Not provided
Not provided
Not provided
Not provided
Not provided
Issues with recruitment and enrollment made the trial impossible or highly impracticable. Termination of this trial was not due to safety reasons.
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Syneos Health | OTHER |
Not provided
Not provided
Not provided
Not provided
The objective of this trial was to provide 6 months of safety follow-up for children and adolescents with dilutional (euvolemic or hypervolemic) hyponatremia who had previously participated in a tolvaptan hyponatremia trial and to assess the efficacy of tolvaptan in increasing serum sodium for those participants who received optional continuing tolvaptan treatment of variable duration (up to 6 months).
Core Safety Follow-up Component
• For all participants: To evaluate the post-treatment safety follow-up of children and adolescent participants with dilutional (euvolemic or hypervolemic) hyponatremia who have previously participated in a tolvaptan hyponatremia trial.
Optional Tolvaptan Treatment Component
• For participants who receive optional tolvaptan treatment: To demonstrate that tolvaptan safely and effectively achieves and maintains increased serum sodium concentrations in children and adolescent participants with dilutional (euvolemic or hypervolemic) hyponatremia when used for both multiple short-term treatments, and/or longer chronic treatments.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Tolvaptan | Experimental | Participants enrolled in this trial were eligible to receive open-label tolvaptan if they had a clinical need as determined by the investigator and met the eligibility criteria for optional tolvaptan treatment. Daily dose levels would have included 3.75 milligrams (mg), 7.5 mg, 15 mg, 30 mg, and 60 mg. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tolvaptan | Drug | Tolvaptan 3.75-, 7.5-, 15-, and 30-mg spray-dried tablets. Dosage: Depending on age and weight, Tablet (3.75 mg - 60 mg daily). Frequency: Once daily. Duration: Participants may be eligible to receive short-term or long-term optional tolvaptan treatment at any time during their participation in the trial, with one or more treatment cycles over a 6-month period. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline At Month 6 In Serum Sodium While Tolvaptan Was Being Administered | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | Baseline, Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Of Participants Who Required Rescue Therapy While On Tolvaptan Treatment | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | Month 6 |
| Percentage Of Participants Who Had Recurrence Of Hyponatremia While On Tolvaptan |
Not provided
Core Safety Follow-up Component
Inclusion Criteria: Participation in a prior pediatric trial with tolvaptan for euvolemic or hypervolemic hyponatremia.
Exclusion Criteria: None
Optional Tolvaptan Treatment Component (per treatment cycle)
Eligibility Criteria:
Ineligibility Criteria:
Had evidence of hypovolemia or intravascular volume depletion (for example, hypotension, clinical evidence of volume depletion, response to saline challenge); if the participant had systolic blood pressure or heart rate outside of the normal range for that age, then volume status was to be specifically clinically assessed to rule out volume depletion.
Had serum sodium < 120 mEq/L (mmol/L), with or without associated neurologic impairment (that is, symptoms such as apathy, confusion, or seizures).
Use of potent CYP3A4 inhibitors in participants ≤ 50 kg or moderate CYP3A4 inhibitors in participants < 20 kg.
Lacked free access to water (inability to respond to thirst) or without ICU-level fluid monitoring and management.
Had a history or current diagnosis of nephrotic syndrome.
Had transient hyponatremia likely to resolve (for example, head trauma or post-operative state).
Had hyperkalemia defined as serum potassium above the upper limit of normal (ULN) for the appropriate pediatric age range.
Had estimated glomerular filtration rate (eGFR) < 30 milliliters (mL)/minute (min)/1.73 meters squared (m^2) calculated by the following equation: eGFR (mL/min/1.73 m^2) = 0.413 x height (centimeter [cm])/serum creatinine (mg/deciliter [dL]).
Had acute kidney injury defined as: Increase in serum creatinine by ≥ 0.3 mg/dL (≥ 26.5 micromoles [μmol]/L) within 48 hours; or increase in serum creatinine to ≥ 1.5 times baseline, which was known or presumed to have occurred within the prior 7 days; or urine volume < 0.5 mL/kg/hour for 6 hours.
Had severe or acute neurological symptoms requiring other intervention (for example, hyperemesis, obtundation, seizures).
Had had treatment for hyponatremia with:
Had anuria or urinary outflow obstruction, unless the participant was, or could be, catheterized during the trial.
Had a history of drug or medication abuse within 3 months prior to the pretreatment visit or current alcohol abuse.
Had a history of hypersensitivity and/or idiosyncratic reaction to benzazepine or benzazepine derivatives (such as benazepril).
Had psychogenic polydipsia (participants with other psychiatric illness may be included per medical monitor approval).
Had uncontrolled diabetes mellitus, defined as fasting glucose > 300 mg/dL (16.7 mEq/L [mmol/L]).
Had screening liver function values > 3 x ULN.
Had cirrhosis and met any of the following conditions: a major gastrointestinal bleed within the past 6 months, evidence of active bleeding (for example, epistaxis, petechiae/purpura, hematuria, or hematochezia), platelet count < 50,000/μL, or use of concomitant medications known to increase bleeding risk.
Had hyponatremia due to the result of any medication that can safely be withdrawn (for example, thiazide diuretics).
Had hyponatremia (for example, hyponatremia in the setting of adrenal insufficiency, untreated hypothyroidism, or hypotonic fluid administration) that is most appropriately corrected by alternative therapies.
Was currently pregnant or breastfeeding.
Had any medical condition that, in the opinion of the investigator, could interfere with evaluation of the trial objectives or safety of the participants.
Was deemed unsuitable for trial participation in the opinion of the investigator.
Participation in another investigational drug trial within the past 30 days, without prior approval from the sponsor medical monitor.
Participants < 4 years of age (or per local Health Authority age restrictions), weight < 10 kg, or who were unable to swallow tablets.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Aurora | Colorado | 80045 | United States | |||
Small studies with less than 25 participants are excluded from data sharing.
Not provided
Not provided
Not provided
Not provided
There was no screening phase. Approximately 100 male or female participants were planned to be enrolled in this trial. A total of 3 participants were enrolled in this trial, but no participant received optional investigational medicinal product during this trial.
Eligible participants included those enrolled in a previous tolvaptan pediatric trial for hyponatremia (NCT02012959); who provided written informed consent at baseline and were able to understand that he/she could withdraw at any time; with the ability to comply with all requirements of the trial; ready to be followed up for 6 months.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Tolvaptan | All participants enrolled first entered a 6-month follow-up trial that evaluated post-treatment safety after participation in a tolvaptan hyponatremia trial. Participants were then eligible to receive open-label tolvaptan if they had a clinical need as determined by the investigator and met the eligibility criteria for optional tolvaptan treatment during the 6-month follow-up period. In this trial, no participants qualified for treatment during the 6-month follow-up period. Daily dose levels would have included 3.75 milligrams (mg), 7.5 mg, 15 mg, 30 mg, and 60 mg. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Core Safety Follow-up Component |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 9, 2015 | Feb 21, 2020 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
|
No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. |
| Month 6 |
| Percentage Of Participants Requiring Continuation Of Tolvaptan Following 30 Days Of Treatment | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | Month 6 |
| Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Total Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in the GCS total score is presented. | Baseline, Month 6 |
| Change From Baseline In PedsQL GCS Physical Health Summary Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in the physical health dimension is presented. | Baseline, Month 6 |
| Change From Baseline In PedsQL GCS Psychosocial Health Summary Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in summed emotional, social, and school dimensions is presented. | Baseline, Month 6 |
| Change From Baseline In PedsQL Multidimensional Fatigue Scale (MFS) Total Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in summed emotional, social, and school dimensions is presented. | Baseline, Month 6 |
| Percentage Of Participants With Overly Rapid Correction In Serum Sodium 24 Hours After The First Dose At Introduction Or Reintroduction Of Tolvaptan | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | Month 6 |
| Plasma Concentrations Of Tolvaptan And Metabolites In Participants Who Had Continued Tolvaptan Therapy For Eight Consecutive Weeks | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | 8 Weeks |
| Participants With A Tanner Staging Score Of 1 At Month 6 | Tanner Staging assessment consists of 2 domains (pubic hair and breast development) for girls and 3 domains (pubic hair, penis development, and testes development) for boys. Staging was based on a single score summarizing the domains (not individual domain scores). Stages range from 1-5, with 1 indicating preadolescent and 5 adult. Participants with a Tanner staging score of 1 (preadolescent) at Month 6 are reported. | Month 6 |
| Change From Baseline In Growth Percentiles For Body Height And Weight At Month 6 | Changes from baseline in growth percentiles for body height and weight were calculated and are reported. | Baseline, Month 6 |
| Change From Baseline In Alanine Aminotransferase (ALT) And Aspartate Aminotransferase (AST) For Participants On Tolvaptan At Month 2 | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. Data reported only include assessments made for ALT and AST during the Core Safety Follow-up Component of the trial. Results are reported in units/liter (U/L). | Baseline, Month 2 |
| Change From Baseline In Bilirubin For Participants On Tolvaptan At Month 2 | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. Data reported only include assessments made for bilirubin during the Core Safety Follow-up Component of the trial. Results are reported in micromoles (umol)/L. | Baseline, Month 2 |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Atlanta | Georgia | 30322 | United States |
| Grand Rapids | Michigan | 49503 | United States |
| Kansas City | Missouri | 64108 | United States |
| New York | New York | 10032 | United States |
| Stony Brook | New York | 11794-8111 | United States |
| The Bronx | New York | 10467 | United States |
| Cleveland | Ohio | 44106 | United States |
| Philadelphia | Pennsylvania | 19104 | United States |
| Seattle | Washington | 98105 | United States |
| Ghent | 9000 | Belgium |
| Prague | 5 150 06 | Czechia |
| Erlangen | Nurnberg | Germany |
| Leipzig | Saxony | 04289 | Germany |
| Genova | 16147 | Italy |
| Naples | 80131 | Italy |
| Palermo | 90134 | Italy |
| Rome | 00165 | Italy |
| Bucharest | 022238 | Romania |
| Sibiu | 550166 | Romania |
| Madrid | 28007 | Spain |
| London | WC1N 3JH | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
| Optional Tolvaptan Treatment Component |
|
All enrolled participants: No enrolled participant received any investigational medicinal product during the study. Due to the low number of participants enrolled, 0 participants are reported due to the risk of reidentification.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Tolvaptan | All participants enrolled first entered a 6-month follow-up trial that evaluated post-treatment safety after participation in a tolvaptan hyponatremia trial. Participants were then eligible to receive open-label tolvaptan if they had a clinical need as determined by the investigator and met the eligibility criteria for optional tolvaptan treatment during the 6-month follow-up period. In this trial, no participants qualified for treatment during the 6-month follow-up period. Daily dose levels would have included 3.75 mg, 7.5 mg, 15 mg, 30 mg, and 60 mg. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Due to the low number of participants enrolled, 0 participants are reported due to the risk of re-identification. | years | |||||||||||||||||||||||||||||
| Sex: Female, Male | Due to the low number of participants enrolled, 0 participants are reported due to the risk of re-identification. | participants |
| ||||||||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Due to the low number of participants enrolled, 0 participants are reported due to the risk of re-identification. | Participants |
| ||||||||||||||||||||||||||||
| Race (NIH/OMB) | Due to the low number of participants enrolled, 0 participants are reported due to the risk of re-identification. | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline At Month 6 In Serum Sodium While Tolvaptan Was Being Administered | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. | Posted | Baseline, Month 6 |
|
| |||||||||||||||||||
| Secondary | Percentage Of Participants Who Required Rescue Therapy While On Tolvaptan Treatment | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. | Posted | Month 6 |
|
| |||||||||||||||||||
| Secondary | Percentage Of Participants Who Had Recurrence Of Hyponatremia While On Tolvaptan | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. | Posted | Month 6 |
|
| |||||||||||||||||||
| Secondary | Percentage Of Participants Requiring Continuation Of Tolvaptan Following 30 Days Of Treatment | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. | Posted | Month 6 |
|
| |||||||||||||||||||
| Secondary | Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Total Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in the GCS total score is presented. | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. | Posted | Mean | Full Range | units on a scale | Baseline, Month 6 |
| |||||||||||||||||
| Secondary | Change From Baseline In PedsQL GCS Physical Health Summary Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in the physical health dimension is presented. | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. | Posted | Mean | Full Range | units on a scale | Baseline, Month 6 |
| |||||||||||||||||
| Secondary | Change From Baseline In PedsQL GCS Psychosocial Health Summary Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in summed emotional, social, and school dimensions is presented. | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. | Posted | Mean | Full Range | units on a scale | Baseline, Month 6 |
| |||||||||||||||||
| Secondary | Change From Baseline In PedsQL Multidimensional Fatigue Scale (MFS) Total Score At Month 6 | The PedsQL GCS was used for quality of life assessment. It is appropriate for at least 2 years of age, however availability may be limited for certain ages and languages. It encompasses 4 dimensions of functioning (physical, emotional, social, school). The age groups covered are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Adolescent (13-18 years). Depending on the participant's age, the questionnaire may be completed by either the participant or the parent/caregiver, as appropriate. For the Toddler group, the PedsQL GCS consists of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consists of 23 items, with a 3-point Likert scale (0, 2, 4) for the Young Child, and a 5-point Likert scale for the Child and Adolescent groups. Scores are transformed on a scale from 0 to 100 and averaged. Higher scores indicate improved quality of life. The change from baseline in summed emotional, social, and school dimensions is presented. | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. | Posted | Mean | Full Range | units on a scale | Baseline, Month 6 |
| |||||||||||||||||
| Secondary | Percentage Of Participants With Overly Rapid Correction In Serum Sodium 24 Hours After The First Dose At Introduction Or Reintroduction Of Tolvaptan | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. | Posted | Month 6 |
|
| |||||||||||||||||||
| Secondary | Plasma Concentrations Of Tolvaptan And Metabolites In Participants Who Had Continued Tolvaptan Therapy For Eight Consecutive Weeks | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected for this outcome measure. | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, no pharmacokinetic analyses were performed. | Posted | 8 Weeks |
|
| |||||||||||||||||||
| Secondary | Participants With A Tanner Staging Score Of 1 At Month 6 | Tanner Staging assessment consists of 2 domains (pubic hair and breast development) for girls and 3 domains (pubic hair, penis development, and testes development) for boys. Staging was based on a single score summarizing the domains (not individual domain scores). Stages range from 1-5, with 1 indicating preadolescent and 5 adult. Participants with a Tanner staging score of 1 (preadolescent) at Month 6 are reported. | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. | Posted | Count of Participants | Participants | Month 6 |
|
| |||||||||||||||||
| Secondary | Change From Baseline In Growth Percentiles For Body Height And Weight At Month 6 | Changes from baseline in growth percentiles for body height and weight were calculated and are reported. | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. | Posted | Mean | Standard Deviation | percentile | Baseline, Month 6 |
|
| ||||||||||||||||
| Secondary | Change From Baseline In Alanine Aminotransferase (ALT) And Aspartate Aminotransferase (AST) For Participants On Tolvaptan At Month 2 | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. Data reported only include assessments made for ALT and AST during the Core Safety Follow-up Component of the trial. Results are reported in units/liter (U/L). | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. | Posted | Mean | Standard Deviation | U/L | Baseline, Month 2 |
|
| ||||||||||||||||
| Secondary | Change From Baseline In Bilirubin For Participants On Tolvaptan At Month 2 | No enrolled participant received any investigational medicinal product during the study. Due to early study termination, efficacy data were not collected. Data reported only include assessments made for bilirubin during the Core Safety Follow-up Component of the trial. Results are reported in micromoles (umol)/L. | All enrolled participants with analyzable data at specified timepoint. No enrolled participant received any investigational medicinal product during the study. | Posted | Mean | Standard Deviation | umol/L | Baseline, Month 2 |
|
|
Baseline to Month 6
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Tolvaptan | All participants enrolled first entered a 6-month follow-up trial that evaluated post-treatment safety after participation in a tolvaptan hyponatremia trial. Participants were then eligible to receive open-label tolvaptan if they had a clinical need as determined by the investigator and met the eligibility criteria for optional tolvaptan treatment during the 6-month follow-up period. In this trial, no participants qualified for treatment during the 6-month follow-up period. Daily dose levels would have included 3.75 milligrams (mg), 7.5 mg, 15 mg, 30 mg, and 60 mg. | 0 | 3 | 1 | 3 | 2 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDra 19.0 | Systematic Assessment |
| |
| Enterovirus Infection | Infections and infestations | MedDra 19.0 | Systematic Assessment |
| |
| Rhinovirus Infection | Infections and infestations | MedDra 19.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDra 19.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDra 19.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDra 19.0 | Systematic Assessment |
| |
| Enterovirus Infection | Infections and infestations | MedDra 19.0 | Systematic Assessment |
| |
| Rhinovirus Infection | Infections and infestations | MedDra 19.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDra 19.0 | Systematic Assessment |
| |
| Viral Upper Respiratory Tract Infection | Infections and infestations | MedDra 19.0 | Systematic Assessment |
|
This trial was prematurely terminated due to challenges in enrollment. The trial was not terminated due to safety reasons. Due to early termination of the study, no efficacy data were collected.
Sponsor reserves the right to review results publications prior to public release and can delay such publications for a period greater than 60 days but no more than 120 days from the date that the publication is submitted to the Sponsor for review. Sponsor can require changes to the publication to protect Sponsor's intellectual property rights and/or confidential information and reserves the right to limit publication timing and scope of data published based on the number of study locations.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Global Clinical Development | Otsuka Pharmaceutical Development & Commercialization, Inc. | 1-609-524-6788 | clinicaltransparency@otsuka-us.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 19, 2017 | Feb 21, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D007010 | Hyponatremia |
| D008659 | Metabolic Diseases |
| D014883 | Water-Electrolyte Imbalance |
| ID | Term |
|---|---|
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000077602 | Tolvaptan |
| ID | Term |
|---|---|
| D001552 | Benzazepines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
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