COMPLETED TRIALS

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 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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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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OBSERVATIONAL TRIALS