A Study to Evaluate Safety, Efficacy and Pharmacokinetics of Paricalcitol For Treatment of Secondary Hyperparathyroidism (SHPT) in Pediatric Participants With Stage 5 Chronic Kidney Disease (CKD)

Study Overview
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Brief Summary
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The main objective of this study is to evaluate the safety, efficacy and pharmacokinetics of paricalcitol oral solution in pediatric participants of ages 0 to 9 years with SHPT associated with stage 5 CKD receiving Peritoneal Dialysis (PD) or Hemodialysis (HD). The 24-week study is divided into two 12-week dosing periods (Dosing Period 1 followed by Dosing Period 2).

Official Title
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A Phase 3, Prospective, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Paricalcitol Oral Solution for the Treatment of Secondary Hyperparathyroidism in Pediatric Subjects Ages 0 to 9 Years With Stage 5 Chronic Kidney Disease Receiving Peritoneal Dialysis or Hemodialysis

Conditions
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Chronic Kidney Disease (CKD), Secondary Hyperparathyroidism (SHPT)

Intervention / Treatment
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* Drug: Paricalcitol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant is currently diagnosed with and/or being treated for secondary hyperparathyroidism (SHPT). Participant must be diagnosed with chronic kidney disease (CKD) stage 5 receiving peritoneal dialysis (PD) or hemodialysis (HD) for at least 30 days prior to initial Screening. For entry into the Washout Period (for vitamin D receptor activator [VDRA] non-naive participants), the participant must meet the appropriate laboratory criteria based upon the participant's age as described in the protocol. For entry into the Dosing Period (for VDRA-naive participants or VDRA non-naive participants who have completed the Washout Period), the participant must meet the appropriate laboratory criteria based upon the participant's age as described in the protocol. Exclusion Criteria: Participant is scheduled to receive a living donor kidney transplant within 3 months of Screening or is a kidney transplant recipient. Participant is expected to discontinue peritoneal dialysis (PD) or hemodialysis (HD) within 3 months of the initial Screening visit. Participant has had a parathyroidectomy within 12 weeks prior to Screening. Participant is taking maintenance calcitonin, bisphosphonates, glucocorticoids (in a dose equivalent to more than > 0.16 mg/kg/day or 5 mg prednisone/day, whichever is lower), 4 weeks prior to Dosing. Participant is receiving calcimimetics at the time of Screening or is expected to initiate calcimimetics at any time throughout the study. Participant is unable to take oral medications.

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 9 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Participants Receiving Paricalcitol<br>Participants will be administered paricalcitol three times a week (TIW) but no more frequently than every other day for 24 weeks | Drug: Paricalcitol<br>* Paricalcitol oral solution (2.5 mcg/mL) will be administered with an oral dispenser<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Who Achieve Positive Response During Dosing Period 1 | Positive response is defined as having two consecutive >= 30% reductions from baseline in intact parathyroid hormone (iPTH) or two consecutive iPTH values in the target range between 150 picograms (pg)/milliliters (mL) to 300 pg/mL (16.5-33.0 picomole[pmol]/L). | Up to Week 12 | 
| Incidence of Hypercalcemia During Dosing Period 1 | Incidence of hypercalcemia is defined as two consecutive, post-baseline, corrected calcium measurements above the normal participants's age-specific upper limit. | Up to Week 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Who Achieve a Positive Response During Dosing Period 2 | Positive response is defined as having two consecutive >= 30% reductions from baseline in iPTH or two consecutive iPTH values in the target range between 150 pg/mL to 300 pg/mL (16.5-33.0 picomole[pmol]/L). | Week 12 through Week 24 | 
| Percentage of Participants Who Achieve a Positive Response During Dosing Periods 1 and 2 Combined | Positive response is defined as having two consecutive >= 30% reductions from baseline in iPTH or two consecutive iPTH values in the target range between 150 pg/mL to 300 pg/mL (16.5-33.0 picomole[pmol]/L). | Up to Week 24 | 
| Percentage of Participants Who Achieve Two Consecutive >= 30% Reductions in iPTH From Baseline During Dosing Period 1 | Participants who achieve two consecutive >= 30% reductions in iPTH will be evaluated. | Up to Week 12 | 
| Percentage of Participants Who Achieve Two Consecutive >= 30% Reductions in iPTH From Baseline During Dosing Period 2 | Participants who achieve two consecutive >= 30% reductions in iPTH will be evaluated. | Week 12 through Week 24 | 
| Percentage of Participants Who Achieve Two Consecutive >= 30% Reductions in iPTH From Baseline During Dosing Periods 1 and 2 Combined | Participants who achieve two consecutive >= 30% reductions in iPTH will be evaluated. | Up to Week 24 | 
| Percentage of Participants Who Achieve Two Consecutive iPTH Values Between 150 pg/mL to 300 pg/mL (16.5 - 33.0 pmol/L) During Dosing Period 1 | Participants who achieve two consecutive iPTH values between 150 pg/mL to 300 pg/mL (16.5 - 33.0 pmol/L) will be evaluated. | Up to Week 12 | 
| Percentage of Participants Who Achieve Two Consecutive iPTH Values Between 150 pg/mL to 300 pg/mL (16.5 - 33.0 pmol/L) During Dosing Period 2 | Participants who achieve two consecutive iPTH values between 150 pg/mL to 300 pg/mL (16.5 - 33.0 pmol/L) will be evaluated. | Week 12 through Week 24 | 
| Percentage of Participants Who Achieve Two Consecutive iPTH Values Between 150 pg/mL to 300 pg/mL (16.5 - 33.0 pmol/L) During Dosing Periods 1 and 2 Combined | Participants who achieve two consecutive iPTH values between 150 pg/mL to 300 pg/mL (16.5 - 33.0 pmol/L) will be evaluated. | Up to Week 24 | 
| Incidence of Hypercalcemia During Dosing Period 2 | Incidence of hypercalcemia is defined as two consecutive, post-baseline, corrected calcium measurements above the normal participants's age-specific upper limit. | Week 12 through Week 24 | 
| Incidence of Hypercalcemia During Dosing Periods 1 and 2 Combined | Incidence of hypercalcemia is defined as two consecutive, post-baseline, corrected calcium measurements above the normal participants's age-specific upper limit. | Up to Week 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic Kidney Disease, Paricalcitol, Hyperparathyroidism, Pediatric Subjects, Peritoneal Dialysis (PD), Hemodialysis (HD), Intact parathyroid hormone (iPTH)