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

NCT02688764 | Terminated Phase 3 Results DMC

• 1 other identifier: PA-CL-PED-01
• Study Type: interventional
• Target: 85
• Locations: 7 countries
• Sites: 38

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).

Conditions

Hyperphosphatemia

Keywords

PA21; phosphate binder

Outcome Measures

Primary Outcomes (3)

• 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 Who Withdrew Due to Treatment Emergent Adverse Events

  Any adverse event Leading to Study Drug Withdrawal is considered.

  through study completion, up to 34 weeks after treatment start date

• Number and Percentage of Participants With Any Treatment Emergent Adverse Event

  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.

  through study completion, up to 34 weeks after treatment start date

Secondary Outcomes (19)

• 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)

• 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

• 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

• 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

• Serum Phosphorus Values at Each Visit

  through study completion, up to 34 weeks after treatment start date

Study Arms (2)

PA21 (Velphoro®)

EXPERIMENTAL

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®)

Calcium Acetate (Phoslyra®)

ACTIVE COMPARATOR

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

Drug: Calcium Acetate (Phoslyra®)

Interventions

PA21 (Velphoro®) 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.

Also known as: sucroferric oxyhydroxide

PA21 (Velphoro®)

Calcium Acetate (Phoslyra®) 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.

Calcium Acetate (Phoslyra®)

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Vifor Fresenius Medical Care Renal Pharma: lead

Study Sites (38)

University of Alabama at Birmingham School of Medicine

Birmingham, Alabama, 35233, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University of Miami - Miller School of Medicine

Miami, Florida, 33136, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Nemours Children's Clinic - Orlando

Orlando, Florida, 32827, United States

Location

Emory-Children's Center

Atlanta, Georgia, 30322-1014, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

University of Michigan Hospital

Ann Arbor, Michigan, 48109, United States

Location

Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601-1914, United States

Location

Saint Barnabas Medical Center

Livingston, New Jersey, 07039-5672, United States

Location

The University of New Mexico

Albuquerque, New Mexico, 87131-0001, United States

Location

Weill Cornell Medical College

New York, New York, 10021, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

University of Oklahoma Medical Center

Oklahoma City, Oklahoma, 73104, United States

Location

Oregon Health and Science University, Doernbecher Children's Hospital

Portland, Oregon, 97239, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

The University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

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

Houston, Texas, 77030, United States

Location

The University of Texas Medical School at Houston

Houston, Texas, 77030, United States

Location

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Hôpital Jeanne de Flandre

Lille, France

Location

Chu de Lyon - Hopital Femme Mere Enfant

Lyon, France

Location

Service de Néphrologie et Endocrinologie pédiatriques

Montpellier, 34295, France

Location

Universitätsklinikum Erlangen

Erlangen, 91054, Germany

Location

Universitätsklinikum Gießen und Marburg GmbH

Marburg, 35043, Germany

Location

Hospital Of Lithuanian University Of Health Sciences Kaunas Clinics

Kaunas, LT-50009, Lithuania

Location

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

Vilnius, 8406, Lithuania

Location

Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku

Bialystok, 15-274, Poland

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

Uniwersytecki Szpital Dzieciecy w Krakowie - Prokocimiu

Krakow, 30-663, Poland

Location

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, Poland

Location

Spitalul Clinic de Urgenţă pentru copii "Maria Sklodowska Curie"

Bucharest, Bucharest, 077120, Romania

Location

Spitalul Clinic Fundeni Bucureşti

Bucharest, Bucharest, 22322, Romania

Location

Children's Republican Clinical Hospital

Kazan', Russia

Location

St. Vladimir Children's City Clinical Hospital

Moscow, Russia

Location

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

Saint Petersburg, Russia

Location

Related Publications (1)

• 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.

  PMID: 40576086 DERIVED

MeSH Terms

Conditions

Hyperphosphatemia

Interventions

sucroferric oxyhydroxide; calcium acetate

Condition Hierarchy (Ancestors)

Phosphorus Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

The study was prematurely ended as a result of the modification of study requirements, as agreed with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Results Point of Contact

• Title: Milica Enoiu / Clinical Research Manager
• Organization: Vifor Fresenius Medical Care Renal Pharma France

milica.enoiu@viforpharma.com

+41588518264

Study Officials

• Larry A Greenbaum, MD; PhD

  Children's Healthcare of Atlanta at Egleston

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 17, 2016
• First Posted: February 23, 2016
• Study Start: May 26, 2016
• Primary Completion: February 21, 2019
• Study Completion: February 21, 2019
• Last Updated: September 10, 2019
• Results First Posted: August 28, 2019

Record last verified: 2019-08

Locations

US (22); PL (4); RU (3); LI (2); FR (3); DE (2); RO (2)