La terapia genica con ATM -130 somministrata ad altri due pazient

Huntington Disease: other two Patients Treated in the AMT-130 Trial


Yesterday 13th October 2020 UniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that two additional patient procedures have been completed in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease.

The ongoing patient enrollment follows a meeting last month of the trial’s independent Data Safety Monitoring Board (DSMB) to review 90-day follow-up data from the first two patients. The DSMB observed no significant safety concerns to prevent further dosing.  The Phase I/II study is a double-blinded, randomized and controlled clinical trial being conducted in the United States. A total of four patients have been enrolled in the study thus far, including two patients treated with AMT-130 and two patients who received imitation surgery.

“We are very pleased with the progress being made to advance this first-in-human AAV gene therapy for Huntington’s disease,” said Ricardo DolmetschPh.D., president of research and development at uniQure. “This is an important achievement that puts us on our original clinical development timeline, making up for the modest delay in the study earlier this year due to COVID-19. In accordance with the study protocol, patient enrollment is expected to continue after a DSMB meeting to review 90-day follow-up data on these two new patients and 6-month data on the first two patients. We expect that this DSMB review will take place early next year and that patient enrollment in the 10-patient first dose cohort will be completed by mid-2021.”

The Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntington’s disease randomized to treatment with AMT-130 or an imitation (sham) surgery across two dose cohorts. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for 5 years after administration of AMT-130.

Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).

Credit by UniQure Press Release 


For more information visit: (NCT04120493)


Clinical Trials