Phase 1/2 Open-label study of Burosumab in Children <12months with XLH
Research type
Research Study
Full title
A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Pediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia (XLH)
IRAS ID
242321
Contact name
Raja Padidela
Contact email
Sponsor organisation
Kyowa Kirin Pharmaceutical Development Ltd.
Eudract number
2019-000469-19
Duration of Study in the UK
years, months, days
Research summary
The purpose of this research study is to find the best starting dose of research medicine, burosumab, for treating X-linked Hypophosphatemia (XLH) in children less than 12 months old. XLH is an inherited genetic disorder that causes low levels of phosphate in the blood. Phosphate is an important mineral for bones and therefore low levels leads to soft and weak bones, which can result in life-long physical disabilities and pain. Children with this condition usually have bowed or bent legs, short in height, bone pain and delayed walking. In addition, children may also have problems with their teeth and hearing loss.
The researchers will see how safe and effective burosumab is in children less than 12 months of age with XLH. The research study will also measure the amount of burosumab in the participant's blood at various times, and the effect burosumab may have on children with XLH including effects on growth and bone abnormalities.
The participants will be in this study for about 80 weeks. Within the UK, the study will be conducted within NHS hospitals (the research site). The participants will need to visit the research site about 20 times during the research study for study visits. As per the research plan (protocol), during study visits, children may receive research medicine and be examined by a research doctor or nurse, who will also perform blood tests and X-rays of the arms and legs. The study medicine will be administered as an injection under the skin of the abdomen, upper arms or thigh of the participant every 2 weeks. For the first 14 weeks, study medicine will be injected at the research site following which, the research medicine could be administered at home by a research clinician.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
20/EM/0215
Date of REC Opinion
3 Nov 2020
REC opinion
Further Information Favourable Opinion