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 ID: STU 032016-048
The study has a 2-stage design. in total, approximately 100 subjects will be randomised to Pa21 treatment and approximately 30 subjects to Phoslyra treatment. in addition, the study will aim to randomise a minimum number of subjects from each age group as detailed in Table a. Table a Minimum number of Randomised Subjects by age Group age Pa21 Phoslyra 0 to [Less Than]1 year 4 1 [GreaterThanorequalTo]1 year to [Less Than]6 years 10 3 [GreaterThanorequalTo]6 years to [Less Than]9 years 10 3 [GreaterThanorequalTo]9 years to [Less Than]18 years 10 3 - Screening period: up to 4 weeks. - Washout period: up to 3 weeks. - Stage 1: open-label, randomised, active-controlled, dose titration of Pa21 and Phoslyra: up to 10 weeks. - Stage 2: open-label long-term safety extension: 24 weeks. - Follow-up: 14 days. Primary efficacy endpoint * Change in serum phosphorus from baseline in the Pa21 group at the end of Stage 1. Primary Safety endpoint * adverse events (aes) profile. * Percentage of withdrawals due to aes. Secondary efficacy endpoints * Change in serum phosphorus from baseline in the Phoslyra group at the end of Stage 1. * Change in serum phosphorus from baseline in the Pa21 and Phoslyra groups at the end of Stage 2. * Percentage of subjects in each stage during which the subject has serum phosphorus levels in the age dependent target ranges. * Serum phosphorus levels at each time point and change from baseline. Secondary Safety endpoints * Serum total corrected calcium levels at each time point and change from baseline. * Percentage of subjects that develop at least 1 episode of sustained hypercalcaemia (as indicated in Table J) during study participation (confirmed by repeat sample 1 week later). * Serum total corrected calcium x phosphorus levels at each time point and change from baseline. * Serum iPTH levels at each time point and change from baseline. * Biochemical/haematological laboratory tests (including blood iron parameters, Vitamin D parameters and bone markers). additional assessments * Patient reported outcomes on palatability and acceptability.
Inclusion Criteria 1. Subjects 0 to <18 years at time of consent. 2. Subjects with hyperphosphataemia i.e., with serum phosphorus levels indicated in the: Age Related Serum Phosphorus Targets for Washout Period Age mmol/L mg/dL 0 to <6 months >2.62 >8.1 >=6 months to <1 year >2.29 >7.1 >=1 year to <6 years >2.02 > 6.3 >=6 years to <13 years >1.77 >5.5 >=13 years to <18 years >1.36 >4.2 3. Subjects who are PB(phosphate binder) naive 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 randomized 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 randomization 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[Right Quote]s opinion.
- Cancer Related
- Healthy Volunteers
- UT Southwestern Principal Investigator
- Mouin Gerios Seikaly
VIFOR FRESENIUS MEDICAL CARE RENAL PHARM