Alder Hey Children's NHS Foundation Trust

Alder Hey is 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.

Clinical Research Division

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.

Other relevant projects

Contact Information

Department Name
Clinical Research Division
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Institute in the Park
Alder Hey Children’s NHS Foundation Trust
Eaton Road, Liverpool L12 2AP

Primary Phone Number
+44 151 252 5570
Primary Email Address
Primary Contact Person
National Institute for Health Research (NIHR) Contact:
Vicky Wilkinson,
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Current Trials:

Study Name Trial Status at Alder Hey Overall Trial Status
Antisense - ATL1102 Fully recruited Fully recruited
Dyne Therapeutics - DYNE-251 Recruiting Recruiting
Pfizer - CIFFREO Fully recruited Fully recruited
Italfarmaco - Givinostat Extension Fully recruited Fully recruited
Sarepta - MOMENTUM Fully recruited Fully recruited
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) Trial complete/terminated Trial complete
Tamoxifen (TAMDMD) Trial complete/terminated Trial complete
Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD. Fully recruited Fully recruited
Sarepta- ESSENCE Fully recruited Fully recruited
Roche- RO7239361 [TERMINATED] Trial complete/terminated Trial terminated
Italfarmaco- Givinostat (EPIDYS) Trial complete/terminated Trial complete
FOR-DMD Trial complete/terminated Trial complete

Contact Listing:

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.

0151 252 5451

Dr Rajesh Madhu is a Consultant in the Neurosciences Department and the Clinical Lead for the Neuromuscular Services at Alder Hey hospital. He completed his Masters degree in Genomic Medicine from the University of Manchester and lead the Genomic Medicine service for Neuromuscular Disorders.  They provide advanced diagnostic investigations for children with suspected Neuromuscular Disorders including whole genome sequencing. They offer multidisciplinary care to children and deliver advanced disease modifying genetic treatments, including gene therapy, for children with neuromuscular disorders.

He ran the specialist clinics for the following conditions

  • Spinal Muscular Atrophy (SMA) - children with this condition get comprehensive evaluation and treatment with Zolgensma gene therapy as well as Nusinersen and Risdiplam genetic treatments
  • Duchenne Muscular Dystrophy 
  • Myotonic Dystophy 
  • Limb girdle Muscular dystrophy
  • Congenital myasthenia syndromes
  • Friedrich's Ataxia 
  • Congenital Myopathies
  • Joint neuropathy 
  • Arthrogryposis 

He also lead the neuromuscular research at Alder Hey. JHe is the Principal investigator/ co- PI for various clinical trials in neuromuscular disorders, including the exon skipping trials, and gene therapy trial in DMD.He is also the Principal investigator for the SOLVE-RD genomic sequencing study.

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.

0151 252 5660;

Clinical lead on the CRF, Dr Hawcutt is one of six consultant paediatric clinical pharmacologists in the UK, and the only one engaged with early phase paediatric clinical trials.

Dan is chair of the Royal College of Paediatrics and Child Health (RCPCH) College Specialty Advisory Committees (CSACs) for Clinical Pharmacology, responsible for training paediatricians in clinical pharmacology and therapeutics in the UK. Alder Hey CRF has one of only two specialist training posts for junior doctors to learn pharmacology, and is the only one that provides CRF and early phase trial experience and training.

Alder Hey supports and hosts the national training programme by hosting a study day. sits on both the Medicines and Healthcare product Regulatory Agency (MHRA) pharmacovigilance and paediatric expert advisory groups.

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.

0151 252 5744

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.

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