First Hemophilia B Patient Enrolled in Potential Breakthrough Phase III Trial

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According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an investigational gene therapy. The treatment is aimed towards those with moderately severe to severe hemophilia B.

Hemophilia B Explained

Hemophilia B is a type of hemophilia that is characterized by a deficiency in the factor IX gene. Hemophilia is a genetically inherited disorder that results in the inability for the blood to form clots. Consequently, those with hemophilia experience long periods of bleeding. Symptoms of hemophilia B include nosebleeds, bruising, urinary tract infections, extended bleeds, and more. There are treatment options available for hemophilia B, mainly targeted around the concentrations of the mission IX factor. Treatment options are also available to be customized for the individual needs of the patient. To learn more about hemophilia B, click here.

More About AMT-061

The treatment AMT-061 has recently been given Breakthrough Therapy Designation through the United States Food and Drug Administration. The novel treatment has also been designated to the Priority Medicines initiative through the European Medicines Agency.

“AMT-061 has the potential to be a major advancement in gene therapy for patients affected by hemophilia B,” said Dr. Pipe from the University of Michigan. Dr. Pipe is also the principal investigator for this clinical trial.

“A one-time treatment, such as AMT-061, could be life-changing for these patients, many of whom struggle to manage ongoing challenges, including compliance with frequent infusions and recurrent episodes of bleeding,” he continued.

Dr. Zelenkofske, the chief medical officer at UniQure, is also optimistic about the trial and the implications it may have on patients in the future.

“We are delighted to have enrolled the first patient in this Phase III pivotal study of a gene therapy for patients with hemophilia B,” he noted.

“This represents a significant milestone for uniQure as we advance a potentially best-in-class gene therapy for patients with this life-altering disorder.  In addition to advancing our pivotal trial, we have also initiated patient recruitment for our 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.”

The Design

In the Phase III HOPE-B pivotal trial, around 50 adults with hemophilia B will participate. Of these 50, the patients will have hemophilia B that is classified as moderately severe to severe. There will be a six month time period when these patients will continue their standard treatment in order to set a baseline control for the study. This period will be strictly observed and nothing new will be implemented throughout this time window.

After this six months of control is over, participants will be given a single intravenous dosage of the treatment AMT-061. This administration is set to begin in early 2019.

Endpoints for the study are to be determined by both Factor IX activity as well as bleed rate of patients. If the study shows success, this could mean a huge difference in the lives of those who have hemophilia B.

To learn more details about the study, click here.


What are your thoughts on this potential breakthrough? Share your stories, thoughts, and hopes with the Patient Worthy community!

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