IMPORTANT

Before participating in a study, talk to your health care provider and learn about the risks and potential benefits.

 The main purpose of the study is to characterize the movement of the drug RO7234292 (RG6042) into, through, and out of the Cerebrospinal fluid (CSF) and plasma in addition to their reaction to the drug. The study will also look at the acute time course and recovery profile of CSF, mHHT lowering in response to RO7234292 (RG6042) treatment after intrathecal (IT) administration of RO7234292 (RG6042) to patients with manifest Huntington’s disease (HD).

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This is a phase III trial where Huntington’s disease (HD) patients get injections in the spine with a drug called Tominersen (previously called RG6042).  The drug is supposed to reduce the amount of Huntingtin in braincells. Some of the participants gets the active drug injected every 2nd months, another group gets the active drug every 4 months and a third group gets injected with the placebo. The potential effect of the study will be measured by testing cognitive, motor and social functioning. 

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The main purpose of this study is to identify whether so-called radioligands (small radioactive particles) can detect mutant Huntingtin (mHTT) in the brain.  This imaging study is done on humans and uses PET scans (images) of the brain to see if the presence of radioligands corresponds with the expression of mutant huntingtin in brain cells.  The objective of the study is to use PET scans of radioligands to measure the presence of mHTT in brain cells.  If proven valid and to be an effective way to measure mHTT, it can be used to measure if huntingtin lowering therapies actually lower mHTT in the brain.  This study is important and needs volunteers because we still don’t have any methods to measure whether the huntingtin lowering therapies actually gets into the brain tissue where it’s needed and whether the mHTT levels are lowered

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PRidopidine Outcome On Function in Huntington Disease (PROOF-HD)

PROOF-HD is a Phase 3 clinical study that evaluates the efficacy and safety of pridopidine in patients with early stage of Huntington Disease. Pridopidine is a small molecule developed by Prilenia for the treatment of neurodegenerative disorders such as HD

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This interventional study is currently in phase 1b/2a and evaluates the safety and tolerability in adult patients with early-manifest HD patients.

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