The Leeds Teaching Hospitals NHS Trust

‘UK first’ Huntington's Disease Clinical Trial starts at Leeds Teaching Hospitals

19 July 2019

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This week (Monday 15th July) Leeds Teaching Hospitals NHS Trust (LTHT) was the first site in the UK to have a patient enrolled into the global Roche Generation-HD1 Study. The study is investigating whether a new compound can be effective in the treatment of patients with Huntington's Disease (HD).

According to Huntington’s Disease Association (HDA) around one in 10,000 people have Huntington's.

Participants in the Phase 3 clinical trial, will undergo lumbar punctures every 2 months for 24 months, where a sample of brain fluid will be taken for analysis, after which, a trial drug (RG6042) or a placebo will be administered into the spinal fluid.

The trial drug is an ASO (antisense oligonucleotide). This is a short strand of genetic code that will bind to the mutant Huntington gene product (mHTT). It is hoped this process will lower the active mHTT levels in the brain and therefore, hopefully slow the progression of HD, and lead to a much-improved quality of life.

11 UK sites along with 15 countries are working towards recruiting 660 patients worldwide into the study, with 50 to 60 recruits coming from the UK.

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Speaking this week, the UK’s first trialist in the study, 63 year old Paul Hammond said:

“It's really exciting to be involved in the study. I have known for around a year and a half that this treatment was on the way. My brother sent me the details of the study after seeing it on the news, so I spoke to my GP and he got me involved with the trial. 

“My mum suffered from Huntington's Disease, and I found out in my 40’s that I was going to get sick. Now there is treatment on trial, it brings me hope, and it changes a lot of things for my family.

“I have two daughters in their 20’s, with one who has started a family of their own, and I feel now that I don't have to worry about their future due to this study.”

M20190712 016 Group shot Medical trial caser study LG7 5495 optCo-ordinating the study at Leeds Teaching Hospitals NHS Trust, Callum Schofield, Genetics Research Practitioner, said:

“Generation-HD1 is the culmination of years of hard work for our HD team here in Leeds.

“It is a hugely ambitious inter-departmental effort, bringing together staff from across the Trust to work on what may be one of the definitive studies in the field of HD research.

“Obviously there is a long way to go yet, but we’re hopeful that this research can finally offer real, tangible hope to our HD families here in Yorkshire, and the HD community as a whole.

“Of course many people will not be able to take part in this research, as the scope for prospective participants is incredibly narrow, but it is important to note that this drug is being tested by other people on their behalf, indeed on behalf of the entire HD community.

“We are exceptionally grateful for our wonderful patients going into this trial, this wouldn’t be possible without them.”

Claire Jarvis, Integrated Franchise Lead for Pipeline, Roche Products Limited said:

“Huntington’s Disease is a devastating and fatal hereditary condition with very limited treatment options. New approaches are desperately needed, which is why Roche is proud to be partnering with Leeds Teaching Hospital here in the UK, as part of a pivotal, global clinical trial programme.

“The programme is looking at whether an innovative new treatment, an antisense oligonucleotide, can reduce the levels of the mutant protein, huntingtin, which is found in the brain of Huntington’s patients and believed to be the underlying cause of the disease.

“The first person to be recruited into the trial in the UK is a significant landmark and we look forward to sharing results of the trial in due course."

More Information about the story: 

About Huntington Disease (HD):

  • Huntington’s disease is a rare genetic, progressive condition that causes the nerve cells in the brain to break down, which severely affects a person’s everyday functions such as mobility and thinking.
  • There is a significant medical need for new therapies for HD as there are no treatments that can prevent, slow or stop progression of the disease. The limited treatments currently available for HD only manage some of its symptom.
  • There is no known cure for the disease and there are currently no approved therapies that can treat the underlying cause.
  • The limited treatments currently approved for HD only manage some of its symptoms, such as reduction in uncontrolled body movements.
  • According to Huntington’s Disease Association (HDA) around one in 8,000 people have Huntington's. It affects men and women in equal proportions. For more information visit

Roche Generation-HD1 study:

The trial is a two-year, three-arm, randomised, multicentre, double-blind, placebo-controlled study, that will take place across 80–90 sites in approximately 15 countries around the world.

Roche plan to enroll 660 patients, aged 25–65 years with clinically diagnosed manifest HD, and will be randomised to either bimonthly or every 4 months 120 mg intrathecal injections of RG6042, or placebo.

UK sites taking part in the study:

  • Aberdeen – Aberdeen Royal Infirmary
  • Birmingham – Birmingham and Solihull Mental Health Foundation Trust
  • Cambridge – Cambridge Centre for Brain Repair
  • Cardiff – University Hospital of Wales
  • Glasgow – Queen Elizabeth University Hospital Glasgow
  • Leeds – Leeds General Infirmary, NIHR Clinical Research Facility 
  • London – National Hospital for Neurology and Neurosurgery
  • Manchester – Central Manchester University Hospitals NHS Foundation Trust
  • Oxford – John Radcliffe Hospital
  • Sheffield – Sheffield Children’s NHS Trust
  • Southampton – University Hospital Southampton NHS Foundation Trust