Canadian Medical Assessment of JINARC™ Outcomes Registry (C-MAJOR)

Canadian Observational Cohort Study of the Real-life Assessment of Tolvaptan (JINARC™) in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

This study is part of the Health Canada approval requirement for JINARC™ (tolvaptan) and is an observational, non-interventional study (NIS) describing the impact of tolvaptan on ADPKD-related burden of illness as measured with a set of Patient Reported Outcome (PRO) Questionnaires. The study is also describing the time to renal replacement therapy (RRT), such as dialysis and transplantation, and the long-term mortality rate and causes (i.e. renal and hepatic), in ADPKD patients treated with JINARC™ (tolvaptan)

Study Overview

• Status: Recruiting
• Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)

• Study Type: Observational
• Enrollment (Estimated): 530

Contacts and Locations

• Study Contact: Name: Annick Laplante; Phone Number: +1 514-654-8616; Email: Annick.Laplante@otsuka-ca.com

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada
      • Recruiting
  • Ontario
    • London, Ontario, Canada
      • Recruiting
    • Ottawa, Ontario, Canada
      • Recruiting
    • Toronto, Ontario, Canada
      • Recruiting
  • Quebec
    • Montreal, Quebec, Canada
      • Recruiting
    • Quebec City, Quebec, Canada
      • Recruiting

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

The study population will be adult patients newly prescribed with JINARC™ (tolvaptan) for the treatment of ADPKD, or patients who are on-treatment with JINARC™ (tolvaptan) at the time of enrolment. Investigator must be ADPKD experts qualified by experience and ability to perform the study.

Description

Inclusion Criteria:

• ADPKD patients ≥18 years old at the time of tolvaptan initiation
• The treating physician must have reached the decision to treat the patient with JINARC™ (tolvaptan) as per the Canadian Product Monograph prior to and independently of soliciting the patient to participate in the study
• The patient or legal guardian must have signed an informed consent indicating the understanding of the study and allowing the use of their anonymous data for the purposes of the study

Exclusion Criteria:

• The patient does not comprehend or refuses to sign the informed consent
• The patient has any contraindications to the use of JINARC™ (tolvaptan) as specified in the Canadian Product Monograph
• The patient has any condition which, as per the judgment of the treating physician, prohibits them from participating in the study

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
ADPKD patients on tolvaptan; ADPKD patients who are newly prescribed with JINARC™ (tolvaptan) or already treated with JINARC™ (tolvaptan) will be eligible.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Changes from baseline in Medical Outcomes Study Short-Form 12 (MOS-SF-12v2) score	Maximum of 120 months
Changes from baseline in Autosomal Dominant Polycystic Kidney Disease-Impact Scale (ADPKD-IS) score	Maximum of 120 months
Changes from baseline in ADPKD - Pain & Discomfort Scale (ADPKD-PDS) score	Maximum of 120 months
Description of Health Care Resource Utilization	Maximum of 120 months
Work and productivity loss measured with the Work Productivity and Activity Impairment (WPAI) questionnaire	Maximum of 120 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to renal replacement therapy		Maximum of 120 months
Long-term mortality rate and causes		Maximum 120 months
Changes in markers of renal function	Markers of renal function: Total Kidney Volume (mL); Kidney Length (cm); estimated Glomerular Filtration Rate (eGFR); Chronic Kidney Disease (CKD) stage	Maximum of 120 months
Adherence to treatment measured with the Medication Adherence Questionnaire		Maximum of 120 months
Time to tolvaptan treatment discontinuation		Maximum of 120 months
Long-term safety profile of tolvaptan	Incidence of adverse events	Maximum of 120 months

Collaborators and Investigators

• Sponsor: Otsuka Canada Pharmaceutical Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2016
• Primary Completion (Estimated): December 1, 2025
• Study Completion (Estimated): June 1, 2026

Study Registration Dates

• First Submitted: September 30, 2016
• First Submitted That Met QC Criteria: October 4, 2016
• First Posted (Estimated): October 5, 2016

Study Record Updates

• Last Update Posted (Actual): February 12, 2024
• Last Update Submitted That Met QC Criteria: February 8, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Quality of Life; Kidney Disease; ADPKD-Related Outcome
• Additional Relevant MeSH Terms: Urologic Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant
• Other Study ID Numbers: 156-203-00047

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Participants consenting to share their data are consented and identified as such in the clinical database.
• IPD Sharing Time Frame: During the course of 2024, a CSR covering the study period from 2016 to the end of 2023 will be produced. Another CSR will be produced in 2026, after the conclusion of the 120-month study period.
• IPD Sharing Supporting Information Type: CSR