IMPORTANT

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

ONGOING INTERVENTIONAL TRIALS

The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring mutant huntingtin protein (mHTT) in the cerebrospinal fluid (CSF) in participants at the early stages of Huntington’s disease (HD).

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The main purpose of this study is to investigate whether Deep Brain stimulation has a positive effect on the motor and cognitive symptoms in Huntingtons patients. Deep brain stimulation (DBS) is a surgery to implant a device that sends electrical signals to brain areas responsible for body movement. Electrodes are placed deep in the brain and are connected to a stimulator device. Similar to a heart pacemaker, a neurostimulator uses electric pulses to regulate brain activity.  DBS treatment has been used for some people with Parkinsons disease.

Both safety and efficacy will be investigated in this study with 50 participants.  25 participants will be stimulated with the DBS for the first period of 3 months and their performance on a variety of tests, will be compared with the 25 participants who don’t get any stimulation.  After the first 3 months, all participants will be treated with DBS for another 3 months.

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The main purpose of this study is to determine whether a drug called Neflamapimod can improve cognitive functions (planning, memory etc) in patients with early-stage Huntington’s disease (HD). The drug is taken orally.  In the study 16 patients will receive the drug for 10 weeks and then be given placebo for 10 weeks or vice versa.  Neither the doctor nor the patient knows in which period they get the active drug or the placebo.  The effect will be tested with different cognitive tests.

Neflamapimod target an enzyme in the brain called p38 alpha. The aim is to reduce expression of the enzyme in neurons and enable the cells to function better.  The drug has proven promising effect in animal tests and it is also currently being tested on patients with Alzheimers Disease. 

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 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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The main purpose of REVHD is to be able to confirm whether a protein called Brain Derived Neurotrophic Factor (BDNF) is a biomarker for Huntington’s disease (HD).

Why is this study important? We have good reasons to believe that BDNF has an active role in protecting damaged brain cells as well as enhancing growth of new ones. The study will provide us with more solid information about the connection between BDNF and symptom development in humans with Huntington’s. If proven to be relevant, BDNF levels will probably be a target for treatment in the future. In this study the BDNF levels in cerebrospinal fluid and blood will hopefully be identified as a biomarker for Huntington’s disease. 

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The purpose of this trial is to evaluate whether the around 60 participants (with early manifest Huntington’s disease) who commit to the physical activity program, manage to follow up and complete the 12 month intervention. The active intervention group will be supervised and encouraged by a trainer/physiotherapist on a regular basis. The results on both motor and cognitive tests, will be compared with a group of around 60 participants who are only monitored doing their physical activities as usual. 

The aim of the study is to see if Huntington’s disease (HD) patients, when being followed up and encouraged in a specialized program for physical activity, follow the program  and benefit from the training.

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This clinical trial is testing a new drug that selectively lowers the mutant Huntington’s disease (HD) protein, whilst leaving the normal huntingtin protein relatively untouched. In order to be able to identify the gene with the mutant Huntingtin, they use a unique method and identify a so-called snip. Wave has identified two different snips that helps locate the mutant Huntingtin and therefore there are two studies (Presicion HD 1 and Presicion HD 2)  with the same treatment, where the only difference is the targeted snip.

The primary objective is to understand whether the drug is safe in a small number of volunteers, before testing in a larger population and collecting evidence that the drug may work. The total of 60 participants receive either placebo or different dosages of the active drug every 4 weeks.  Both the active drug and the placebo is delivered through a spinal injections. 

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This clinical trial is testing a new drug that selectively lowers the mutant Huntington’s disease (HD) protein, whilst leaving the normal huntingtin protein relatively untouched. In order to be able to identify the gene with the mutant Huntingtin, they use a unique method and identify a so-called snip. Wave has identified two different snips that helps locate the mutant Huntingtin and therefore there are two studies (Presicion HD 1 and Presicion HD 2)  with the same treatment, where the only difference is the targeted snip.

The primary objective is to understand whether the drug is safe in a small number of volunteers, before testing in a larger population and collecting evidence that the drug may work. The total of 60 participants receive either placebo or different dosages of the active drug every 4 weeks.  Both the active drug and the placebo is delivered through a spinal injections. 

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

PROOF-HD is a Phase 3, randomized, double-blind, placebo-controlled study evaluating 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. Pridopidine binds and activates a protein (Sigma-1 receptor or S1R) that is expressed at high levels within the brain.

The purpose of this study is to evaluate the effect of pridopidine 45mg twice daily on functional capacity, as well as on motor and behavioral features in participants with early-stage manifestation HD.

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In this trial a compound called Triheptanoin is tested to see if it has a positive impact on Huntington’s disease (HD) patients functions in all areas but with a special emphasis on cognitive functions. Trhiheptanoion is artificially produced and it is a fat.  It is almost tasteless and colorless and is already used as medical food to treat certain diseases related to the bodies energy production, socalled metabolic disorders. We know that the brain cells energy production is negatively affected in HD.

100 participants is included in the trial. 50 will get Triheptanoin each day for 12 months.  The other 50 participants will for the first 6 months receive placebo.  But for the following 6 months they will get Trheptanoin.

Potential effect will be tested with MRS to get an image of whether the energy production has improved or at least not decreased. MRI images will also be taken to see any effect on brain volume.  In addition the participants will be tested with a battery of motor and cognitive tests as well as questionnaires related to general function and psychiatric status.

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