Trial · NCT01214421
Tolvaptan Extension Study in Participants With ADPKD
NCT01214421, Phase 3, Completed, Otsuka Pharmaceutical Development & Commercialization, Inc.; results first posted 2021-10-25
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Tolvaptan Extension Study in Participants With ADPKD. Trials, record NCT01214421. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01214421
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Tolvaptan Extension Study in Participants With ADPKD
- NCT number
- NCT01214421
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 1,083 participants, actual
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Conditions
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- Interventions
- Tolvaptan
- First posted
- 2010-10-05
- Last updated
- 2021-10-25
- Other registries' IDs
- eudract:2010-018401-10
- Results first posted
- 2021-10-25
- Limitations and caveats
The trial did not meet its primary endpoint as both groups showed similar decreases in the rate of TKV growth due in part to unforeseen limitations in the trial design. However, early-treatment resulted in cumulative improvements in renal function.
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From the Baseline in Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Study Baseline (Prior to Day 1 in Study 156-04-251) to Month 24 in this study (Study 156-08-271) | Total kidney volume is a measure of disease progression in the ADPKD participants. 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, radiologists used proprietary software to measure the volume of both kidneys in participants continuing from previous study (156-04-251) at Month 24 of this study (156-08-271) comparing change in TKV for the early-treated (those previously treated with tolvaptan) to delayed-treated (those previously treated with placebo). The percent change in the volume of both kidneys combined was analysed using mixed-effect model repeated measures (MMRM) analysis and reported. This outcome measure was analyzed only in the participants enrolled from the previous study - 156-04-251, as pre-specified in the protocol. |
| Secondary | Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Study Baseline (Prior to Day 1 in Study 156-04-251) to Month 24 in this study (Study 156-08-271) | Estimated Glomerular Filtration Rate (eGFR) according to CKD-EPI is calculated using the CKD-EPI equation, expressed as a single equation, is GFR = 141 × min (serum creatinine \[Scr\]/κ, 1)α × max(Scr/κ, 1)\^-1.209 × 0.993 Age × 1.018 (if female) × 1.159 (if black), where Scr is serum creatinine, κ is 0.7 for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/ĸ or 1, and max indicates the maximum of Scr/κ or 1 in participants continuing from previous study (156-04-251) at Month 24 of this study (156-08-271) comparing change in eGFR for the early-treated (those previously treated with tolvaptan) to delayed-treated (those previously treated with placebo). MMRM was used for the analysis. This outcome measure was analyzed only in the participants enrolled from the previous - 156-04-251, as pre-specified in the protocol. |
| Secondary | Annualized Slope of Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Study Baseline (Prior to Day 1 in Study 156-08-271) to Month 24 in this study (Study 156-08-271) | Annualized slope of TKV is a measure of renal function and disease progression in ADPKD participants. The annualized slope is calculated as percentage of growth in TKV (measured in mL by MRI) divided by each participant's years of participation for all participants was calculated using MMRM analysis in participants continuing from previous study (156-04-251) at Month 24 of this study (156-08-271) comparing annualized slope of TKV for the early-treated (those previously treated with tolvaptan) to delayed-treated (those previously treated with placebo). This outcome measure was analyzed only in the participants enrolled from the previous study - 156-04-251, as pre-specified in the protocol. |
| Secondary | Annualized Slope of eGFR (CKD-EPI) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Study Baseline (Prior to Day 1 in Study 156-08-271) to Month 24 (Study 156-08-271) | Annualized slope of eGFR (CKD-EPI) is a measure of renal function and disease progression in ADPKD participants. The annualized slope of eGFR (CKD-EPI) (divided by each participant's years of participation) for all participants was calculated using MMRM analysis in participants continuing from previous study (156-04-251) at Month 24 of this study (156-08-271) comparing change in TKV for the early-treated (those previously treated with tolvaptan) to delayed-treated (those previously treated with placebo). eGFR was calculated using the Chronic Kidney Disease-Epidemiology (CKD-EPI) formula. This outcome measure was analyzed only in the participants enrolled from the previous study - 156-04-251, as pre-specified in the protocol. |
| Secondary | Annualized TKV Slope for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271 | Tolvaptan, Delayed Treated: Baseline to Month 24 in Study 156-08-271; Placebo: Baseline to Month 36 in Study 156-04-251 | Annualized slope of TKV is a measure of renal function and disease progression in ADPKD participants. The annualized slope is calculated as percentage of growth in TKV (measured in mL by MRI) divided by each participant's years of participation, using MMRM analysis to compare annualized slope of TKV for the participants who received placebo in previous study (156-04-251) to annualized slope of TKV for the same participants who received tolvaptan in this study (156-08-271). This outcome measure was analyzed only in the participants enrolled from the previous study - 156-04-251 and who received placebo in the previous study, as pre-specified in the protocol. |
| Secondary | Annualized Slope of Renal Function (eGFRCKD-EPI) for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271 | Tolvaptan, Delayed Treated: Baseline to Month 24 in Study 156-08-271; Placebo: Baseline to Month 36 in Study 156-04-251 | Annualized slope of eGFR (CKD-EPI) is a measure of renal function and disease progression in ADPKD participants. The annualized slope of eGFR (calculated using CKD-EPI formula) divided by each participant's years of participation, using MMRM analysis to compare annualized slope of eGFR (CKD-EPI) for the participants who received placebo in previous study (156-04-251) to the annualized slope of eGFR (CKD-EPI) for the same participants who received tolvaptan in this study (156-08-271). This outcome measure was analyzed only in the participants enrolled from the previous study - 156-04-251 who received placebo in previous study and received tolvaptan in this study, as pre-specified in the protocol. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From the Baseline in Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Study Baseline (Prior to Day 1 in Study 156-04-251) to Month 24 in this study (Study 156-08-271) | The Efficacy Sample consisted of all enrolled participants who took at least 1 dose of study drug and completed 3 years of study drug treatment in previous Study 156-04-251. Overall number of participants analyzed signifies the number of participants who were evaluable for this outcome measure. | percent change | MEAN |
| SECONDARY | Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Study Baseline (Prior to Day 1 in Study 156-04-251) to Month 24 in this study (Study 156-08-271) | The Efficacy Sample consisted of all enrolled participants who took at least 1 dose of study drug and completed 3 years of study drug treatment in previous Study 156-04-251. Overall number of participants analyzed signifies the number of participants who were evaluable for this outcome measure. | mL/min/1.73 square meters (m^2) | LEAST_SQUARES_MEAN |
| SECONDARY | Annualized Slope of Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Study Baseline (Prior to Day 1 in Study 156-08-271) to Month 24 in this study (Study 156-08-271) | The Efficacy Sample consisted of all enrolled participants who took at least 1 dose of study drug and completed 3 years of study drug treatment in previous Study 156-04-251. Overall number of participants analyzed signifies the number of participants who were evaluable for this outcome measure. | percent change in TKV/participant-years | NUMBER |
| SECONDARY | Annualized Slope of eGFR (CKD-EPI) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Study Baseline (Prior to Day 1 in Study 156-08-271) to Month 24 (Study 156-08-271) | The Efficacy Sample consisted of all enrolled participants who took at least 1 dose of study drug and completed 3 years of study drug treatment in previous Study 156-04-251. Overall number of participants analyzed signifies the number of participants who were evaluable for this outcome measure. | ml/min/1.73m^2/participant-years | NUMBER |
| SECONDARY | Annualized TKV Slope for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271 | Tolvaptan, Delayed Treated: Baseline to Month 24 in Study 156-08-271; Placebo: Baseline to Month 36 in Study 156-04-251 | The Efficacy Sample consisted of all enrolled participants who took at least 1 dose of study drug and completed 3 years of study drug treatment in previous Study 156-04-251. Overall number of participants analyzed signifies the number of participants who were evaluable for this outcome measure. | percent change in TKV/participant-years | NUMBER |
| SECONDARY | Annualized Slope of Renal Function (eGFRCKD-EPI) for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271 | Tolvaptan, Delayed Treated: Baseline to Month 24 in Study 156-08-271; Placebo: Baseline to Month 36 in Study 156-04-251 | The Efficacy Sample consisted of all enrolled participants who took at least 1 dose of study drug and completed 3 years of study drug treatment in previous Study 156-04-251. Overall number of participants analyzed signifies the number of participants who were evaluable for this outcome measure. | ml/min/1.73m^2/participant-years | NUMBER |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From the Baseline in Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Tolvaptan, Early Treated (From Study 156-04-251: Tolvaptan) | 28.66 | Standard Deviation 26.14 | percent change | |
| Percent Change From the Baseline in Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Tolvaptan, Delayed Treated (From Study 156-04-251: Placebo) | 30.58 | Standard Deviation 27.21 | percent change | |
Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Tolvaptan, Early Treated (From Study 156-04-251: Tolvaptan) | -16.77 | Standard Deviation 13.16 | mL/min/1.73 square meters (m^2) | |
Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) | Tolvaptan, Delayed Treated (From Study 156-04-251: Placebo) | -19.92 | Standard Deviation 16.40 | mL/min/1.73 square meters (m^2) | |
| Annualized Slope of Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Tolvaptan, Early Treated (From Study 156-04-251: Tolvaptan) | 6.164 | percent change in TKV/participant-years | ||
| Annualized Slope of Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Tolvaptan, Delayed Treated (From Study 156-04-251: Placebo) | 4.960 | percent change in TKV/participant-years | ||
| Annualized Slope of eGFR (CKD-EPI) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Tolvaptan, Early Treated (From Study 156-04-251: Tolvaptan) | -3.255 | ml/min/1.73m^2/participant-years | ||
| Annualized Slope of eGFR (CKD-EPI) for Study 156-04-251 Participants Enrolled in Study 156-08-271 | Tolvaptan, Delayed Treated (From Study 156-04-251: Placebo) | -3.142 | ml/min/1.73m^2/participant-years | ||
| Annualized TKV Slope for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271 | Tolvaptan, Delayed Treated (In Study 156-08-271) | 4.779 | percent change in TKV/participant-years | ||
| Annualized TKV Slope for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271 | Placebo (In Study 156-04-251) | 5.627 | percent change in TKV/participant-years |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 557 | |
| Overall Study | STARTED | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 314 | |
| Overall Study | STARTED | Tolvaptan (From Other Studies: Tolvaptan, Early Treated) | 181 | |
| Overall Study | STARTED | Tolvaptan (From Other Studies: Placebo, Delayed Treated) | 31 | |
| Overall Study | COMPLETED | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 507 | |
| Overall Study | COMPLETED | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 256 | |
| Overall Study | COMPLETED | Tolvaptan (From Other Studies: Tolvaptan, Early Treated) | 143 | |
| Overall Study | COMPLETED | Tolvaptan (From Other Studies: Placebo, Delayed Treated) | 22 | |
| Overall Study | NOT COMPLETED | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 50 | |
| Overall Study | NOT COMPLETED | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 58 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events980
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 (From Study 156-04-251: Tolvaptan, Early Treated) | 35 | 557 | |
| other | Anaemia | Blood and lymphatic system disorders | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 20 | 314 | |
| other | Anaemia | Blood and lymphatic system disorders | Tolvaptan (From Other Studies: Tolvaptan, Early Treated) | 13 | 181 | |
| other | Anaemia | Blood and lymphatic system disorders | Tolvaptan (From Other Studies: Placebo, Delayed Treated) | 0 | 31 | |
| other | Abdominal Pain | Gastrointestinal disorders | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 37 | 557 | |
| other | Abdominal Pain | Gastrointestinal disorders | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 15 | 314 | |
| other | Abdominal Pain | Gastrointestinal disorders | Tolvaptan (From Other Studies: Tolvaptan, Early Treated) | 13 | 181 | |
| other | Abdominal Pain | Gastrointestinal disorders | Tolvaptan (From Other Studies: Placebo, Delayed Treated) | 0 | 31 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 23 | 557 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 16 | 314 |
1 to 10 of 980Source ClinicalTrials.gov
See all 980 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 5 | 557 |
| Total, all-cause mortality | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 0 | 314 |
| Total, all-cause mortality | Tolvaptan (From Other Studies: Tolvaptan, Early Treated) | 0 | 181 |
| Total, all-cause mortality | Tolvaptan (From Other Studies: Placebo, Delayed Treated) | 0 | 31 |
| Total, other adverse events | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 528 | 557 |
| Total, other adverse events | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 310 | 314 |
| Total, other adverse events | Tolvaptan (From Other Studies: Tolvaptan, Early Treated) | 175 | 181 |
| Total, other adverse events | Tolvaptan (From Other Studies: Placebo, Delayed Treated) | 29 | 31 |
| Total, serious adverse events | Tolvaptan (From Study 156-04-251: Tolvaptan, Early Treated) | 143 | 557 |
| Total, serious adverse events | Tolvaptan (From Study 156-04-251: Placebo, Delayed Treated) | 79 | 314 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers6
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.
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/NCT01214421
GET https://conductscience.org/api/trials/v1/NCT01214421/events?page=2