X-Linked Hypophosphatemia Registry
Research type
Research Database
Full title
An international, multicentre, prospective, non-interventional observational registry for patients with X-linked hypophosphatemia (XLH)
IRAS ID
229425
Sponsor organisation
Strakan International (SA)
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
X-linked Hypophosphatemia (XLH) is a rare disorder characterised by renal phosphate wasting, hypophosphatemia and defective bone mineralisation. The two main pathological conditions resulting from hypophosphatemia are rickets and osteomalacia.
To date there is little data available on the natural history of XLH such as the number of those affected (incidence and prevalence), the risk factors, co-morbidities (secondary conditions also affecting XLH patients), treatments being prescribed and mortality rates.
The X-linked- Hypophosphatemia (XLH) Registry will be an international, multi-centre, prospective, non-interventional observational registry of patients with X-linked hypophosphatemia. The primary objective of the registry is to collect natural history data to characterise the treatment, progression and long-term outcomes of XLH. The registry will be open to individuals of both sexes aged ≥0 years of age with a diagnosis of XLH.
Data entered into the registry will reflect clinical investigations, assessments and treatment undertaken/prescribed as standard care/routine practice at each centre. Patients will be recruited in centres from across Europe, with an expected total of 1200 patients recruited over 5 years. The secondary objective of the registry is to describe the efficacy and safety of treatments used to manage the symptoms and signs of XLH.
Each countries Chief Investigator (CI) will form part of the XLH Registry Steering Committee in conjunction with Sponsor representatives who will assess projects wishing to access registry data for research.
REC name
North West - Greater Manchester West Research Ethics Committee
REC reference
17/NW/0530
Date of REC Opinion
8 Sep 2017
REC opinion
Favourable Opinion
Research database title
X-linked Hypophosphatemia (XLH) is a rare disorder characterised by renal phosphate wasting, hypophosphatemia and defective bone mineralisation. The two main pathological conditions resulting from hypophosphatemia are rickets and osteomalacia. To date there is little data available on the natural history of XLH such as the number of those affected (incidence and prevalence), the risk factors, co-morbidities (secondary conditions also affecting XLH patients), treatments being prescribed and mortality rates. The X-linked- Hypophosphatemia (XLH) Registry will be an international, multi-centre, prospective, non-interventional observational registry of patients with X-linked hypophosphatemia. The primary objective of the registry is to collect natural history data to characterise the treatment, progression and long-term outcomes of XLH. The registry will be open to individuals of both sexes aged ≥0 years of age with a diagnosis of XLH. Data entered into the registry will reflect clinical investigations, assessments and treatment undertaken/prescribed as standard care/routine practice at each centre. Patients will be recruited in centres from across Europe, with an expected total of 1200 patients recruited over 5 years. The secondary objective of the registry is to describe the efficacy and safety of treatments used to manage the symptoms and signs of XLH. Each countries Chief Investigator (CI) will form part of the XLH Registry Steering Committee in conjunction with Sponsor representatives who will assess projects wishing to access registry data for research.