The Registered Clinical Trials Unit (CTU) Network is proud to support International Clinical Trials Day (20 May).

The date marks an important moment back in 1747, when Royal Navy surgeon James Lind set out to better understand scurvy. His study was small, just 12 men over six days, but it showed that citrus fruits could prevent the disease, and is often seen as one of the first clinical trials.

Now, the anniversary is used around the world to highlight the importance of clinical trials and recognise the people who make them happen. Registered CTUs across the Network take the opportunity to share their work in schools, hospitals, and universities around the country or hold internal events to meet and thank participants and funders. You can read about a small fraction of these activities below. To find out about more about what CTUs do click here.

If you’re interested in taking part in health and care research across the UK, the Be Part of Research website can help you explore ongoing clinical trials by condition or location.

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Staff from the NHS Blood and Transplant Clinical Trials Unit will be running a mini randomised controlled trial at the NHSBT site where our statisticians are based. We aim to test the hypothesis that paper aeroplanes made from A7 paper will fly further than those made from A6 paper. Participants (NHSBT colleagues) will be randomly allocated a paper size, build a plane, and test its flight distance. To explore the reliability of self‑reported data, participants will rate their paper‑aeroplane‑making skills beforehand. We will share the results with colleagues at the site.

Find out more about the work of the NHS Blood and Transplant Clinical Trials Unit here.

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To mark ICTD, staff from Keele Clinical Trials Unit will be taking part in a creative research methods showcase at our university (19 May 2026), demonstrating the participant experience in a randomised controlled trial. On the day itself, we’ll be collaborating with MPFT NHS Trust on their research bus in Newcastle-under-Lyme town centre, engaging the public and promoting opportunities to take part and get involved in health research. We will also be showcasing our mock trial at an event at Queen Elizabeth Hospital, Birmingham, highlighting the value of high-quality clinical trials.

Find out more about the work of the Keele Clinical Trials Unit here.

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On 20 May 2026, leading researchers from the Lancashire Clinical Trials Unit will be presenting at the University of Lancashire’s annual Research and Knowledge Exchange Festival.  As part of a day dedicated to health and wellbeing, they will be discussing some of their ground-breaking clinical trials and innovative methodological studies that are driving the future of health research.

Find out more about the work of the Lancashire Clinical Trials Unit here.

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To help us celebrate International Clinical Trials Day, Leeds Clinical Trials Research Unit will be getting a visit from the family of former Leeds United footballer, World Cup winner and PFA Player of the Year Norman Hunter. Norman spent seven years receiving care in Leeds following his diagnosis with Chronic Lymphocytic Leukaemia (CLL) until his death in 2020.

His wife Sue has been raising money ever since and some of the over £170,000 raised has gone to help fund lab work related to the FLAIR and STATIC trials. In the past the family have visited the labs to meet the team and learn about the impact of their fundraising but this year they will be visiting LICTR to see where the trials are coordinated, meet the team and hear more about what is involved in running clinical trials.

Find out more about the work of the Leeds Clinical Trials Research Unit here.

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The UK Clinical Research Collaboration (UKCRC) Clinical Trials Unit (CTU) Network was created in 2007 in response to a shared recognition among researchers and funders that the UK needed greater capacity and consistency in the design and delivery of high‑quality academic clinical trials. Established alongside the growth of the National Institute for Health and Care Research (NIHR), the Network provides a coordinated, nationally accessible system that connects CTUs with funders, regulators and other key stakeholders, while supporting best practice through shared expertise and independent review.

Central to this mission is the Network’s registration process. Registration serves as a visible and trusted benchmark of quality for non‑commercial academic clinical trials, enabling funders, collaborators and participants to identify CTUs with the expertise, infrastructure and systems required to deliver robust and ethical research. In our current rapidly evolving trials landscape, clear and credible standards remain as essential as ever.

This review placed strong emphasis on engagement with a broad range of stakeholders, including CTU staff, research participants, funders and regulators. Their input was vital in testing whether the existing criteria continued to reflect quality and competence, and in identifying opportunities to future‑proof the process.

Stakeholders were invited to review how clear and effective the current criteria are, covering areas such as portfolio, staff expertise, infrastructure, quality assurance, information systems, research strategy, national standards (e.g. PPI&E), and statistics. Patient and public contributors took part in several workshops, allowing more detailed feedback to be gathered. Views were also sought on renewal timelines for existing members and on the approach to open calls for new CTUs. All feedback was considered by the Criteria Review Group, set up to oversee the process, as well as by the Executive Group and the independent International Registration Review Committee.

Overall, respondents supported the current approach, with no major structural changes recommended. However, several thoughtful refinements were agreed. These included clarifying expectations around the completion and publication of randomised controlled trials, broadening the way CTUs can evidence contribution beyond named authorship, strengthening PPI&E criteria, introducing an Equality, Diversity and Inclusion element, updating language in the Information Systems and Statistics sections to reflect current practice, and making the frequency of open calls clearer and more predictable.

This review has reaffirmed the strength of the existing registration framework and highlighted its continued value. By maintaining a clear marker of excellence across all stages of non‑commercial trials, the CTU Network continues to play a vital role in supporting high‑quality research and safeguarding the UK’s global reputation for clinical trials excellence.

Of the registration process, Otavio Berwanger, Chair of the International Registration Review Committee said:

“Being part of the UK Registered Clinical Trials Unit (CTUs) Network isn’t just about recognition; it genuinely strengthens how CTUs design and deliver trials. The registration criteria help ensure the right expertise, processes, and governance are in place to run studies that are both robust and reliable, ultimately improving the quality of the evidence we generate.”

To view the updated criteria, and the associated guidance, please click here.

The UK Clinical Trials Units (CTU) Network has published new guidance to support researchers considering running clinical trials beyond the UK, offering practical advice for navigating the complexities of international collaboration.

The document takes the form of a “Things to Consider” guide, reflecting the wide variation in experiences across UK-based CTUs involved in international studies. It aims to provide a flexible and practical list of important areas for trial teams to bear in mind at the planning stage of multi-national research.

Supporting global trial delivery

Interest in conducting clinical trials across borders has grown significantly in recent years, driven by the need for larger, more diverse participant populations and the opportunity to address global health challenges. However, running trials outside the UK introduces a range of operational, regulatory, and cultural considerations.

The CTU Network highlights that experiences of international working differ considerably depending on the country, the trial design, and the timing of the work. As a result, producing rigid, one-size-fits-all guidance was seen as neither feasible nor helpful.

A practical approach

The new document draws on the shared experience of the UK CTU community to outline key areas that research teams should consider when planning international activity. These include costing and funding arrangements, understanding cultural and local contexts, navigating contracts and legal frameworks, and managing ethics and regulatory approvals across different jurisdictions.

The guidance also provides signposts to a range of existing resources developed by other organisations, helping researchers access more detailed or country-specific information where needed.

Developed through shared expertise

The guidance has been developed by a group of senior trialists from across the UK CTU Network, bringing together experience of designing and delivering trials in a wide range of international settings. This collaborative approach reflects the Network’s commitment to sharing knowledge and promoting best practice across registered CTUs, ensuring that collective learning benefits the wider trial community.

Learning from shared experience

A key message is the value of learning from colleagues. The CTU Network encourages researchers to connect with teams who have direct experience of working in particular countries, recognising the practical insights this can provide when navigating unfamiliar systems.

Researchers interested in speaking with colleagues from other CTUs can contact the Network, which will seek to connect them with individuals who have relevant international experience.

A living resource

By taking a principles-based approach, the Network hopes that the document will remain useful over time in a rapidly evolving landscape for global clinical research. Rather than attempting to keep pace with changing regulations and processes in every country, it provides a framework for thinking through key issues and making informed decisions.

The guidance is intended for anyone involved in trial design and delivery, including trial managers, statisticians, and chief investigators considering international expansion.

You can download the guidance here.

A personal reflection on dementia, research, and advocacy by Simon Denegri OBE, Chair of the CTU Network Executive Group, Chair of Dementia Disco, and Chair of Sense About Science.

My story with dementia begins as a teenager in the late eighties. My grandmother was staying with us to give my aunt, her carer, a break. My parents had gone out after helping my grandmother to bed. There was a loud crash from the bedroom next door to mine and I found her lying on the floor, confused and disorientated. To this day I remember the frailty of her body in my arms and her vulnerability as I placed her back in bed. It was the moment when dementia invaded my young world with shocking reality.

That is the beginning of my story with dementia and I am choosing to tell it – as I hope so many others will – as part of Dementia Action Week and the Alzheimer’s Society’s ‘Forget-Me-Not campaign this week (18 – 25 May).

My personal experience of dementia has had a strong bearing on my career from the early nineties when I was the Alzheimer’s first parliamentary officer and public awareness of the disease was only just stirring. Right up to the present day and my role as Chair of Dementia Disco, a small but rapidly growing charity which aims to use music to inspire memories, conversation and friendships in and between people living with dementia.

Dementia was my introduction to research and to public involvement. The Alzheimer’s Society was the first charity to pioneer in the nineties involving patients and carers in decisions about research funding. Seeing this unfold had a formative influence on me: public involvement is about bringing social justice and democracy to one of the most important human endeavours – science – and making it more relevant to society. I am lucky to have had the chance to make this happen more widely in the UK and globally with some amazing patient partners, clinicians and researchers.

It is therefore a happy coincidence that this week we also celebrate International Clinical Trials Day (ICTD) on 20 May. For some time ICTD has been used by NIHR, charities and members of the UKCRC CTU Network as well as many others to raise public awareness of the importance of clinical trials. More importantly to encourage people to volunteer to take part in studies. Hundreds of thousands of people are now signed up to NIHR’s ‘Be Part of Research’ matchmaking service which enables patients and researchers to connect about opportunities. But we need many more volunteers in research in the future and it is important that the Government has made a commitment to make this easier to do as part of its NHS 10-Year Plan.

The dementia community has again been a pioneer here. It is eleven years since Join Dementia Research was set up. Over 100,000 people have registered and hundreds of studies have recruited volunteers by using this service. It has undoubtedly been an important element in the exciting developments now taking place in UK dementia research. We may still have a long way to go before we cure dementia but research and improvements in treatments and care mean that many people can now live fuller lives for longer – perhaps even connect with others at a Dementia Disco.

In recent months there have been several public attitudes surveys showing that public trust in science remains high but is fragile. Also that they are often overwhelmed by the way in which science is communicated. But that telling stories enable them to connect with and see its importance to their life and that of their community.

In dementia, in clinical research, across science in general we must continue to tell stories which bring opportunity and hope to our fellow human beings. It was a pleasure to share my story and I hope you will pass it on. Better still please tell your own story.

Simon Denegri OBE

A newly published academic paper has shed light on how central data monitoring is currently undertaken across registered clinical trial units (CTUs) in the UK, highlighting both strengths in existing practice and areas where greater consistency could support trial quality.

What Is Central Data Monitoring?

Central data monitoring refers to a range of activities carried out by trial staff while a study is ongoing, with the aim of safeguarding data quality, integrity and reliability. Unlike on-site monitoring, these activities are performed remotely and often involve routine review of trial data, processes and performance indicators. While CDM is widely recognised as an essential component of good trial conduct, there is limited regulatory guidance on which activities should be undertaken or who should be responsible for them.

Findings and Recommendations

To better understand current practices, the research team distributed a structured survey to all UKCRC-registered CTUs on 25 April 2025. The survey asked respondents to indicate whether 16 defined central monitoring activities were carried out on all trials, some trials or not at all, and which staff roles were typically involved in delivering each activity.

The results show considerable variation across CTUs. Central data monitoring is commonly undertaken by statisticians, trial managers and data managers, alongside other trial team members depending on the activity. While some monitoring practices were widely and consistently applied, others were implemented more selectively, often influenced by trial design, risk profile or available resources.

Notably, the authors highlight the lack of detailed regulatory direction as a key factor contributing to this variability. In response, the paper proposes a core set of eight central data monitoring activities that the authors recommend should be considered for inclusion in all clinical trials, providing a potential baseline for more standardised practice.

The authors express their sincere thanks to all CTUs and colleagues who contributed to the survey, recognising the time and expertise required to share detailed information on internal processes.

Read the full paper here.

Find more guidance on monitoring issues here.

Dementia Action Week is an awareness campaign led by the Alzheimer’s Society, bringing people and organisations together to take action on dementia.

Dementia is the UK’s leading cause of death, and around one in three people born in the UK today will develop it during their lifetime.

Across the country, many Registered Clinical Trials Units (CTUs) are carrying out vital research into dementia, from prevention and treatment to better support for carers. Below, you can read about just a few of these important studies.

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SleepBoost: Could improving sleep help prevent Alzheimer’s dementia?

The SleepBoost study, led by Professor Coulthard and the Bristol Trials Centre at the University of Bristol, will focus on a phase of sleep known as slow-wave sleep, which plays a key role in removing harmful proteins from the brain. The study is currently in set up with an aim to begin recruitment later in the year.

The study aims to assess whether improving sleep helps prevent Alzheimer’s dementia. The study will specifically improve the quality of slow-wave (deep) sleep and measure the effect on Alzheimer’s markers in the blood. Recruitment will take place across 11 locations in the UK, with a focus on including South Asian communities who may be more affected by poor sleep. By using sleep recordings, brain activity monitoring, scans, blood and spinal fluid samples, and memory tests to measure impact, we hope our findings will lead to a better understanding of the links between sleep and Alzheimer’s. Our vision is a new class of tailored Alzheimer’s disease treatments based on a new mechanism of neurodegeneration.

Find out more here: https://www.bristol.ac.uk/news/2026/january/165-million-to-investigate-neurodegenerative-diseases.html

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Rare Dementia (RD) – TALK

BELIDE assesses the effectiveness of an online education course programme (“Better Living with non-memory led dementia”) in improving the psychological wellbeing of carers of people with rarer dementias. IWARF examines the effectiveness of a digital programme (“Improving Wellbeing Associated with Rare Familial dementias”) in improving the psychological wellbeing of people at risk of genetic dementia.

NWORTH CTU (https://www.bangor.ac.uk/nworth), which is a member of the UK Registered CTU Network, manages the data, statistical and methodological aspects of both the BELIDE and IWARF RCTs. Dr Joshua Stott is the Chief Investigator and the Trial Statistician is Dr Andrew Brand.

For further information about RD-TALK research programme, please visit: https://www.raredementiasupport.org/research/rd-talk/

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The CareCoach Study: Empowering Carers of People Living with Dementia

CareCoach is a support package that can be delivered remotely. This allowed the research team to recruit carers from across the UK remotely via social media and healthcare services. Advertising the study on social media proved to be a successful way to reach carers. So successful that 18% of participants (73 carers) joined the study through social media!

Managing the trial centrally at the Norwich CTU was key in ensuring data was high quality and strong recruitment. This streamlined approach helped the study meet its recruitment target (404 carers) on time within 12 months. In fact, CareCoach was one of the fastest recruiting trials funded by NIHR in 2025-2026 in the UK.

Strong interest from carers wanting to take part in the study highlights the need for more support for those caring for people living with dementia. Results are expected later this year, with plans to explore making CareCoach available to carers who need it most.

Read more about CareCoach: https://carecoachtimes.org/carecoach/

A participant shares their CareCoach experience: https://news.joindementiaresearch.nihr.ac.uk/how-dementia-research-is-helping-support-carers/

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“Being Kind to Ourselves” study – a full-scale Randomised Controlled Trial (RCT) evaluating Compassion Focused Therapy (CFT) for improving depression and anxiety in individuals with dementia

The feasibility phase data will serve as an internal pilot and will be incorporated into the final trial dataset. The full RCT requires a total sample of 304 participants. Importantly, 73 participants have already been successfully recruited and randomised during the feasibility phase. Therefore, the primary recruitment goal for this next phase is securing a further 231 participants to reach the required sample size. Recruitment is scheduled to commence in January 2026.

The trial is building upon the established infrastructure of the initial study. While 7 sites participated in the feasibility trial, some of which will continue with the RCT, complemented by the opening of new sites to accelerate participant enrolment.

The first time this model of funding has been used by NWORTH CTU and offers an exciting opportunity to optimise existing resources and accelerate the RCT timeline.

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Read a personal reflection on dementia, research, and advocacy by Simon Denegri OBE, Chair of the CTU Network Executive Group, Chair of Dementia Disco, and Chair of Sense About Science here.

The environmental impact of clinical trials

Climate change is the biggest health threat facing humanity. Yet healthcare, including clinical trials, contributes to the problem by producing 4-5% of global greenhouse gas emissions. Promoting greener, more sustainable practices in clinical research is essential to reducing the environmental impact of our work.

Developing a method to footprint clinical trials

As a first step towards lower carbon trial design, a method and guidance to carbon footprint publicly funded clinical trials was developed by UKCRC Registered CTU trialists based at the ICR-CTSU and the University of Liverpool. The initial guidance was piloted on two CRUK-funded, ICR-CTSU managed trials https://pubmed.ncbi.nlm.nih.gov/38267250/.

Collaboration with 7 more UKCRC Registered CTUs (Centre for Trials Research, Centre for Healthcare Randomised Trials, Edinburgh Clinical Trials Unit, Imperial Clinical Trials Unit, Newcastle Clinical Trials Unit, UCL Innovative Clinical Trials Unit (formerly MRC CTU at UCL), Liverpool Clinical Trials Centre) and 3 international CTUs (Cancer Trials Ireland, Trinity College Dublin and CTN Diabetes and Institute for Clinical Trials, and The George Institute for Global Health) further tested and refined the applicability and usability of the method developed.

Each CTU identified a trial from their portfolio and a member of their team who could perform the footprinting of the trial. The work was performed by a variety of team members from each CTU (trial managers, MSC students, PhD students, clinicians). The work took between 5 and 60 hours per trial depending on the complexity of the trial, and how familiar the person completing the carbon footprinting was with the trial itself. This was conducted alongside existing routine workload, without specific funding to support it.

The results showed that the carbon hotspots of these trials varies but there are some key themes starting to emerge in terms of where the majority of emissions are seen.  You can read more about this work here: What is the carbon footprint of academic clinical trials? A study of hotspots in 10 trials – PubMed.

Carbon footprinting drop‑in clinics

We now need to turn carbon footprinting into action and promote a paradigm shift to lower carbon trial design and delivery.

For more information, contact cict-icrctsu@icr.ac.uk or visit the MRC NIHR TMRP Greener Trials website

Building the evidence base

The accumulating data on publicly funded clinical trial footprints is being collated through the trials footprinted at the clinics. These data are critical to our knowledge base and to supporting research around lower carbon trial design.

Each UKCRC CTU agreeing to share footprinting data with the Greener Trials Toolkit is contributing towards the update and refinement of the guidance, which increases its applicability to a wider variety of future trialists. The more data we have, the faster and easier we can make the calculations for future users.

We’re delighted that more and more UKCRC CTUs are attending the clinics and sharing their data with us, most recently the Rapid Eczema Trials which are being delivered in collaboration with Nottingham CTU, and the STRATA trial delivered in collaboration with the Bristol Trials Centre and footprinted by  Bristol Medical School.

The data are also building an evidence base of where hotspots lie in trials in different therapeutic areas or using different designs and interventions. We need this information to facilitate research into mitigation strategies to reduce those hotspots, without compromising the quality of trials and integrity of trial data.

The Greener Trials Toolkit

To help speed up carbon footprinting calculations and collation of this critical data, Wellcome are also funding the development of an online, free to use, open science and fully referenced Greener Trials Toolkit.

The Toolkit, planned for release in 2026, will speed up carbon footprinting calculations and provide users with information on mitigation strategies for the hotspots identified. The Toolkit will also include a publicly accessible database of carbon emission factors and curate the data that users share with the Greener Trials Data Consortium.

Supporting sustainability across the CTU Network

The Network is also supporting CTUs in the consideration of sustainability. The UKCRC CTU Network Sustainability thematic group was recently conceived, and the group have held their first meeting and are excitedly planning the ways in which it can bring the CTU network together to share knowledge, best practice and practical experience of considering and implementing Greener Trials.

We’d like to express our huge thanks to the 10 UKCRC Registered Clinical Trial Units and affiliates who have already footprinted trials and shared their data with the Greener Trials Data Consortium and look forward to hearing from others who may be interested in considering the carbon footprint of their trials.

To get involved, please contact cict-icrctsu@icr.ac.uk.

The Network became a supporter of the Concordat for the Environmental Sustainability of Research and Innovation Practice in June 2024.  Learn more here.

The UKCRC Registered Clinical Trials Unit (CTU) Network is seeking a highly experienced public contributor with expertise in clinical trials to contribute to the strategic work of its Executive Group.

The successful candidate will have a wealth of experience, knowledge, and acumen within the field of non-commercial/academic clinical trials research, demonstrate an excellent understanding of best practice as well as current drivers for change.

As the collective voice of non-commercial and academic clinical trials units, the Network plays an important role in engaging with Government, funders, regulators, and patients to improve and strengthen clinical research in the UK. The Network is seen internationally as an exemplar for the development of national clinical trials support. It was established in 2007, and its members are committed to setting and sharing good practice and improving standards in clinical trials.

The CTU Network is funded by its members, and major funders have representation on the Executive Group, this includes; Cancer Research UK, NIHR, Arthritis UK, Chief Scientist Office Scotland, and Health and Social Care Research Wales.

The deadline for applications is 6pm on 29 May 2026.

Don’t miss your chance to register for the next ‘How to be a Great Chief Investigator for Clinical Trials’ workshop taking place in Nottingham on 5 May 2026.

The workshop is designed to equip researchers with the skills and knowledge necessary to excel as Chief Investigators (CIs), through:

–  Expert guidance from experienced CIs with a diverse range of backgrounds and specialties.
–  The opportunity to meet and gain insights from specialists at Registered CTUs, including statisticians and trial managers.
–  Interactive activities to develop practical understanding of the issues.
–  A collaborative learning environment.

You’ll also be able to begin building your own peer support network.

Previous workshops have been awarded 5 external (category 1) CPD credits by the Federation of the Royal College of Physicians, and an application for CPD accreditation is currently in progress for this event.

Find out more and register here

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What did previous attendees think of the How to be a Great Chief Investigator workshop?

Vital rare disease research is being undertaken by many Registered Clinical Trials Units (CTUs).

There are thousands of rare diseases that collectively affect millions of people in the UK. Because each condition itself is rare, it is challenging to do clinical trials to show whether new treatment approaches are safe and effective. Sadly, this means many rare diseases lack effective treatment with no research ongoing to give patients hope. Read more about the importance of research into rare diseases here.

Registered CTUs are helping to tackle these challenges by improving how rare disease research is designed and delivered. You can explore a few of these important and innovative trials below.

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rEECur: Balancing clinical efficacy against toxicity

There are currently 2 arms open to recruitment; IFOS and IFOS combined with lenvatinib (IFOS-L). Lenvatinib belongs to a class of drugs which has shown promising activity in relapsed disease, with an acceptable toxicity profile. A new treatment, trabectedin plus irinotecan is being added into rEECur with the aim of opening in the UK in summer 2026.  The inclusion of trabectedin is underpinned by promising data which has demonstrated clinical benefit in ES. These drugs offer hope to patients who might not otherwise have access to potentially more effective and less toxic drug combinations, providing better quality of life. The Cancer Research Clinical Trials Unit (CRCTU) at the University of Birmingham provides the infrastructure and expertise needed to deliver rEECur including involvement in protocol development and design and regulatory navigation ensuring the trial is scientifically robust and ethically compliant. Delivery is supported through supply of drugs unavailable via local hospital supply, centralised data management, monitoring and safety reporting which ensures the integrity of the results and the wellbeing of participants. By managing these logistical and administrative requirements, the CRCTU ensures that trials are conducted to the highest standards.

Acknowledgements: Prof Martin McCabe (Chief Investigator), Cancer Research UK (funder)

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CF-10: Identifying a better treatment for cystinosis

Cystinosis is a rare inherited recessive condition, with a frequency of 1 in 100,000-200,000 live births, that, if untreated, causes kidney failure and death. There are only about 2-3 new cases each year in the UK.  Standard treatment is cysteamine, which has unpleasant side effects and needs to be taken very frequently, so better treatments are needed. CF-10 is a cysteamine prodrug that was designed to eliminate the deleterious side effects of cysteamine, and does not need to be taken so frequently, and hence may be a superior treatment for cystinosis.

The CF-10 trial is the initial step in the evaluation of CF-10; it is a Phase 1 clinical trial, funded by the Medical Research Council, to estimate the best dose and safety of this potential new treatment. The trial is co-ordinated by the Cancer Research UK CTU in Birmingham, and is expected to open to patient recruitment next year.

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Funded by the EU’s Horizon programme and coordinated by Newcastle CTU, recruitment opened across multiple international sites. Conducting an international trial in an ultra-rare paediatric condition presents considerably challenges, but such a collaboration is necessary for extremely rare diseases. The CTU model provides the operational coordination, statistical expertise and governance oversight needed to deliver this type of complex global study.

Find out more here.

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Because PBC is rare, feasibility is critical. OPERA employs advanced statistical analysis approaches that allowed the sample size required to be reduced by 30% without compromising scientific robustness. This is making the trial more feasible to complete.

Find out more here.

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TreatWolfram: Evaluating a promising treatment for Wolfram syndrome

Find out more here.

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To find out more about the work of Registered CTUs, including ongoing rare disease research, follow the Network on LinkedIn and Bluesky.