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

NCT ID: NCT02688764

Last Updated: 2019-09-10

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

85 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-05-26

Study Completion Date

2019-02-21

Brief Summary

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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

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Conditions

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Hyperphosphatemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

Group Type EXPERIMENTAL

PA21 (Velphoro®)

Intervention Type DRUG

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.

Calcium Acetate (Phoslyra®)

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

Group Type ACTIVE_COMPARATOR

Calcium Acetate (Phoslyra®)

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type 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.

Intervention Type DRUG

Other Intervention Names

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sucroferric oxyhydroxide

Eligibility Criteria

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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)
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vifor Fresenius Medical Care Renal Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Larry A Greenbaum, MD; PhD

Role: PRINCIPAL_INVESTIGATOR

Children's Healthcare of Atlanta at Egleston

Locations

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University of Alabama at Birmingham School of Medicine

Birmingham, Alabama, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

University of Miami - Miller School of Medicine

Miami, Florida, United States

Site Status

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status

Nemours Children's Clinic - Orlando

Orlando, Florida, United States

Site Status

Emory-Children's Center

Atlanta, Georgia, United States

Site Status

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

University of Michigan Hospital

Ann Arbor, Michigan, United States

Site Status

Washington University School of Medicine in St. Louis

St Louis, Missouri, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Saint Barnabas Medical Center

Livingston, New Jersey, United States

Site Status

The University of New Mexico

Albuquerque, New Mexico, United States

Site Status

Weill Cornell Medical College

New York, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

University of Oklahoma Medical Center

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health and Science University, Doernbecher Children's Hospital

Portland, Oregon, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Texas Children's Hospital - Texas Children's Feigin Center

Houston, Texas, United States

Site Status

The University of Texas Medical School at Houston

Houston, Texas, United States

Site Status

Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Hôpital Jeanne de Flandre

Lille, , France

Site Status

Chu de Lyon - Hopital Femme Mere Enfant

Lyon, , France

Site Status

Service de Néphrologie et Endocrinologie pédiatriques

Montpellier, , France

Site Status

Universitätsklinikum Erlangen

Erlangen, , Germany

Site Status

Universitätsklinikum Gießen und Marburg GmbH

Marburg, , Germany

Site Status

Hospital Of Lithuanian University Of Health Sciences Kaunas Clinics

Kaunas, , Lithuania

Site Status

Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos

Vilnius, , Lithuania

Site Status

Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku

Bialystok, , Poland

Site Status

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Site Status

Uniwersytecki Szpital Dzieciecy w Krakowie - Prokocimiu

Krakow, , Poland

Site Status

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, , Poland

Site Status

Spitalul Clinic de Urgenţă pentru copii "Maria Sklodowska Curie"

Bucharest, Bucharest, Romania

Site Status

Spitalul Clinic Fundeni Bucureşti

Bucharest, Bucharest, Romania

Site Status

Children's Republican Clinical Hospital

Kazan', , Russia

Site Status

St. Vladimir Children's City Clinical Hospital

Moscow, , Russia

Site Status

St. Petersburg GBUZ "Children's City Hospital No. 1"

Saint Petersburg, , Russia

Site Status

Countries

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United States France Germany Lithuania Poland Romania Russia

References

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Natale P, Green SC, Ruospo M, Craig JC, Vecchio M, Elder GJ, Strippoli GF. Phosphate binders for preventing and treating chronic kidney disease-mineral and bone disorder (CKD-MBD). Cochrane Database Syst Rev. 2025 Jun 27;6(6):CD006023. doi: 10.1002/14651858.CD006023.pub4.

Reference Type DERIVED
PMID: 40576086 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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PA-CL-PED-01

Identifier Type: -

Identifier Source: org_study_id

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