Trial · NCT04705051
Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat
NCT04705051, Phase 3, Terminated, Sanofi; results first posted 2022-10-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat. Trials, record NCT04705051. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04705051
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat
- NCT number
- NCT04705051
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 24 participants, actual
- Lead sponsor
- Sanofi
- Conditions
- Congenital Cystic Kidney Disease
- Interventions
- Venglustat GZ402671
- First posted
- 2021-01-12
- Last updated
- 2025-09-17
- Other registries' IDs
- eudract:2020-004400-34
- Results first posted
- 2022-10-06
- Limitations and caveats
The study was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392. Termination decision was due to lack of efficacy in ADPKD population and not linked to safety findings with venglustat.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change in Total Kidney Volume (TKV) | From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823 | Total kidney volume is a measure of disease progression in the ADPKD participants. Study LTS15823 was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392 (NCT03523728). Study EFC15392 was terminated based on the results of the planned prespecified futility analysis performed for this study. Termination decision was due to a lack of efficacy in the ADPKD population and not linked to safety findings with venglustat. Due to the early termination of study LTS15823 and low enrollment numbers, efficacy analysis was not performed. |
| Secondary | Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) | From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823 | eGFR is a measure of kidney function assessed through blood/serum. Study LTS15823 was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392 (NCT03523728). Study EFC15392 was terminated based on the results of the planned prespecified futility analysis performed for this study. Termination decision was due to a lack of efficacy in the ADPKD population and not linked to safety findings with venglustat. Due to the early termination of study LTS15823 and low enrollment numbers, efficacy analysis was not performed. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From the first administration of the IMP in LTS15823 study up to the last IMP administration in LTS15823 study + 30 days (i.e., up to approximately 20 weeks) | Adverse event (AE) was defined as any untoward medical occurrence in participant who received investigational medicinal product (IMP) and did not necessarily have a causal relationship with treatment. All reported AEs were TEAEs that developed/worsened during 'TEAE period' (defined as the time from the first administration of the IMP in LTS15823 study up to the last IMP administration + 30 days). A SAEs was defined as any AE that at any dose results in: death; is life threatening; requires initial or prolonged inpatient hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; is a suspected transmission of any infectious agent via an authorized medicinal product; is a medically important event. |
| Secondary | Change From Baseline in Lens Clarity During the Open-label Extension Treatment-emergent Period of LTS15823 Study | Baseline (EFC15392 study Baseline); from first administration of IMP up to last administration of IMP of open-label extension study LTS15823 + 30 days (i.e., up to approximately 20 weeks), in comparison to the EFC15392 study Baseline | Change from EFC15392 study Baseline in the lens clarity was assessed by ophthalmological examination with World Health Organization (WHO) simplified cataract grading system during the open label extension treatment-emergent period of LTS15823 study. Study LTS15823 was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392 (NCT03523728). Study EFC15392 was terminated based on the results of the planned prespecified futility analysis performed for this study. Termination decision was due to a lack of efficacy in the ADPKD population and not linked to safety findings with venglustat. Baseline was defined as the values from EFC15392 study Baseline. |
| Secondary | Effect on Mood With Change From Baseline in Beck Depression Inventory (BDI-II) Score During the Open-label Extension Treatment-emergent Period of LTS15823 Study | Baseline (EFC15392 study Baseline); from first IMP administration up to 3 months in study LTS15823, in comparison to the EFC15392 study Baseline | BDI-II was a validated self-reported instrument of 21 questions which were each scored on a Likert scale of 0 to 3. Total score was calculated as sum of all scores of 21 questions. Total scores ranged from 0 (minimal depression) to 63 (severe depression), with higher score totals indicating more severe depression symptoms (0 to 13: indicates minimal depression;14 to 19: indicates mild depression; 20 to 28: indicates moderate depression; and 29 to 63: indicates severe depression). A negative change from baseline indicated an improvement. Baseline was defined as the values from EFC15392 study Baseline. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change in Total Kidney Volume (TKV) | From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823 | For study LTS15823, due to low number of enrollments as compared to the planned numbers and very limited data available post-baseline (only 2 participants reached the planned Month 3 visit), the analysis was carried out only on the available safety and tolerability data. Due to early termination of the study, no efficacy data was available at Month 24 hence the efficacy analysis was not performed. | ||
| SECONDARY | Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) | From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823 | For study LTS15823, due to low number of enrollments as compared to the planned numbers and very limited data available post-baseline (only 2 participants reached the planned Month 3 visit), the analysis was carried out only on the available safety and tolerability data. Due to early termination of the study, no efficacy data was available at Month 24 hence the efficacy analysis was not performed. | ||
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From the first administration of the IMP in LTS15823 study up to the last IMP administration in LTS15823 study + 30 days (i.e., up to approximately 20 weeks) | Analysis was performed on Long-term extension safety population which included all participants who had received at least one dose of study medication. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Lens Clarity During the Open-label Extension Treatment-emergent Period of LTS15823 Study | Baseline (EFC15392 study Baseline); from first administration of IMP up to last administration of IMP of open-label extension study LTS15823 + 30 days (i.e., up to approximately 20 weeks), in comparison to the EFC15392 study Baseline | For study LTS15823, due to early termination of the study, no ophthalmological data was collected at the planned visits as per protocol and thus the planned analysis was not performed. | ||
| SECONDARY | Effect on Mood With Change From Baseline in Beck Depression Inventory (BDI-II) Score During the Open-label Extension Treatment-emergent Period of LTS15823 Study | Baseline (EFC15392 study Baseline); from first IMP administration up to 3 months in study LTS15823, in comparison to the EFC15392 study Baseline | Analysis was performed on long-term extension safety population. BDI score was collected for 16 participants at Baseline and up to 3 months at the end of trial visit i.e., within 30 days after last dose of study drug following the decision for early termination of the study. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group4
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) and Serious Adverse Events (SAEs) | Venglustat 15mg | Any TEAEs | 2 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Venglustat 15mg | SAEs | 1 | Participants | |
| Effect on Mood With Change From Baseline in Beck Depression Inventory (BDI-II) Score During the Open-label Extension Treatment-emergent Period of LTS15823 Study | Venglustat 15mg | Baseline | 2.188 | Standard Deviation 4.086 | score on a scale |
| Effect on Mood With Change From Baseline in Beck Depression Inventory (BDI-II) Score During the Open-label Extension Treatment-emergent Period of LTS15823 Study | Venglustat 15mg | Change from Baseline | -0.875 | Standard Deviation 4.455 | score on a scale |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Venglustat 15mg | 24 | |
| Overall Study | Treated | Venglustat 15mg | 23 | |
| Overall Study | COMPLETED | Venglustat 15mg | 0 | |
| Overall Study | NOT COMPLETED | Venglustat 15mg | 24 |
Source ClinicalTrials.gov
Adverse events1
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 |
|---|---|---|---|---|---|---|
| serious | Wound Infection | Infections and infestations | Venglustat 15mg | 1 | 23 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Venglustat 15mg | 0 | 23 |
| Total, other adverse events | Venglustat 15mg | 0 | 23 |
| Total, serious adverse events | Venglustat 15mg | 1 | 23 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT04705051
GET https://conductscience.org/api/trials/v1/NCT04705051/registered