Trial · NCT02004691
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
NCT02004691, Phase 2, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2022-05-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency. Trials, record NCT02004691. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02004691
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
- NCT number
- NCT02004691
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 36 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- placebo (saline), Olipudase alfa
- First posted
- 2013-12-09
- Last updated
- 2024-11-07
- Other registries' IDs
- eudract:2015-000371-26
- Results first posted
- 2022-05-24
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline | Baseline (Day 1) | Percent predicted Hb and Altitude-adjusted DLco was calculated as: 100\*Adjusted DLco/Predicted DLco in unit of mL CO/min/mmHg where, adjusted DLco = Observed DLco (in mL CO/min/mmHg) times Hemoglobin-adjusted factor times Altitude-adjustment factor. |
| Primary | Percent Change From Baseline in Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Week 52 | Baseline, Week 52 | Percent predicted Hb and Altitude-adjusted DLco was calculated as: 100\*Adjusted DLco/Predicted DLco in unit of mL CO/min/mmHg where, adjusted DLco = Observed DLco (in mL CO/min/mmHg) times Hemoglobin-adjusted factor times Altitude-adjustment factor. |
| Primary | Combination Spleen Endpoint: Component 1: Spleen Volume (in MN) at Baseline | Baseline (Day 1) | Spleen volume was assessed by abdominal magnetic resonance imaging (MRI) to quantitate the degree of splenomegaly in multiples of normal (MN). |
| Primary | Combination Spleen Endpoint: Component 1: Percent Change From Baseline in Spleen Volume (in MN) at Week 52 | Baseline, Week 52 | Spleen volume was assessed by abdominal MRI to quantitate the degree of splenomegaly in MN. |
| Primary | Combination Spleen Endpoint (Primary for US Only): Component 2: Splenomegaly-Related Score (SRS) at Baseline | Baseline (Day 1) | The SRS rates 5 items: abdominal pain, abdominal discomfort, early satiety, dissatisfaction with abdominal body image, and difficulty to bend down using a numerical rating scale of 0 (absent) to 10 (worst imaginable). The total score of SRS ranges from 0 to 50, with higher scores (50) indicated worst imaginable rating. It was pre-specified as secondary endpoint for countries outside of US. |
| Primary | Combination Spleen Endpoint (Primary for US Only): Component 2: Change From Baseline in Splenomegaly-Related Score (SRS) at Week 52 | Baseline, Week 52 | The SRS rates 5 items: abdominal pain, abdominal discomfort, early satiety, dissatisfaction with abdominal body image, and difficulty to bend down using a numerical rating scale of 0 (absent) to 10 (worst imaginable). The total score of SRS ranges from 0 to 50, with higher scores (50) indicated worst imaginable rating. It was pre-specified as secondary endpoint for countries outside of US. |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) up to Week 52 | From the first infusion of investigational medicinal product (IMP) up to 52 weeks | An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered with a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TESAEs was defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. Reported AEs are treatment-emergent AEs that developed, worsened or became serious during the treatment period. |
| Secondary | Number of Participants With Adverse Events of Special Interest (AESIs) up to Week 52 | From the first infusion of IMP up to 52 weeks | An AESI was defined as an 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. AESIs included any pregnancy, symptomatic overdose, dose-limiting toxicities (DLTs) as defined in protocol and infusion associated reactions (IARs). 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. |
| Secondary | Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Liver Function Tests up to Week 52 | From Baseline (Day 1) up to 52 weeks | PCSA criteria: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST): \>3 x upper limit of normal (ULN), \>5 x ULN, \>10 x ULN and \>20 x ULN. Alkaline phosphatase (ALP) \> 1.5 x ULN. Total bilirubin \>1.5 x ULN and \>2 x ULN. ALT and total bilirubin: ALT \> 3 x ULN and total bilirubin \> 2 x ULN. Baseline was defined as the last non-missing value prior to the first infusion of study treatment. |
| Secondary | Number of Participants Who Developed Anti-Drug Antibodies (ADA) to Olipudase Alfa up to Week 52 | Baseline (Day 1) and Week 52 | Number of participants with treatment-emergent ADA are presented. Baseline was defined as the last non-missing value prior to the first infusion of study treatment. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline | Baseline (Day 1) | Analysis was performed on mITT population. | % Predicted DLco | MEAN |
| PRIMARY | Percent Change From Baseline in Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Week 52 | Baseline, Week 52 | Analysis was performed on mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | percent change | LEAST_SQUARES_MEAN |
| PRIMARY | Combination Spleen Endpoint: Component 1: Spleen Volume (in MN) at Baseline | Baseline (Day 1) | Analysis was performed on mITT population. | multiples of normal (MN) | MEAN |
| PRIMARY | Combination Spleen Endpoint: Component 1: Percent Change From Baseline in Spleen Volume (in MN) at Week 52 | Baseline, Week 52 | Analysis was performed on mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | percent change | LEAST_SQUARES_MEAN |
| PRIMARY | Combination Spleen Endpoint (Primary for US Only): Component 2: Splenomegaly-Related Score (SRS) at Baseline | Baseline (Day 1) | Analysis was performed on mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | score on a scale | MEAN |
| PRIMARY | Combination Spleen Endpoint (Primary for US Only): Component 2: Change From Baseline in Splenomegaly-Related Score (SRS) at Week 52 | Baseline, Week 52 | Analysis was performed on mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) up to Week 52 | From the first infusion of investigational medicinal product (IMP) up to 52 weeks | The safety population was a subset of the randomized population and included randomized participants who received at least 1 infusion (partial or total). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Adverse Events of Special Interest (AESIs) up to Week 52 | From the first infusion of IMP up to 52 weeks | The safety population was a subset of the randomized population and included randomized participants who received at least 1 infusion (partial or total). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Liver Function Tests up to Week 52 | From Baseline (Day 1) up to 52 weeks | The safety population was a subset of the randomized population and included randomized participants who received at least 1 infusion (partial or total). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Anti-Drug Antibodies (ADA) to Olipudase Alfa up to Week 52 | Baseline (Day 1) and Week 52 | The safety population was a subset of the randomized population and included randomized participants who received at least 1 infusion (partial or total). | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group62
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline | Placebo | 48.45 | Standard Deviation 10.76 | % Predicted DLco | |
| Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline | Olipudase Alfa | 49.44 | Standard Deviation 10.99 | % Predicted DLco | |
| Percent Change From Baseline in Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Week 52 | Placebo | 2.961 | Standard Error 3.3832 | percent change | |
| Percent Change From Baseline in Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Week 52 | Olipudase Alfa | 21.968 | Standard Error 3.3362 | percent change | |
| Combination Spleen Endpoint: Component 1: Spleen Volume (in MN) at Baseline | Placebo | 11.21 | Standard Deviation 3.84 | multiples of normal (MN) | |
| Combination Spleen Endpoint: Component 1: Spleen Volume (in MN) at Baseline | Olipudase Alfa | 11.69 | Standard Deviation 4.92 | multiples of normal (MN) | |
| Combination Spleen Endpoint: Component 1: Percent Change From Baseline in Spleen Volume (in MN) at Week 52 | Placebo | 0.481 | Standard Error 2.5002 | percent change | |
| Combination Spleen Endpoint: Component 1: Percent Change From Baseline in Spleen Volume (in MN) at Week 52 | Olipudase Alfa | -39.446 | Standard Error 2.4294 | percent change | |
| Combination Spleen Endpoint (Primary for US Only): Component 2: Splenomegaly-Related Score (SRS) at Baseline | Placebo | 28.05 | Standard Deviation 10.56 | score on a scale | |
| Combination Spleen Endpoint (Primary for US Only): Component 2: Splenomegaly-Related Score (SRS) at Baseline | Olipudase Alfa | 24.55 | Standard Deviation 11.13 | score on a scale |
1 to 10 of 62Source ClinicalTrials.gov
See all 62 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| PAP: Up to 52 Weeks | STARTED | Placebo | 18 | |
| PAP: Up to 52 Weeks | STARTED | Olipudase Alfa | 18 | |
| PAP: Up to 52 Weeks | COMPLETED | Placebo | 17 | |
| PAP: Up to 52 Weeks | COMPLETED | Olipudase Alfa | 18 | |
| PAP: Up to 52 Weeks | NOT COMPLETED | Placebo | 1 | |
| PAP: Up to 52 Weeks | NOT COMPLETED | Olipudase Alfa | 0 | |
| ETP: From Week 52 up to 5 Years | STARTED | Placebo | 17 | |
| ETP: From Week 52 up to 5 Years | STARTED | Olipudase Alfa | 18 | |
| ETP: From Week 52 up to 5 Years | COMPLETED | Placebo | 13 | |
| ETP: From Week 52 up to 5 Years | COMPLETED | Olipudase Alfa | 16 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events132
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 | Covid-19 | Infections and infestations | PAP: Placebo | 0 | 18 | 0 |
| other | Covid-19 | Infections and infestations | PAP: Olipudase Alfa | 0 | 18 | 0 |
| other | Covid-19 | Infections and infestations | ETP: Placebo/Olipudase Alfa | 7 | 17 | 8 |
| other | Covid-19 | Infections and infestations | ETP: Olipudase Alfa/Olipudase Alfa | 8 | 18 | 9 |
| other | Nasopharyngitis | Infections and infestations | PAP: Placebo | 6 | 18 | 8 |
| other | Nasopharyngitis | Infections and infestations | PAP: Olipudase Alfa | 8 | 18 | 18 |
| other | Nasopharyngitis | Infections and infestations | ETP: Placebo/Olipudase Alfa | 5 | 17 | 10 |
| other | Nasopharyngitis | Infections and infestations | ETP: Olipudase Alfa/Olipudase Alfa | 7 | 18 | 22 |
| other | Upper Respiratory Tract Infection | Infections and infestations | PAP: Placebo | 4 | 18 | 6 |
| other | Upper Respiratory Tract Infection | Infections and infestations | PAP: Olipudase Alfa | 6 | 18 | 8 |
1 to 10 of 132Source ClinicalTrials.gov
See all 132 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PAP: Placebo | 0 | 18 |
| Total, all-cause mortality | PAP: Olipudase Alfa | 0 | 18 |
| Total, all-cause mortality | ETP: Placebo/Olipudase Alfa | 0 | 17 |
| Total, all-cause mortality | ETP: Olipudase Alfa/Olipudase Alfa | 0 | 18 |
| Total, other adverse events | PAP: Placebo | 13 | 18 |
| Total, other adverse events | PAP: Olipudase Alfa | 17 | 18 |
| Total, other adverse events | ETP: Placebo/Olipudase Alfa | 9 | 17 |
| Total, other adverse events | ETP: Olipudase Alfa/Olipudase Alfa | 12 | 18 |
| Total, serious adverse events | PAP: Placebo | 4 | 18 |
| Total, serious adverse events | PAP: Olipudase Alfa | 3 | 18 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Olipudase Alfa vs Placebo: Number of Participants Who Developed Anti-Drug Antibodies (ADA) to Olipudase Alfa up to Week 52 |
Source 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/NCT02004691
GET https://conductscience.org/api/trials/v1/NCT02004691/results?page=2