Trial · NCT00413777
Tolvaptan Open-label Pilot Efficacy, Tolerability, and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT00413777, Phase 2, 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 Open-label Pilot Efficacy, Tolerability, and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD). Trials, record NCT00413777. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00413777
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Tolvaptan Open-label Pilot Efficacy, Tolerability, and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- NCT number
- NCT00413777
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 46 participants, actual
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Conditions
- Polycystic Kidney, Autosomal Dominant
- Interventions
- Tolvaptan
- First posted
- 2006-12-20
- Last updated
- 2017-07-02
- Results first posted
- 2017-07-02
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | AEs were recorded from screening (ICF was signed) until 7-Day follow-up | An AE was defined as any new medical problem, or exacerbation of an existing problem, experienced by a participant while enrolled in a study , whether or not it was considered drug-related by the study physician. A treatment-emergent AE (TEAE) was defined as an AE that started after start of study drug treatment; or if the event was continuous from Baseline and was serious, study drug related, or resulted in death, discontinuation. |
| Secondary | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose. | Baseline to Month 36 | Spot urine osmolality at trough was determined for urine samples collected immediately prior to morning dosing for Day 1 (Baseline), Months 2, 6, 12, 24, 36 for all participants. Sample was taken after the first morning's void and was provided as a mid-stream, clean catch sample. During the titration period (Weeks 1, 2, 3 and 4) and at Month 6, additional samples were collected for the preceding day immediately preceding the 2nd daily dose and at bed-time. These samples allowed derivation of an average nadir of spot urine osmolality concentrations at each dose level and while at steady state during extended tolvaptan administration. At the Month 36 visit, participants were given a urine container and brought back the specimen at Extension Day 1. All participants were fasting. |
| Secondary | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Second Daily Dose. | Baseline to Month 24 | Urine osmolality at steady state (after at least 4 days of dosing) including "absolute trough" prior to the second daily dose. Samples for this assessment were taken as closely coincident to PK blood sample as practical. During Weeks 1, 2, 3 and 4 of the Titration Period and at Month 6, additional samples were collected for the preceding day immediately preceding the second daily dose. These samples allowed derivation of an average nadir of spot urine osmolality concentrations at each dose level and while at steady state during extended tolvaptan administration. |
| Secondary | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Bedtime. | Baseline to Month 24 | Urine osmolality at steady state (after at least 4 days of dosing) including "average of troughs" (the mean urine osmolality prior to bedtime). Samples for this assessment were to be taken as closely coincident to PK blood sample as practical. During Weeks 1, 2, 3 and 4 of the Titration Period and at Month 6, additional samples were collected for the preceding day at bedtime. These samples allowed derivation of an average nadir of spot urine osmolality concentrations at each dose level and while at steady state during extended tolvaptan administration. |
| Secondary | Percent Change From Baseline in Renal Volume. | Baseline to Month 36 | Total Kidney Volume (TKV) was assessed by the central magnetic resonance imaging (MRI) rater. |
| Secondary | Change From Pre-dose Baseline in Renal Function Estimated by Glomerular Filtration Rate (GFR). | Baseline to Month 36 | GFR was estimated using reciprocal serum creatinine formula. The formula does not adjust for body weight or height, but this may be done to normalize to body surface area. The formula for reciprocal Serum creatinine is: 1/Pcr. (Pcr = serum creatinine concentration \[mg/dL\]). Clinic weight scales were calibrated at least yearly. |
| Secondary | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose- Extension. | Baseline to Months 2, 6, 12, 24, 36, Extension Day 1, Extension Month 12 | Spot urine osmolality at trough was determined for urine samples collected immediately prior to morning dosing for Day 1 (Baseline), Months 2, 6, 12, 24, 36, Extension Day 1, and Extension Month 12 for all participants. Sample was taken after the first morning's void and was provided as a mid-stream, clean catch sample. During the titration period (Weeks 1, 2, 3 and 4) and at Month 6, additional samples were collected for the preceding day immediately preceding the 2nd daily dose and at bed-time. These samples allowed derivation of an average nadir of spot urine osmolality concentrations at each dose level and while at steady state during extended tolvaptan administration. At the Month 36 visit, participants were given a urine container and brought back the specimen at Extension Day 1. All participants were fasting. |
| Secondary | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Second Daily Dose- Extension. | Baseline to Month 24 | Urine osmolality at steady state (after at least 4 days of dosing) including "absolute trough" prior to the second daily dose. Samples for this assessment were taken as closely coincident to PK blood sample as practical. During Weeks 1, 2, 3 and 4 of the Titration Period and at Month 6, additional samples were collected for the preceding day immediately preceding the second daily dose. These samples allowed derivation of an average nadir of spot urine osmolality concentrations at each dose level and while at steady state during extended tolvaptan administration. |
| Secondary | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Bedtime- Extension. | Baseline to Month 24 | Urine osmolality at steady state (after at least 4 days of dosing) including "average of troughs" (the mean urine osmolality prior to bedtime). Samples for this assessment were to be taken as closely coincident to PK blood sample as practical. During Weeks 1, 2, 3 and 4 of the Titration Period and at Month 6, additional samples were collected for the preceding day at bedtime. These samples allowed derivation of an average nadir of spot urine osmolality concentrations at each dose level and while at steady state during extended tolvaptan administration. |
| Secondary | Percent Change From Baseline in Renal Volume-Extension. | Baseline to Months 2, 12, 24, 36, Extension Day 1, Extension Month 12 | TKV was assessed by the central magnetic resonance imaging (MRI) rater. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | AEs were recorded from screening (ICF was signed) until 7-Day follow-up | Safety dataset was defined as all participants who consumed at least 1 dose of study medication. Safety variables analyzed included physical examinations, laboratory tests, vital signs, ECG's and AEs. | participants | NUMBER |
| SECONDARY | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose. | Baseline to Month 36 | The intent-to-treat (ITT) dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. Observed cases (OC) dataset were used. | mOsm/kg | MEAN |
| SECONDARY | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Second Daily Dose. | Baseline to Month 24 | The ITT dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. OC dataset were used. | mOsm/kg | MEAN |
| SECONDARY | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Bedtime. | Baseline to Month 24 | The ITT dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. OC dataset were used. | mOsm/kg | MEAN |
| SECONDARY | Percent Change From Baseline in Renal Volume. | Baseline to Month 36 | The ITT dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. OC dataset were used. | Percentage change per month | MEAN |
| SECONDARY | Change From Pre-dose Baseline in Renal Function Estimated by Glomerular Filtration Rate (GFR). | Baseline to Month 36 | The ITT dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. OC dataset were used. | dL/mg | MEAN |
| SECONDARY | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose- Extension. | Baseline to Months 2, 6, 12, 24, 36, Extension Day 1, Extension Month 12 | The intent-to-treat (ITT) dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. Observed cases (OC) dataset were used. | mOsm/kg | MEAN |
| SECONDARY | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Second Daily Dose- Extension. | Baseline to Month 24 | The ITT dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. OC dataset were used. | mOsm/kg | MEAN |
| SECONDARY | Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Bedtime- Extension. | Baseline to Month 24 | The ITT dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. OC dataset were used. | mOsm/kg | MEAN |
| SECONDARY | Percent Change From Baseline in Renal Volume-Extension. | Baseline to Months 2, 12, 24, 36, Extension Day 1, Extension Month 12 | The ITT dataset was defined as a dataset that included data from all participants who enrolled to the study with observations at Baseline and Post Baseline. OC dataset were used. | Percentage change per month | MEAN |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group318
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | Tolvaptan 45+15 mg | Participants with serious TEAEs | 3 | participants | |
| Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | Tolvaptan 60+30 mg | Participants with serious TEAEs | 8 | participants | |
| Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | Tolvaptan 45+15 mg | Participants with severe TEAEs | 6 | participants | |
| Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | Tolvaptan 60+30 mg | Participants with severe TEAEs | 10 | participants | |
| Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | Tolvaptan 45+15 mg | Participants discontinued due to AEs | 3 | participants | |
| Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. | Tolvaptan 60+30 mg | Participants discontinued due to AEs | 1 | participants | |
| Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose. | Tolvaptan 45+15 mg | Month 2 (N= 21, 22) | -274.10 | Standard Deviation 223.14 | mOsm/kg |
| Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose. | Tolvaptan 60+30 mg | Month 2 (N= 21, 22) | -228.00 | Standard Deviation 238.19 | mOsm/kg |
| Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose. | Tolvaptan 45+15 mg | Month 6 (N= 19, 23) | -288.42 | Standard Deviation 219.55 | mOsm/kg |
| Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose. | Tolvaptan 60+30 mg | Month 6 (N= 19, 23) | -263.78 | Standard Deviation 221.05 | mOsm/kg |
1 to 10 of 318Source ClinicalTrials.gov
See all 318 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Fixed-dose Period | STARTED | Tolvaptan 45+15 mg | 22 | |
| Fixed-dose Period | STARTED | Tolvaptan 60+30 mg | 24 | |
| Fixed-dose Period | COMPLETED | Tolvaptan 45+15 mg | 18 | |
| Fixed-dose Period | COMPLETED | Tolvaptan 60+30 mg | 21 | |
| Fixed-dose Period | NOT COMPLETED | Tolvaptan 45+15 mg | 4 | |
| Fixed-dose Period | NOT COMPLETED | Tolvaptan 60+30 mg | 3 | |
| Extension Period | STARTED | Tolvaptan 45+15 mg | 17 | |
| Extension Period | STARTED | Tolvaptan 60+30 mg | 18 | |
| Extension Period | COMPLETED | Tolvaptan 45+15 mg | 17 | |
| Extension Period | COMPLETED | Tolvaptan 60+30 mg | 18 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events134
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 45+15 mg | 3 | 22 | |
| other | Anaemia | Blood and lymphatic system disorders | Tolvaptan 60+30 mg | 1 | 24 | |
| other | Palpitations | Cardiac disorders | Tolvaptan 45+15 mg | 3 | 22 | |
| other | Palpitations | Cardiac disorders | Tolvaptan 60+30 mg | 1 | 24 | |
| other | Dry eye | Eye disorders | Tolvaptan 45+15 mg | 3 | 22 | |
| other | Dry eye | Eye disorders | Tolvaptan 60+30 mg | 2 | 24 | |
| other | Abdominal distension | Gastrointestinal disorders | Tolvaptan 45+15 mg | 3 | 22 | |
| other | Abdominal distension | Gastrointestinal disorders | Tolvaptan 60+30 mg | 2 | 24 | |
| other | Abdominal pain | Gastrointestinal disorders | Tolvaptan 45+15 mg | 2 | 22 | |
| other | Abdominal pain | Gastrointestinal disorders | Tolvaptan 60+30 mg | 5 | 24 |
1 to 10 of 134Source ClinicalTrials.gov
See all 134 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tolvaptan 45+15 mg | ||
| Total, all-cause mortality | Tolvaptan 60+30 mg | ||
| Total, other adverse events | Tolvaptan 45+15 mg | 20 | 22 |
| Total, other adverse events | Tolvaptan 60+30 mg | 23 | 24 |
| Total, serious adverse events | Tolvaptan 45+15 mg | 3 | 22 |
| Total, serious adverse events | Tolvaptan 60+30 mg | 8 | 24 |
Source ClinicalTrials.gov
Papers6
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 45+15 mg vs Tolvaptan 60+30 mg: 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/NCT00413777
GET https://conductscience.org/api/trials/v1/NCT00413777/registered?page=2