Actively Recruiting

Motor Neurone Disease Clinical Trials

Find actively recruiting clinical trials for motor neurone disease (MND/ALS) in the UK. Explore emerging treatments from gene therapy to neuroprotective agents.

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Motor Neurone Disease Treatment Pathway

See where clinical trials fit into your treatment journey

Newly Diagnosed

First-line treatment and supportive care optimisation

Standard: Riluzole, Edaravone, multidisciplinary team care

Disease Modification

Trials aiming to slow or stop disease progression

Standard: Riluzole + supportive care. Tofersen for SOD1 mutations

Gene Therapy

Targeted treatments for genetic forms of MND

Standard: Tofersen (SOD1), emerging gene therapies for other mutations

Symptom Management

Trials addressing breathing, nutrition, and quality of life

Standard: NIV, gastrostomy, occupational therapy, palliative care

Key Biomarkers

SOD1 Mutation

About 20% of familial ALS cases have SOD1 mutations. Tofersen (an antisense oligonucleotide) targets SOD1 and is available through NHS and ongoing trials.

C9orf72 Expansion

The most common genetic cause of MND/ALS. Multiple gene therapy trials targeting C9orf72 are underway, including antisense oligonucleotide approaches.

Neurofilament Light Chain (NfL)

A blood biomarker reflecting neuronal damage. Used in clinical trials to measure disease activity and treatment response.

FUS Mutation

A rarer genetic cause of ALS. Several trials explore targeted therapies for FUS-associated MND.

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Frequently Asked Questions

There is currently no cure for MND/ALS, but clinical trials are making significant progress. Gene therapies targeting SOD1 and C9orf72 mutations have shown promise, and several neuroprotective agents are in advanced trials.

Tofersen is an antisense oligonucleotide that targets the SOD1 gene. It is approved for SOD1-mutant ALS and has been shown to reduce SOD1 protein levels and slow disease progression. Ongoing trials explore longer-term outcomes.

Discuss trial options with your MND specialist or neurologist. The NIHR supports an active MND research network in the UK. You can also search TrialConnect to find recruiting studies and contact trial sites directly.

How to Join a Motor Neurone Disease Clinical Trial

1

Search

Use our search above to find trials that match your condition and location. Review the eligibility criteria carefully.

2

Discuss

Talk to your GP or specialist about any trials you are interested in. They can help determine if a trial is appropriate for you.

3

Contact

Reach out to the trial team directly using the contact information on the ClinicalTrials.gov listing. They will guide you through screening.

4

Enrol

If you meet the criteria and decide to participate, you will go through informed consent and begin the trial process.

Related Comparisons

πŸ—ΊοΈ Related Pathway: Neurological Pathway