Phase 2 N=46 Randomized Treatment

Tolvaptan Open-label Pilot Efficacy, Tolerability, and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Polycystic Kidney, Autosomal Dominant

Bottom Line

View on ClinicalTrials.gov: NCT00413777 ↗

Enrolled (actual)

Serious AEs

23.9%

Results posted

Jul 2017

Primary outcome: Primary: Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion. — 3; 8; 6; 10 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Tolvaptan (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.
Primary completion: Jun 2010

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety Assessments Based on Vital Signs, Electrocardiogram (ECG's), Clinical Laboratory Tests, Physical Examinations Are Reported as Adverse Events (AEs) Upon Study Physician Discretion.	3; 8; 6; 10; 3; 1	—
SECONDARY Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose.	-274.10; -228.00; -288.42; -263.78; -227.29; -178.43	—
SECONDARY Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Second Daily Dose.	-263.95; -298.71; -321.78; -294.13; -288.20; -234.65	—
SECONDARY Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Bedtime.	-275.00; -291.09; -327.41; -301.83; -283.21; -245.83	—
SECONDARY Percent Change From Baseline in Renal Volume.	-1.0; -1.3; 0.3; 2.4; 4.6; 1.0	—
SECONDARY Change From Pre-dose Baseline in Renal Function Estimated by Glomerular Filtration Rate (GFR).	-0.09; -0.05; -0.03; -0.01; -0.01; -0.03	—
SECONDARY Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to First Daily Dose- Extension.	-328.06; -176.88; -270.94; -223.50; -234.56; -155.65	—
SECONDARY Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Second Daily Dose- Extension.	-281.63; -254.82; -305.25; -261.61; -288.20; -190.69	—
SECONDARY Mean Change From Baseline in Trough Urine Osmolality at Steady State Prior to Bedtime- Extension.	-322.27; -258.88; -309.73; -264.50; -283.21; -206.00	—
SECONDARY Percent Change From Baseline in Renal Volume-Extension.	-1.3; -1.4; -0.7; 1.9; 3.2; 0.2	—
SECONDARY Change From Pre-dose Baseline in Renal Function Estimated by GFR- Extension.	-0.09; -0.04; -0.03; -0.00; -0.01; -0.01	—
SECONDARY Mean Change From Baseline in Systolic Blood Pressure (sBP) for Hypertension Assessment.	-3.2; -3.0; -3.6; -5.0; -4.6; -3.5	—
SECONDARY Mean Change From Baseline in Diastolic Blood Pressure (dBP) for Hypertension Assessment.	-4.0; -0.2; -3.6; -2.5; -4.8; -2.8	—
SECONDARY Mean Change From Baseline in Mean Arterial Pressure (MAP) for Hypertension Assessment.	-3.7; -1.0; -3.6; -3.3; -4.7; -3.0	—
SECONDARY Mean Change From Baseline in sBP for Hypertension Assessment- Extension.	-1.6; -2.7; -3.4; -5.6; -4.1; -4.3	—
SECONDARY Mean Change From Baseline in dBP for Hypertension Assessment- Extension.	-5.5; 0.3; -4.5; -2.4; -5.3; -2.9	—
SECONDARY Mean Change From Baseline in MAP for Hypertension Assessment- Extension.	-4.1; -0.6; -4.2; -3.4; -4.8; -3.4	—
SECONDARY Mean Change From Baseline in Patient-assessed Renal Pain Scale.	0.6; 0.0; 0.4; -0.1; 1.2; -0.1	—
SECONDARY Mean Change From Baseline in Patient-assessed Renal Pain Scale- Extension.	0.9; 0.3; 0.8; -0.2; 1.2; -0.3	—
SECONDARY Mean Change From Baseline in Abdominal Girth Measurement.	0.6; -2.3; 1.4; 0.5; 1.3; -2.8	—
SECONDARY Mean Change From Baseline in Abdominal Girth Measurement- Extension.	-0.8; -2.8; 0.3; 0.3; 1.1; -3.5	—

Summary

This study's purpose is to evaluate the long-term safety of open-label tolvaptan regimens to determine the maximally-tolerated dose and acquire pilot efficacy data in patients with autosomal dominant polycystic kidney disease (ADPKD).

Eligibility Criteria

Inclusion Criteria

Prior participation in designated tolvaptan ADPKD studies (156-04-248, 156-04-249).
Able to give Informed Consent.

Exclusion Criteria

Women who are breast feeding and females of childbearing potential who are not using acceptable contraceptive methods.
In the opinion of the study investigator or sponsor may present a safety risk.
Patients who are unlikely to adequately comply with study procedures.
Patients who at Day 1 have an estimated glomerular filtration rate (GFR) below 30 mL/min or who anticipate renal-replacement therapy within one year of study entry.
Patients having contraindications to magnetic resonance imaging (MRI) or gadolinium contrast will be eligible but will not be able to participate in MRI.
Patients taking a diuretic within 1 week of enrollment or likely to need diuretic therapy prior to Month 2.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00413777). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.