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
- Conditions
- Hyperphosphatemia
- Interventions
- 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
- 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.
- 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/L25-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/LSerum 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)
Saint Barnabas Medical Center
🇺🇸Livingston, New Jersey, United States
Spitalul Clinic de Urgenţă pentru copii "Maria Sklodowska Curie"
🇷🇴Bucureşti, Bucharest, Romania
Spitalul Clinic Fundeni Bucureşti
🇷🇴București, Bucharest, Romania
St. Vladimir Children's City Clinical Hospital
🇷🇺Moscow, Russian Federation
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
University of Michigan Hospital
🇺🇸Ann Arbor, Michigan, United States
Children's Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku
🇵🇱Białystok, Poland
Instytut "Pomnik - Centrum Zdrowia Dziecka"
🇵🇱Warszawa, Poland
Oregon Health and Science University, Doernbecher Children's Hospital
🇺🇸Portland, Oregon, United States
St. Petersburg GBUZ "Children's City Hospital No. 1"
🇷🇺Saint Petersburg, Russian Federation
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
University of Alabama at Birmingham School of Medicine
🇺🇸Birmingham, Alabama, United States
University of Miami - Miller School of Medicine
🇺🇸Miami, Florida, United States
Emory-Children's Center
🇺🇸Atlanta, Georgia, United States
Hôpital Jeanne de Flandre
🇫🇷Lille, France
Washington University School of Medicine in St. Louis
🇺🇸Saint Louis, Missouri, United States
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
Nemours Children's Clinic - Orlando
🇺🇸Orlando, Florida, United States
University of Oklahoma Medical Center
🇺🇸Oklahoma City, Oklahoma, United States
Chu de Lyon - Hopital Femme Mere Enfant
🇫🇷Lyon, France
The University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Service de Néphrologie et Endocrinologie pédiatriques
🇫🇷Montpellier cedex 5, France
Universitätsklinikum Gießen und Marburg GmbH
🇩🇪Marburg, Germany
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
The University of Texas Medical School at Houston
🇺🇸Houston, Texas, United States
The University of New Mexico
🇺🇸Albuquerque, New Mexico, United States
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
Weill Cornell Medical College
🇺🇸New York, New York, United States
Hospital Of Lithuanian University Of Health Sciences Kaunas Clinics
🇱🇹Kaunas, Lithuania
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
Universitätsklinikum Erlangen
🇩🇪Erlangen, Germany
Texas Children's Hospital - Texas Children's Feigin Center
🇺🇸Houston, Texas, United States
Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos
🇱🇹Vilnius, Lithuania
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdansk, Poland
Uniwersytecki Szpital Dzieciecy w Krakowie - Prokocimiu
🇵🇱Krakow, Poland
Children's Republican Clinical Hospital
🇷🇺Kazan, Russian Federation
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States