The following research trials are currently open to patients with peripheral nerve diseases in the UK.
This list is not yet complete. See also https://clinicaltrials.gov/ which is the international registry of all clinical trials, searchable by disease and country, which also provides further technical information. There is advice to help patients navigate this site HERE
Most clinical trials will pay reasonable travel expenses for patients to attend the hospital. You will have to attend the hospital for monitoring visits which may be quite frequent.
Intervention
A global Phase 3, randomized, double-blind, double-dummy trial evaluating riliprubart versus standard intravenous immunoglobulin (IVIg) in adults with CIDP.
INCLUSION CRITERIA
Adults with CIDP who are already receiving maintenance IVIg but continue to have residual disability.
Locations
Oxford
General information
Open
Most CIDP patients who respond to IVIg do not achieve complete recovery and often remain with weakness, sensory symptoms, fatigue, or functional impairment. VITALIZE is designed to determine whether riliprubart can provide better disease control than continued IVIg treatment.
Contact
Dr Simon Rinaldi (UK CI); Sanofi (Pharma company)
Intervention
The investigational treatment is acoramidis, a small-molecule TTR stabilizer that binds tightly to transthyretin and helps prevent dissociation of the TTR tetramer, the key step that leads to amyloid formation.
Inclusion criteria
People who carry a pathogenic TTR mutation but have not yet developed symptoms. The study enrols:
Locations
London
General information
Open
ACT-EARLY (Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young) is a Phase 3 prevention study testing whether acoramidis, a transthyretin (TTR) stabilizer, can prevent or delay the onset of hereditary ATTR amyloidosis (ATTRv). Current ATTRv treatments are typically started after symptoms appear, such as neuropathy or cardiomyopathy. ACT-EARLY is one of the first large trials asking whether treatment should begin before disease onset, analogous to preventive approaches in genetic conditions like Huntington's disease or familial Alzheimer's disease.
Contact
Eidos Therapeutics (BridgeBio pharma)
Intervention
The IMAGiNe study is not a drug trial. It is an international, prospective observational registry designed to improve understanding of IgM paraproteinaemic neuropathies, particularly neuropathy associated with anti-myelin-associated glycoprotein (anti-MAG) antibodies.
Inclusion criteria
International multicentre registry with prospective observational study. No investigational treatment is assigned; patients receive routine clinical care. Includes patients with:
LOCATIONS
Countrywide
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