Clinical trial NCT02688764: 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
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Hyperphosphatemia
Keywords: PA21
phosphate binder
Lead sponsor: Vifor Fresenius Medical Care Renal Pharma
Sponsor class: INDUSTRY
First posted: 2016-02-23
Enrollment: 85
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility 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)

Primary outcomes:
measure: Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 Group
timeFrame: From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
measure: Number and Percentage of Participants Who Withdrew Due to Treatment Emergent Adverse Events
timeFrame: through study completion, up to 34 weeks after treatment start date
measure: Number and Percentage of Participants With Any Treatment Emergent Adverse Event
timeFrame: through study completion, up to 34 weeks after treatment start date
Secondary outcomes:
measure: Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the Phoslyra Group
timeFrame: From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
measure: Change in Serum Phosphorus Level From Baseline to the End of Stage 2 in Both Groups
timeFrame: From baseline to study completion, up to 34 weeks after treatment start date
measure: Participants With Serum Phosphorus Levels Within the Age-dependent Target Range in Each Stage
timeFrame: through study completion, up to 34 weeks after treatment start date
measure: Participants With Serum Phosphorus Levels Within the Age Related Normal Range in Each Stage
timeFrame: through study completion, up to 34 weeks after treatment start date
measure: Serum Phosphorus Values at Each Visit
timeFrame: through study completion, up to 34 weeks after treatment start date
measure: Serum Total Corrected Calcium at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Participants With Sustained Hypercalcaemia
timeFrame: through study completion, up to 34 weeks after treatment start date
measure: Serum Total Corrected Calcium-Phosphorus Product at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Serum iPTH Levels at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Ferritin Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Unsaturated Iron Binding Capacity Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Iron Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Transferrin Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: 25-Hydroxy Vitamin D Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Bone Specific Alkaline Phosphatase Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Type I Collagen C-Telopeptides Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Fibroblast Growth Factor 23 Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Osteocalcin-CL Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
measure: Tartrate-resistant Acid Phosphatase 5b Values at Each Time Point and Change From Baseline
timeFrame: From baseline through study completion, up to 34 weeks after treatment start date
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham School of Medicine
state: Alabama
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Miami
country: United States
facility: University of Miami - Miller School of Medicine
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Clinic - Orlando
state: Florida
city: Atlanta
country: United States
facility: Emory-Children's Center
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Ann Arbor
country: United States
facility: University of Michigan Hospital
state: Michigan
city: Saint Louis
country: United States
facility: Washington University School of Medicine in St. Louis
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Livingston
country: United States
facility: Saint Barnabas Medical Center
state: New Jersey
city: Albuquerque
country: United States
facility: The University of New Mexico
state: New Mexico
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Oklahoma City
country: United States
facility: University of Oklahoma Medical Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health and Science University, Doernbecher Children's Hospital
state: Oregon
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Dallas
country: United States
facility: The University of Texas Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital - Texas Children's Feigin Center
state: Texas
city: Houston
country: United States
facility: The University of Texas Medical School at Houston
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Lille
country: France
facility: Hôpital Jeanne de Flandre
city: Lyon
country: France
facility: Chu de Lyon - Hopital Femme Mere Enfant
city: Montpellier cedex 5
country: France
facility: Service de Néphrologie et Endocrinologie pédiatriques
city: Erlangen
country: Germany
facility: Universitätsklinikum Erlangen
city: Marburg
country: Germany
facility: Universitätsklinikum Gießen und Marburg GmbH
city: Kaunas
country: Lithuania
facility: Hospital Of Lithuanian University Of Health Sciences Kaunas Clinics
city: Vilnius
country: Lithuania
facility: Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos
city: Białystok
country: Poland
facility: Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Krakow
country: Poland
facility: Uniwersytecki Szpital Dzieciecy w Krakowie - Prokocimiu
city: Warszawa
country: Poland
facility: Instytut "Pomnik - Centrum Zdrowia Dziecka"
city: Bucureşti
country: Romania
facility: Spitalul Clinic de Urgenţă pentru copii "Maria Sklodowska Curie"
state: Bucharest
city: București
country: Romania
facility: Spitalul Clinic Fundeni Bucureşti
state: Bucharest
city: Kazan
country: Russian Federation
facility: Children's Republican Clinical Hospital
city: Moscow
country: Russian Federation
facility: St. Vladimir Children's City Clinical Hospital
city: Saint Petersburg
country: Russian Federation
facility: St. Petersburg GBUZ "Children's City Hospital No. 1"
Study officials:
affiliation: Children's Healthcare of Atlanta at Egleston
name: Larry A Greenbaum, MD; PhD
role: PRINCIPAL_INVESTIGATOR
References: