Michela Guglieri
The DMD Hub Central Recruitment Database, a model for effective recruitment to Rare Disease clinical trials
Autori
- MICHELA GUGLIERI (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK – NEUROLOGIA)
- PHILLIP CAMMISH (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK – )
- PIETRO RIGUZZI (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK. DEPARTMENT OF NEUROSCIENCES, UNIVERSITY OF PADOVA, PADOVA, ITALY. – )
- ALEX JOHNSON (DUCHENNE UK – )
- KERRY SHIPPEY (DUCHENNE UK – )
- KATE ADCOCK (MUSCULAR DYSTROPHY UK – )
- SUZANNE GLOVER (PATHFINDERS NEUROMUSCULAR ALLIANCE – )
- EMILY REUBEN (DUCHENNE UK – )
- MARIA ELENA FARRUGIA (INSTITUTE OF NEUROLOGICAL SCIENCES, GLASGOW, SCOTLAND, UK – )
- ROSALINE QUINLIVAN (DEPARTMENT OF NEUROMUSCULAR DISEASES, UCL QUEEN SQUARE INSTITUTE OF NEUROLOGY AND THE NATIONAL HOSPITAL FOR NEUROLOGY AND NEUROSURGERY, LONDON, UK – )
- TRACEY WILLIS (WOLFSON CENTRE FOR INHERITED NEUROMUSCULAR DISEASES AND DEPARTMENT OF MUSCULOSKELETAL HISTOPATHOLOGY, RJAH ORTHOPAEDIC HOSPITAL, OSWESTRY, UK – )
- ANNE-MARIE CHILDS (LEEDS TEACHING HOSPITALS NHS TRUST – )
- MARIACRISTINA SCOTO (GREAT ORMOND STREET HOSPITAL NHS TRUST – )
- CATHY TURNER (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK – )
- MEGAN MCNIFF (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK – )
- DAN RISEBOROUGH (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK – )
- VOLKER STRAUB (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK – )
- EMMA HESLOP (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK – )
Presentatore
MICHELA GUGLIERI (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, TRANSLATIONAL AND CLINICAL RESEARCH INSTITUTE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK)
Modalità
Poster Session
Abstract
“Aims: The DMD Hub (www.dmdhub.org), a network of sites dedicated to clinical trials for Duchenne Muscular Dystrophy (DMD) in the UK, has established the Central Recruitment Database (CRD) (www.dmdhubrecruits.org), a centrally coordinated national recruitment contact database to ensure fair access to clinical research for people with DMD and support trial sites in identifying potentially eligible participants.
Methods: Information on a participant’s condition, including genetic diagnosis and motor abilities, and preferences relating to participation in research studies are being collected via an online questionnaire completed by the participant. Participants are asked to consent to share the information provided directly with trial sites who are looking for potential participants for a specific research study.
Results: The CRD was launched on the 31st March 2022 and has over 230 participants with a confirmed genetic diagnosis of DMD. The CRD has so far facilitated the successful recruitment of 31 participants to 9 research studies (including gene therapy and exon skipping trials) across the UK.
Following this success, the Becker Muscular Dystrophy (BMD) Hub (www.bmd-hub.org) and CRD (www.bmdhubrecruits.org) were launched on the 29th February 2024, aiming to replicate these achievements in promoting clinical trial readiness for BMD research studies.
Conclusions: The CRD has notably improved access to research opportunities for people with DMD regardless of participants’ geographical location and has actively supported UK clinical trial sites in recruiting efforts. The CRD model demonstrates an effective approach to enhance equity and efficacy in participant recruitment for muscular dystrophy research, with potential applicability to other rare diseases.”