La prima parte dell’arruolamento nella sperimentazione con ATM-130 per l'Huntington si è conclusa con successo e lo studio arriverà presto in Europa.

Buone notizie da UniQure sulla sperimentazione con ATM-130 per la malattia di Huntington

Finally Good news for the Huntington's community!

We received yesterday the official communication from uniQure, announcing that the enrollment of the first patient cohort in the study of ATM-130 for the treatment of Huntington's disease has concluded, ahead of schedule.

But the good news don't end here: a new clinical trial with AMT-130 will be initiated in Europe by the end of the year. 

"This communication, which came at the tail end of the Easter holidays, restores our hope and reminds us that research is not stopping: the possible therapeutic strategies to be explored are still many and potentially valid. The entire Huntington's community continues to have by its side a network of researchers at the forefront of the common battle against the disease" - says Barbara D'Alessio, President and CEO of the Italian League for Huntington's Disease Research.


The conclusion of the enrollment of the first cohort of participants

Nine centers in the United States are ready to begin enrollment of the next cohort of participants, which, however, will not begin until after the evaluation of data by the Data Safety Monitoring Board, expected by mid-year. 

As a reminder, the ongoing Phase I/II clinical trial with AMT-130 is a randomized, placebo-controlled, double-blind study to explore the safety, tolerability, and proof-of-concept of AMT-130 in patients with early-stage Huntington's disease.  The study involves two dose cohorts with a total of 26 patients, some treated with AMT-130 and others with a similar, but sham, surgical procedure. 

The first dose cohort, the one that just completed recruitment, consists of 10 patients, six of whom received treatment with AMT-130 and four of whom received a sham surgery. 

The second cohort consists of 16 patients, of whom 10 will receive treatment with AMT-130 and six will receive sham surgery. The study consists of a 12-month blinded period followed by long-term open-label (placebo-free) follow-up for 5 years after AMT-130 administration. 

Launch of an open-label study of ATM-130 in Europe

A Phase Ib/II study of AMT-130 will begin in Europe in the second half of 2021. It will be an open-label, i.e., placebo-free, study and will enroll 15 patients with early-stage Huntington's disease in two dose cohorts. Along with the U.S. study, the European study aims to test the safety, proof-of-concept, and optimal dose of AMT-130 to be continued in a subsequent, desirable Phase III or confirmatory study if an accelerated registration pathway is feasible. In Europe, there are an estimated 75,000 people with Huntington's disease.

Remember that AMT-130 is introduced into the brain through a neurosurgical procedure called CED (Convention-Enhanced Delivery) performed with the help of MRI, which involves a single administration to a specific region of the brain, the striatum.


For more information on the clinical study:

See the UniQure Site

See the study sheet on our website 

Read the Official Press Release