A Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent Chronic Kidney Disease (CKD) Patients With Hyperphosphataemia

Phase 3

Terminated

Conditions: Hyperphosphatemia

Interventions: Drug: Calcium Acetate (Phoslyra®)
Drug: PA21 (Velphoro®)

Registration Number: NCT02688764

Lead Sponsor: Vifor Fresenius Medical Care Renal Pharma

Brief Summary: This is a Phase 3, Open-label, Randomised, Active-controlled, Parallel Group, Multicentre Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients with Hyperphosphataemia. The aim of this Phase 3 clinical study is to demonstrate similar efficacy of PA21 (Velphoro) in paediatric and adolescent patients with CKD, and to provide safety and dosing information for this patient population. The Phoslyra (comparator) group provides information for a descriptive comparison of PA21 against a commonly used calcium-based phosphate binder (calcium acetate).

Detailed Description: Not available

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 85

Inclusion Criteria

Subjects 0 to <18 years at time of consent.
Subjects with hyperphosphataemia
Subjects ≥1 year with CKD Stages 4-5 defined by a glomerular filtration rate <30 mL/min/1.73 m2 or with CKD Stage 5D receiving adequate maintenance haemodialysis (HD) or peritoneal dialysis (PD) for at least 2 months prior to screening.
Subjects <1 year must have CKD.
Appropriate written informed consent and, where appropriate/required assent, have been provided.

Exclusion Criteria

Subjects with hypercalcaemia at screening
Subjects with intact parathyroid hormone (iPTH) levels >700 pg/mL at screening.
Subjects who are PB naïve who weigh <5 kg at screening. Subjects receiving stable doses of PBs who weigh <6 kg at screening
Subjects requiring feeding tube sizes ≤6 FR (French catheter scale).
Subjects with history of major gastrointestinal surgery or significant gastrointestinal disorders.
Subjects with hypocalcaemia (serum total corrected calcium <1.9 mmol/L; <7.6 mg/dL) at screening.
Subject is pregnant (e.g., positive human chorionic gonadotropin test) or breast feeding.
Subject has a significant medical condition(s)

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Calcium Acetate (Phoslyra®)	Calcium Acetate (Phoslyra®)	Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.
PA21 (Velphoro®)	PA21 (Velphoro®)	PA21 (Velphoro®), chewable tablets 500 mg iron PA21 (Velphoro®), chewable tablets 250 mg iron PA21 (Velphoro®), powder for oral suspension 500 mg iron PA21 (Velphoro®), powder for oral suspension 250 mg iron PA21 (Velphoro®), powder for oral suspension 125 mg iron

Primary Outcome Measures

Name	Time	Method
Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 Group	From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
Number and Percentage of Participants With Any Treatment Emergent Adverse Event	through study completion, up to 34 weeks after treatment start date	Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAEs until end of stage 2. Please refer to the detailed tables included on the Adverse Event Module for specifics.
Number and Percentage of Participants Who Withdrew Due to Treatment Emergent Adverse Events	through study completion, up to 34 weeks after treatment start date	Any adverse event Leading to Study Drug Withdrawal is considered.

Secondary Outcome Measures

Name	Time	Method
Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the Phoslyra Group	From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
Participants With Serum Phosphorus Levels Within the Age Related Normal Range in Each Stage	through study completion, up to 34 weeks after treatment start date	Number and percentages of participants with serum phosphorus levels below, within and above age-dependent normal ranges at baseline, at the end of Stage 1 and at the end of Stage 2. The age related normal ranges for serum phosphorus levels are: * 0 to \<1year 1.36 - 2.62 mmol/L * 1 year to \<6 years 1.03 - 1.97 mmol/L * 6 years to \<9 years 1.03 - 1.97 mmol/L * 9 years to \<10 years 1.03 - 1.97 mmol/L * 10 years to \<15 years 1.00 - 1.94 mmol/L * 15 years to ≤18 years 0.71 - 1.65 mmol/L
25-Hydroxy Vitamin D Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Serum Phosphorus Values at Each Visit	through study completion, up to 34 weeks after treatment start date
Unsaturated Iron Binding Capacity Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Change in Serum Phosphorus Level From Baseline to the End of Stage 2 in Both Groups	From baseline to study completion, up to 34 weeks after treatment start date
Serum iPTH Levels at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Ferritin Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Fibroblast Growth Factor 23 Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Osteocalcin-CL Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Participants With Serum Phosphorus Levels Within the Age-dependent Target Range in Each Stage	through study completion, up to 34 weeks after treatment start date	Number and percentages of participants with serum phosphorus levels below, within and above age-dependent target ranges at baseline, at the end of Stage 1 and at the end of Stage 2. The age target ranges for serum phosphorus levels are: * 0 to \<1 year 1.62-2.52 mmol/L * 1 year to \<6 years 1.45-2.10 mmol/L * 6 years to \<13 years 1.16-1.87 mmol/L * 13 years to ≤18 years 0.74-1.45 mmol/L
Serum Total Corrected Calcium at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Participants With Sustained Hypercalcaemia	through study completion, up to 34 weeks after treatment start date	Number and percentages of participants with at least 1 episode of sustained hypercalcaemia (defined as total calcium value above the upper safety limit confirmed by repeat sample 1 week later) during the study
Transferrin Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Type I Collagen C-Telopeptides Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Tartrate-resistant Acid Phosphatase 5b Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Serum Total Corrected Calcium-Phosphorus Product at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date	Summary statistics of Serum total corrected calcium-phosphorus product at each time point and change from baseline, where serum total corrected calcium-phosphorus product correspond to the product of serum total calcium and Phosphorus, expressed in mmol\^2/L\^2
Iron Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date
Bone Specific Alkaline Phosphatase Values at Each Time Point and Change From Baseline	From baseline through study completion, up to 34 weeks after treatment start date

Trial Locations

Locations (38): University of Alabama at Birmingham School of Medicine
🇺🇸
Birmingham, Alabama, United States
Children's National Medical Center
🇺🇸
Washington, District of Columbia, United States
University of Miami - Miller School of Medicine
🇺🇸
Miami, Florida, United States
Nicklaus Children's Hospital
🇺🇸
Miami, Florida, United States
Nemours Children's Clinic - Orlando
🇺🇸
Orlando, Florida, United States
Emory-Children's Center
🇺🇸
Atlanta, Georgia, United States
University of Iowa Hospitals and Clinics
🇺🇸
Iowa City, Iowa, United States
University of Michigan Hospital
🇺🇸
Ann Arbor, Michigan, United States
Washington University School of Medicine in St. Louis
🇺🇸
Saint Louis, Missouri, United States
Hackensack University Medical Center
🇺🇸
Hackensack, New Jersey, United States
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University of Alabama at Birmingham School of Medicine
🇺🇸Birmingham, Alabama, United States