Phase 3
N=1,445
Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Polycystic Kidney Disease, Autosomal Dominant
Bottom Line
View on ClinicalTrials.gov: NCT00428948 ↗Enrolled (actual)
1,445
Serious AEs
18.8%
Results posted
Jul 2017
Primary outcome: Primary: Percentage Change Per Year in Total Kidney Volume From Baseline to Month 36 — 2.777; 5.608 Percentage change per year — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Tolvaptan (Drug); Placebo (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Primary completion
- Jan 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Change Per Year in Total Kidney Volume From Baseline to Month 36 |
2.777; 5.608 | <0.0001 sig |
| SECONDARY Number of ADPKD Clinical Progression Events Per 100 Follow-up Years From Baseline to Month 36 |
43.94; 50.04 | 0.0095 sig |
| SECONDARY Change in Renal Function Per Year From Week 3 to Month 36 |
-2.555; -3.682 | <0.0001 sig |
| SECONDARY Change in Mean Arterial Blood Pressure Per Year in Non-hypertensive Participants From Baseline to Month 36 |
2.561; 2.592 | 0.5520 |
| SECONDARY Area Under the Concentration-time Curve of Change in Renal Pain From Baseline to Month 36 |
-0.00; 0.08 | 0.1604 |
| SECONDARY Number of Hypertensive Events Per 100 Follow-up Years in Non-hypertensive Participants From Baseline to Month 36 |
31.80; 29.60 | 0.9704 |
| SECONDARY Percentage of Participants With a Clinically Sustained Decrease of Blood Pressure Leading to a Sustained Reduction in Antihypertensive Therapy From Baseline to Month 36 |
6.24; 5.62 | 0.7532 |
Summary
This study's purpose is to evaluate the long-term safety and efficacy of tolvaptan versus placebo in patients with ADPKD.
Eligibility Criteria
Inclusion Criteria
- Legal adult age and able to give Informed Consent.
- Adult subjects with a diagnosis of ADPKD. A diagnosis of ADPKD (age 18 or 20-50) required several cysts in each kidney (3 if by sonography, 5 if by CT or MRI) in those with a family history of ADPKD and 10 cysts (by any radiologic method) in each kidney and exclusion of other cystic kidney diseases if there was no family history.
- Willingness to comply with reproductive precautions, if female.
- Estimated creatinine clearance ≥ 60 mL/min. Estimated from serum creatinine during screening using Cockcroft-Gault with correction for gender and race, where possible.
- Rapidly progressive kidney growth (total volume ≥ 750 cc) by magnetic resonance imaging (MRI) at randomization.
Exclusion Criteria
- Prior exposure to tolvaptan or other experimental PKD therapies.
- Currently taking medication for purpose of affecting PKD cysts.
- 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 or confound study objectives.
- Patients who are unlikely to adequately comply with study procedures.
- Patients having contraindications to MRI.
- Patients taking medications or having any illnesses likely to affect ADPKD outcomes.
Data sourced from ClinicalTrials.gov (NCT00428948). 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.