A Phase 3 Study Shows Encouraging Results for a Potential Treatment for Charcot-Marie-Tooth Type 1A

Encouraging topline results from a pivotal Phase 3 study into a drug for Charcot-Marie-Tooth type 1A disease have been shared by Pharnext. To find out about this news in more detail, you can view Pharnext’s press release at AccessWire by clicking here.

About Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth disease (CMT) is a progressive genetic condition that damages the peripheral nerves. These nerves play an important role in communicating messages from the brain about muscle movement, and sensory information (such as hot, cold, touch, and pain) back to the brain. People with CMT may experience muscle weakness, changes to their gait, and numbness, amongst other symptoms.

There are multiple types of CMT, which are given numbers. These categories are then often sub-divided using letters into different forms within the types. CMT1A, which the study investigated, is thought to be the most common form of CMT, and affects about six out of every ten patients with CMT1.

The Study

A Phase 3 pivotal clinical trial is studying the effects of the investigational drug PXT3003 in patients with mild to moderate CMT1A. The fifteen-month long study involved more than three hundred patients, who were either given a high dose of the drug, a lower dose, or a placebo.

The Results

The study showed encouraging results supporting the effectiveness and the safety of the drug. Patients taking the higher dosage showed an average reduction of 0.4 on the Overall Neuropathy Limitation Scale used to assess disability (a 0.3 reduction was judged to be meaningful). The higher dosage group also met the secondary aim of the study, which was an improvement in the 10-meter walk test. People in this group showed a reduction of 0.5 seconds.

Following these results, Pharnext says that it plans to apply for market approval in both the United States and the European Union.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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