NCT03581591

Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets

An Open Label Trial to Assess the Safety and Efficacy of Burosumab (KRN23), an Investigational Antibody to FGF23, in a Single Pediatric Patient With Epidermal Nevus Syndrome(ENS) and Associated Hypophosphatemic Rickets

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Redwood Dermatology Sciences · Academic / Other
• Sex: Female
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A 52 week, open label trial to assess the safety and efficacy of KRN23, an investigational antibody to FGF23, in a single pediatric patient with Epidermal Nevus Syndrome(ENS) and associated hypophosphatemic rickets A 26 weeks extension to original study to monitor patient lab results for her safety.

Detailed description

1.1 Primary Objective The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets 1.2 Secondary Objectives 1. The PD profile of KRN23 as assessed by changes from baseline over time 1,25(OH)2D, iPTH, Serum Calcium, TRP and TmP/GFR (the ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate) 2. Changes in underlying skeletal disease/rickets as assessed by standard radiographs utilizing the Radiographic Global Impression of Change (RGI-C) rating scales 3. Effects of KRN23 on biochemical markers of bone turnover that reflect rickets severity, alkaline phosphatase (ALP) 4. Walking ability as assessed by 6-Minute Walk Test (6MWT) 5. Patient/parent-Reported Outcomes as assessed by PROMIS and FPS-R rating scales 1.3 Exploratory Objective 1\. Dual-energy X-ray absorptiometry (DXA) 1.4 Safety Objective Assess the safety of KRN23 administration in a single patient with ENS-associated hypophosphatemic rickets, based on adverse events (AEs), laboratory assessments, cardiac imaging and renal ultrasound.

Conditions

• Hypophosphatemia
• Hypophosphatemic Rickets
• Pain, Chronic

Interventions

Type	Name	Description
BIOLOGICAL	Burosumab	recombinant human IgG1 monoclonal antibody to fibroblast growth factor 23)

Timeline

• Start date: 2018-01-31
• Primary completion: 2019-12-06
• Completion: 2019-12-06
• First posted: 2018-07-10
• Last updated: 2020-01-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study