Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00428948
• Date of registration: 26/01/2007
• Prospective Registration: No
• Primary sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.
• Public title: Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD) TEMPO3:4
• Scientific title: A Phase 3, Multi-center, Double-blind, Placebo-controlled, Parallel-arm Trial to Determine Long-term Safety and Efficacy of Oral Tolvaptan Tablets Regimens in Adult Subjects With Autosomal Dominant Polycystic Kidney Disease
• Date of first enrolment: January 2007
• Target sample size: 1445
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00428948
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Poland, Romania, Russian Federation, United Kingdom, United States

Contacts

• Name: Vicente Torres, MD, PhD
• Affiliation: Mayo Medical Center

• Name: Osamu Sato
• Affiliation: Otsuka Pharmaceutical Corporation, Ltd. Japan

• Name: Frank Czerwiec, MD, PhD
• Affiliation: Otsuka Pharmaceutical Development and Commercialization, Inc.

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 50 Years
Gender: All

Health Condition(s) or Problem(s) studied

Polycystic Kidney Disease, Autosomal Dominant

Intervention(s)

Drug: Placebo

Drug: Tolvaptan

Primary Outcome(s)

Percentage Change Per Year in Total Kidney Volume From Baseline to Month 36 [Time Frame: Baseline to Month 36]

Secondary Outcome(s)

Change in Mean Arterial Blood Pressure Per Year in Non-hypertensive Participants From Baseline to Month 36 [Time Frame: Baseline to Month 36]

Percentage of Participants With a Clinically Sustained Decrease of Blood Pressure Leading to a Sustained Reduction in Antihypertensive Therapy From Baseline to Month 36 [Time Frame: Baseline to Month 36]

Area Under the Concentration-time Curve of Change in Renal Pain From Baseline to Month 36 [Time Frame: At screening, Baseline, Day 1, every 4 months up to month 36/early tremination (ET), follow-up visit 1 and 2]

Change in Renal Function Per Year From Week 3 to Month 36 [Time Frame: Week 3 to Month 36]

Number of ADPKD Clinical Progression Events Per 100 Follow-up Years From Baseline to Month 36 [Time Frame: Baseline to Month 36]

Number of Hypertensive Events Per 100 Follow-up Years in Non-hypertensive Participants From Baseline to Month 36 [Time Frame: Baseline to Month 36]

Secondary ID(s)

156-04-251

2006-002768-24

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Otsuka Pharmaceutical Co., Ltd.

Results

• Results available: Yes
• Date Posted: 02/07/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00428948