Monday, January 8, 2018

Update Pharnext PXT 3003 Clinical Trial


"To be of use in the world is the only way to happiness." -Hans Christian Anderson

I had my one year follow up visit in November.  I have been part of a Phase III clinical trial of 300 CMT Type 1A patients at 30 sites world wide.

I have been going to St. Paul for my visits with Dr. Walk and his staff at the University of Minnesota.

The drug in the trial is Pharnext PXT 3003. It is a mixture of baclofen, naltrexone and D-sorbital.  I take my medicine twice a day.  The trial is a double bind study so I may be part of the placebo group.

This last visit was a full evaluation. They take a medical history and ask about side effects. I have had none. I seem to have a bit more energy and my toes are straighter.

Then my hand and foot strength are measured. I do a timed walk and a timed test to put and remove pins from a board.  Dr. Walk did a nerve conduction test.

I go back in a few weeks for the end of this phase of the trial. I have the option to continue and will be get the medicine. I plan on continuing since I have had no side effects so far.

The thought was this drug would stop progression and maybe provide a bit of improvement. Since CMT progresses slowly there may not be any large changes. I would be happy if I could keep my CMT from progressing . I would love to retain the ability to walk and run.

The drug was designed to help even though like me with a milder expression. I was happy to volunteer for the trial hoping it will help everyone with CMT 1A. That includes other members of my own famly.

I've been involved in studies before.  One of my doctors at the MDA clinic took DNA to study because I am so active.  The first appointment I had in St. Paul for this study I donated DNA again this time to a study at the University of Florida. I hope there is a clue in my DNA that shows why I can be so active and others struggle with daily pain and activities.

According to Pharnext the drug works by:


  • Inhibition of the PMP 22 gene over expression associated with de-myelination.
  • Direct nerve protection
  • Provide positive effects to other cellular types: muscle cells, neuro-muscular junctions and nerve cells.
In September it was announced the trial would continue because of the positive results so far. The philosophy of Pharnext has been development of innovative drugs based on combination and re-purposing of known drugs.  Pharnext 3003 is currently used to treat MS.

The trial started in December of 2015.

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Chris Wodke
Founder & Manager Team CMT
www.run4cmt.com

Chris is a triathlete and long distance runner. She is a three time participant of the Boston Marathon.  In 2012 she finished 2nd at Boston in the Mobility Impaired Division. She was on the course in 2013 when the bombs exploded.

She has appeared three times at the Paratriathlon National Triathlon Sprint Championship. She was the 2012 and 2014 National Champion Paratriathlon Open Division Champion.

In 2014 she was the PC Open Champion at the Duathlon National Championship and at the Aqua bike National Championship in 2016. She represented Team USA at the Aquathon ITU World Championship in Chicago in 2015 and at the World championship in Cozumel in 2016.  

 In 2014 she represented the U.S. as a paratriathlete at the Pan-American Triathlon Championship in Dallas, Texas.  She has won state championships in cycling and triathlon as a senior Olympian. In 2017 she placed 2nd in her age group at the Winter Triathlon National Championship, earning a spot on Team USA for the World Championship.

She travels around the country raising awareness of CMT.

She is the author of the book, “Running for My Life” that details her experience as a CMT affected athlete.

You may visit her author page at:
http://www.amazon.com/Christine-Wodke/e/B00IJ02HX6

Team CMT is a group of athletes and supporters working to raise awareness and to find a cure for CMT. We have 212 members in 38 states. We also have members in Australia, England, Scotland, Canada, Vietnam, Turkey, Finland, Scotland, France, Poland, Iran, Norway and Sweden. If you wish to join us visit our web site; www.run4cmt.com or www.hnf-cure.org

CMT or Charcot-Marie-Tooth is the most commonly inherited peripheral neuropathy. It affects over 155,000 Americans (as many as MS).  It is a disease of the nerves that control the muscles. It is slowly progressive, causing loss of normal function and or sensation in the lower legs/feet and arms/hands.

Symptoms include; muscle wasting in the lower legs and feet leading to foot drop, poor balance and gait problems Atrophy in the hands causes difficulty with manual dexterity.

Structural foot deformities such as high arches and hammer toes are common.

Poor tolerance for cool or cold temperatures and many people have chronically cold hands and feet.

Additional symptoms may include fatigue, sleep apnea, breathing difficulties and hearing loss.

Additional Link
Follow CMT affected Paratriathlete Timmy Dixon
http://cmtamputee.wordpress.com/

Follow CMT Author Chris Steinke
https://cmtandmesite.wordpress.com/2017/01/30/what-is-charcot-marie-tooth-disease/


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