| Primary | Phase A: Change From Baseline in Spot Urine Osmolality (Pre-morning Dose) | 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. | 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. | Posted | | Mean | Standard Deviation | milliosmoles per kilogram (mOsm/kg) | | Baseline, and Week 1 of Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
| | | Title | Denominators | Categories |
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| Baseline | - ParticipantsOG00048
- ParticipantsOG00143
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| Primary | Phase A: Change From Baseline in Specific Gravity (Pre-morning Dose) | 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. | 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. | Posted | | Mean | Standard Deviation | ratio | | Baseline, and Week 1 of Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo |
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| Secondary | Phase A: Percent Change From Phase A Baseline in Height-Adjusted Total Kidney Volume (htTKV) as Measured by Magnetic Resonance Imaging (MRI) | htTKV is used in participants with autosomal dominant polycystic kidney disease to predict the onset of renal insufficiency. | 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. | Posted | | Mean | Standard Deviation | percent change | | Baseline, and Month 12 of Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A and B: Mean 24-hour Fluid Balance Prior to Week 1 | Participants were instructed to record all fluid taken and all urine output for the 24-hour period. | 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. | Posted | | Mean | Standard Deviation | milliliter | | Prior to Week 1 in Phase A and B | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A: Change From Baseline in Renal Function (Estimated Glomerular Filtration Rate [eGFR] by Schwartz Formula) at Each Clinic Visit in Phase A | 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. | 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. | Posted | | Mean | Standard Deviation | mL/min/1.73 m^2 | | Phase A Baseline, Months 1, 6, and 12 | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 |
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| Secondary | Phase B: Change From Phase B Baseline in Renal Function (eGFR by Schwartz Formula) at Each Clinic Visit in Phase B | 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 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. | Posted | | Mean | Standard Deviation | mL/min/1.73 m^2 | | Phase B Baseline, Week 1, Months 1, 6, 12, 18, and 24 | | | | ID | Title | Description |
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| OG000 | Phase B: Prior Tolvaptan | Qualified participants (defined as those who were willing to continue in the trial and who did not have any adverse events [AEs] that would require investigational medicinal product [IMP] discontinuation) who received tolvaptan and completed Phase A were enrolled in Phase B and received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting dose based on their body weight as per following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 24 months. Doses may be titrated down dependent upon participant tolerability. | |
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| Secondary | Phase B: Percent Change From Phase B Baseline in htTKV as Measured by MRI at Month 12 and Month 24 | htTKV is used in participants with autosomal dominant polycystic kidney disease to predict the onset of renal insufficiency. | 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. | Posted | | Mean | Standard Deviation | percent change | | Phase B Baseline, Months 12 and 24 | | | | ID | Title | Description |
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| OG000 | Phase B: Prior Tolvaptan | Qualified participants (defined as those who were willing to continue in the trial and who did not have any adverse events [AEs] that would require investigational medicinal product [IMP] discontinuation) who received tolvaptan and completed Phase A were enrolled in Phase B and received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting dose based on their body weight as per following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 24 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase B: Prior Placebo |
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| Secondary | Phase A: 24-hour Urine Volume | Urine volume refers to the quantity of urine produced per unit of time. | 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. | Posted | | Mean | Standard Deviation | milliliter per 24 hours post dose | | 24 hours post dose after Month 1 on study medication in Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A: 24-hour Fluid Intake | Daily fluid intake (total water) is defined as the amount of water consumed from foods, plain drinking water, and other beverages. | 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. | Posted | | Mean | Standard Deviation | milliliter | | 24 hours post dose after Month 1 on study medication in Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A: 24-hour Fluid Balance | 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. | 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. | Posted | | Mean | Standard Deviation | milliliter | | 24 hours post dose after Month 1 on study medication in Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A: 24-hour Sodium Clearance | Data was categorized and reported based on the total daily dose for the given time point of 0-24 hours. | 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. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | | Mean | Standard Deviation | milliliter per minute (mL/min) | | 24 hours post dose after Month 1 on study medication in Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A: 24-hour Creatinine Clearance | Creatinine is produced from the metabolism of protein, when muscles burn energy. Most creatinine is filtered out of the blood by the kidneys and excreted in urine. The creatinine clearance value is determined by measuring the concentration of endogenous creatinine (that which is produced by the body) in both plasma and urine. Data was categorized and reported based on the total daily dose for the given time point of 0-24 hours. | 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. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | | Mean | Standard Deviation | mL/min | | 24 hours post dose after Month 1 on study medication in Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 |
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| Secondary | Phase A: 24-hour Free Water Clearance | Data was categorized and reported based on the total daily dose for the given time point of 0-24 hours. | 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. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | | Mean | Standard Deviation | mL/min | | 24 hours post dose after Month 1 on study medication in Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A: Percentage of Each Tanner Stage by Gender and Age Compared to Normative Populations | Tanner stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Only those categories with at least one participant with event are reported. | The Phase A Safety Set included all participants who were randomized and received at least 1 dose of IMP in Phase A. | Posted | | Number | | percentage of participants | | At Baseline, Months 6 and 12 of Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase B: Percentage of Each Tanner Stage by Gender and Age Compared to Normative Populations | Tanner stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Only those categories with at least one participant with event are reported. | The Phase B Safety Set included all participants who were enrolled in Phase B and received at least 1 dose of IMP in Phase B. | Posted | | Number | | percentage of participants | | At Baseline, Months 6, 12, 18, and 24 of Phase B | | | | ID | Title | Description |
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| OG000 | Phase B: Prior Tolvaptan | Qualified participants (defined as those who were willing to continue in the trial and who did not have any adverse events [AEs] that would require investigational medicinal product [IMP] discontinuation) who received tolvaptan and completed Phase A were enrolled in Phase B and received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting dose based on their body weight as per following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 24 months. Doses may be titrated down dependent upon participant tolerability. | |
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| Secondary | Phase A: Change From Baseline in Growth Percentile by Gender and Age | The growth percentile was based on the assessment of height and weight. | The Phase A Safety Set included all participants who were randomized and received at least 1 dose of IMP in Phase A. Number analyzed is the number of participants with data available for analysis at the given time point. | Posted | | Mean | Standard Deviation | growth percentile | | At Baseline, Months 6 and 12 of Phase A | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase B: Change From Baseline in Growth Percentile by Gender and Age | The growth percentile was based on the assessment of height and weight. | The Phase B Safety Set included all participants who were enrolled in Phase B and received at least 1 dose of IMP in Phase B. Number analyzed is the number of participants with data available for analysis at the given time point. | Posted | | Mean | Standard Deviation | growth percentile | | At Baseline, Months 6, 12, 18, and 24 of Phase B | | | | ID | Title | Description |
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| OG000 | Phase B: Prior Tolvaptan | Qualified participants (defined as those who were willing to continue in the trial and who did not have any adverse events [AEs] that would require investigational medicinal product [IMP] discontinuation) who received tolvaptan and completed Phase A were enrolled in Phase B and received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting dose based on their body weight as per following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 24 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase B: Prior Placebo | Qualified participants (defined as those who were willing to continue in the trial and who did not have any AEs that would require IMP discontinuation) who received matching-placebo and completed Phase A, were enrolled in Phase B and received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), based on their current body weight as per following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 24 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A: Change From Baseline in Creatinine Value | Phase A Baseline is the last pre-dose evaluation. The Last Visit is the last available post-baseline evaluation including early term. As prespecified in the protocol, data for safety is reported by the treatment group (Phase A: Tolvaptan and Phase A: Placebo) | The Phase A Safety Set included all participants who were randomized and received at least 1 dose of IMP in Phase A. Number analyzed is the number of participants with data available for analysis at the given time point. | Posted | | Mean | Standard Deviation | milligrams per deciliter (mg/dL) | | Baseline, Week 1, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, Follow Up Day 7, End of Treatment, and Last Visit | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. | | OG001 | Phase A: Placebo | Participants received matching-placebo tablets, orally as a split-dose (with the first dose taken upon awakening and second dose taken approximately 8 hours later), and starting dose based on their weight as per the following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase B: Change From Baseline in Creatinine Value | Phase B Baseline is the last evaluation prior to the first dose in Phase B. The Last Visit is the last available post-baseline evaluation including early term. As prespecified in the protocol, data for safety is reported by the treatment group (Phase B: Prior Tolvaptan and Phase B: Prior Placebo). | The Phase B Safety Set included all participants who were enrolled in Phase B and received at least 1 dose of IMP in Phase B. Number analyzed is the number of participants with data available for analysis at the given time point. | Posted | | Mean | Standard Deviation | mg/dL | | Baseline, Week 1, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Follow Up Day 7, End of Treatment, and Last Visit | | | | ID | Title | Description |
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| OG000 | Phase B: Prior Tolvaptan | Qualified participants (defined as those who were willing to continue in the trial and who did not have any adverse events [AEs] that would require investigational medicinal product [IMP] discontinuation) who received tolvaptan and completed Phase A were enrolled in Phase B and received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting dose based on their body weight as per following specifications: ≥20 to <45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 24 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A and B: Percentage of Participants With Potentially Clinically Significant Abnormalities in Vital Signs | Vital sign measurements included measurements of respiratory rate, blood pressure, body temperature and pulse. Any value outside the normal range was flagged for the attention of the investigator who assessed whether or not a flagged value is of clinical significance. Only those categories with at least one participant with event are reported. As prespecified in the protocol, data for safety is reported by the treatment group (Phase A: Tolvaptan, Phase A: Placebo, Phase B: Prior Tolvaptan and Phase B: Prior Placebo). | The Phase A Safety Set included all participants who were randomized and received at least 1 dose of IMP in Phase A. The Phase B Safety Set included all participants who were enrolled in Phase B and received at least 1 dose of IMP in Phase B. Number analyzed is the number of participants with at least one post-baseline numeric result for the given vital sign parameter. | Posted | | Number | | percentage of participants | | From first dose of study drug up to 14 days post last dose (up to approximately 37 months) | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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| Secondary | Phase A and B: Percentage of Participants With Potentially Clinically Significant Abnormalities in Laboratory Test Results Including Liver Function Tests (LFTs) | Laboratory parameters=haematology,chemistry,urinalysis,& LFTs.Criteria for laboratory abnormalities along with their test result grade=Increased creatinine level: Baseline(BSL):Grade 0,>BSL-1.5xBSL:1,>1.5-3xBSL:2,>3-6xBSL:3,>6xBSL:4.Decreased glucose level: <30:-4,30-<40:-3, 40-<55:-2, 55-<65:-1,>=65:0; Increased:<=115:0,>115-160:1,>160-250:2,>250-500:3,>500:4.Decreased potassium level: <2.5:-4,2.5-<3:-3,3-<lower limit of normal(LLN):-1,LLN:0; Increased:upper limit of normal(ULN):0,>ULN-5.5:1,>5.5-6:2,>6-7:3,>7:4.Decreased sodium level: <120:-4,120-124:-3,125-129:-2,130-135:-1,>=136:0; Increased:<=145:0,146-150:1,151-155:2,156-160:3,>160:4. Increased triglyceride level:ULN:0,>ULN-2.5xULN:1,>2.5-5xULN:2,>5-6xULN:3,>6xULN:4. Decreased Neutrophils:<0.5:-4,0.5-<1:-3,1-<1.5:-2,1.5-<LLN:-1,LLN:0. Potentially clinically significant increase or decrease was defined as Baseline grade 0,1,-1 and post-baseline grade >1, <-1 or Baseline grade >1,<-1 and post-baseline grade >or\ | The Phase A Safety Set included all participants who were randomized and received at least 1 dose of IMP in Phase A. The Phase B Safety Set included all participants who were enrolled in Phase B and received at least 1 dose of IMP in Phase B. Number analyzed is the number of participants with at least one post-baseline numeric result for the given laboratory parameter. Only those categories with at least one participant with event are reported. | Posted | | Number | | percentage of participants | | From first dose of study drug up to 14 days post last dose (up to approximately 37 months) | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan |
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| Secondary | Phase A and B: Percentage of Participants With Aquaretic Adverse Events (AEs) | An AE was defined as any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Aquaretic AEs included Medical Dictionary for Regulatory Activities [MedDRA] preferred terms of thirst, polyuria (production of large volumes of dilute urine), nocturia (need to wake up to urinate at night), pollakiuria (abnormally frequent urination), and polydipsia (excessive thirst). As prespecified in the protocol, data for safety is reported by the treatment group (Phase A: Tolvaptan, Phase A: Placebo, Phase B: Prior Tolvaptan and Phase B: Prior Placebo). | The Phase A Safety Set included all participants who were randomized and received at least 1 dose of IMP in Phase A. The Phase B Safety Set included all participants who were enrolled in Phase B and received at least 1 dose of IMP in Phase B. | Posted | | Number | | percentage of participants | | From first dose of study drug up to 14 days post last dose (up to approximately 37 months) | | | | ID | Title | Description |
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| OG000 | Phase A: Tolvaptan | Participants received tolvaptan tablets, orally as a split dose (with the first dose taken upon awakening and the second dose taken approximately 8 hours later), and starting doses based on their weight as per the following specifications: ≥20 to 45 kg: 15/7.5 mg; ≥45 to ≤75 kg: 30/15 mg; >75 kg: 45/15 mg, for 1 week. The starting dose was up-titrated (≥20 to <45 kg: 30/15 mg; ≥45 to ≤75 kg: 45/15 mg; >75 kg: 60/30 mg) after 1 week based upon tolerability and thereafter participants continued the same dose for 12 months. Doses may be titrated down dependent upon participant tolerability. |
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