Alder Hey one of Europe’s largest children’s hospitals and is a leading paediatric research centre. Its aim is to be the premier world class centre for research, innovation and education, and we aim to pioneer and develop new treatments for children and young people.
This is made possible by a comprehensive range of paediatric specialist and general clinical services, its expert healthcare professional workforce, a large multidisciplinary and experienced clinical research delivery workforce and its extensive access to a supra-regional population of children and young people. At any time, there are over 200 clinical research studies taking place, ranging from observational studies to complex, interventional clinical trials supported by the Clinical Research Division, including the NIHR Alder Hey Clinical Research Facility. Alder Hey is the UK’s largest centre for volume of recruitment into children’s clinical research studies.
Alder Hey is one of the larger neuromuscular centres in the UK and has recently been again awarded centre of clinical excellence status by the National Audit Commissioned by MDUK.
Institute in the Park
Alder Hey Children’s NHS Foundation Trust
Eaton Road, Liverpool L12 2AP
|Study Name||Trial Status at Alder Hey||Overall Trial Status|
|Pfizer - DMD C3391003 Gene Therapy||Not yet recruiting||Recruiting|
|Italfarmaco - Givinostat Extension||Enrolling by invitation||Enrolling by invitation|
|Sarepta - MOMENTUM||Recruiting||Recruiting|
|DYSTANCE 51 [TERMINATED]||Trial complete/terminated||Trial terminated|
|Patient Registry Translarna (Ataluren)||Fully recruited||Fully recruited|
|Wave Life Sciences Exon 51||Trial complete/terminated||Trial complete|
|Vamorolone Phase 2b (VISION-DMD)||Fully recruited||Fully recruited|
|Tamoxifen (TAMDMD)||Fully recruited||Fully recruited|
|Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD.||Fully recruited||Recruiting|
|Roche- RO7239361 [TERMINATED]||Trial complete/terminated||Trial terminated|
|Italfarmaco- Givinostat (EPIDYS)||Fully recruited||Fully recruited|
|FOR-DMD||Fully recruited||Fully recruited|
Dr Spinty studied medicine at the University of Hamburg, Germany. Initial clinical training was in Germany but subsequently in the UK.
He completed his specialist Paediatric and Paediatric Neurology training in Leeds, UK and was appointed as Consultant Paediatric Neurologist and lead clinician for the neuromuscular services at Alder Hey Children’s Hospital in 2004. The service was again granted “centre of clinical excellence” status in December 2015.
Since 2010, Dr Spinty has been the lead for neuromuscular research at Alder Hey. He is the Principal Investigator for currently 9 studies related to Duchenne Muscular Dystrophy.
He has founded and is the chair of the North West Adult and Paediatric Neuromuscular Network that achieved excellence status in December 2016.
Aiste started her journey to biomedical research in Lithuania, where she got an equivalent of the 1st class honours degree in Biochemistry from Vilnius University. She then took a PhD offer from the University of Manchester and moved to the UK to investigate electrophysiological properties of excitable cells with the main focus on vascular smooth muscle. She was awarded a PhD in Physiology in 2012 and then took a post-doctoral project at The University of Oxford to study pharmacology and biophysics of specialised proteins called ion channels that are embedded in cell membranes and regulate their function. Upon successful completion of the project Aiste was awarded an Early Investigator’s Prize for her publication on ion channel characteristics in the Journal of Physiology.
Aiste then moved back to the University of Manchester, where she joined a cardiac research group. She was investigating structural and functional properties of ovine atrial transverse tubules, i.e. extensions of the cell membrane penetrating towards the interior of the cell. In parallel to this project, she also got an Associated Fellowship Recognition by the Higher Education Academy for her contributions to teaching.
After successfully completing two biomedical research projects, Aiste moved to clinical research and took a Clinical Trials Coordinator’s role at Alder Hey Children’s Hospital. She manages different aspects of the Duchenne muscular dystrophy trials portfolio from trial initiation to completion. Aiste is extremely pleased to see new pharmacological targets being identified for the treatment of this muscle wasting disease. She hopes that new therapies would lead to a life-changing breakthrough in clinical practice.
Originally from Cork, Ireland, Sarah moved to Bristol in September 2000 to study physiotherapy at the University of West of England in Bristol. After qualifying in 2003, Sarah took up her first post as a junior physiotherapist in Hope Hospital in Salford until moving to Alder Hey Children’s Hospital, Liverpool in 2006 as a Senior Physiotherapist. She worked across all acute and community paediatric specialities gaining an interest in Neurology. In 2010, Sarah started a MRes degree at the University of Liverpool, which allowed her to complete research projects of interest to her and her area of clinical work. After a break for maternity leave, Sarah moved into the Neuromuscular Physiotherapy team in 2012, initially as a temporary position and then became a permanent member of the team in 2014. In 2015, she completed her MRes in clinical Science with distinction. Having gained a keen interest in research during her MRes studies, Sarah took the opportunity to take up the research physiotherapist post as part of the Alder Hey Neuromuscular team, in collaboration with Duchenne UK.
Tim has been appointed as a Duchenne muscular dystrophy Research Nurse at Alder Hey. He qualified as a staff nurse at Alder Hey in 2003 and worked on the regional oncology unit at Alder Hey.
He became a research nurse at the Clinical Research Facility in 2012, where he has lead on multiple early phase research studies, including oncology studies, studies in theatre, and gastroenterology studies amongst others. Also, he has led on 2 studies that recruited the first global patients to the study.
Away from work he is a family man and spends most of his weekends watching his children play multiple sports! He is a keen cyclist and plays football socially once a week.
Rebecca graduated from Edge Hill University in 2013, and was awarded with a bachelor of Science degree in Children’s Nursing. She received her first nursing post at a Alder Hey Children’s Hospital, working as a staff nurse on a medical specialty ward from 2013-2018. She then joined the clinical research team in 2018 and was assigned to the DMD research post in March 2020, before she was seconded to assist with COVID-19 research studies.
Rebecca is currently working with the Duchenne Muscular Dystrophy Research team. She co-leads on numerous DMD trials and works alongside a team of DMD research professionals. Her role helps to assist the study sponsors in finding better and safer treatments for children and young people with DMD, and hopefully in the future to find a cure.