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
25 to 50 years old.
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