Vifor PA-CL-PED-01

Principal Investigator: Raoul  Nelson
Keywords: hyperphosphataemia , Calcium Acetate , CKD Department: Pediatric Administration
IRB Number: 00093772 Co Investigator:  
Specialty: Pediatrics, General
Sub Specialties: Adolescent Medicine
Recruitment Status: Recruiting

Contact Information

Jeana McKenney
Jeana.mckenney@hsc.utah.edu
801/583-3497

Brief Summary

STUDY OBJECTIVES
Primary Objective
• To evaluate the efficacy of PA21 in reducing serum phosphorus levels in paediatric and adolescent subjects with CKD at the end of Stage 1.
 
Secondary Objectives
• To evaluate the efficacy of PA21 in maintaining the serum phosphorus lowering effects in paediatric and adolescent subjects with CKD at the end of Stage 2.
• To evaluate the safety of PA21 in paediatric and adolescent subjects with CKD.
• To evaluate the efficacy of Phoslyra in reducing and maintaining serum phosphorus levels in paediatric and adolescent subjects with CKD at the end of Stages 1 and 2.
• To evaluate the safety of Phoslyra in paediatric and adolescent subjects with CKD.

Inclusion Criteria

1. Subjects 0 to <18 years at time of consent.
2. Subjects with hyperphosphataemia i.e., with serum phosphorus levels indicated in Table 28.
3. Subjects who are PB naïve or have been receiving stable doses of a PB(s) for at least 1 month prior to screening. Subjects may be on stable doses of a maximum of 2 PBs. Subjects who have been receiving PBs will enter an obligatory washout period and may be randomised once their serum phosphorus levels are as indicated in Table 28. Subjects already receiving a PB but with serum phosphorus levels indicated in Table 28 may be eligible for randomisation without a washout period.
4. 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 HD or PD for at least 2 months prior to screening.
5. Subjects <1 year must have CKD. 
6. PD subjects must have had 1 month of unchanged PD prescription (volume and number of exchanges). Home HD subjects may be included (no nocturnal HD (overnight stay at site) will be allowed).
7. Appropriate written informed consent and, where appropriate/required assent, have been provided. Written informed consent (and, where appropriate/required, assent) must be provided before any study-specific procedures are performed, including screening procedures.
8. Parents/legal guardians and, where appropriate/required, subjects with the ability to provide written informed consent, and where appropriate/required, assent, to understand the requirements of the study and abide by the study restrictions, and who agree to return for the required assessments, in the Investigator’s opinion.

Exclusion Criteria

1. Subjects with hypercalcaemia at screening as indicated in Table 9.
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. (In order to comply with maximum blood sample volumes in paediatric clinical trials [75]).
4. Subjects requiring feeding tube sizes ≤6 FR (French catheter scale).
5. Subjects with planned or expected parathyroidectomy within the next 12 months, in the Investigator’s opinion.
6. Subjects with history of:
− Major GI surgery which, in the Investigator’s opinion, is likely to influence the
outcome of treatment with PBs.
− Significant GI disorders.
7. Subjects with estimated life expectancy of less than 12 months.
8. Subjects with known seropositivity to human immunodeficiency virus.
9. Subjects with a history of haemochromatosis or other iron accumulation disorders.
10. Subjects on PD with a history of peritonitis in the last 3 months or ≥3 episodes in the last 12 months.
11. Subjects with hypocalcaemia (serum total corrected calcium <1.9 mmol/L; <7.6 mg/dL) at screening.
12. Subjects with raised alanine aminotransferase or aspartate aminotransferase >3 times the upper limit of the normal range based on central laboratory results at screening.
13. Subjects taking more than 2 PBs concomitantly prior to screening.
14. Subjects taking any prohibited medication(s) (See Section 7.7).
15. Subject has known hypersensitivity and/or intolerance to any of the active substances or to any of the excipients to be administered.
16. Subject has previously been randomised into this study.
17. Subject is currently enrolled in or has completed any other investigational device or drug study <30 days prior to screening, or is receiving other investigational agent(s).
18. Subject is pregnant (e.g., positive human chorionic gonadotropin test) or breast feeding.
19. If of child-bearing potential, subject is not using adequate contraceptive precautions. Subject must agree to use a highly effective method of birth control during the study and for 1 month after the last dose of study medication. Adequate methods of birth control are defined as those which result in a low failure rate (i.e., <1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptives, some intra-uterine devices, sexual abstinence or vasectomised partner.
Non-child-bearing potential includes being surgically sterilised at least 6 months prior to the study.
20. Subject has a history of drug or alcohol abuse within 2 years prior to screening.
21. Subject has a significant medical condition(s) e.g., uncontrolled diabetes, known hepatitis B surface antigen positivity and/or hepatitis C virus ribonucleic acid positivity or anticipated need for major surgery during the study, or any other kind of disorder that may be associated with increased risk to the subject, or may interfere with study assessments or outcomes.