Randomised, double-blind, placebo-controlled study to evaluate the effect of metformin, an AMPK activator, on cognitive measures of progression in Huntington's disease patients.

Trial details

Who runs it

IIS La Fe

Trial code

EU 2024-518875-73-00

Record updated

2024. december 11.

Original description

The main objective of this study is to evaluate the effect of metformin on the scores obtained in different cognitive subtests that make up the Unified Huntington's Disease Rating Scale (UHDRS) and that have been shown to be very sensitive to disease progression from early stages and even in the pre-symptomatic phase.Read full original descriptionShow less

The main objective of this study is to evaluate the effect of metformin on the scores obtained in different cognitive subtests that make up the Unified Huntington's Disease Rating Scale (UHDRS) and that have been shown to be very sensitive to disease progression from early stages and even in the pre-symptomatic phase.

Inclusion: Positive symptomatic clinical diagnosis of HD; They should be able to travel to the study site, and in the judgement of the investigator, show that they are likely to be able to continue to travel for the duration of the study; Availability and willingness of a carer, informant, or family member to provide information during study visits assessing PBAs. It is recommended that the carer be someone who attends the patient at least 2-3 times per week and for at least 3 hours per occasion. The suitability of the carer should be judged by the researcher; Presence of ≥ 36 CAG repeats (or more) in the huntingtin gene based on centralised CAG analysis; Males or females between 21-65 years of age, inclusive, with an onset of HD at 18 years of age or older; Women of childbearing age (women who are not postmenopausal or who have not undergone surgical sterilisation) should be using a method of contraception for 30 days prior to starting study treatment, and should maintain at least two methods of birth control for the duration of the study and until 30 days after the last dose of treatment is taken; A sum of > 4 points on the UHDRS-TMS scale and a diagnostic confidence level of 4; Independence scale ≥ 75 %; A score on the UHDRS-TFC scale ≥ 8 at the screening visit; Must be able and willing to provide written informed consent prior to any study-related procedures being performed at the screening visit. Patients with a legal guardian must be authorised according to local requirements; They should be able and willing to take oral medication and should be able to comply with study-specific procedures.

Exclusion: Taken metformin in the last three months prior to the start of the study; You have been treated for heart failure or recently had a heart attack, have severe problems with circulation, or have difficulty breathing; Has been diagnosed with oncological disease; Have diabetes of any type; Have any medical condition other than HD (metabolism, kidney function, liver function, heart problems, etc.), or any contraindication against metformin; Having an uncontrolled psychiatric condition; You are allergic to metformin or any of the other ingredients of this medicine; It presents a serious infection; Have renal [creatinine clearance <60 ml/min calculated by Cockcroft - Gault formula] or hepatic problems; Drinking > 6 units/day of alcohol (alcoholism); Presents suicidal propensity, with affirmative response in items 4 or 5 in the C-SSRS in the screening visit; You are pregnant or breastfeeding; Tiene deshidratación, por diarrea prolongada o severa, o ha vomitado varias veces.

EU CTIS 2024-518875-73-00