Development of a PRO and ObsRO in Pyruvate Kinase Deficiency

• Research type

  Research Study

• Full title

  Development of Patient- and Observer-Reported Outcome Measures in Pyruvate Kinase Deficiency

• IRAS ID

  244086

• Contact name

  Siobhan McDonold

• Contact email

  siobhan.mcdonold@endpointoutcomes.com

• Sponsor organisation

  Agios Pharmaceuticals

• Duration of Study in the UK

  0 years, 4 months, 18 days

• Research summary

  Patient Interview Study: What are the signs, symptoms, and impacts of Pyruvate Kinase Deficiency?

  Pyruvate kinase (PK) deficiency is a type of rare haemolytic anaemia. Common signs and symptoms may include: fatigue, dyspnoea, jaundice, and splenomegaly.
  
  The aim of this non-interventional, qualitative study is to create patient and caregiver questionnaires of the signs, symptoms, and impacts of PK deficiency and to better understand children’s experiences with PK deficiency. Seven to 10 children and seven to 10 parents/caregivers living in the United Kingdom will be interviewed about their/their child’s experiences with PK Deficiency. Well-trained employees of Endpoint Outcomes from Boston, MA, USA, will conduct interviews at King’s College under the principal investigator, Professor David Rees. Following consent, children and their caregivers will talk about the signs/symptoms and impacts of PK deficiency on the child’s overall quality of life. Participants will be asked to report how bothersome each sign/symptom and impact is. Interviews last approximately 30-40 minutes for children and 60 minutes for adults. Phone interviews may also be conducted in the event that scheduling or other issues do not permit for an in-person interview.

  To participate, children should currently (i.e., within the last month) be experiencing at least one anaemia symptom (e.g., tiredness, weakness, exhaustion) and experience either a second anaemia symptom distinct from the first or another PK deficiency symptom (e.g., jaundice, pallor, abdominal pain) in the past.

  No medications are given as part of this study. This is a study where we would like to better understand what it is like to have PK deficiency. By participating in this interview, participants can help to ensure that the perspective of patients with PK deficiency is accurately captured in future clinical trial research.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  18/NE/0250

• Date of REC Opinion

  10 Aug 2018

• REC opinion

  Favourable Opinion