Terazosin RepUrposing STudy in ALS (TRUST)
Research type
Research Study
Full title
Terazosin RepUrposing STudy in ALS (TRUST): a pilot study targeting PGK1 with terazosin in ALS patients
IRAS ID
301752
Contact name
Alexander Thompson
Contact email
Sponsor organisation
University of Oxford / Research Governance, Ethics and Assurance Team
Eudract number
2021-003345-38
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), is a neurodegenerative disease in which motor neurons deteriorate leading to progressive weakness. Treatment options are very limited. Laboratory evidence suggests that an enzyme called phosphoglycerate kinase 1 (PGK1) might help to protect motor neurons by increasing their energy availability. The drug terazosin is known to activate PGK1, and researchers at the University of Oxford and University of Edinburgh have shown that terazosin helps motor neuron survival in laboratory models of the disease. Terazosin is a drug that is routinely prescribed to patients either for high blood pressure or for symptoms arising from an enlarged prostate gland in men, where it acts through a different mechanism from PGK1 activation.
As motor neurons deteriorate in ALS, they release substances that can be measured in body fluids and give an indication of how active the disease is. These are called biomarkers. In this pilot study, we will test whether terazosin treatment significantly lowers the levels of biomarkers in the blood, spinal fluid and urine at intervals over the course of 6 months. We will also monitor clinical measurements of disease progression, such as the ALS Functional Rating Score and the spirometry ‘breathing’ test, which are carried out as part of your routine clinical appointments. If this study shows that terazosin alters biomarker levels, suggesting that it potentially slows the disease process in patients with ALS, it would then make a strong case for a larger-scale clinical trial.REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
21/EM/0251
Date of REC Opinion
21 Dec 2021
REC opinion
Further Information Favourable Opinion