Evaluation of oral acceptability of RLF-OD032 for children with PKU

• Research type

  Research Study

• Full title

  A prospective, single-arm, open-label study to evaluate the oral acceptability of RLF-OD032 oral suspension in paediatric patients (aged 2-14 years) with Phenylketonuria (PKU)

• IRAS ID

  367772

• Contact name

  Suresh Vijay

• Contact email

  suresh.vijay1@nhs.net

• Sponsor organisation

  APR Applied Pharma Research sa

• Duration of Study in the UK

  1 years, 0 months, 0 days

• Research summary

  A prospective, single-arm, open-label study in 10 evaluable paediatric patients (aged 2-14 years) with PKU to assess oral acceptability. Participants will temporarily use a sapropterin oral suspension (RLF-OD032) in place of their usual formulation for 7 days. Data on their current drug will be collected via the Current Formulation Evaluation Questionnaire and following a 7-day study period of the new formulation, a daily diary and the RLF-OD032 Oral Acceptability Questionnaire will allow oral acceptability to be evaluated.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  26/NW/0052

• Date of REC Opinion

  30 Mar 2026

• REC opinion

  Further Information Favourable Opinion