Clinical Trial: Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypoph
Brief Summary: UX023-CL301 is a multicenter, randomized, open-label, Phase 3 study comparing the efficacy and safety of KRN23 with active control (oral phosphate/active vitamin D therapy) in children with XLH (aged 1 to ≤12 years) who have radiographic evidence of rickets, open epiphyses, and have received oral phosphate/active vitamin D therapy for ≥ 6-12 consecutive months prior to screening. Approximately 60 subjects will be randomized 1:1 to receive open-label KRN23 administered by subcutaneous injection or oral phosphate and active vitamin D therapy for a total of 64 weeks.
Detailed Summary:
Sponsor: Ultragenyx Pharmaceutical Inc
Current Primary Outcome: Improvement in rickets in children with XLH [ Time Frame: 40 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change in Serum Phosphorous [ Time Frame: 64 weeks ]Effect of KRN23 as compared with active control by change from baseline in serum phosphorous
- Change in Serum 1,25(OH)D [ Time Frame: 64 weeks ]Effect of KRN23 as compared with active control by change from baseline in serum 1,25(OH)D
- Walking ability using the Six Minute Walk Test (6MWT) [ Time Frame: 24, 40 and 64 weeks ]Change from baseline in total distance walked
- Growth [ Time Frame: 24, 40 and 64 weeks ]Change from baseline in standing height
- Patient/Parent reported pain, fatigue and physical function/mobility [ Time Frame: 24, 40 and 64 weeks ]Change from baseline using the Patient Reported Outcomes Measurement Information System (PROMIS)
- Phamacokinetics throughout the dosing cycle [ Time Frame: 64 weeks ]Measure the concentration of KRN23
- Incidence, frequency, and severity of AE's and SAE's [ Time Frame: 64 weeks ]Incidence compared against active control group
Original Secondary Outcome: Same as current
Information By: Ultragenyx Pharmaceutical Inc
Dates:
Date Received: May 23, 2016
Date Started: September 2016
Date Completion: September 2018
Last Updated: May 2, 2017
Last Verified: May 2017