Trial · NCT00428948
Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT00428948, Phase 3, Completed, Otsuka Pharmaceutical Development & Commercialization, Inc.; results first posted 2017-07-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD). Trials, record NCT00428948. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00428948
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- NCT number
- NCT00428948
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 1,445 participants, actual
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Conditions
- Polycystic Kidney Disease, Autosomal Dominant
- Interventions
- Tolvaptan, Placebo
- First posted
- 2007-01-30
- Last updated
- 2017-07-02
- Other registries' IDs
- eudract:2006-002768-24
- Results first posted
- 2017-07-02
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Change Per Year in Total Kidney Volume From Baseline to Month 36 | Baseline to Month 36 | Kidney volume was assessed in T1-weighted magnetic resonance images collected at each study site and sent to a central reviewing facility. At the central reviewing facility, blinded radiologists used proprietary software to measure the volume of both kidneys. |
| Secondary | Number of ADPKD Clinical Progression Events Per 100 Follow-up Years From Baseline to Month 36 | Baseline to Month 36 | These ADPKD events in the key secondary Outcome Measure were selected on the basis of their potential relationship to progressing cystogenesis. Reducing the rate of cyst development and expansion would likely slow the progression of ADPKD. The 4 events were: (1) Onset or progression of hypertension (someone is hypertensive if they have \> 139 mmHg systolic blood pressure \[BP\], \> 89 mmHg diastolic BP, or if they are taking antihypertensive medication at any BP level); (2) severe renal pain requiring medical intervention; (3) worsening albuminuria (by category, see below); and (4) worsening renal function, defined as a 25% decrease in 1/serum creatinine from Baseline. Albuminuria was assessed using spot urine albumin/creatinine ratio measurements (all measurements in mg/mmol). Categories included normal (\< 2.8 female or \< 2.0 male), microalbuminuria (2.8-28 female or 2.0-20 male), and overt proteinuria (\> 28 female or \> 20 male. |
| Secondary | Change in Renal Function Per Year From Week 3 to Month 36 | Week 3 to Month 36 | Renal function was assessed using serum creatinine measurements and was estimated using 1/serum creatinine. The formula for 1/serum creatinine is: 1/Pcr, where Pcr = serum creatinine concentration (mg/dL). The change in renal function per year was based on the slope of change, obtained by regressing renal function data against time by subject. |
| Secondary | Change in Mean Arterial Blood Pressure Per Year in Non-hypertensive Participants From Baseline to Month 36 | Baseline to Month 36 | For participants who were non-hypertensive (systolic BP ≤ 139 mmHg and diastolic BP ≤ 89 mmHg without taking antihypertensive medications) at baseline, mean arterial blood pressure was measured at scheduled clinic visits up to the point of exposure to antihypertensive therapy for any reason. The change in mean arterial blood pressure per year was based on the slope of blood pressure, obtained by regressing blood pressure against time by subject. |
| Secondary | Area Under the Concentration-time Curve of Change in Renal Pain From Baseline to Month 36 | At screening, Baseline, Day 1, every 4 months up to month 36/early tremination (ET), follow-up visit 1 and 2 | Change from baseline in renal pain was assessed by a 0 to 10 pain scale as average area under the concentration-time curve (AUC) between baseline and the last trial visit or the last visit prior to initiating medical (eg, narcotic or anti-nociceptives \[eg, tricyclic antidepressants\]) or surgical therapy for pain. In the pain scale, score 0 represented no pain at all and score 10 represented the worst pain. A negative change score indicates less pain. AUC of renal pain was derived from renal pain scores within treatment period and was calculated using the trapezoidal rule, by dividing the number of days between the first and last assessment. |
| Secondary | Number of Hypertensive Events Per 100 Follow-up Years in Non-hypertensive Participants From Baseline to Month 36 | Baseline to Month 36 | A hypertensive event was defined as a change from non-hypertensive (systolic BP ≤ 139 mmHg and diastolic BP ≤ 89 mmHg without taking antihypertensive medications) status to 1 of 3 conditions: (1) High pre-hypertensive (systolic BP \[sBP\] \> 129 mmHg and/or diastolic BP \[dBP\] \> 84 mmHg), (2) hypertensive (sBP \> 139 mmHg and/or dBP \> 89 mmHg), or (3) requiring antihypertensive therapy. |
| Secondary | Percentage of Participants With a Clinically Sustained Decrease of Blood Pressure Leading to a Sustained Reduction in Antihypertensive Therapy From Baseline to Month 36 | Baseline to Month 36 |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Change Per Year in Total Kidney Volume From Baseline to Month 36 | Baseline to Month 36 | Intent-to-treat population: All randomized participants who had Baseline and post-baseline observations of total kidney volume. Only participants with available data were included in the analysis. | Percentage change per year | MEAN |
| SECONDARY | Number of ADPKD Clinical Progression Events Per 100 Follow-up Years From Baseline to Month 36 | Baseline to Month 36 | Intent-to-treat population: All randomized participants. Only participants with available data were included in the analysis. | Events/100 follow-up years | NUMBER |
| SECONDARY | Change in Renal Function Per Year From Week 3 to Month 36 | Week 3 to Month 36 | Intent-to-treat population: All randomized participants with at least 4 months of follow-up. Only participants with available data were included in the analysis. | (mg/mL)^-1 per year | MEAN |
| SECONDARY | Change in Mean Arterial Blood Pressure Per Year in Non-hypertensive Participants From Baseline to Month 36 | Baseline to Month 36 | Intent-to-treat population: All randomized participants with at least 4 months of follow-up. Only non-hypertensive participants with available data were included in the analysis. | mmHg | MEAN |
| SECONDARY | Area Under the Concentration-time Curve of Change in Renal Pain From Baseline to Month 36 | At screening, Baseline, Day 1, every 4 months up to month 36/early tremination (ET), follow-up visit 1 and 2 | Intent-to-treat population: All randomized participants with at least 4 months of follow-up. Only participants with available data were included in the analysis. | units on a scale | MEAN |
| SECONDARY | Number of Hypertensive Events Per 100 Follow-up Years in Non-hypertensive Participants From Baseline to Month 36 | Baseline to Month 36 | Intent-to-treat population: All randomized participants with at least 4 months of follow-up. Only non-hypertensive participants with available data were included in the analysis. | Events/100 follow-up years | NUMBER |
| SECONDARY | Percentage of Participants With a Clinically Sustained Decrease of Blood Pressure Leading to a Sustained Reduction in Antihypertensive Therapy From Baseline to Month 36 | Baseline to Month 36 | Intent-to-treat population: All randomized participants with at least 4 months of follow-up. Only participants taking antihypertensive medication at Baseline with available data were included in the analysis. | Percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Change Per Year in Total Kidney Volume From Baseline to Month 36 | Tolvaptan | 2.777 | Standard Deviation 5.659 | Percentage change per year | |
| Percentage Change Per Year in Total Kidney Volume From Baseline to Month 36 | Placebo | 5.608 | Standard Deviation 5.330 | Percentage change per year | |
| Number of ADPKD Clinical Progression Events Per 100 Follow-up Years From Baseline to Month 36 | Tolvaptan | 43.94 | Events/100 follow-up years | ||
| Number of ADPKD Clinical Progression Events Per 100 Follow-up Years From Baseline to Month 36 | Placebo | 50.04 | Events/100 follow-up years | ||
| Change in Renal Function Per Year From Week 3 to Month 36 | Tolvaptan | -2.555 | Standard Deviation 9.767 | (mg/mL)^-1 per year | |
| Change in Renal Function Per Year From Week 3 to Month 36 | Placebo | -3.682 | Standard Deviation 6.361 | (mg/mL)^-1 per year | |
| Change in Mean Arterial Blood Pressure Per Year in Non-hypertensive Participants From Baseline to Month 36 | Tolvaptan | 2.561 | Standard Deviation 9.798 | mmHg | |
| Change in Mean Arterial Blood Pressure Per Year in Non-hypertensive Participants From Baseline to Month 36 | Placebo | 2.592 | Standard Deviation 7.095 | mmHg | |
| Area Under the Concentration-time Curve of Change in Renal Pain From Baseline to Month 36 | Tolvaptan | -0.00 | Standard Deviation 1.29 | units on a scale | |
| Area Under the Concentration-time Curve of Change in Renal Pain From Baseline to Month 36 | Placebo | 0.08 | Standard Deviation 1.43 | units on a scale |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Tolvaptan | 961 | |
| Overall Study | STARTED | Placebo | 484 | |
| Overall Study | Received Treatment | Tolvaptan | 961 | |
| Overall Study | Received Treatment | Placebo | 483 | |
| Overall Study | COMPLETED | Tolvaptan | 740 | |
| Overall Study | COMPLETED | Placebo | 417 | |
| Overall Study | NOT COMPLETED | Tolvaptan | 221 | |
| Overall Study | NOT COMPLETED | Placebo | 67 |
Source ClinicalTrials.gov
Adverse events504
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 | Anaemia | Blood and lymphatic system disorders | Tolvaptan | 30 | 961 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 26 | 483 | |
| other | Abdominal distension | Gastrointestinal disorders | Tolvaptan | 49 | 961 | |
| other | Abdominal distension | Gastrointestinal disorders | Placebo | 17 | 483 | |
| other | Abdominal pain | Gastrointestinal disorders | Tolvaptan | 63 | 961 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 32 | 483 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Tolvaptan | 63 | 961 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 43 | 483 | |
| other | Constipation | Gastrointestinal disorders | Tolvaptan | 81 | 961 | |
| other | Constipation | Gastrointestinal disorders | Placebo | 12 | 483 |
1 to 10 of 504Source ClinicalTrials.gov
See all 504 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tolvaptan | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Tolvaptan | 793 | 961 |
| Total, other adverse events | Placebo | 372 | 483 |
| Total, serious adverse events | Tolvaptan | 177 | 961 |
| Total, serious adverse events | Placebo | 95 | 483 |
Source ClinicalTrials.gov
Papers22
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 |
|---|
| Tolvaptan vs Placebo: serious adverse events |
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/NCT00428948
GET https://conductscience.org/api/trials/v1/NCT00428948/events?page=2