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Clinical Trial Information


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Huntington's disease

Phase I/II study of AMT-130 in Huntington's disease

AMT-130 is a novel gene therapy candidate for the treatment of Huntington’s disease, utilizing our miQURE™ proprietary gene-silencing platform and incorporating an AAV vector carrying a microRNA specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment. 

This 5-year trial consists of a blinded 18-month Core Study Period to evaluate the safety and potential impact of AMT-130 on disease progression and an unblinded 3.5-year Long-Term Period with annual follow-up visits to evaluate the safety of AMT-130 and disease progression.  

Learn more about the Phase I/II study of AMT-130 here.  

 > Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington Disease



Phase III HOPE-B pivotal study of etranacogene dezaparvovec (AMT-061) in hemophilia B  

uniQure has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. 

Patients in the Phase III HOPE-B clinical study were initially enrolled into a prospective, observational lead-in period of at least six months during which bleeding events and FIX replacement therapy usage were monitored. All patients required prophylactic routine FIX replacement prior to entering the clinical trial, and patients were not excluded from the trial based on pre-existing NAbs to AAV5. The primary endpoints in the study include FIX activity at 26-weeks and 52-weeks after dosing and annualized bleeding during the 52 weeks after dosing.

> HOPE-B Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients 


Phase IIb dose-confirmation study of etranacogene dezaparvovec (AMT-061) in hemophilia B   

The Phase IIb study of etranacogene dezaparvovec is an open-label, single-dose, single-arm, multi-center trial being conducted in the United States. Three patients with severe hemophilia (endogenous Factor IX (FIX) activity less than or equal to one percent) were enrolled in the study and received a single intravenous infusion of 2x1013 gc/kg. Prior to the administration of etranacogene dezaparvovec, all three patients showed low levels of pre-existing neutralizing antibodies to AAV5 but were not excluded from the trial on that basis. The patients in the Phase IIb study were followed for 52 weeks to assess FIX activity, bleeding rates and usage of FIX replacement therapy, and will be monitored for five years to evaluate the safety of etranacogene dezaparvovec.


Phase I/II study of AMT-060 in hemophilia B   

AMT-061 and AMT-060 are identical gene therapies apart from two nucleotide substitutions in the coding sequence for FIX. The gene variant, referred to as FIX-Padua, has been reported to provide an approximate 8 to 9-fold increase in FIX activity compared to the wild-type FIX protein used in AMT-060. All other critical quality attributes of AMT-061 are expected to be comparable to those of AMT-060, as AMT-061 utilizes the same AAV5 capsid and proprietary insect cell-based manufacturing platform.  

The AMT-060 study is an ongoing Phase I/II, open-label, multi-center study including 10 patients with documented severe or moderately-severe hemophilia B.  uniQure will follow all 10 patients in the study for a period of five years to further assess long-term safety and durability.