DMD Hub Central Recruitment Pilot Project

Information for participants

The DMD Hub Central Recruitment Pilot Project is looking to create a national contact list of children and adults with DMD who are interested in participating research studies.

Over the past 10 years, there has been an increase in the number of DMD clinical trials and research studies  evaluating the effect of new interventions and treatments, better understanding the natural course of the disease, and exploring new markers to measure disease progression or treatment effect. Due to the complexity of the disease and of setting up clinical sites to conduct them, these studies are often run in a limited number of centres in the UK. Inevitably this creates some challenges to both clinicians and patients and families.

The objective of the DMD Hub Central Recruitment Pilot Project is to assess the benefit to patients and trial sites of maintaining a centrally coordinated national recruitment contact list of children and adults with DMD who are interested in participating in DMD research studies.

People with a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) and who are interested in participating in research studies in DMD that are taking place in the UK. Children (under 16 years) must be registered by their parent or guardian. This study is only open to people who live in the UK.

We ask for your personal details, such as name, address, date of birth, NHS number and where you are treated for your DMD. We also ask about your genetic diagnosis, motor function, wheelchair use, medication taken and preferences for participation in research studies (including type of study and travel preferences). You will be asked to update this information every six months.

The DMD Hub Central Recruitment Pilot Project is looked after by Dr Michela Guglieri at Newcastle University.

About the DMD Hub Central Recruitment Pilot Project

Here you will find out more information about why we have a registry and how your data will be used.

What is the DMD Hub Central Recruitment Pilot Project?

The DMD Hub Central Recruitment Pilot Project collects information about people with DMD, who are interested in participating in research studies. The objective of the database is to facilitate fair and equitable access to clinical research, by supporting trial sites in identifying potentially eligible participants to take part in DMD research studies in the UK.

Since launching on 31 March 2022, the CRD has been used by DMD Hub trial sites to support their patient recruitment.

How do I benefit from registering?

Although one of the main objectives of this project is to facilitate recruitment in research studies for people living with Duchenne Muscular Dystrophy regardless of their geographical location, participation will not guarantee that somebody will be recruited (or approached to be recruited) in a study. Recruitment in a research study will depend on national recruitment targets, eligibility criteria and will remain a responsibility of the Principal Investigator for the study at each clinical trial site.

If a site is looking for potential eligible participants for a specific research study, a Health Care Professional at the site will be able to contact the DMD Hub to identify potentially eligible people based upon the information provided to the DMD Hub Central Recruitment Pilot Project. Potential study participants will be identified by the DMD Hub based on the information provided as part of the pilot study. Information provided by participants will be passed on to the Health Care Professional at the trial site for them to contact the individual and their local neuromuscular specialist to discuss the specific trial and potential recruitment. All people identified as potentially eligible for a particular study will then be assessed against the study specific inclusion and exclusion criteria to confirm eligibility. As sites often have limits on the number of people that they can recruit to a research study, any people registered on the DMD Hub Recruitment Pilot Project who match the recruitment criteria will be selected at random, to ensure a fair process for all.

I want to be involved in a research study (e.g., clinical trial or natural history study). If I register, is this guaranteed?

There is no guarantee that registering your details will mean you will be automatically approached to take part in a research study. Health Care Professionals looking to recruit people to a study will have the opportunity to review the details you have given and if the study appears to be suitable for you, they may contact you to discuss potential recruitment. All people contacted regarding a particular study will then be assessed in greater detail and at this stage it may be clear that other developments in your health or details not recorded on the DMD Hub Central Recruitment Pilot Project mean that the study is not a suitable one for you.

Who can register?

People with a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) and who are interested in participating in research studies in DMD that are taking place in the UK. Children (under 16 years) must be registered by their parent or guardian. This study is only open to people who live in the UK.

How do you register?

Registration involves completing an online questionnaire; you can view the questions you will be asked.

Are my details safe?

All information we receive from you will be treated confidentially. The information that you enter into The DMD Hub Central Recruitment Pilot Project online platform about yourself will be stored on a secure server located in the UK. Only members of the DMD Hub Central Recruitment Project team will be given specific permission and will be allowed to look at this information. If we publish any research or other documents based on information from this project, this will not identify you by name.

A key aspect of the DMD Hub Central Recruitment Pilot Project is that we are able to share your information with clinical trial sites within the UK, in order to help with recruitment to DMD studies that you have notified us that you are interested in. In order to do this, we will be required to share your information (including your personal information and information about your condition) with these trial sites. To do this we will verify the trial sites requesting to utilise the DMD Hub Central Recruitment Pilot Project and then use a secure file drop off service between the University and a trial site. You will have the opportunity to give your permission for sharing your data in this way on the participant consent form.

If you would like more information about how we manage personal data more generally, including your rights under law, and the contact details of the University’s Data Protection Officer, please see our website: http://www.ncl.ac.uk/data.protection/

Who looks after my data?

The DMD Hub Central Recruitment Pilot Project is looked after by Dr Michela Guglieri at Newcastle University. The DMD Hub Central Recruitment Pilot Project is governed by a study Steering Committee. Your data will be stored securely, and no unauthorised persons will be able to gain access to any information about your child.

Who has allowed this data to be collected?

This study has been reviewed and approved by the relevant ethics committee at Newcastle University, to ensure we are not doing anything harmful to you or your data in this project.

Participant information sheets