Hoffmann-La Roche



This study evaluates the safety, biomarkers and efficacy trends of different dose levels of investigational drug tominersen in people aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD. Tominersen is an investigational drug that has been studied in several clinical trials since 2015, including in a Phase III study called GENERATION HD1 that tested two different dose regimens of tominersen in adults with manifest HD.

The new Phase II of the GENERATION HD2 trial is a ‘placebo-controlled’ clinical trial, which means that one of the groups will be given a substance with no active ingredients (also known as a ‘placebo’); it looks like the drug being tested but does not contain any real medicine. Comparing results from the different groups helps the researchers know whether any changes seen result from the drug or occur by chance.

This is a double-blinded trial, which means that neither the participant nor the clinical trial doctor can choose or know the group the participant is in until the trial is over. This approach helps to prevent bias. However, the participant’s clinical trial doctor can find out which group the participant is in, if their safety is at risk.

WEBINAR on the updates of GENERATION HD2 trial

Ages Eligible
for Study:

25 to 50 years old.

Sexes Eligible
for Study:


Accepts Healthy Volunteers:


People can take part in this trial if they fit certain criteria, including if they:

  • Are aged 25 to 50 years (at the start of the trial)
  • Have a CAP score (a research calculation based on age and the number of times the mutated section within the HD gene repeats itself – known as the CAG number) of
    400 to 500
  • Have been diagnosed with early manifest HD or are carriers of the abnormal huntingtin gene who are starting to show very early, subtle signs of HD (known as prodromal HD). This may only be clear during a detailed examination by a physician
  • Can tolerate giving blood, having lumbar punctures and MRIs
  • Have a person who can act as a ‘study companion’ throughout the trial

People may not be able to take part in this trial if they:

  • Cannot walk without help
  • Cannot have MRI scans
  • Have an implanted shunt or catheter for the central nervous system
  • Are currently receiving or have had certain treatments before, including those for HD that may affect HTT levels
  • Have certain other medical conditions, including chronic migraines, certain mental health issues or certain infections
  • Are pregnant or breastfeeding, or are planning to become pregnant during or soon after the clinical trial


Denmark, France, Germany, UK, Poland, Italy, Spain, Portugal and Switzerland.

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The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study’s objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.

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The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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Inclusion Criteria

A type of eligibility criteria. These are the reasons that a person is allowed to participate in a clinical study.

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biomarker, or biological marker is a measurable indicator of some biological state or condition. It can be a level of something in your blood or hormon levels for instance.  Biomarkers are often measured and evaluated to examine normal biological processes – like measuring the level of hemoglobin in your blood or your blood pressure.  In reference to research on Huntingtons disease, a biomarker is an indicator of disease progression.  Examples are changes within the cells related to so-called metabolism (energy processes) or levels of mutant Huntingtin in nervecells. It can also be changes in organs – like loss of muscle or brain volume.  We use biomarkers to measure how the disease progress and potentially how a treatment influence the disease progression. 

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An inactive substance or treatment that looks the same as, and is given in the same way as, an active drug or intervention/treatment being studied.

The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work. (from

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CAG repeat

The stretch of DNA at the beginning of the HD gene, which contains the sequence CAG repeated many times, and is abnormally long in people who will develop HD. (from

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Exclusion Criteria

A type of eligibility criteria. These are reasons that a person is not allowed to participate in a clinical study.