Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics Study of Tolvaptan in Pediatric Congestive Heart Failure (CHF) Patients With Volume Overload

NCT03255226 | Completed Phase 3 Results

• 2 other identifiers: 156-102-00123JapicCTI-173674
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

To determine the efficacy, safety, and dose and regimen of tolvaptan in pediatric CHF patients with volume overload

Conditions

Pediatric Congestive Heart Failure (CHF) Patients With Volume Overload

Outcome Measures

Primary Outcomes (1)

• Percentages of Subjects Whose Was Decreased by 1.7% or More Body Weight From Baseline

  The primary endpoint of this trial was the percentage of subjects whose body weight on the day after the third day of treatment with tolvaptan at the evaluation dose (the third day of administration at the evaluation dose) was decreased by 1.7% or more from the weight measured before breakfast (baseline) on the first day of the treatment period (the initial tolvaptan administration day), under the condition that the mean daily urine volume for the 3 days of treatment with tolvaptan at the evaluation dose was higher than the daily urine volume for the pretreatment observation period. The percentage of subjects as well as the exact 95% confidence interval (CI) based on binomial distribution were calculated.

  Day after Day 3 at evaluation dose

Secondary Outcomes (4)

• Change Rate From Baseline in Daily Urine Volume

  Baseline, Day1, Day2 and Day3 of administration at evaluation dose

• Percent Changes From Baseline in Body Weight (kg)

  Day after Day 3 at evaluation dose

• Improvement Rates of Lower Limb Edema

  Day after Day 3 at evaluation dose

• Improvement Rates of Pulmonary Congestion

  Day after Day 3 at evaluation dose

Study Arms (1)

Tolvaptan

EXPERIMENTAL

Tolvaptan 1% granules or tolvaptan 15 mg tablet with water once daily.

Drug: Tolvaptan

Interventions

Tolvaptan DRUG

Tolvaptan 1% granules or tolvaptan 15 mg tablet with water once daily.

Tolvaptan

Eligibility Criteria

• Age: 6 Months - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patients with volume overload despite having received any of the following diuretic therapies in whom sufficient effects cannot be expected even if the dose of the diuretics is increased or in whom the investigator or subinvestigator judges that increasing the dose of the diuretics is difficult due to concerns regarding electrolyte abnormalities or other side effects
• Furosemide (oral administration) ≥0.5 mg/kg/day. Azosemide 30 mg and torasemide 4 mg will be calculated as equivalent to furosemide 20 mg.
• Hydrochlorothiazide ≥2 mg/kg/day
• Trichlormethiazide ≥0.05 mg/kg/day
• Spironolactone ≥ 1 mg/kg/day
• Patients capable of complaining of thirst. Patients unable to complain of thirst due to their young age can also be enrolled in the trial if strict management of fluid intake and excretion is conducted. However, even if such fluid management is possible, the patients in whom the investigator or subinvestigator judges that tolvaptan cannot be safely administered are to be excluded
• Patients who can be hospitalized from at least 3 days before start of tolvaptan administration until 2 days after the final administration.
• others

You may not qualify if:

• Patients whose volume overload status shows improvement during the screening period or pretreatment observation period
• Patients who are unable to drink fluid (including patients who are unable to sense thirst)
• Patients whose circulatory blood flow is suspected to be decreased
• Patients with an assisted circulation apparatus
• Patients with hypernatremia (serum or blood sodium concentration exceeding 145 mEq/L) others

Sponsors & Collaborators

• Otsuka Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Unknown Facility

Kanto Region, Japan

Location

MeSH Terms

Conditions

Heart Failure

Interventions

Tolvaptan

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Director of Clinical Trials
• Organization: Otsuka Pharmaceutical Co., Ltd.

CL_OPCJ_RDA_Team@otsuka.jp

+81363617366

Study Officials

• Takehisa Matsumaru, Mr

  Otsuka Pharmaceutical Co., Ltd.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 18, 2017
• First Posted: August 21, 2017
• Study Start: March 7, 2018
• Primary Completion: July 11, 2021
• Study Completion: July 15, 2021
• Last Updated: August 23, 2024
• Results First Posted: August 23, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data will be available after marketing approval in global markets, or beginning 1-3 years following article Publication. There is no end date to the availability of the data.
• Access Criteria: Otsuka will share data on an Otsuka-owned remotely accessible data sharing platform with Python and R analytical software. Research requests should be directed to clinicaltransparency@Otsuka-us.com.

Locations

JP (1)