Trial · NCT04064346
Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease
NCT04064346, Phase 3, Terminated, Palladio Biosciences; results first posted 2023-02-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease. Trials, record NCT04064346. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04064346
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease
- NCT number
- NCT04064346
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 12 participants, actual
- Lead sponsor
- Palladio Biosciences
- Conditions
- Autosomal Dominant Polycystic Kidney, ADPKD
- Interventions
- Lixivaptan, Placebo
- First posted
- 2019-08-21
- Last updated
- 2023-02-06
- Results first posted
- 2023-02-06
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized Change in Estimated Glomerular Filtration Rate (eGFR) - Part 1 | Baseline to the end of Follow-up Period I (up to 71 weeks) | The annualized change in eGFR will be calculated from the Chronic Kidney Disease Epidemiology Collaboration eGFR creatinine equation refit without the race variable (CKD-EPIcr\_R) equation for serum creatinine from baseline (mean of 3 eGFR determinations obtained during Screening and Placebo Run-in Periods \[Visits 1a/1b (if required), Visit 2, and Visit 3\]) to final assessment (mean of 3 eGFR determinations obtained during Follow-up Period I or, for participants who discontinue treatment prior to Visit 22, during the Follow-up Period 8 to 28 days following study drug treatment discontinuation). |
| Primary | Annualized Change in Estimated Glomerular Filtration Rate (eGFR) - Part 2 | Baseline to the end of Follow-up Period II (up to 64 weeks) | The annualized change in eGFR will be calculated from the CKD-EPIcr\_R equation for serum creatinine from baseline (mean of 3 eGFR determinations obtained during obtained during Follow-up Period I) to final assessment (mean of 3 eGFR determinations obtained during Follow-up Period II or, for participants who discontinue treatment prior to Visit 43, during the Follow-up Period 8 to 28 days following study drug treatment discontinuation). |
| Secondary | Number of Participants With Serum Alanine Aminotransferase (ALT) Levels >3 × the Upper Limit of Normal (ULN) - Part 1 | From the end of the Single-blind Lixivaptan Titration Period to the end of Follow-up Period I (maximum of 102 days) | Number of participants randomized to lixivaptan with serum ALT levels \>3 × the ULN compared to those randomized to placebo. The normal range of ALT was defined as 0-33 or 0-44 U/L, depending on the lab used. |
| Secondary | Number of Participants With Serum ALT Levels >3 × the ULN - Part 2 | Duration of the Lixivaptan Re-titration Period and the Maintenance Treatment Period (56 weeks) and Follow-up Period II (4 weeks) | Number of participants randomized to lixivaptan with serum ALT levels \>3 × ULN compared to those randomized to placebo. The normal range of ALT was defined as 0-33 or 0-44 U/L, depending on the lab used. |
| Secondary | eGFR Slope - Part 1 | Baseline to the end of Double-blind Randomized Treatment Period (up to 67 weeks), with changes from baseline calculated every 4 weeks during the Double-blind Randomized Treatment Period | Annualized rate of change (slope) in eGFR for serum creatinine, based on all eGFR determinations from baseline to all visits with on-treatment eGFR values during the Double-blind, Randomized Treatment Period. The baseline value will be calculated as the mean of 3 eGFR determinations obtained during Screening and Placebo Run-in Periods (Visits 1a/1b \[if required\], Visit 2, and Visit 3). |
| Secondary | eGFR Slope - Part 2 | Baseline to the end of the Maintenance Treatment Period (up to 60 weeks), with changes from baseline calculated every 4 weeks during the Maintenance Treatment Period | Annualized rate of change (slope) in eGFR for serum creatinine, based on all eGFR determinations from baseline to all visits with on-treatment eGFR values during the Maintenance Treatment Period. The baseline value will be calculated as the mean of 3 eGFR determinations obtained during obtained during Follow-up Period I. |
| Secondary | Height-adjusted Total Kidney Volume (htTKV) - Part 1 | Baseline to the end of Follow-up Period I (up to 71 weeks) | Annualized percent change in htTKV, determined by magnetic resonance imaging (MRI), from baseline (obtained at Screening \[Visit 1a\]) to the end of Follow-up Period I. |
| Secondary | Height-adjusted Total Kidney Volume (htTKV) - Part 2 | Baseline to the end of Follow-up Period II (up to 60 weeks) | Annualized percent change in htTKV, determined by MRI from baseline (obtained at Visit 25/Week 56) to the end of Follow-up Period II. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Lixivaptan Titration Period in Part 1 | Up to 6 weeks | Number of participants with TEAEs during the Lixivaptan Titration Period. |
| Secondary | Number of Participants With TEAEs After Randomization in Part 1 | From Randomization to study completion (up to 102 days) | Number of participants with TEAEs by treatment group after Randomization (Double-blind Randomized Treatment Period and Follow-up Period I) |
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 Change in Estimated Glomerular Filtration Rate (eGFR) - Part 1 | Baseline to the end of Follow-up Period I (up to 71 weeks) | Efficacy data were not analyzed as only 5 participants were randomized, and all 5 participants were prematurely terminated from the study drug by the sponsor less than 1 month following randomization. One month after randomization was the first scheduled post-randomization visit during which efficacy parameters would have been collected. | ||
| PRIMARY | Annualized Change in Estimated Glomerular Filtration Rate (eGFR) - Part 2 | Baseline to the end of Follow-up Period II (up to 64 weeks) | Efficacy data were not analyzed as only 5 participants were randomized, and all 5 participants were prematurely terminated from the study drug by the sponsor less than 1 month following randomization. One month after randomization was the first scheduled post-randomization visit during which efficacy parameters would have been collected. The study terminated prior to any participants entering Part 2 of study. | ||
| SECONDARY | Number of Participants With Serum Alanine Aminotransferase (ALT) Levels >3 × the Upper Limit of Normal (ULN) - Part 1 | From the end of the Single-blind Lixivaptan Titration Period to the end of Follow-up Period I (maximum of 102 days) | All participants who entered the Double-blind Randomized Treatment Period. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Serum ALT Levels >3 × the ULN - Part 2 | Duration of the Lixivaptan Re-titration Period and the Maintenance Treatment Period (56 weeks) and Follow-up Period II (4 weeks) | The study terminated prior to any participants entering Part 2 of study. | ||
| SECONDARY | eGFR Slope - Part 1 | Baseline to the end of Double-blind Randomized Treatment Period (up to 67 weeks), with changes from baseline calculated every 4 weeks during the Double-blind Randomized Treatment Period | Efficacy data were not analyzed as only 5 participants were randomized, and all 5 participants were prematurely terminated from the study drug by the sponsor less than 1 month following randomization. One month after randomization was the first scheduled post-randomization visit during which efficacy parameters would have been collected. | ||
| SECONDARY | eGFR Slope - Part 2 | Baseline to the end of the Maintenance Treatment Period (up to 60 weeks), with changes from baseline calculated every 4 weeks during the Maintenance Treatment Period | Efficacy data were not analyzed as only 5 participants were randomized, and all 5 participants were prematurely terminated from the study drug by the sponsor less than 1 month following randomization. One month after randomization was the first scheduled post-randomization visit during which efficacy parameters would have been collected. The study terminated prior to any participants entering Part 2 of study. | ||
| SECONDARY | Height-adjusted Total Kidney Volume (htTKV) - Part 1 | Baseline to the end of Follow-up Period I (up to 71 weeks) | Efficacy data were not analyzed as only 5 participants were randomized, and all 5 participants were prematurely terminated from the study drug by the sponsor less than 1 month following randomization. One month after randomization was the first scheduled post-randomization visit during which efficacy parameters would have been collected. | ||
| SECONDARY | Height-adjusted Total Kidney Volume (htTKV) - Part 2 | Baseline to the end of Follow-up Period II (up to 60 weeks) | Efficacy data were not analyzed as only 5 participants were randomized, and all 5 participants were prematurely terminated from the study drug by the sponsor less than 1 month following randomization. One month after randomization was the first scheduled post-randomization visit during which efficacy parameters would have been collected. The study terminated prior to any participants entering Part 2 of study. | ||
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Lixivaptan Titration Period in Part 1 | Up to 6 weeks | All participants entering the Lixivaptan Titration Period. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With TEAEs After Randomization in Part 1 | From Randomization to study completion (up to 102 days) | All participants who were randomized. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group14
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 Serum Alanine Aminotransferase (ALT) Levels >3 × the Upper Limit of Normal (ULN) - Part 1 | Lixivaptan | 0 | Participants | ||
| Number of Participants With Serum Alanine Aminotransferase (ALT) Levels >3 × the Upper Limit of Normal (ULN) - Part 1 | Placebo | 1 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Lixivaptan Titration Period in Part 1 | Lixivaptan Titration Period Participants | 3 | Participants | ||
| Number of Participants With TEAEs After Randomization in Part 1 | Lixivaptan | 1 | Participants | ||
| Number of Participants With TEAEs After Randomization in Part 1 | Placebo | 1 | Participants | ||
| Number of Participants With Potentially Clinically Important Clinical Laboratory Findings During the Lixivaptan Titration Period in Part 1 | Lixivaptan Titration Period Participants | 0 | Participants | ||
| Number of Participants With Potentially Clinically Important Clinical Laboratory Findings After Randomization in Part 1 | Lixivaptan | 0 | Participants | ||
| Number of Participants With Potentially Clinically Important Clinical Laboratory Findings After Randomization in Part 1 | Placebo | 0 | Participants | ||
| Number of Participants With Potentially Clinically Important Vital Signs Findings During the Lixivaptan Titration Period in Part 1 | Lixivaptan Titration Period Participants | 0 | Participants | ||
| Number of Participants With Potentially Clinically Important Vital Signs Findings After Randomization in Part 1 | Lixivaptan | 0 | Participants |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow63
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Pre-randomization Single-blind Period | STARTED | All Subjects Pre-randomization | 12 | |
| Pre-randomization Single-blind Period | STARTED | Lixivaptan | 0 | |
| Pre-randomization Single-blind Period | STARTED | Placebo | 0 | |
| Pre-randomization Single-blind Period | Treated in Placebo Run-in | All Subjects Pre-randomization | 12 | |
| Pre-randomization Single-blind Period | Treated in Placebo Run-in | Lixivaptan | 0 | |
| Pre-randomization Single-blind Period | Treated in Placebo Run-in | Placebo | 0 | |
| Pre-randomization Single-blind Period | Treated in Lixivaptan Titration Period | All Subjects Pre-randomization | 8 | |
| Pre-randomization Single-blind Period | Treated in Lixivaptan Titration Period | Lixivaptan | 0 | |
| Pre-randomization Single-blind Period | Treated in Lixivaptan Titration Period | Placebo | 0 | |
| Pre-randomization Single-blind Period | COMPLETED | All Subjects Pre-randomization | 5 |
1 to 10 of 63Source ClinicalTrials.gov
See all 63 participant flowAdverse events39
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 distension | Gastrointestinal disorders | Lixivaptan Titration Period | 1 | 8 | 1 |
| other | Abdominal distension | Gastrointestinal disorders | Lixivaptan (Randomized Treatment Period) | 0 | 4 | 0 |
| other | Abdominal distension | Gastrointestinal disorders | Placebo (Randomized Treatment Period) | 0 | 1 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Lixivaptan Titration Period | 1 | 8 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Lixivaptan (Randomized Treatment Period) | 0 | 4 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo (Randomized Treatment Period) | 0 | 1 | 0 |
| other | Nasopharyngitis | Infections and infestations | Lixivaptan Titration Period | 1 | 8 | 1 |
| other | Nasopharyngitis | Infections and infestations | Lixivaptan (Randomized Treatment Period) | 0 | 4 | 0 |
| other | Nasopharyngitis | Infections and infestations | Placebo (Randomized Treatment Period) | 0 | 1 | 0 |
| other | Influenza A virus test positive | Investigations | Lixivaptan Titration Period | 1 | 8 | 1 |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lixivaptan Titration Period | 0 | 8 |
| Total, all-cause mortality | Lixivaptan (Randomized Treatment Period) | 0 | 4 |
| Total, all-cause mortality | Placebo (Randomized Treatment Period) | 0 | 1 |
| Total, other adverse events | Lixivaptan Titration Period | 3 | 8 |
| Total, other adverse events | Lixivaptan (Randomized Treatment Period) | 1 | 4 |
| Total, other adverse events | Placebo (Randomized Treatment Period) | 1 | 1 |
| Total, serious adverse events | Lixivaptan Titration Period | 0 | 8 |
| Total, serious adverse events | Lixivaptan (Randomized Treatment Period) | 0 | 4 |
| Total, serious adverse events | Placebo (Randomized Treatment Period) | 0 | 1 |
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 comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Lixivaptan vs Placebo: Number of Participants With Potentially Clinically Important Vital Signs Findings After Randomization in Part 1 | Trial comparisons |
| Lixivaptan vs Placebo: Number of Participants With Serum Alanine Aminotransferase (ALT) Levels >3 × the Upper Limit of Normal (ULN) - Part 1 | Trial comparisons |
| Lixivaptan vs Placebo: Number of Participants With Potentially Clinically Significant 12-lead ECG Findings After Randomization in Part 1 | Trial comparisons |
| Lixivaptan vs Placebo: Number of Participants With TEAEs After Randomization in Part 1 | Trial comparisons |
| Lixivaptan vs Placebo: Number of Participants With Potentially Clinically Important Clinical Laboratory Findings After Randomization in Part 1 | 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.
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Read this trial from software.
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