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

iMagemHTT

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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EIP19-NFD-401

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 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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DOMINO-HD

DOMINO-HD will focus on aspects of life for people with Huntington’s that haven’t been studied in depth up until now to improve quality of life for HD patients and families. The main purpose of this study is to look at how sleep, nutrition and physical activity impacts the progression of Huntington´s disease so as to develop new ways to support and manage fundamental aspects of HD patients. The study will also explore how digital technologies, such as wearable fitness trackers, can be used to support people with HD.

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DIACCIMEX

The main objective of the study is to develop and validate an analytical and clinical Non-Invasive Prenatal Diagnosis (NIPD) test for triplet repeat diseases by isolated circulating fetal trophoblastic cells (CFTC) analysis from maternal blood, searching for the familial mutation in families at risk of having one of the following triplet repeat diseases: Huntington's disease, Steinert Myotonic dystrophy, Fragile X syndrome, spinocerebellar ataxia (SCA) 1, 2 and 3.

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GEN-PEAK

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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GENERATION HD1

This is a phase III trial where 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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HD-DBS

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. The potential effect of the study will be measured by testing cognitive, motor and social functioning.

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LCR-MH

The main purpose of this study is to be able to confirm whether a protein called BDNF is a biomarker for the expression of HD in humans. The study will recruit 135 participants who will be followed for a period of 2 years. Their levels of BDNF will be measured in samples taken both from the cerebrospinal fluid (CNF) and blood. The symptom development in the 2 year trial period, will also be monitored with brain imaging and by testing cognitive functions. The study will investigate how the levels of BDNF in the blood and the CSF differ or correspond. The study will also try to identify the connection between BDNF levels and symptom development. 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 HD. If proven to be relevant, BDNF levels will probably be a target for treatment in the future. In this study the BDNF levels in CSF and blood will hopefully be identified as a biomarker for HD. We need biomarkers to measure if a treatment has positive effect or not.

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PACE-HD

The purpose of this trial is to evaluate whether the around 60 participants (with early manifest HD) 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 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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Precision HD1

This clinical trial is testing a new drug that selectively lowers the mutant Huntington’s disease 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. The study will investigate how the levels of BDNF in the blood and the CSF differ or correspond. The study will also try to identify the connection between BDNF levels and symptom development. 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 HD. If proven to be relevant, BDNF levels will probably be a target for treatment in the future. In this study the BDNF levels in CSF and blood will hopefully be identified as a biomarker for HD. We need biomarkers to measure if a treatment has positive effect or not.

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Precision HD2

This clinical trial is testing a new drug that selectively lowers the mutant Huntington’s disease 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. The study will investigate how the levels of BDNF in the blood and the CSF differ or correspond. The study will also try to identify the connection between BDNF levels and symptom development. 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 HD. If proven to be relevant, BDNF levels will probably be a target for treatment in the future. In this study the BDNF levels in CSF and blood will hopefully be identified as a biomarker for HD. We need biomarkers to measure if a treatment has positive effect or not.

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REVHD

The main purpose of this study is to evaluate the therapeutic potential of Resveratrol on the caudate volume in HD patients, using volumetric MRI. This trial aims to test for a reduction in neurodegeneration among HD patients.

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TRIHEP3

In this trial a compound called Triheptanoin is tested to see if it has a positive impact on 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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