X-Linked Hypophosphatemia Registry
Research type
Research Database
IRAS ID
326836
Contact name
Nikola Misikova
Contact email
Research summary
An international, multicentre, prospective, non-interventional observational registry for patients with X-linked hypophosphatemia (XLH)
REC name
North West - Greater Manchester West Research Ethics Committee
REC reference
23/NW/0160
Date of REC Opinion
14 Jul 2023
REC opinion
Favourable Opinion
Data collection arrangements
Data entered into the registry will reflect clinical investigations, assessments and treatment undertaken/prescribed as standard care/routine practice at each centre for individuals of both sexes and any age with a diagnosis of XLH, who are naïve
to treatment, treated with conventional therapy (oral phosphate and/or active vitamin D analogue), treated with burosumab, or currently untreated. Participants will be asked to complete Quality of Life scales (including, but not limited to,
Short Form 36 [SF-36] and Paediatric Quality of Life [PedsQL]); these are
optional/recommended and not mandated as per this protocol.Research programme
The Registry is an international, multicentre, prospective, non-interventional observational Registry, open to individuals of both sexes and any age with a diagnosis of XLH, who are naïve to treatment, treated with conventional therapy (oral phosphate and/or active vitamin D analogue), treated with burosumab, or currently untreated. A subset of the Registry data will be used to fulfil a Post-Authorisation Safety Study (PASS) of burosumab as requested by the EMA’s Committee for Medical Products for Human Use (CHMP).
Research database title
An international, multicentre, prospective, non-interventional observational registry for patients with X-linked hypophosphatemia (XLH)
Establishment organisation
Kyowa Kirin International plc.
Establishment organisation address
Galabank Business Park
Galashiels
TD1 1QH