Trial · NCT03523728
A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients
NCT03523728, Phase 2, Phase 3, Terminated, Genzyme, a Sanofi Company; results first posted 2022-11-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients. Trials, record NCT03523728. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03523728
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients
- NCT number
- NCT03523728
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 478 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Polycystic Kidney, Autosomal Dominant
- Interventions
- Venglustat, Placebo
- First posted
- 2018-05-14
- Last updated
- 2023-02-03
- Other registries' IDs
- eudract:2017-004084-12
- Results first posted
- 2022-11-09
- Limitations and caveats
Following decision to discontinue EFC15392 based on the futility analysis, the two-steps analysis initially planned were not applicable, therefore only the final analysis of all safety endpoints (laboratory, vital sign, ECG, physical examination, BDI-II, and lens opacity) were performed on extended combined Stage 1 and Stage 2 safety population only.
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1 | From Baseline to Month 18 | Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using magnetic resonance imaging (MRI). The annualized slope of change in TKV (in percentage \[%\] per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment \* time interaction and mayo imaging classification \* time interaction and included random intercept and slope. |
| Primary | Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2 | From Baseline to Month 24 | An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR in each treatment group was obtained from the linear mixed effect model including the fixed categorical effects of treatment group (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per interactive response technology \[IRT\]: 1C, 1D, 1E), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope. |
| Secondary | Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1 | From Baseline to Month 24 | An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR was obtained from the linear mixed effect model including the fixed categorical effects of treatment group, mayo imaging classification (as per IRT), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope. Due to early termination of study for futility, the two-steps analysis initially planned was not applicable, then the annualized rate of change from baseline in eGFR in Stage 1 population were assessed using all data available up to database lock (i.e., including Month 24 assessment). As this is a slope, it allowed to have more data and to reduce variability. |
| Secondary | Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Combined Stage 1 and Stage 2 | From Baseline to Month 18 | Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using MRI. The annualized slope of change in TKV (in % per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment \* time interaction and mayo imaging classification \* time interaction and included random intercept and slope. |
| Secondary | Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | From Baseline to Month 18 | The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a numeric rating scale (NRS) to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicates greater intensity of pain. Least-squares (LS) means, and standard errors (SE) were estimated from mixed-effect model with repeated measures (MMRM) analysis. |
| Secondary | Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | From Baseline to Month 24 | The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a NRS to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicate greater intensity of pain. LS means and SE were estimated from MMRM analysis. |
| Secondary | Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | From Baseline to Month 18 | The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to 'Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis. |
| Secondary | Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | From Baseline to Month 24 | The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to "Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis. |
| Secondary | Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 | Day 1: 3 hours Post-Dose, Month 1: Pre-Dose and 3 hours Post-Dose, Month 6: Pre-Dose, Month 18: Pre-Dose | Venglustat plasma concentrations was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm. |
| Secondary | Pharmacokinetics: Plasma Concentration of Venglustat: Stage 2 | Month 1: Pre-dose and 3 hours Post-dose | Venglustat plasma concentrations was determined using a validated LC-MS/MS method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1 | From Baseline to Month 18 | Analysis was performed on Stage 1 intent-to-treat (ITT) population that includes all participants randomized in Stage 1 and were analyzed according to the intervention allocated by randomization (venglustat 15 mg, venglustat 8 mg or placebo). Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. | percent change in TKV/year | GEOMETRIC_LEAST_SQUARES_MEAN |
| PRIMARY | Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2 | From Baseline to Month 24 | Analysis was performed on Combined Stage 1 and Stage 2 ITT population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m\^2 at screening who are randomized in Stage 1 or Stage 2 and were analyzed according to the intervention allocated by randomization (venglustat 15 mg, venglustat 8 mg or placebo). Data for this outcome measure (OM) was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | mL/min/1.73 m^2/year | LEAST_SQUARES_MEAN |
| SECONDARY | Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1 | From Baseline to Month 24 | Analysis was performed on Stage 1 ITT population. | mL/min/1.73 m^2/year | LEAST_SQUARES_MEAN |
| SECONDARY | Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Combined Stage 1 and Stage 2 | From Baseline to Month 18 | As no post-baseline MRI data were collected in Stage 2 due to early study discontinuation, the analysis of TKV in Combined Stage 1 and Stage 2 was not done. | ||
| SECONDARY | Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | From Baseline to Month 18 | Analysis was performed on Stage 1 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | From Baseline to Month 24 | Analysis was performed on Combined Stage 1 and Stage 2 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | From Baseline to Month 18 | Analysis was performed on Stage 1 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | From Baseline to Month 24 | Analysis was performed on Combined Stage 1 and Stage 2 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 | Day 1: 3 hours Post-Dose, Month 1: Pre-Dose and 3 hours Post-Dose, Month 6: Pre-Dose, Month 18: Pre-Dose | Analysed on Stage 1 pharmacokinetic (PK) population that included all participants randomized in Stage 1 who took at least one dose of study drug and had at least one post-baseline PK assessment; analyzed according to the intervention they actually received. Here, number analyzed signifies participants with available data for each specified category. | nanograms per milliliter | MEAN |
| SECONDARY | Pharmacokinetics: Plasma Concentration of Venglustat: Stage 2 | Month 1: Pre-dose and 3 hours Post-dose | Analysis was performed on Stage 2 PK population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m\^2 at screening who were randomized in Stage 2, who took at least one dose of study drug and who had at least one post-baseline PK assessment. Participants were analyzed according to study drug they actually received. Here, number analyzed signifies participants with available data for each specified category. | nanograms per milliliter | MEAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group384
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1 | Stage 1- Placebo | 6.35 | 95% Confidence Interval 5.10 to 7.62 | percent change in TKV/year | |
| Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1 | Stage 1- Venglustat 8 mg | 7.71 | 95% Confidence Interval 6.46 to 8.98 | percent change in TKV/year | |
| Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1 | Stage 1- Venglustat 15 mg | 6.38 | 95% Confidence Interval 5.11 to 7.66 | percent change in TKV/year | |
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2 | Placebo | -2.40 | 95% Confidence Interval -3.30 to -1.49 | mL/min/1.73 m^2/year | |
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2 | Venglustat 8 mg | -4.82 | 95% Confidence Interval -5.82 to -3.83 | mL/min/1.73 m^2/year | |
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2 | Venglustat 15 mg | -4.89 | 95% Confidence Interval -5.80 to -3.99 | mL/min/1.73 m^2/year | |
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1 | Stage 1- Placebo | -3.14 | 95% Confidence Interval -4.09 to -2.18 | mL/min/1.73 m^2/year | |
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1 | Stage 1- Venglustat 8 mg | -4.74 | 95% Confidence Interval -5.68 to -3.80 | mL/min/1.73 m^2/year | |
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1 | Stage 1- Venglustat 15 mg | -4.59 | 95% Confidence Interval -5.53 to -3.65 | mL/min/1.73 m^2/year | |
| Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | Stage 1- Placebo | -0.09 | Standard Error 0.15 | score on a scale |
1 to 10 of 384Source ClinicalTrials.gov
See all 384 results by groupParticipant flow45
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Stage 1 (24 Months) | STARTED | Stage 1- Placebo | 78 | |
| Stage 1 (24 Months) | STARTED | Stage 1- Venglustat 8 mg | 78 | |
| Stage 1 (24 Months) | STARTED | Stage 1- Venglustat 15 mg | 80 | |
| Stage 1 (24 Months) | STARTED | Stage 2- Placebo | 0 | |
| Stage 1 (24 Months) | STARTED | Stage 2- Venglustat 15 mg | 0 | |
| Stage 1 (24 Months) | COMPLETED | Stage 1- Placebo | 12 | |
| Stage 1 (24 Months) | COMPLETED | Stage 1- Venglustat 8 mg | 10 | |
| Stage 1 (24 Months) | COMPLETED | Stage 1- Venglustat 15 mg | 12 | |
| Stage 1 (24 Months) | COMPLETED | Stage 2- Placebo | 0 | |
| Stage 1 (24 Months) | COMPLETED | Stage 2- Venglustat 15 mg | 0 |
1 to 10 of 45Source ClinicalTrials.gov
See all 45 participant flowAdverse events390
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 | Stage 1- Placebo | 5 | 78 | 5 |
| other | Covid-19 | Infections and infestations | Stage 1- Venglustat 8 mg | 6 | 78 | 6 |
| other | Covid-19 | Infections and infestations | Stage 1- Venglustat 15 mg | 1 | 80 | 1 |
| other | Covid-19 | Infections and infestations | Stage 2- Placebo | 3 | 122 | 3 |
| other | Covid-19 | Infections and infestations | Stage 2- Venglustat 15 mg | 7 | 119 | 7 |
| other | Conjunctivitis | Infections and infestations | Stage 1- Placebo | 4 | 78 | 5 |
| other | Conjunctivitis | Infections and infestations | Stage 1- Venglustat 8 mg | 0 | 78 | 0 |
| other | Conjunctivitis | Infections and infestations | Stage 1- Venglustat 15 mg | 1 | 80 | 1 |
| other | Conjunctivitis | Infections and infestations | Stage 2- Placebo | 1 | 122 | 1 |
| other | Conjunctivitis | Infections and infestations | Stage 2- Venglustat 15 mg | 1 | 119 | 1 |
1 to 10 of 390Source ClinicalTrials.gov
See all 390 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Stage 1- Placebo | 0 | 78 |
| Total, all-cause mortality | Stage 1- Venglustat 8 mg | 0 | 78 |
| Total, all-cause mortality | Stage 1- Venglustat 15 mg | 0 | 80 |
| Total, all-cause mortality | Stage 2- Placebo | 0 | 122 |
| Total, all-cause mortality | Stage 2- Venglustat 15 mg | 0 | 119 |
| Total, other adverse events | Stage 1- Placebo | 42 | 78 |
| Total, other adverse events | Stage 1- Venglustat 8 mg | 46 | 78 |
| Total, other adverse events | Stage 1- Venglustat 15 mg | 48 | 80 |
| Total, other adverse events | Stage 2- Placebo | 32 | 122 |
| Total, other adverse events | Stage 2- Venglustat 15 mg | 48 | 119 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 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.
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