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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: 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
  1. Subjects 0 to <18 years at time of consent.
  2. Subjects with hyperphosphataemia
  3. 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.
  4. Subjects <1 year must have CKD.
  5. Appropriate written informed consent and, where appropriate/required assent, have been provided.
Exclusion Criteria
  1. Subjects with hypercalcaemia at screening
  2. Subjects with intact parathyroid hormone (iPTH) levels >700 pg/mL at screening.
  3. Subjects who are PB naïve who weigh <5 kg at screening. Subjects receiving stable doses of PBs who weigh <6 kg at screening
  4. Subjects requiring feeding tube sizes ≤6 FR (French catheter scale).
  5. Subjects with history of major gastrointestinal surgery or significant gastrointestinal disorders.
  6. Subjects with hypocalcaemia (serum total corrected calcium <1.9 mmol/L; <7.6 mg/dL) at screening.
  7. Subject is pregnant (e.g., positive human chorionic gonadotropin test) or breast feeding.
  8. Subject has a significant medical condition(s)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
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
NameTimeMethod
Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 GroupFrom 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 Eventthrough 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 Eventsthrough study completion, up to 34 weeks after treatment start date

Any adverse event Leading to Study Drug Withdrawal is considered.

Secondary Outcome Measures
NameTimeMethod
Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the Phoslyra GroupFrom 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 Stagethrough 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 BaselineFrom baseline through study completion, up to 34 weeks after treatment start date
Serum Phosphorus Values at Each Visitthrough study completion, up to 34 weeks after treatment start date
Unsaturated Iron Binding Capacity Values at Each Time Point and Change From BaselineFrom 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 GroupsFrom baseline to study completion, up to 34 weeks after treatment start date
Serum iPTH Levels at Each Time Point and Change From BaselineFrom baseline through study completion, up to 34 weeks after treatment start date
Ferritin Values at Each Time Point and Change From BaselineFrom baseline through study completion, up to 34 weeks after treatment start date
Fibroblast Growth Factor 23 Values at Each Time Point and Change From BaselineFrom baseline through study completion, up to 34 weeks after treatment start date
Osteocalcin-CL Values at Each Time Point and Change From BaselineFrom 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 Stagethrough 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 BaselineFrom baseline through study completion, up to 34 weeks after treatment start date
Participants With Sustained Hypercalcaemiathrough 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 BaselineFrom 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 BaselineFrom 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 BaselineFrom 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 BaselineFrom 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 BaselineFrom baseline through study completion, up to 34 weeks after treatment start date
Bone Specific Alkaline Phosphatase Values at Each Time Point and Change From BaselineFrom 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

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