CATEGORIES: Clinical Trials | Featured
HNF Centers of Excellence

Are You a Patient With CMT 1A? New Clinical Trial Opportunity Available For You!

by | Oct 31, 2016 | 9 comments

For the first time investigators are looking for patients to participate in a pivotal Phase 3 clinical trial of Pharnext’s lead investigational pleodrug, PXT-3003 for the potential treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). The study which expects to enroll approximately 300 patients is taking place in the USA, Europe and Canada.

Nine of HNF’s Centers of Excellence are now accepting patients for trial enrollment. Please visit: to find out which centers in your area are recruiting and for contact information.

To learn about inclusion and exclusion criteria and for a complete list of sites visit: and enter protocol # NCT02579759. Be sure to check back periodically as more sites are added, including Canada. BY GETTING INVOLVED YOU CAN HELP CHANGE THE FUTURE! Please consider signing up—and help us get the word out to others so they, too, can get involved. Together, let’s be a part of the first potential treatment for CMT!

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  1. Amy

    What would be the closest site conducting the studies to Georgia?

    • courtney

      Hi Amy,

      The trial is now closed for new patients. Keep checking HNF’s site for more updated clinical trials.

  2. Ralph Wiseman

    CMT is not a fun disease. Hopefully a cure will come soon.

    • Ralph Wiseman

      Participation would be greatly appreciated.

  3. Nicholas Fried

    I have type 1A and so does my son. I am in London England and would very much like to contact any bodies doing these trials here. Can you advise?

  4. Kathy Ransom

    I don’t see any participating Canadian centers, are any coming up?

  5. Tracy Roberts

    I don’t which type I am, are any sites conducting the genetic testing?

      • Tracy Roberts

        Hi Courtney,

        You asked to contact you retarding genetic testing. I would love to take part in any cmt study.



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