Therapeutic Clinical Trials

Manchester is a leading centre for rare conditions clinical trials. Numerous clinical trials are currently taking place across Manchesteroffering patients access to developing treatments not yet widely available, while advancing our collective understanding of rare conditions. These trials span new and improved therapies, existing care pathways, and deeper insight into how rare conditions develop, ultimately aiming to improve quality of life for those living with them. Use the table below to explore the clinical trials currently taking place and find out how to get involved.

Highlighted Clinical Trials

Metformin in Li-Fraumeni Syndrome (MILI) Trial

This clinical trial is exploring the use of metformin, a drug commonly used to treat diabetes, as a way of reducing the risk of cancer in people with Li-Fraumeni syndrome (LFS).

LFS is a rare genetic condition caused by an inherited faulty gene called TP53. This is known as one of the most crucial tumour-suppressing genes, however, is faulty throughout the body in people with LFS. Meaning people living with LFS are at an extremely high risk of developing cancer anywhere in their body. Currently there are no available treatments to reduce this risk.

A study was performed where mice with LFS were given metformin and it proved to reduce their risk of developing cancer. It is thought that metformin achieves this by working to change the metabolism of the cells with the faulty gene, so that they act like normal cells. Although the study was ground-breaking, it is not enough evidence to be sure that metformin ultimately reduces the risk in humans. The MILI trial aims to enlist 224 people with LFS, in the UK, to treat half of them with metformin every day for 5 years and half without. This trial will aid researchers to understand whether metformin can reduce the risk of cancer in people with LFS.

Read more about this clinical trial: https://www.isrctn.com/ISRCTN16699730

Gene Therapy for Children with Mucopolysaccharidosis II (MPS II)

This clinical trial is testing a new gene therapy treatment for young children with mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. MPS II is a rare genetic condition that affects how the body breaks down certain substances, leading to progressive damage in various parts of the body. This happens to people with MPS II due to a missing gene which is supposed to code for a specific enzyme responsible for breaking down waste products in the cell. At present the only approved treatment for MPS II is enzyme replacement therapy. This available treatment replaces the deficient enzyme (due to the missing gene) and alleviates symptoms rather than treating the dysfunctional cell. The gene therapy aims to provide a working copy of the faulty gene in the hope of forming and delivering much more of the needed enzyme to slow down the disease and improve long-term health outcomes for affected children.

Read more about this trial in our article: https://www.mrcc.org.uk/our-impact/new-hope-for-children-with-devastating-rare-genetic-disorder-thanks-to-world-first-research-in-manchester/

Read more about this clinical trial: https://www.isrctn.com/ISRCTN12458940

Manchester Clinical Trials database

 Below is a table containing rare disease clinical trials currently taking place in Manchester. The table includes the name of each trial along with links to more information about the study. Clicking on the links will take you directly to the relevant trial pages for more information, allowing you to explore ongoing research, read about different treatments being tested, and find further details about who may be able to take part. Last update: May 2026

Credits:

Isabella Carpio Gonzalez – University of Manchester medical student