Clinical Trial: Tolvaptan Open-Label Pilot Efficacy, Tolerability and Safety Study in ADPKD

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase 2, Multi-center, Open-label Study to Determine Long-term Safety, Tolerability and Efficacy of Split-dose Oral Regimens of Tolvaptan Tablets in a Range of 30 to 120 mg/d in Patients With

Brief 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 ADPKD.

Detailed Summary:

Autosomal Dominant Polycystic Kidney Disease is a genetic disease classified by the formation of fluid-filled cysts in the kidneys. The accumulation of these cysts causes the kidneys to enlarge several times the normal size and leads to the eventual loss of renal function and ultimately results in renal failure in end-stage patients. This is a disease with life-threatening implications to those who have it and their family members who may also be affected. Aside from early antihypertensive control and dietary protein restriction, which are presumed to offer a modest degree of protection, most surviving patients require renal replacement therapy (dialysis and transplant) and suffer from high morbidity and mortality.

A rationale for use of tolvaptan in these genetic disorders has been proven, in principle, through use of a variety of animal models. In these models, tolvaptan is effective in halting or reversing the progression of this renal disease.

The current study is being undertaken in order to evaluate whether tolvaptan, an oral AVP inhibitor, will maintain an adequate safety profile and show a potential clinical benefit by reducing total renal volume in the hopes of making an impact upon disease progression.


Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

Current Primary Outcome: Long-term Safety [ Time Frame: 4 Years ]

Adverse events by assigned intervention


Original Primary Outcome:

  • The evaluation of long-term safety of tolvaptan in patients with ADPKD with various dosing regimens:
  • Safety:Adverse events,vital signs,clinical laboratory tests,12- lead electrocardiograms.


Current Secondary Outcome:

  • Trough Urine Osmolality at steady state [ Time Frame: 36 Months ]
    Change from baseline at each study visit.
  • Change in Total Kidney Volume (TKV) [ Time Frame: 36 Months ]
  • Renal function by estimated GFR [ Time Frame: 36 Months ]
  • Trough Urine Osmolality at Steady State [ Time Frame: Extension Day 1, Extension Year 1 ]
  • Change in Total Kidney Volume [ Time Frame: Extension Day 1, Extension Year 1 ]
  • Renal function by estimated GFR [ Time Frame: Extension Day 1, Extension Year 1 ]


Original Secondary Outcome: Change from baseline for:1. Trough Urine Osmolality at steady state. 2. Renal function by estimated GFR 3. Combined volume of the kidneys. 4. Hypertension Assessment. 5. Renal Pain Assessment. 6. Abdominal Girth Assessment 7.PKD Outcomes Survey.

Information By: Otsuka Pharmaceutical Development & Commercialization, Inc.

Dates:
Date Received: December 18, 2006
Date Started: December 2005
Date Completion:
Last Updated: May 30, 2012
Last Verified: May 2012