COMPLETED TRIALS

Here is a list of the latest completed Observationals and Interventional trials in Europe

INTERVENTIONAL TRIALS

The main objective of DIACCIMEX was 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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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 reason of this study is to look at the movement of the drug, Tominerson into, through, and out of the CSF and plasma and their reaction to the drug. The study will also look at a time line and recovery profile of mHTT in CSF.

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GEN-EXTEND is the open-label extension study for the Phase 3 trial, GENERATION HD1. Whereas GENERATION HD1 is designed to evaluate the effects of Roche’s HD treatment and help decide if it should be made available to patients, GEN-EXTEND evaluates the long-term safety of the same treatment, to understand long-term side effects. Not everyone is able to be a part of this study, only patients who have been involved in the earlier trials in the programme can join an open-label extension.

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This WAS a phase 3 trial where HD participants were injected in the spine with a drug called Tominersen, an ASO. The drug was believed to lower the amount of mHHT in brain cells. The delivery of the drug was stopped after it was advised by an independent committee. The participants were asked to remain and continue with data collection for the remaining period of the trial. 85% chose to continue.   

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The main purpose of REVHD was to evaluate the therapeutic potential of Resveratrol on the caudate volume in Huntington’s disease patients, using volumetric MRI. 

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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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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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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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OBSERVATIONAL 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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BIOHD is studying ‘biomarkers’ that predicts the progression of Huntington’s disease. The study is therefore trying to find markers in the blood, through imaging, and psychological tests. In doing so, clinicians might be better at predicting how Huntington’s disease will progress in people who carry the mutated gene. 

What is a biomarker? It is a measurable indicator of a disease or condition. It can for instance be a level of something in your blood or hormon levels. 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. 

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The main purpose of DOMINO-HD 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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