Blood phenylalanine control of patients with Phenylketonuria in the UK

• Research type

  Research Study

• Full title

  Blood phenylalanine control of patients with Phenylketonuria in the UK

• IRAS ID

  237853

• Contact name

  Anita Macdonald

• Contact email

  Anita.Macdonald@nhs.net

• Sponsor organisation

  Birmingham Women's and Children's Hospital

• Clinicaltrials.gov Identifier

  To be requested, To be requested

• Duration of Study in the UK

  1 years, 3 months, 31 days

• Research summary

  In phenylketonuria (PKU), it is known that generally patients are able to achieve good blood phenylalanine (PHE) control in childhood but compliance decreases with age.
  In 2002, blood PHE control was reported over 6 years from 1994-2000 in 4 centres: 3 UK, 1 Australian. Patients aged <10y had 70% of blood PHE within target range but this decreased to 30% in patients >15y. Treatment strategies and dietary products have improved in recent years. In 2017, the new European PKU guidelines suggested lower blood PHE target concentrations (age 0-12y, 120-360μmol/L; and age >13y, 120-600μmol/L).
  We aim to evaluate blood PHE control in patients with PKU from the main pediatrics and adult centres in the UK. Data will be collected over a period of 7 years and good blood PHE control will be defined according to the European PKU guidelines.

• REC name

  West Midlands - Black Country Research Ethics Committee

• REC reference

  18/WM/0188

• Date of REC Opinion

  28 Jun 2018

• REC opinion

  Favourable Opinion