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Clinical Trial Information

Hemophilia B clinical trials

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If you are a patient interested in more information about the Phase III trial of AMT-061 in hemophilia B,
please contact us at


Phase III HOPE-B pivotal study of AMT-061  

uniQure has begun enrolling patients in the Phase III HOPE-B pivotal study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.

The Phase III HOPE-B pivotal trial is a multinational, multi-center, open-label, single-arm study to evaluate the safety and efficacy of AMT-061. Approximately 50 adult hemophilia B patients classified as severe or moderately severe will be enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients will receive a single intravenous administration of AMT-061. Dosing of patients in the HOPE-B pivotal trial is expected to start early in the first quarter of 2019. 

The primary endpoint of the study will be based on the Factor IX (FIX) activity level achieved following the administration of AMT-061, and the secondary endpoints will measure annualized FIX replacement therapy use rate and annualized bleed rate. 

Patients enrolled in the HOPE-B trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers. Previous studies performed by uniQure suggest that AAV5 gene therapies may be viable treatments for at least 97% of patients. 

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


Phase IIb dose-confirmation study of AMT-061   

Concurrent with the lead-in phase of the HOPE-B pivotal study, uniQure will also conduct a short, Phase IIb dose-confirmation study of AMT-061 in approximately three patients. Patients will receive a single dose of 2x1013 vc/kg and be evaluated for a period of approximately six to eight weeks to determine FIX activity and confirm the dose of AMT-061 for the pivotal study.  Patients enrolled in this dose-confirmation study will be followed for one year.

In addition to advancing our pivotal trial, we have also initiated patient recruitment for the Phase IIb dose-confirmation study and expect to commence enrollment in July. We look forward to announcing top-line FIX data from the dose-confirmation study before the end of the year.


Phase I/II study of AMT-060   

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.  In July 2017, the Company presented updated clinical data from our Phase I/II trial (view press release).   

The data from this ongoing study demonstrate clinically significant and sustained increases in FIX activity, substantial reductions in FIX replacement usage and a near cessation of spontaneous bleeding episodes.  

At both doses evaluated, AMT-060 appears to be safe and well-tolerated with no loss of FIX activity, no activation of T-cell response and no development of inhibitors for any of the 10 patients in the study.  None of the patients treated have lost FIX activity for up to one year post administration. To date, our insect-cell manufactured AAV5 gene therapies have been administered to 22 patients across three clinical studies without any evidence of AAV5 capsid-specific cellular immune responses or long-term safety complications.  

All patients screened in the study tested negative for pre-existing neutralizing antibodies against the AAV5 vector uniQure is using in the trial. This collective data set suggests that a large proportion of the hemophilia patient population may be eligible for treatment with AMT-061, which uses the same AAV5 capsid.


Phase I/II study of AMT-130

AMT-130 is an investigational, single administration gene therapy intended to modify the disease course for HD. Preclinical studies have shown that AMT-130 lowers huntingtin protein and improved Huntington disease signs in animal models.

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.  

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