lymph nodes, heart, lungs, abdomen, and extremities/joints. Shift from Baseline was monitored. An abbreviated physical exam (general appearance only) was only performed pre- and post-infusion for those who did not do complete exam. |
| Primary | For Adults: Number of Participants With Abnormality in Extended Neurological Examinations | Baseline (Day 1 of original study), Month 78 | Extended neurological examination for adults included examination of mental status. Shift from Baseline was monitored. |
| Primary | For Pediatrics: Number of Participants With Abnormality in Extended Neurological Examinations | Baseline (Day 1 of original study), Month 84 | The neurological examination in pediatric participants included examination of mental status, cranial nerve examination, motor examination: tone, reflexes examination, sensory examination, coordination, gait and coordination, and strength examination. Shift from Baseline was monitored. |
| Primary | Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Vital Signs | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | PCSA: Heart Rate: High: \>=120 beats per minute (bpm) (adults \[ad\], adolescents \[ado\], children \[chi\]), \>=140 bpm (early chi \[ec\]), \>=175 bpm (infants\[inf\]) \& increase from baseline (IFB)\>=20 bpm for all age ranges (AAR), HR Low: \<=50 bpm (ad, ado, chi), \<=75 bpm (ec), \<=80 bpm (inf) \& decrease from baseline (DFB) \>=20 bpm for AAR. Systolic blood pressure: High: \>= 160 millimeters of mercury (mmHg) (ad); \>=119 mmHg (ado), \>=108 mmHg (chi), \>=101 mmHg (ec),\>=98 mmHg (inf) \& IFB \>=20 mmHg for AAR. SBP Low: \<=95 mmHg (ad), \<=90 mmHg (ado), \<= 80mm Hg (chi), \<=70 mmHg (ec), \<=70 mmHg (inf) \& DFB \>=20 mmHg for AAR. Diastolic blood pressure: High:\>110 mmHg (ad), \>=78 mmHg (ado), \>=72 mmHg (chi), \>=59 mmHg (ec), \>=54 mmHg (inf) and IFB \>=10 mmHg for AAR. DBP Low:\<=45 mmHg (ad), \<=54 mmHg (ado), \<=48 mmHg (chi), \<=34mmHg (ec and inf) \& DFB\>=10 mmHg for AAR. |
| Primary | Number of Participants With PCSA in Clinical Chemistry Parameters | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | PCSA: Creatinine High, category 1: \>=150 micromole/L (umol) (adults), \>=132 umol/L or 1.5 mg/deciliter (dL) (adolescents), \>=90 umol/litre (L) or 1.1 mg/dL (children), \>53 umol/L or 0.6 mg/dL (early children and infants); category 2: \>=30% IFB (adults). Blood Urea Nitrogen: \>=17 millimole (mmol)/L (adults), \>=6.4 mmol/L or 18 mg/dL (pediatrics \[ped\]). Glucose Low\<=3.9 mmol/L and \<lower limit of normal (adults), \<2.7 mmol/L (ped), High: \>=11.1 mmol/L (unfasted), \>7 mmol/L (fasted) (adults), \>=7 mmol/L (fasted after \>12 hours of fast); \>=10.0 mmol/L (unfasted) (ped). |
| Primary | Number of Participants With PCSA in Liver Function Tests | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | PCSA: Alanine aminotransferase (ALT) \>3 x upper limit of normal (ULN). Aspartate aminotransferase (AST) \>3 x ULN. Alkaline phosphatase (ALP) \> 1.5 x ULN. Total Bilirubin \>1.5 x ULN (ad) and \>1.3 x ULN (ped). ALT and total bilirubin: ALT \> 3 x ULN and total bilirubin \> 2 x ULN. |
| Primary | Number of Participants With PCSA in Hematology Parameters | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | PCSA: White blood cells (WBC): Low: \<3.0 Giga (G)/L (Non-Black \[NB\])/\<2.0 G/L (Black \[B\])(ad), \<4.5 G/L (ado), \<5.0 G/L(chi), \<3.0 G/L (ec), \<4.0 G/L (inf), High: \>=16.0 G/L(ad), \>13.5 G/L (ado), \>17.0 G/L(chi), \>16 G/L (ec), \>20 G/L (inf). Hemoglobin-ad: Low-1: \<=115 g/dL (Male \[M\])/\<=95 g/dL (Female \[F\]), 20% DFB for both, High:\>=185 g/L (M)/\>=165 g/L (F), Low-2: DFB\>=20 g/L (ad), \<10 g/dL (ado, chi, ec); \<9.0 g/dL (inf); 20% DFB for all. Platelets: Low: \<100 G/L (AAR) and 20% DFB and High: \>=700 G/L (ad and ped). |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 registered outcomesOutcomes27
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), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With IARs | From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | For Adults: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam | Baseline (Day 1 of original study), Month 78 | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. Only those participants with data collected at specified timepoints are reported. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | For Pediatrics: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam | Baseline (Day 1 of original study), Month 84 | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. Only those participants with data collected at specified timepoints are reported. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | For Adults: Number of Participants With Abnormality in Extended Neurological Examinations | Baseline (Day 1 of original study), Month 78 | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. Only those participants with data collected at specified timepoints are reported. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | For Pediatrics: Number of Participants With Abnormality in Extended Neurological Examinations | Baseline (Day 1 of original study), Month 84 | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. Only those participants with data collected at specified timepoints are reported. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Vital Signs | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With PCSA in Clinical Chemistry Parameters | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With PCSA in Liver Function Tests | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With PCSA in Hematology Parameters | Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first | Results are based on the Safety analysis set which consisted of all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 outcomesResults by group177
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), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13412 (Adults) | Any TEAE | 5 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | Any TEAE | 20 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13412 (Adults) | Any TESAE | 1 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | Any TESAE | 10 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13412 (Adults) | AESI: Any Pregnancies | 0 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | AESI: Any Pregnancies | 0 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13412 (Adults) | AESI: Symptomatic Overdose | 0 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | AESI: Symptomatic Overdose | 0 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13412 (Adults) | AESI: Dose-limiting toxicities | 0 | Participants | |
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | AESI: Dose-limiting toxicities | 10 | Participants |
1 to 10 of 177Source ClinicalTrials.gov
See all 177 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Olipudase Alfa: Participants From DFI13412 (Adults) | 5 | |
| Overall Study | STARTED | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 20 | |
| Overall Study | COMPLETED | Olipudase Alfa: Participants From DFI13412 (Adults) | 5 | |
| Overall Study | COMPLETED | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 20 | |
| Overall Study | NOT COMPLETED | Olipudase Alfa: Participants From DFI13412 (Adults) | 0 | |
| Overall Study | NOT COMPLETED | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 0 |
Source ClinicalTrials.gov
Adverse events64
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 | Pyrexia | General disorders | Olipudase Alfa: Participants From DFI13412 (Adults) | 4 | 5 | 97 |
| other | Pyrexia | General disorders | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 18 | 20 | 98 |
| other | Contusion | Injury, poisoning and procedural complications | Olipudase Alfa: Participants From DFI13412 (Adults) | 4 | 5 | 55 |
| other | Contusion | Injury, poisoning and procedural complications | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 9 | 20 | 148 |
| other | Headache | Nervous system disorders | Olipudase Alfa: Participants From DFI13412 (Adults) | 5 | 5 | 178 |
| other | Headache | Nervous system disorders | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 13 | 20 | 91 |
| serious | Pancytopenia | Blood and lymphatic system disorders | Olipudase Alfa: Participants From DFI13412 (Adults) | 0 | 5 | 0 |
| serious | Pancytopenia | Blood and lymphatic system disorders | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 1 | 20 | 1 |
| serious | Talipes | Congenital, familial and genetic disorders | Olipudase Alfa: Participants From DFI13412 (Adults) | 0 | 5 | 0 |
| serious | Talipes | Congenital, familial and genetic disorders | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 1 | 20 | 1 |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Olipudase Alfa: Participants From DFI13412 (Adults) | 0 | 5 |
| Total, all-cause mortality | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 0 | 20 |
| Total, other adverse events | Olipudase Alfa: Participants From DFI13412 (Adults) | 5 | 5 |
| Total, other adverse events | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 20 | 20 |
| Total, serious adverse events | Olipudase Alfa: Participants From DFI13412 (Adults) | 1 | 5 |
| Total, serious adverse events | Olipudase Alfa: Participants From DFI13803 (Pediatrics) | 10 | 20 |
Source ClinicalTrials.gov
Papers4
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Olipudase Alfa: Participants From DFI13412 (Adults) vs Olipudase Alfa: Participants From DFI13803 (Pediatrics): deaths from any cause | Trial comparisons |
| Olipudase Alfa: Participants From DFI13412 (Adults) vs Olipudase Alfa: Participants From DFI13803 (Pediatrics): serious adverse events | Trial comparisons |
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/NCT02004704
GET https://conductscience.org/api/trials/v1/NCT02004704/registered?page=2