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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02688764
Recruitment Status : Terminated (The study was prematurely ended due to the modification of study requirements by the US Food and Drug Administration and the European Medicines Agency)
First Posted : February 23, 2016
Results First Posted : August 28, 2019
Last Update Posted : September 10, 2019
Sponsor:
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Hyperphosphatemia Drug: PA21 (Velphoro®) Drug: Calcium Acetate (Phoslyra®) Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 85 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Randomised, Active-controlled, Parallel Group, Multicentre, Phase 3 Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients With Hyperphosphataemia
Actual Study Start Date : May 26, 2016
Actual Primary Completion Date : February 21, 2019
Actual Study Completion Date : February 21, 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 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

Drug: PA21 (Velphoro®)

During Stage 1 (Open-Label Dose Titration; up to 10 weeks), PA21 subjects will receive PA21 at a starting dose based on their age. Dose of PA21 will be increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject has been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

During Stage 2 (Open-Label Safety Extension, 24 week safety extension),subjects will continue on the dose received at the end of Stage 1, unless a dose change is required. Doses may be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject has been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time during Stage 2.

Other Name: sucroferric oxyhydroxide

Active Comparator: Calcium Acetate (Phoslyra®)
Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.
Drug: Calcium Acetate (Phoslyra®)

During Stage 1 (Open-Label Dose Titration; up to 10 weeks), Phoslyra subjects will receive Phoslyra either at a starting dose based on their weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose of Phoslyra will be increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject has been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

During Stage 2 (Open-Label Safety Extension, 24 week safety extension),subjects will continue on the dose received at the end of Stage 1, unless a dose change is required. Doses may be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject has been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time during Stage 2.





Primary Outcome Measures :
  1. Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 Group [ Time Frame: From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date) ]
  2. Number and Percentage of Participants Who Withdrew Due to Treatment Emergent Adverse Events [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
    Any adverse event Leading to Study Drug Withdrawal is considered.

  3. Number and Percentage of Participants With Any Treatment Emergent Adverse Event [ Time Frame: 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.



Secondary Outcome Measures :
  1. Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the Phoslyra Group [ Time Frame: From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date) ]
  2. Change in Serum Phosphorus Level From Baseline to the End of Stage 2 in Both Groups [ Time Frame: From baseline to study completion, up to 34 weeks after treatment start date ]
  3. Participants With Serum Phosphorus Levels Within the Age-dependent Target Range in Each Stage [ Time Frame: 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

  4. Participants With Serum Phosphorus Levels Within the Age Related Normal Range in Each Stage [ Time Frame: 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

  5. Serum Phosphorus Values at Each Visit [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
  6. Serum Total Corrected Calcium at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  7. Participants With Sustained Hypercalcaemia [ Time Frame: 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

  8. Serum Total Corrected Calcium-Phosphorus Product at Each Time Point and Change From Baseline [ Time Frame: 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

  9. Serum iPTH Levels at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  10. Ferritin Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  11. Unsaturated Iron Binding Capacity Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  12. Iron Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  13. Transferrin Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  14. 25-Hydroxy Vitamin D Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  15. Bone Specific Alkaline Phosphatase Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  16. Type I Collagen C-Telopeptides Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  17. Fibroblast Growth Factor 23 Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  18. Osteocalcin-CL Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  19. Tartrate-resistant Acid Phosphatase 5b Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02688764


Locations
Hide Hide 38 study locations
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United States, Alabama
University of Alabama at Birmingham School of Medicine
Birmingham, Alabama, United States, 35233
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Florida
University of Miami - Miller School of Medicine
Miami, Florida, United States, 33136
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Nemours Children's Clinic - Orlando
Orlando, Florida, United States, 32827
United States, Georgia
Emory-Children's Center
Atlanta, Georgia, United States, 30322-1014
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Michigan
University of Michigan Hospital
Ann Arbor, Michigan, United States, 48109
United States, Missouri
Washington University School of Medicine in St. Louis
Saint Louis, Missouri, United States, 63110
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601-1914
Saint Barnabas Medical Center
Livingston, New Jersey, United States, 07039-5672
United States, New Mexico
The University of New Mexico
Albuquerque, New Mexico, United States, 87131-0001
United States, New York
Weill Cornell Medical College
New York, New York, United States, 10021
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Oklahoma
University of Oklahoma Medical Center
Oklahoma City, Oklahoma, United States, 73104
United States, Oregon
Oregon Health and Science University, Doernbecher Children's Hospital
Portland, Oregon, United States, 97239
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
The University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Texas Children's Hospital - Texas Children's Feigin Center
Houston, Texas, United States, 77030
The University of Texas Medical School at Houston
Houston, Texas, United States, 77030
United States, Utah
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
France
Hôpital Jeanne de Flandre
Lille, France
Chu de Lyon - Hopital Femme Mere Enfant
Lyon, France
Service de Néphrologie et Endocrinologie pédiatriques
Montpellier cedex 5, France, 34295
Germany
Universitätsklinikum Erlangen
Erlangen, Germany, 91054
Universitätsklinikum Gießen und Marburg GmbH
Marburg, Germany, 35043
Lithuania
Hospital Of Lithuanian University Of Health Sciences Kaunas Clinics
Kaunas, Lithuania, LT-50009
Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos
Vilnius, Lithuania, 8406
Poland
Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku
Białystok, Poland, 15-274
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland, 80-952
Uniwersytecki Szpital Dzieciecy w Krakowie - Prokocimiu
Krakow, Poland, 30-663
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Warszawa, Poland
Romania
Spitalul Clinic de Urgenţă pentru copii "Maria Sklodowska Curie"
Bucureşti, Bucharest, Romania, 077120
Spitalul Clinic Fundeni Bucureşti
București, Bucharest, Romania, 22322
Russian Federation
Children's Republican Clinical Hospital
Kazan, Russian Federation
St. Vladimir Children's City Clinical Hospital
Moscow, Russian Federation
St. Petersburg GBUZ "Children's City Hospital No. 1"
Saint Petersburg, Russian Federation
Sponsors and Collaborators
Vifor Fresenius Medical Care Renal Pharma
Investigators
Layout table for investigator information
Principal Investigator: Larry A Greenbaum, MD; PhD Children's Healthcare of Atlanta at Egleston
  Study Documents (Full-Text)

Documents provided by Vifor Fresenius Medical Care Renal Pharma:
Study Protocol  [PDF] September 22, 2015
Statistical Analysis Plan  [PDF] March 20, 2019


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Responsible Party: Vifor Fresenius Medical Care Renal Pharma
ClinicalTrials.gov Identifier: NCT02688764    
Other Study ID Numbers: PA-CL-PED-01
First Posted: February 23, 2016    Key Record Dates
Results First Posted: August 28, 2019
Last Update Posted: September 10, 2019
Last Verified: August 2019
Keywords provided by Vifor Fresenius Medical Care Renal Pharma:
PA21
phosphate binder
Additional relevant MeSH terms:
Layout table for MeSH terms
Hyperphosphatemia
Phosphorus Metabolism Disorders
Metabolic Diseases
Calcium, Dietary
Ferric Compounds
Calcium
Calcium acetate
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Bone Density Conservation Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Hematinics