UX023-CL301 Phase 3 KRN23 (3106-0010)
Research type
Research Study
Full 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 Hypophosphatemia (XLH)
IRAS ID
210749
Contact name
Raja Padidela
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc
Eudract number
2016-000600-29
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
The purpose of this research study is to assess if an experimental drug called KRN23 is an effective and safe treatment for prepubescent children ages 1 to 12 years with X linked hypophosphatemia (XLH) compared to standard treatment (oral phosphate/active vitamin D therapy).
Approximately 60 patients with XLH will be randomised at a ratio of 1:1 to receive open-label KRN23 administered by subcutaneous (SC) injection or standard treatment with oral phosphate and active vitamin D therapy for a total of 64 weeks. The starting dose of KRN23 will be 0.8 mg/kg once every 2 weeks (Q2W). The dose may be increased to 1.2 mg/kg at any time during the study if a subject meets the dose-adjustment criteria. Standard treatment will be administered on an individualised basis at the discretion of the investigator. However, general guidance will be provided based on expert recommendations in Europe.
All participants will discontinue oral phosphate and active vitamin D therapy for 7 days prior to randomisation. Patients randomised to receive KRN23 will also need to discontinue oral phosphate and vitamin D metabolite therapy throughout the duration of the study.
Participants from either treatment groups who complete the active-controlled Treatment Period of the study (64 weeks) may be eligible for an extension study and receive KRN23 treatment for up to an additional 96 weeks or until the study drug is commercially available.
The study will be conducted in a paediatric population; as such, where possible, measures to minimise pain and distress to the patient have been considered including the use of home health visits (KRN23 treatment arm only). At every home health visit time points all subjects randomised to the active control arm will receive a phone call to assess adverse events and concomitant medications. Where possible, timing of assessments will be coordinated with standard safety laboratory tests.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
16/NW/0617
Date of REC Opinion
3 Nov 2016
REC opinion
Further Information Favourable Opinion