Trial · NCT02964273
Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)
NCT02964273, Phase 3, Completed, Otsuka Pharmaceutical Development & Commercialization, Inc.; results first posted 2023-01-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease). Trials, record NCT02964273. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02964273
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)
- NCT number
- NCT02964273
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 91 participants, actual
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Conditions
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- Interventions
- Tolvaptan, Tolvaptan Matching-placebo
- First posted
- 2016-11-16
- Last updated
- 2023-01-03
- Other registries' IDs
- eudract:2016-000187-42
- Results first posted
- 2023-01-03
Source ClinicalTrials.gov
Registered outcomes22
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Phase A: Change From Baseline in Spot Urine Osmolality (Pre-morning Dose) | Baseline, and Week 1 of Phase A | Urine osmolality is a measure of urine concentration, measured by osmometer, which evaluates the freezing point depression of a solution and supplies results as milliosmoles per kilogram of water. Spot urine osmolality was determined for urine samples collected immediately prior to morning dosing for Day 1 (Baseline), and Week 1 for all participants. Sample was taken after the first morning's void and was provided as a mid-stream, clean catch sample. All participants were fasting. |
| Primary | Phase A: Change From Baseline in Specific Gravity (Pre-morning Dose) | Baseline, and Week 1 of Phase A | Urine specific gravity is a measure of the concentration of solutes in the urine and provides information on the kidney's ability to concentrate urine. Spot urine sample for determination of specific gravity was collected immediately prior to morning dosing for Day 1 (Baseline), and Week 1 for all participants. Sample was taken after the first morning's void and was provided as a mid-stream, clean catch sample. All participants were fasting. |
| Secondary | Phase A: Percent Change From Phase A Baseline in Height-Adjusted Total Kidney Volume (htTKV) as Measured by Magnetic Resonance Imaging (MRI) | Baseline, and Month 12 of Phase A | htTKV is used in participants with autosomal dominant polycystic kidney disease to predict the onset of renal insufficiency. |
| Secondary | Phase A and B: Mean 24-hour Fluid Balance Prior to Week 1 | Prior to Week 1 in Phase A and B | Participants were instructed to record all fluid taken and all urine output for the 24-hour period. |
| Secondary | Phase A: Change From Baseline in Renal Function (Estimated Glomerular Filtration Rate [eGFR] by Schwartz Formula) at Each Clinic Visit in Phase A | Phase A Baseline, Months 1, 6, and 12 | Renal function was assessed by estimated eGFR calculated by the Schwartz formula (eGFR = 0.413 × height \[cm\] /serum creatinine mg/dL), expressed as mean change in eGFR at the specified time points. The units for the data reported are milliliter per minute per 1.73 meter square (mL/min/1.73 m\^2). The baseline was the evaluation done at Week 1 in Phase A for this outcome measure. |
| Secondary | Phase B: Change From Phase B Baseline in Renal Function (eGFR by Schwartz Formula) at Each Clinic Visit in Phase B | Phase B Baseline, Week 1, Months 1, 6, 12, 18, and 24 | Renal function was assessed by estimated eGFR calculated by the Schwartz formula (eGFR = 0.413 × height \[cm\] /serum creatinine mg/dL), expressed as mean change in eGFR at the specified time points. |
| Secondary | Phase B: Percent Change From Phase B Baseline in htTKV as Measured by MRI at Month 12 and Month 24 | Phase B Baseline, Months 12 and 24 | htTKV is used in participants with autosomal dominant polycystic kidney disease to predict the onset of renal insufficiency. |
| Secondary | Phase A: 24-hour Urine Volume | 24 hours post dose after Month 1 on study medication in Phase A | Urine volume refers to the quantity of urine produced per unit of time. |
| Secondary | Phase A: 24-hour Fluid Intake | 24 hours post dose after Month 1 on study medication in Phase A | Daily fluid intake (total water) is defined as the amount of water consumed from foods, plain drinking water, and other beverages. |
| Secondary | Phase A: 24-hour Fluid Balance | 24 hours post dose after Month 1 on study medication in Phase A | Fluid balance is a term used to describe the balance of the input and output of fluids in the body to allow metabolic processes to function correctly. |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 registered outcomesOutcomes22
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Phase A: Change From Baseline in Spot Urine Osmolality (Pre-morning Dose) | Baseline, and Week 1 of Phase A | The Full Analysis Set (FAS) included all participants who were randomized to a treatment group, received at least 1 dose of the investigational medicinal product (IMP), and had both a Phase A baseline and at least 1 postbaseline efficacy evaluation. Number analyzed is the number of participants with data available for analysis at the given time point. | milliosmoles per kilogram (mOsm/kg) | MEAN |
| PRIMARY | Phase A: Change From Baseline in Specific Gravity (Pre-morning Dose) | Baseline, and Week 1 of Phase A | The FAS included all participants who were randomized to a treatment group, received at least 1 dose of the IMP, and had both a Phase A baseline and at least 1 postbaseline efficacy evaluation. Number analyzed is the number of participants with data available for analysis at the given time point. | ratio | MEAN |
| SECONDARY | Phase A: Percent Change From Phase A Baseline in Height-Adjusted Total Kidney Volume (htTKV) as Measured by Magnetic Resonance Imaging (MRI) | Baseline, and Month 12 of Phase A | The FAS included all participants who were randomized to a treatment group, received at least 1 dose of the IMP, and had both a Phase A baseline and at least 1 postbaseline efficacy evaluation. Overall number analyzed are the number of participants with data available for analyses. | percent change | MEAN |
| SECONDARY | Phase A and B: Mean 24-hour Fluid Balance Prior to Week 1 | Prior to Week 1 in Phase A and B | The FAS for Phase A included all participants who were randomized to a treatment group, received at least 1 dose of the IMP, and had both a Phase A Baseline and at least 1 postbaseline efficacy evaluation. The FAS for Phase B included all participants who enrolled to Phase B, received at least 1 dose of the IMP, and had both a baseline and at least 1 postbaseline efficacy evaluation in Phase B. Overall number analyzed are the number of participants with data available for analyses. | milliliter | MEAN |
| SECONDARY | Phase A: Change From Baseline in Renal Function (Estimated Glomerular Filtration Rate [eGFR] by Schwartz Formula) at Each Clinic Visit in Phase A | Phase A Baseline, Months 1, 6, and 12 | The FAS included all participants who were randomized to a treatment group, received at least 1 dose of the IMP, and had both a Phase A baseline and at least 1 postbaseline efficacy evaluation. Number analyzed is the number of participants with data available for analysis at the given time point. | mL/min/1.73 m^2 | MEAN |
| SECONDARY | Phase B: Change From Phase B Baseline in Renal Function (eGFR by Schwartz Formula) at Each Clinic Visit in Phase B | Phase B Baseline, Week 1, Months 1, 6, 12, 18, and 24 | The FAS for Phase B included all participants who enrolled to Phase B, received at least 1 dose of the IMP, and had both a baseline and at least 1 postbaseline efficacy evaluation in Phase B. Number analyzed is the number of participants with data available for analysis at the given time point. | mL/min/1.73 m^2 | MEAN |
| SECONDARY | Phase B: Percent Change From Phase B Baseline in htTKV as Measured by MRI at Month 12 and Month 24 | Phase B Baseline, Months 12 and 24 | The FAS for Phase B included all participants who enrolled to Phase B, received at least 1 dose of the IMP, and had both a baseline and at least 1 postbaseline efficacy evaluation in Phase B. Number analyzed is the number of participants with data available for analysis at the given time point. | percent change | MEAN |
| SECONDARY | Phase A: 24-hour Urine Volume | 24 hours post dose after Month 1 on study medication in Phase A | The Dense Pharmacokinetic/Pharmacodynamic (PK/PD) Population included a subset of participants half on tolvaptan and half on placebo, in the 12 to 17 year old age group who had dense PK sampling after at least 1 month on IMP. Overall number analyzed are the number of participants with data available for analyses. | milliliter per 24 hours post dose | MEAN |
| SECONDARY | Phase A: 24-hour Fluid Intake | 24 hours post dose after Month 1 on study medication in Phase A | The Dense PK/PD Population included a subset of participants half on tolvaptan and half on placebo, in the 12 to 17 year old age group who had dense PK sampling after at least 1 month on IMP. Overall number analyzed are the number of participants with data available for analyses. | milliliter | MEAN |
| SECONDARY | Phase A: 24-hour Fluid Balance | 24 hours post dose after Month 1 on study medication in Phase A | The Dense PK/PD Population included a subset of participants half on tolvaptan and half on placebo, in the 12 to 17 year old age group who had dense PK sampling after at least 1 month on IMP. Overall number analyzed are the number of participants with data available for analyses. | milliliter | MEAN |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 outcomesResults by group603
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Phase A: Change From Baseline in Spot Urine Osmolality (Pre-morning Dose) | Phase A: Tolvaptan | Baseline | 635 | Standard Deviation 252 | milliosmoles per kilogram (mOsm/kg) |
| Phase A: Change From Baseline in Spot Urine Osmolality (Pre-morning Dose) | Phase A: Placebo | Baseline | 646 | Standard Deviation 250 | milliosmoles per kilogram (mOsm/kg) |
| Phase A: Change From Baseline in Spot Urine Osmolality (Pre-morning Dose) | Phase A: Tolvaptan | Change From Baseline at Week 1, Phase A | -386 | Standard Deviation 284 | milliosmoles per kilogram (mOsm/kg) |
| Phase A: Change From Baseline in Spot Urine Osmolality (Pre-morning Dose) | Phase A: Placebo | Change From Baseline at Week 1, Phase A | -93 | Standard Deviation 332 | milliosmoles per kilogram (mOsm/kg) |
| Phase A: Change From Baseline in Specific Gravity (Pre-morning Dose) | Phase A: Tolvaptan | Baseline | 1.017 | Standard Deviation 0.006 | ratio |
| Phase A: Change From Baseline in Specific Gravity (Pre-morning Dose) | Phase A: Placebo | Baseline | 1.017 | Standard Deviation 0.006 | ratio |
| Phase A: Change From Baseline in Specific Gravity (Pre-morning Dose) | Phase A: Tolvaptan | Change From Baseline at Week 1, Phase A | -0.009 | Standard Deviation 0.007 | ratio |
| Phase A: Change From Baseline in Specific Gravity (Pre-morning Dose) | Phase A: Placebo | Change From Baseline at Week 1, Phase A | -0.002 | Standard Deviation 0.008 | ratio |
| Phase A: Percent Change From Phase A Baseline in Height-Adjusted Total Kidney Volume (htTKV) as Measured by Magnetic Resonance Imaging (MRI) | Phase A: Tolvaptan | 2.28 | Standard Deviation 8.75 | percent change | |
| Phase A: Percent Change From Phase A Baseline in Height-Adjusted Total Kidney Volume (htTKV) as Measured by Magnetic Resonance Imaging (MRI) | Phase A: Placebo | 6.11 | Standard Deviation 7.48 | percent change |
1 to 10 of 603Source ClinicalTrials.gov
See all 603 results by groupParticipant flow28
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Phase A: Double-blind Period (12 Months) | STARTED | Phase A: Tolvaptan | 48 | |
| Phase A: Double-blind Period (12 Months) | STARTED | Phase A: Placebo | 43 | |
| Phase A: Double-blind Period (12 Months) | STARTED | Phase B: Prior Tolvaptan | 0 | |
| Phase A: Double-blind Period (12 Months) | STARTED | Phase B: Prior Placebo | 0 | |
| Phase A: Double-blind Period (12 Months) | Dense Pharmacokinetic/Pharmacodynamic (PK/PD) Population | Phase A: Tolvaptan | 12 | |
| Phase A: Double-blind Period (12 Months) | Dense Pharmacokinetic/Pharmacodynamic (PK/PD) Population | Phase A: Placebo | 8 | |
| Phase A: Double-blind Period (12 Months) | Dense Pharmacokinetic/Pharmacodynamic (PK/PD) Population | Phase B: Prior Tolvaptan | 0 | |
| Phase A: Double-blind Period (12 Months) | Dense Pharmacokinetic/Pharmacodynamic (PK/PD) Population | Phase B: Prior Placebo | 0 | |
| Phase A: Double-blind Period (12 Months) | COMPLETED | Phase A: Tolvaptan | 44 | |
| Phase A: Double-blind Period (12 Months) | COMPLETED | Phase A: Placebo | 40 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 participant flowAdverse events324
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 | Ear pain | Ear and labyrinth disorders | Phase A: Tolvaptan | 3 | 48 | |
| other | Ear pain | Ear and labyrinth disorders | Phase A: Placebo | 2 | 43 | |
| other | Ear pain | Ear and labyrinth disorders | Phase B: Prior Tolvaptan | 2 | 42 | |
| other | Ear pain | Ear and labyrinth disorders | Phase B: Prior Placebo | 2 | 39 | |
| other | Abdominal pain | Gastrointestinal disorders | Phase A: Tolvaptan | 6 | 48 | |
| other | Abdominal pain | Gastrointestinal disorders | Phase A: Placebo | 3 | 43 | |
| other | Abdominal pain | Gastrointestinal disorders | Phase B: Prior Tolvaptan | 4 | 42 | |
| other | Abdominal pain | Gastrointestinal disorders | Phase B: Prior Placebo | 12 | 39 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Phase A: Tolvaptan | 5 | 48 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Phase A: Placebo | 4 | 43 |
1 to 10 of 324Source ClinicalTrials.gov
See all 324 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Phase A: Tolvaptan | 0 | 48 |
| Total, all-cause mortality | Phase A: Placebo | 0 | 43 |
| Total, all-cause mortality | Phase B: Prior Tolvaptan | 0 | 42 |
| Total, all-cause mortality | Phase B: Prior Placebo | 0 | 39 |
| Total, other adverse events | Phase A: Tolvaptan | 45 | 48 |
| Total, other adverse events | Phase A: Placebo | 42 | 43 |
| Total, other adverse events | Phase B: Prior Tolvaptan | 38 | 42 |
| Total, other adverse events | Phase B: Prior Placebo | 39 | 39 |
| Total, serious adverse events | Phase A: Tolvaptan | 1 | 48 |
| Total, serious adverse events | Phase A: Placebo | 6 | 43 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers4
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/NCT02964273
GET https://conductscience.org/api/trials/v1/NCT02964273/registered?page=2