Non-interventional Study to Collect Real-world Effectiveness, Safety, and Adherence Data on Extended-release Calcifediol (Rayaldee®) in Non-dialysis CKD Patients With Secondary Hyperparathyroidism. Protocol Version 2.0, 06DEC2023
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Vifor (International) Inc.
- Enrollment
- 110
- Locations
- 16
- Primary Endpoint
- Change in serum phosphate level
Study Overview
Brief Summary
The overall study objective is to collect real-world data on the safety and effectiveness of ERC to gradually increase 25D to the level required by Stage 3 and 4 CKD patients.
ERC (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 25D safely and to a high enough magnitude to reliably reduce elevated PTH in patients with non-dialysis chronic kidney disease (ND-CKD). Clinical studies show that ERC is an effective, well tolerated treatment for secondary hyperparathyroidism (SHPT) in ND-CKD patients with Vitamin D insufficiency or deficiency. ERC 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 ERC results exclusively from patients from the US and mainly from patients who have participated in clinical trials. It is therefore of major interest to observe the value of ERC in daily use outside of the controlled trial settings in the US as well as in Europe. (Protocol v.2.0,06Dec2023).
Detailed Description
Non-interventional, prospective, multicentre, cohort study. Approximately 100 patients diagnosed with ND-CKD with SHPT being treated with ERC according to the SmPC will be included.
The scheduled total study duration is 2.5 years, with a recruitment phase of approximately 1.5 years. The individual prospective observational period per patient is scheduled to last up to 12 months, or up to 18 months for pre-treated patients. The number of observational time points for an individual patient will be dependent on the individual observational period and is assumed to be approximately every 3 months.
A patient is eligible if ERC treatment initiation can coincide with the date of patient inclusion in the study or can happen up to 6 months before patient inclusion in the study. The observational period of ERC treatment will be retrospective if it occurs before study inclusion and prospective for the period after study inclusion. The decision to initiate treatment remains with the treating physician, in line with their normal standard of care, in accordance with the SMPC.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Retrospective
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Signed informed consent
- •Age ≥18 years
- •Indication for ERC treatment in accordance with the currently approved SmPC.
- •Starting ERC treatment or being on ERC treatment for a maximum of 6 months before inclusion in the study.
- •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 ERC
Outcomes
Primary Outcomes
Change in serum phosphate level
Time Frame: From up to 6 months prior of ERC treatment start to up to 18 months after treatment
mg/mL or nmol/L
Change in 25-hydroxyvitamin D (25D) and iPTH levels
Time Frame: From up to 6 months prior of ERC treatment start to up to 18 months after treatment
ng/mL or nmol/L, pg/mL or pmol/L
Change in serum calcium level
Time Frame: From up to 6 months prior of ERC treatment start to up to 18 months after treatment
mg/mL or nmol/L
Secondary Outcomes
No secondary outcomes reported