D-IMPACT
Improving Measurement and Priorities for Assessment in Clinical Trials and studies of non-degenerative childhood Dystonia
We are setting agreed priorities for what should be measured in clinical trials of childhood-onset dystonia.
At a glance
- Study type
- Core outcome set development (scoping review, qualitative study, professional survey, Delphi consensus)
- Population
- Children and young people with non-degenerative dystonia, their families, clinicians and researchers
- Setting
- International, online
- Funder
- NIHR Advanced Fellowship (NIHR303636)
- Registration
- COMET — Study 3483
- Ethical approval
- Camden and King's Cross Research Ethics Committee (25/LO/0150) and Queen Mary University of London Research Ethics Committee
- Dates
- 2024 to 2028
What and why
When children take part in research on dystonia treatments, studies measure many different things in many different ways. That makes it hard to compare results, to know whether what's being measured is what truly matters to children and families, and to work out which treatments help the most. D-IMPACT brings together children, families, and clinicians to agree what a clinical trial or study involving children with non-degenerative childhood dystonia should always measure.
The result will be an international core outcome set: a minimum agreed list of outcomes that future trials and studies should report. Core outcome sets reduce waste in research, improve evidence synthesis, and put the priorities of children and families at the centre of trial design.
How the study works
D-IMPACT runs in three phases and combines several research methods.
Phase 1 has three parallel work streams and gathers evidence from three sources:
- a scoping review of the outcomes already used in dystonia research,
- interviews with children and their families about what matters most to them,
- an international survey of professionals across health, social care, and education.
Phase 2 is an international e-Delphi study with three rounds, building on what Phase 1 found. It brings together two groups to reach consensus on which outcomes belong in the core set:
- a professional panel, and
- experts with lived experience (children and parents).
Phase 3 identifies the measurement tools available for the agreed outcomes and recommends how each should be assessed.
Take part in the Delphi study
Help set the priorities for measuring outcomes in dystonia trials.
- Open to clinicians, young people with dystonia, parents and carers, and researchers.
- Three online rounds.
- About 20 minutes per round.
Team
Hortensia Gimeno, Childhood Participation Lab, Queen Mary University of London, UK
Katherine Knighting, Childhood Participation Lab, Edge Hill University, UK
Hannah Scott, Childhood Participation Lab, Queen Mary University of London, UK
Khamani Edwards, Childhood Participation Lab, Queen Mary University of London, UK
Eleanor P Redding, Childhood Participation Lab, Queen Mary University of London, UK
Enna Thea Kul-Want, Childhood Participation Lab, Queen Mary University of London, UK
Grace Stynes, Childhood Participation Lab, Queen Mary University of London, UK
Georgia A Vine, Childhood participation lab, University of Huddersfield, UK
Ellie Simpson, Childhood Participation Lab, Queen Mary University of London, UK
Rob Molloy, Childhood Participation Lab, Queen Mary University of London, UK
Claire Higgins, Childhood Participation Lab, Queen Mary University of London, UK
Bhooma R. Aravamuthan, Washington University School of Medicine, USA
Paula S C Chagas, Universidade Federal de Juiz de Fora, Brazil
Marina de Koning-Tijssen, University of Groningen, Netherlands
Hendriekje Eggink, University of Groningen, Netherlands
Darcy Fehlings, University of Toronto, Canada
Emmanuel Roze, Sorbonne University, France
Adrienne Harvey, Murdoch Children's Research Institute, Australia
Jean-Pierre Lin, Evelina London Children's Hospital, UK
Daniel E. Lumsden, Evelina London Children's Hospital, UK
Jonathan W. Mink, Dystonia Medical Research Foundation, USA
Eva Maria Navarrete Muñoz, Miguel Hernández University, Spain
Belen Perez Dueñas, Universitat Autònoma de Barcelona, Spain
Peter Rosenbaum, McMaster University, Canada
Kirsty Stewart, The Children's Hospital at Westmead, Australia
Outputs
- [coming soon…]
Acknowledgements
Funded by NIHR Advanced Fellowship (NIHR303636). With thanks to our experts with lived experience.
