RAYALDEE Non Interventional Study (NIS) on Effectiveness in ND-CKD SHPT Patients

Study Overview
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Brief Summary
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The purpose of this study is to generate data on the effectiveness of Rayaldee® to gradually increase 25-hydroxyvitamin D (25D) safely and to a high enough magnitude to reliably reduce elevated parathyroid hormone in patients with non-dialysis chronic kidney disease. Rayaldee®, a prolonged-release calcifediol (PRC) formulation, is an orally administered prohormone of active vitamin D (1,25-dihydroxyvitamin D [1,25D]) designed to increase serum total 25-hydroxyvitamin D (25D) safely and to a high enough magnitude to reliably reduce elevated parathyroid hormone (PTH) in patients with non-dialysis chronic kidney disease (ND-CKD). Clinical studies show that PRC is an effective, well tolerated treatment for secondary hyperparathyroidism (SHPT) in ND-CKD patients with vitamin D insufficiency or deficiency. PRC gradually raises serum 25D levels, resulting in physiologically regulated increases in serum 1,25D and sustained and progressive reductions in PTH levels, while avoiding clinically meaningful increases in serum phosphate and calcium. To date, experience with the use of Rayaldee® results exclusively from patients from the United States and mainly from patients who have participated in clinical trials. It is therefore of major interest to observe the value of Rayaldee® in daily use outside of controlled trial settings with a larger sample size and in Europe.

Detailed Description
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This study is a non-interventional, prospective, multicentre, multinational (European), cohort study. A maximum of 500 patients diagnosed with ND-CKD and SHPT and initiated on Rayaldee® treatment will be included. The scheduled total study duration is 2 years per country, with a recruitment phase of 1 year. The individual prospective observational period per patient is scheduled to last at least 12 months. The observational period of Rayaldee® (both retrospective and prospective) is scheduled up to 18 months after treatment start. Treatment with Rayaldee® will follow the physician's prescription. A patient is eligible if Rayaldee® treatment has been initiated within maximal 6 months upfront to or at study inclusion. Inclusion of patients with Rayaldee® initiation maximal 6 months prior to study inclusion are possible as long as the following laboratory parameters are available at the initiation of Rayaldee® treatment: 25D, intact parathyroid hormone (iPTH), serum calcium, and serum phosphate.

Official Title
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Non-interventional Study to Investigate the Real-world Effectiveness, Safety, and Adherence to Rayaldee® in Non-dialysis CKD Patients With Secondary Hyperparathyroidism (SHPT)

Conditions
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Chronic Kidney Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed informed consent Age ≥18 years Indication for Rayaldee® treatment in accordance with the currently approved Summary of Product Characteristics (SmPC) Stable kidney function in the medical judgment of the investigator Exclusion Criteria: Parallel participation in an interventional study • Enrolment in a prior clinical trial with Rayaldee®

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Single Cohort<br>Data from medical records up to 3 months prior treatment start will be retrospectively collected (if available), to provide baseline data and to serve as a basis for evaluating the treatment decision. If relevant baseline data is not available within 3 months preceding treatment start, retrospective documentation can extend to up to 6 months. The observational period (both retrospective and prospective) is scheduled up to 18 months after treatment start. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in 25-hydroxyvitamin D (25D) and iPTH levels | ng/mL or nmol/L, pg/mL or pmol/L | From up to 6 months prior to Rayaldee treatment start to up to 18 months after treatment | 
| Change in serum calcium level | mg/mL or nmol/L | From up to 6 months prior to Rayaldee treatment start to up to 18 months after treatment | 
| Change in serum phosphate level | mg/mL or nmol/L | From up to 6 months prior to Rayaldee treatment start to up to 18 months after treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hyperparathyroidism, calcifediol