Anti-FGF23 antibody, KRN23, in pediatric patients with XLH

• Research type

  Research Study

• Full title

  A Randomized, Open-Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the anti-FGF23 antibody, KRN23, in Paediatric Patients with X-linked Hypophosphatemia (XLH)

• IRAS ID

  153048

• Contact name

  Raja Padidela

• Contact email

  raja.padidela@cmft.nhs.uk

• Sponsor organisation

  Ultragenyx Pharmaceutical

• Eudract number

  2014-000406-35

• ISRCTN Number

  NA

• Clinicaltrials.gov Identifier

  NA

• Research summary

  The purpose of this research study is to asses if an experimental drug called KRN23 is an effective and safe treatment for prepubescent children ages 5-12 years with X-linked hypophosphatemia (XLH). KRN23 will be administered via subcutaneous (SC) injections monthly (Q4)or biweekly (Q2) for a total of 64 weeks. The study consists of a 16-week individual dose Titration Period, followed by a 48-week Treatment Period. The study will enrol approximately 30 paediatric patients with XLH and radiographic evidence of bone disease. Subjects will need to discontinue oral phosphate and vitamin D metabolite therapy prior to randomization and throughout the duration of the study.

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  14/EM/0174

• Date of REC Opinion

  28 May 2014

• REC opinion

  Further Information Favourable Opinion