The document provides an update on efforts to increase clinical trial capacity for Duchenne Muscular Dystrophy (DMD) since a workshop held in July 2015. Patient organizations have provided funding for priority staff positions identified by clinical trial sites. A pump priming model is being explored to allow charities to recoup funding used to support staff upfront. Plans are also underway to develop a DMD clinical trials hub based on the successful Trial Acceleration Programme in Birmingham, to further aid in accelerating DMD clinical trials. Next steps include consolidating the hub proposal and securing funding to advance the model quickly.

1. Newcastle Plan Update December 18th
2015
Kim Down and Katie Bushby

2. United Kingdom & Ireland Patient Organisation Participants:
Action Duchenne
Alex’s Wish
DMD Pathfinders
Duchenne Ireland
Duchenne Now
Harrison’s Fund
Joining Jack
Join our Boys Trust
Muscular Dystrophy UK
The Duchenne Children’s Trust
The Duchenne Family Support Group
The Duchenne Research Fund
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3. UPDATE
PATIENT ORGANISATIONS FUNDING URGENT NEEDS
There have been significant achievements in putting in place the means to
increase clinical trial capacity for Duchenne Muscular Dystrophy since the
United Kingdom National Workshop on Clinical Trial Capacity was held on
the 10th
of July. The Newcastle Plan which was the output of the meeting
addressed short, medium and long term priorities to increase clinical trial
capacity for DMD which was fully embraced by the patient organisations
and representatives of the National Institute for Health Research (NIHR).
We just wanted to update everyone on what has been achieved since that
meeting was held.
In August a questionnaire was sent to sites currently undertaking the
largest number of DMD clinical trials asking them to identify the posts that
they felt were urgently needed in the short term. This included an outline
budget and a justification for the posts as well as information about
matched funding etc. MDUK also set in place a broader audit of trial
capacity across a larger range of sites and neuromuscular diseases. They
have also set in place the mechanism to enhance funding for the North Star
Clinical network
A meeting was then held on the 28th of September attended by the patient
organisations with the aim of progressing the one year actions from the
Newcastle Plan. Specifically for that meeting the Patient Organisations
wanted to ensure that the sites identified had the staff immediately needed
so that no further clinical trials were turned away from those sites.
The patient organisations agreed at that meeting that they would make
funding available for the sites identified and an application for funding the
prioritised posts at those sites was sent out. Funding decisions have
subsequently been made by the patient organisations based on those
applications and site visits made to the applicants and the results of the
MDUK audit.
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4. NATIONAL INSTITUTE OF HEALTH RESEARCH AND PUMP PRIMING PLANS
The generous funding allocated to sites has been offered regardless of the
pump priming model suggested by William Van't Hoff (Consultant
Paediatric Nephrologist, Head of Clinical Research Facility, NIHR CRN:
National Specialty Lead for Children, North Thames LCRN: Clinical Research
and Specialty Lead Children) which may allow charities to recoup and
reinvest the money that they put into funding staff up front for trials.
William attended the meeting and shared his proposal on charities pump
priming posts with the Patient Organisations in attendance.
A further meeting was held on October 19th
involving William, Katie Bushby,
Gillian Chater, Assistant Finance Director – Research and Development (The
Newcastle upon Tyne Hospitals NHS Foundation Trust), Stephanie
Blacklock, RM & G Manager (The Newcastle upon Tyne Hospitals NHS
Foundation Trust), Matthew Peak, Director of Research Clinical Lead NIHR
CRN: Children, North West Coast Co-Director: Alder Hey NIHR CRF for
Experimental Medicine, Becky Davis, Clinical Trials Coordinator (The John
Walton Muscular Dystrophy Research Centre, Newcastle University) and
Michela Guglieri, Research Fellow/Honorary Consultant (The John Walton
Muscular Dystrophy Research Centre, Newcastle University). The purpose
of the discussion was to progress and brainstorm the proposal for charities
and NIHR/CRN/Trust resources to share the burden of getting specialised
input to trial set up and support. The various Trusts involved now
understand the urgency of the issue of trial capacity and lack of posts and
are considering all possible avenues to overcoming the problem, including
the pump priming proposal.
A subsequent teleconference including the Patient Organisation
Representatives (Nic Bungay from Muscular Dystrophy UK, Emily Crossley
from Duchenne Children's Trust and Alex Johnson from Joining Jack) took
place on the 3rd
of December. This teleconference highlighted the need for
the Trusts and the NIHR to look at a sustainable model for keeping these
posts in the longer term. The pump priming proposal is being taken
forward in the first instance with the GOSH charity and some legal aspects
as well as practical financial aspects need to be worked out. The outcome
of this process will be of huge interest both to charities and to sites and it is
a great opportunity to ensure clinical trials are adequately resourced into
the future.
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5. CLINICAL RESEARCH HUB
Development of a clinical research hub to aid in accelerating clinical trials in
DMD is also very much underway. Emily Crossley, Alex Johnson, Kim Down
and Gillian Kenyon (a Clinical Trial Coordinator from Newcastle) visited
Birmingham to look at the Trial Acceleration Programme (TAP for
Leukaemia & Lymphoma). We met with Shamyla Siddique (the Trial
Manager and Team Leader) at Queen Elizabeth hospital to discuss the Trial
Acceleration Programme from Bloodwise (Leukaemia & Lymphoma
Research). They received core funding for a hub and spoke model for
clinical trials and have dedicated TAP staff in 13 clinical trial centres around
the UK. Their focus is on non-commercial trials led by clinicians in the sites
(mostly for repurposing low risk drugs already available) so slightly different
from the model needed for DMD. However, several good lessons were
taken away from their experiences. The first was the need to ensure that
there is a champion for DMD clinical trials at each of the sites. The second
was to ensure that sure that staff understand their link to the hub. The
third was the importance of engaging each of the sites.
We are currently working on a proposal for a DMD Clinical Trial Hub on the
back of that meeting, involving the resources of the MRC Centre at London
and Newcastle including TREAT-NMD and the North Star Network, and in
close collaboration with representatives from the NIHR to ensure that the
hub is successful.
The aims of our hub will be:
• To ensure that all patients with DMD, including children and adults,
have access to clinical research opportunities.
• To work with sites to ensure that they have core support to
undertake DMD clinical trials.
• Coordination of industry enquiries.
• To work with industry to ensure that they are aware of site
capabilities (site feasibility) through use of the Care and Trial Site
Registry (CTSR).
• Potential to link with registry and natural history data sets.
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6. • To integrate with the North Star network in order to optimise the
offering for patients and industry.
• To act as a training resource and hands on help-line for sites getting
up and running.
 To organise training and certification (e.g. in outcome
measures)
NEXT STEPS
The next steps will be to consolidate the planning of the hub and a post will
also be funded by Duchenne Children’s Trust to take the model of the hub
forward quickly. There has been great engagement and collaboration so far
with this process and hopefully this will continue into 2016. The other
element which is crucial to the idea of a hub going ahead is the integration
of ongoing initiatives like the MRC Centre, North Star Network and TREAT-
NMD.
So we would like to say a huge thank you to everyone who has been
involved both in the workshop itself and in all the work that has gone on
subsequently.
As Emily Crossley stated in the workshop way back in July:
“History is made in small moments of time – opportunities
grasped that can change the landscape of DMD for this
and future generations of children diagnosed with DMD
for the better. Let’s make this one of those moments.”
Emily Crossley, Founder and Director of Duchenne
Children’s Trust and mother of Eli, who has DMD.
We hope you agree that we have made the most of the opportunities that
the workshop offered and are assured that we will continue to do whatever
we can to ensure that clinical trial capacity is not a barrier to anyone who
wishes to take part in a DMD clinical trial in the future.
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