UCL HUNTINGTON’S DISEASE RESEARCH

PADDINGTON

At a glance

May involve psychology tests

May involve blood tests

May involve brain scans

Official website: http://www.paddingtonproject.eu

Please note- the Paddington study is now fully recruited.

About the Study

The aim of the PADDINGTON project is to develop and apply a range of biomarkers that will help in the clinical development of a novel drug (SEN0014196, Siena Biotech) with disease-modifying potential in Huntington’s Disease.

PADDINGTON began in February 2011 and involves research sites in London, Manchester, Leiden (Netherlands), Paris (France) and Ulm (Germany), Bochum (Germany), Krakow (Poland) and Warsaw (Poland). Professor Sarah Tabrizi is the Principal Investigator for the London Site, based here at UCL.

There are several studies called ‘work packages’, that make up the PADDINGTON Project:

Work package 1

Work package 1 is an exploratory phase IB clinical trial testing the safety and tolerability of the drug in a small group of people with early symptoms of Huntington’s disease.

Work package 1: what is involved?

60 volunteers with early Huntington’s Disease across 6 sites will be given SEN0014196 once daily for two weeks. Participants will be required to stay overnight in our Clinical Trial Centre at the beginning and end of the two week period. During this period, several tests will be administered to monitor the participants’ responses to the drug:

Blood samples will be taken
A medical interview and neurological examination by a neurologist
Safety assessments, including a physical examination
Behavioural and cognitive (thinking) tasks

Work package 1: who can take part?

Work package 1 is now fully recruited.

Work package 2

Work package 2 is an imaging biomarker project aiming to develop and get in motion a feasible assessment package for tracking the progression of early stage Huntington’s disease. This assessment package may provide a useful tool for future trials. Work package 2 is an observational study, not a drug trial.

However, it is hoped that, if found to be well tolerated and safe, the clinical trial with SEN0014196 could be expanded into a Phase III trial using the framework developed in work package 2.

Work package 2: what is involved?

60 volunteers with early Huntington’s Disease and 40 control participants will be enrolled over 4 sites (London, Leiden, Paris and Ulm). Assessments will last roughly 3 hours and will include:

An MRI scan (lasting approximately 45 minutes)
Two blood samples (the first being a fasting blood sample before breakfast)
A medical interview and neurological examination by a neurologist
Cognitive (thinking) tasks

Work package 2: who can take part?

Work package 2 is now fully recruited.

Work package 3

Work package 3 is a separate study that will be using the blood samples taken in work packages 1 and 2 to investigate using bloods as markers of disease progression. These markers may be useful in future clinical trials testing drug efficacy.