Pharnext PXT 3003 Clinical Trial

This spring the Hereditary Neuropathy Foundation (HNF) and Pharnext made an announcement that many of us in the CMT have been waiting for.

They announced the start of Phase 3 trials of PXT 3003 for CMT 1A,  This drug is a blend of baclofen, naltrexone and sorbitol. It has been used to treat MS. There was a successful phase 2 trial of 80 CMT patients and the drug was well tolerated.

The phase three trials will be conducted with 300 patients at 27 sites in the United States and Europe.

I volunteered for the study at the University of Minnesota lead by Dr. Walk.

I emailed the contact on the HNF site that listed all the study locations.

I was contacted by the site study coordinator Valarie Ferment.  I was worried that I would not be accepted because I am mildly affected and so active.  I shared this with Valarie.  I was asked a number of screening questions about my age, plans for surgery and whether I had CMT 1A confirmed by a genetic test.

I was sent an application and asked to provide a copy of my genetic test and diagnosis.

I was told there would be no compensation for participating in the study but that travel expenses would be paid. That was good news for me because I live in Milwaukee, a six hour drive from the study site.  The team working on the project reviews each application.

I got  meet  Dr. Walk at the HNF patient conference in New York city this October. He was on one of the panels and was sitting at my table. There was poster by Pharnext at the conference that said the rejection rate for the study was 25%. When I him about it, he said one of the qualifiers was foot drop and weakness in the foot.  Since we were on break he did a quick check of my feet and said I would meet that criteria.  He said my acceptance would depend on the whole screening process, but that he remembered discussing my case. They were interested because of my mild case and athletic activity.

I shared with Dr. Walk that because I was so active one of my doctors at MDA clinic in Milwaukee paid for my genetic test. She felt there may be something in my DNA that might be different than someone more severely affected with CMT. Dr. Walk shared with me that there was a study in Florida collecting DNA from patients like me to study. He asked if I would agree to submit a blood sample to the study when I was in Minneapolis for my study screening appointment. I eagerly agreed. Even if  I was not accepted for the Pharnext study, I would be able to help another research project.

On October 17th I had my initial screening appointment. It was like the normal neurological exam we get every year. I had to do the nerve conduction test which I hate.

Plus I had an EKG, I am guessing to be sure I was healthy enough. Blood was also drawn and lots of questions about if I had any plans for surgery or had any surgery.  You cannot have had surgery in the last 2 years or plan to have surgery. You could not be on high dosage of vitamin C in the last two months. It had to be less than 750 milligrams a day. I added up all my supplements and I was just under.

There were disclaimers and waivers to sign.

I also was enrolled in the Florida study so that mean waivers and another blood draw.

 The first assessment took about 4 hours and I was told I was accepted to the study.

The following week there were more tests. I did a test of grip and foot strength, a test were I did a timed walk and where I had to put 9 pegs in a hole.

After all the testing was done, Valarie went to draw my sample. There are two different doses and a placebo. It is a double blind study that will last 15 months.

I was given a 3 month supply of an oral  solution that I have to take twice a day. The medicine has to be kept refrigerated which will put a crimp in my traveling. I take it in the morning and in the evening right after eating.

It does not taste very good, but I am getting used to it.  It is delivered in a syringe, so I put the medicine as far back in my mouth as I can.   I am asked to save all the bottles and return them when I go back in three months.  I was given a card to carry in my wallet saying I was taking the drug and to contact Dr. Walk if I am treated for any medical emergency. I have to get permission to take any new drug or supplement.  I am allowed to leave the study at any time.

So far I have had absolutely no side affects. I have noticed that I have much more energy. Fatigue has been one of my biggest problems.  I am encouraged because it is so early in the study. I am really hopeful by the results so far. I also noticed that I have more energy at night, so it has been harder to get to sleep. The good news is I have really good energy during the day despite that. It is a good energy, not jittery at all. I feel much more normal and able to function.

I will go back every three months just for a short assessment and to pick up another 3 month supply of medicine. At 12 months and 15 months I will be assessed with the same tests I had at the first two appointments. Those tests include:

• Nerve Conduction velocity
• Sensory Nerve Potential Amplitude
• Nine hole peg test
• Compound Muscle Amplitude Potential
• Hand Grip and foot dorsiflexion
• CMT neuropathy score
• Disability measured by change in overall neuropathy limitation scale

I am sure I may have missed something, but you get the idea of what is being screened and the process. The study will be expanded if all goes well and I would encourage anyone with CMT 1A to participate. There still may be room at some of the study site.  Sites include:

• California, Cedars-Sinai Medical Center
• Connecticut, Charles H. Kamen Neurosmuscular Center
• Florida, University of Florida Health and University of Miami
• Kansas, University of Kansas Medical Center
• Massachusetts, Brigham and Women's Hospital
• Michigan, University of Michigan
• Minnesota, University of Minnesota Health
• Missouri, St. Louis Medical Center
• New Jersey, Hckensack University Medical Center
• New York City, Columbia University
• Washington, St. Luke's Rehabilitation Institute Spokane

To join, send an email to the site coordinator. Some of the sites are full, but check in case there are openings.

************************************

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 will represent the US 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.

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.

http://www.henschelhausbooks.com/catalog/memoir-biography/215-running-for-my-life-winning-for-cmt-9781595982827.html

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 207 members in 38 states. We also have members in Australia, England, Scotland, Canada, Vietnam, Turkey, Finland, Scotland, France, Poland, Iran 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/

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