Clinical Research

With almost 20 years of experience in delivering clinical trials, the John Walton Muscular Dystrophy Research Centre (JWMDRC) is an internationally recognised leader in neuromuscular clinical research.

Aligned with the JWMDRC mission, the clinical research team delivers high standard clinical research project, including observational and natural history studies, interventional trials with advanced therapies, and investigational drugs and devices. We aspire to advance in the knowledge of NMDs, outcome measures and treatment for patients offering the opportunity to participate in research to as many patients as possible.

The Clinical Research team includes neuromuscular clinicians, expert physiotherapists, research nurses, project managers, clinical trial coordinators, data managers and clinical trial and project assistants.

We have extensive experience and expertise in setting up and delivering academic and commercial clinical trials, from Phase I to Phase III studies, as well as developing clinical research projects aiming to accelerate translational research in neuromuscular diseases and provide access to innovative and advance therapies to patients with a broad range of neuromuscular diseases, across paediatric and adult patient populations.

Over the past 10 years we have delivered over 30 natural history/observational studies, nearly 40 clinical trials with new investigational drugs, and 6 advanced therapy trials.

Our priorities are clinical research studies focused on advanced therapies and innovative approaches, academic collaborations to advance care, translational research and treatment options targeting patient populations or areas of specific unmet needs.

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Clinical Research Studies

Based on our clinical trial and neuromuscular expertise, we are the national leading site for several academic and commercial clinical research studies and trials. This includes the first gene therapy studies in Duchenne Muscular Dystrophy and Pompe disease coming to the UK.

Clinical studies and trials run at the JWMDRC

Exon skipping approaches:

  • DELIVER:
    Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
  • ESSENCE:
    Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

Gene therapy approaches:

Other interventional trials:

Natural history and observational studies:

Clinical Research projects led by the JWMDRC

We have been leading national and international clinical research projects and collaborations aiming to advance diagnosis, standards of care development, outcome measures and drug development for patients with neuromuscular diseases including:

Adult SMA Reach

Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular Atrophy (SMA).

AFO-CCD

This pilot study aimed to identify the sample size required to power a larger scale study to compare AFOs and CCDs in managing ankle range of movement and function in boys with DMD. It will also explore adherence and patient satisfaction for the two devices.

BIND

The BIND study aims to improve the learning and behavioural challenges in individuals with DMD or BMD.

FOR-DMD

The FOR DMD study was an international, double blind, controlled study which has been established to assess the relative effectiveness and adverse event profiles of the 3 most frequently prescribed corticosteroid regimes in DMD.

ICGNMD

This centre aims to further neuromuscular research in partnership by building diverse genetically diagnosed international cohorts, increasing access to genomic medicine and clinical trials and importantly investing in training the next generation of NMD researchers.

COS2

The International Clinical Outcome Study for Dysferlinopathy (COS) has been running since September 2012 with the generous support of the Jain Foundation.

Muscle MRI in Pompe

This study aims to address the critical need for early detection and treatment monitoring in LOPD by employing a new MRI technique, Carbon Spectroscopy, which detects the Carbon present in the glycogen of the muscles and therefore allows us to measure glycogen levels in patients.

Myo-Guide

Myo-Guide is an artificial intelligence (AI) based algorithm that supports the diagnosis of patients with neuromuscular diseases through an analysis of the pattern of fat replacement on muscle MRI.

Participation in Clinical Trials

The Patient Journey

We are involved in clinical trials and research projects in different neuromuscular diseases across the full life span, from paediatric to adulthood.

Equitable/fair access to clinical research is our priority. Patients are identified through our clinic list but we also receive referrals from other clinical centres and collaborate with patient registries and databases to ensure that access to clinical trials is not limited to patients follow up at our centre.

I am always treated with respect, I feel part of the team
It's great to see people who understand totally, have empathy but still positive. Great to be able to discuss any concerns without seeming silly. Invaluable to have the link with my doctors to get updates and referrals. Cannot fault or undervalue the importance of how the research has helped me and my family
The whole experience has been life changing, from the care given to the people I have met and kept contact with. I feel far more connected to those researching my condition and a deep understanding of how this is progressing

Locations

Our research study and clinical trial activity is delivered at specialist centres within the Newcastle University NHS Trust at locations across Newcastle City Centre.

The Clinical Research Facility (CRF)

This purpose-built facility is located in the Leazes Wing of the Royal Victoria Infirmary in the centre of Newcastle Upon Tyne. The highly trained and dedicated team actively deliver all stages of cutting-edge and complex clinical research in both adults and children. The hospital is accessible by road, bus and the Tyne & Wear metro service and has on-site parking facilities.

Great North Children’s Hospital (GNCH)

The GNCH is located within the Royal Victoria Infirmary in the centre of Newcastle Upon-Tyne. It is one of only 14 major children’s centres in the UK and has the most extensive range of children’s services anywhere outside of London.  It has specialist wards, operating theatres, outpatients and day case suites tailored for children and plays a vital role in the delivery of paediatric clinical trials and safety surveillance.

Newcastle Magnetic resonance Centre (NMRC)

The Newcastle Magnetic Resonance Centre (Newcastle Magnetic Resonance Centre - Newcastle Magnetic Resonance Centre - Newcastle University (ncl.ac.uk)) is a world leading centre for research in Neuromuscular Diseases.

Clinical Research conducted in collaboration between the John Walton Muscular Dystrophy Research Centre and the Newcastle Magnetic Resonance (NMR) Centre includes:

  • Creation and delivery of the largest longitudinal imaging study of Limb Girdle Muscular Dystrophies (link to COS page).

  • Development and application of novel approaches to investigate muscle pathology and metabolism with multinuclear magnetic resonance, including 13C measurements of energy storage, 31P measurements of energy handling, and 23Na measurements of ion homeostasis.

  • Development and application of advanced scan acceleration methods for higher scan quality and shorter duration scans in quantitative MRI.

  • Leadership of International Collaborations to including the Myo-MRI and Bioimage Projects.  

  • MRI Specification and Analysis Services for Clinical Trials using quantitative MRI as an endpoint.

For further details Muscle - Newcastle Magnetic Resonance Centre - Newcastle University (ncl.ac.uk)

MYO-MRI Conference – Imaging in Neuromuscular Disease Conference

Meet the Clinical Research team

Photo by Scott Graham on Unsplash

The Clinical Trial Coordination team

EXPERTISE:

  • Set up of clinical research studies, ranging from observational studies through the clinical trials of investigational medicinal products to advanced therapies in a broad spectrum of neuromuscular diseases

  • Support for advanced therapy studies

  • Set up and delivery of international multi-centre clinical trials

  • Grant applications support

  • Development of and support for the implementation of national guidance on costing for clinical trial in neuromuscular diseases

Dan Riseborough

Clinical Trial Coordinator

Clinical Research

Ellie Drummond

Clinical Trial Coordinator

Clinical Research

Georgina Boyle

Clinical Trial Coordinator

Clinical Research

Katie McConville

Study Support Assistant

Clinical Research

The Research Nurses

EXPERTISE:

  • Provide ongoing support and information to participants on their journey through an academic research study or clinical trial.

  • As well as delivering the nursing activities required for a study or trial, they will collect, record and verify all study data with a high degree of accuracy to ensure high quality research.

  • Act in the best interests of the research participants and will oversee and maintain effective communication between the research team and the study participants, their relatives and the multidisciplinary team involved in their care.

Laura Behar

Research Nurse

Clinical Research

Sara Wilkinson

Research Nurse

Clinical Research

The Physiotherapy team

EXPERTISE:

  • Leaders in developing clinical outcome assessments used in NMD trials and clinics internationally including the North Star Ambulatory Assessment, Performance of Upper limb 2.0 and North Star Assessment for limb girdle type muscular dystrophies

  • Advise on trial design and outcome measure selection in natural history studies and clinical trials

  • Collect primary and secondary endpoints across paediatric and adult natural history studies and interventional trials

Dionne Moat

Neuromuscular Research Physiotherapist

Clinical Care

Emma Grover

Physiotherapist

Clinical Care
Clinical Research

Emma Robinson

Physiotherapist

Clinical Care
Clinical Research

Jassi Michell-Sodhi

Clinical Specialist Physiotherapist

Clinical Care

Karen Wong

Neuromuscular Research Physiotherapist

Clinical Care

Meredith James

Clinical Specialist Research Physiotherapist

Clinical Care
Clinical Research

Michelle McCallum

Physiotherapy Technical Instructor

Clinical Care

Robert Muni Lofra

Consultant Physiotherapist

Clinical Care
Clinical Research

The Data Management team

EXPERTISE:

  • Maintenance of national and international patient databases and post-marketing registries

  • Data quality

  • Implement innovative methods to monitor large data sets

  • Data quality

  • Implement innovative methods to monitor large data sets

Elena Karkkainen

Data Manager

Clinical Research

Jamie Mitchell

Data Manager

Clinical Research

Jess Page

SMA Dataset Project Coordinator

Clinical Research

Julie Bohill

Registry Project Manager

Networking

PhD Students

Our PhD students are key to our clinical research. We have a long history of supervising successful Clinical PhD students across neuromuscular disease.

Current PhD students include:
  • Carla Bolaño Diaz

    "The role of magnetic resonance imaging in the study of dysferlinopathies"
  • Jose Verdú-Díaz

    "Design and implementation of a fully automatic pipeline for muscle MRI segmentation, fat fraction, quantification and neuromuscular disease diagnostics"
  • Marianela Schiava

    "The results of the FOR DMD study and the impact of early corticosteroids in DMD: implications for clinical care and clinical trial design"
  • Pietro Riguzzi

    University of Padova: "Translational Specialistic Medicine: Dystrophinopathies - deep phenotyping"

Collaborations

We have a well-established framework working together with the other strands at the JWMDRC such as the Clinical Team, Basic Research Team, Networking Team as well as the The John Walton Muscular Dystrophy Research Centre Biobank. We have strong collaborations with both academic partners, pharmaceutical companies, national and international networks as well as disease registries, patient organisations and advocacy groups.

The Clinical Research Facility

We deliver most of our studies at the NIHR Clinical Research Facility (CRF) at the Royal Victoria Infirmary (RVI) in Newcastle. The CRF provides a team of dedicated research nurses, data managers and trial coordinators with the experience of setting up and delivering all phases of clinical trials in accordance with Good Clinical Practice Standards.

The Royal Victoria Infirmary & Great North Children Hospital

The Great North Children Hospital (GNCH) multi-disciplinary team support our clinical and clinical research activities and cover different areas of expertise including paediatric neurology, respiratory medicine, cardiology, radiology and surgery.

Within the Royal Victoria Infirmary (RVI) and GNCH, we have established the paediatric and adult gene therapy safety task force which includes expert clinicians to cover multi-disciplinary areas of care (neurology, cardiology, immunology, haematology, nephrology, hepatology, respiratory medicine, pharmacy, intensive and emergency care) to support the delivery of gene therapy trials as well as approved advanced therapies.

DMD Hub

We are the coordinating centre for the DMD Hub and one of the leading DMD Hub trial site. Our team members are members of the DMD Hub networks (clinical trial coordinators, research nurses, Principal Investigators, young investigators) and actively contribute to the development of resources to support other clinical trial sites and promoting harmonisation of clinical trial conduct in the UK.

Biobank

Dan Cox working in the lab

An integral part of our clinical and clinical research activities is the collection of samples (blood, urine, saliva, muscle biopsy, skin biopsy, etc.) for the JWMDRC Research Biobank to be used in research projects across the world.

The Northern Alliance Advanced Therapies Treatment Centre

We are part of The Northern Alliance Advanced Therapies Treatment Centre (NAATTC), a consortium of twenty industry, NHS and academic organisations led by Newcastle Hospitals and Scottish National Blood Transfusion Service (SNBTS). The purpose of the NATTC is to develop the systems and infrastructure required to support the delivery of cell and gene therapies with the ultimate aim of increasing patient access to advanced therapy medicinal products (ATMPs) on a national level.

Biomedical Research Centre

We are part of the National Institute for Health and Care Research (NIHR) Newcastle Biomedical Research Centre (BRC) which brings together world-leading researchers and clinicians to turn new scientific breakthroughs into treatments for patients.

Our centre is a co-lead on the Neuromuscular Disease, Rare Diseases and Mitochondrial Dysfunction theme. Our aim is to systematically develop and evaluate novel approaches to diagnosing, monitoring and treating neuromuscular disease (NMD) and mitochondrial dysfunction, and to extend the application of these technologies across the broader spectrum of rare diseases.

Muscle Immunoanalysis Unit

Our team works in close collaboration with the Muscle Immunoanalysis Unit (MIU) with regards to the sample processing for clinical trials.

Northstar Clinical Network

We are proud to be active collaborators in the North Star Clinical Network.

The North Star Project was set up in 2003 to help drive improvements in services and set national standards of care for children living with Duchenne Muscular Dystrophy (DMD). The overall aim is to optimise the care of young patients with Duchenne Muscular Dystrophy (DMD) by achieving and practising consensus on best clinical management, with agreed assessment and treatment protocols, irrespective of the treating clinical centre.

The project aims to:

  • Provide best support to patients by agreeing protocols for assessment and best practice treatment options

  • Assist clinicians working with muscle disease by developing a national clinical network and providing a discussion forum to promote best patient care

  • Standardise and optimise steroid therapy in ambulant children with DMD throughout the UK

  • Ensure a standard assessment protocol for newly diagnosed children and those due to start corticosteroid treatment

Integrating research in clinical care

As a translational research centre the insights we gain from both clinical practice and research activity continuously feed into one another ensuring we maintain strong evidence based practice. Within the clinical strand (link) our clinicians are continuously striving to improve patient care and experiences through developing, implementing and offering new research to our patients.

We currently deliver several research studies within the clinical service including the Creatine D3 trial (link), SMA Adult (link) and the AFO-CCD study (static night ankle foot orthosis vs contracture control devices) (link). We also recruit and collect samples for The John Walton Muscular Dystrophy Research Centre Biobank (link) of rare, inherited neuromuscular and neurodegenerative diseases.

Working with patient organisations

We work extensively in partnership with patient organisations nationally and internationally to drive research, improve care and engage with the neuromuscular community. We are always pleased to discuss opportunities for collaboration and discuss your priorities.


“We have been working closely with the team at the John Walton Muscular Dystrophy Research Centre since 2012 on the International Clinical Outcome Study for Dysferlinopathy.

Hundreds of genetically confirmed dysferlinopathy patients have participated globally in COS and COS2 as  well as over 135 clinicals and medical staff.  A total of 1248 clinic visits were completed world wide. The high level of patient retention is an important measure of their success.“

Sarah Shira Emmons VP Patient Affair, The Jain Foundation


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