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CMT1A & PREMIER Trial

What you need to know

HNF Partner Pharnext is Actively Recruiting CMT1A Patients for PXT3003 Phase III Trial 

Pharnext, a French biopharmaceutical company, is conducting an international study called PREMIER. They are developing a potential new treatment for subjects living with  CMT1A.  The purpose of the PREMIER study is to find out if a study drug, called PXT3003, is safe and effective at treating people with CMT1A. It is hoped that PXT3003 can improve how the nerves function, thereby improving the symptoms of CMT1A. 

Pharnext are currently looking for people age 16-65 years with a confirmed genetic diagnosis, who may be interested in joining the PREMIER study. The PREMIER study will take approximately 17 months to complete. During the study, PXT3003 will be compared to a placebo (an inactive or “dummy” drug). Subjects will be assigned to one of two treatment groups as follows: 

  1. PXT3003 taken orally, twice daily for 15 months 
  2. Placebo taken orally, twice daily for 15 months

The treatment groups will be selected at random by a computer. Subjects will be split evenly across the two groups, so they will have a 1 in 2 chance of receiving the study drug. The study is double-blinded, which means that for the entire study neither the subjects nor the study doctors will know if they are receiving PXT3003 or placebo.

Eligible subjects will attend the clinic every 3 months (6 visits in total), and the study team will contact them by telephone at least twice between these visits. They will also attend a final clinic visit, known as a Safety Follow-Up Visit, to assess their ongoing health and well-being.

For more information, visit www.premiercmt1a.com/ where you’ll be prompted to answer six questions to check if you may be eligible for the PREMIER study. Based on your answers, a pop up will let you know if you are eligible and you can view a map listing sites that are enrolling patients with the contact information. Or you can fill out the form on this page and someone at HNF will reach out to you or direct your email to info@hnf-cure.org.

Contact Us About the PREMIER Trial

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