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About PREMIER Am I Eligible?

About PREMIER

Thank you for your interest in the PREMIER study.

PREMIER is a clinical study of a study drug called PXT3003. The study is sponsored by Pharnext.

PREMIER is now enrolling people with CMT1A.

Charcot-Marie Tooth disease (CMT) is a group of rare genetic disorders and CMT1A is one of the types of CMT. CMT1A affects the nerves in arms, hands, legs, and feet. These nerves control muscles and carry information to and from the brain, but in people with CMT1A, the nerves are prevented from functioning properly. This is why people with CMT1A may have muscle weakness and numbness, particularly in the muscles of the lower legs. It can also cause hand weakness, sensory loss, balance problems, or highly arched or very flat feet. As CMT1A is a progressive disease, the symptoms get worse over time.

There is currently no approved medicine for CMT1A and treatment consists of supportive care such as orthotics (e.g. leg braces), physical and occupational therapy, or surgery.

The purpose of the PREMIER study is to discover if PXT3003 is effective at treating CMT1A and to find out how safe it is to take. In the study, PXT3003 will be compared against a placebo (an inactive or “dummy” drug), so subjects in the study may end up receiving a placebo instead of PXT3003.

The study will involve subjects aged 16 to 65 years of age.

PREMIER is a Phase 3 study.

Approximately 50 study centers around the world

Approximately
350 subjects

Approximately
17 months

Find a Study Center

Please use the map below to find a study center near you. Study centers will be added to the map once they begin enrolling subjects. If there is no center that is enrolling in your location at the moment, please check back at a later stage.

Your PREMIER Journey

The PREMIER study will take you approximately 17 months to complete and is divided into three periods as follows:

Screening Period – Up to 35 Days

The purpose of the Screening period is to ensure that you are eligible to take part in the study.

You will visit the study center to take part in the informed consent process. During this process, study doctors will tell you everything you need to know about the study, provide you with written information to read, and answer any questions that you may have.

If you are happy to continue and provide informed consent, doctors will then perform a number of screening assessments to assess your eligibility.

Treatment Period – Up to 15 Months

If you are eligible to take part in the study, you will progress to the Treatment period and be assigned to one of two treatment groups as follows:

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

The treatment group that you are assigned to will be selected at random by a computer. Study subjects will be split evenly across these groups, so you 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 you nor the study doctors will know if you are receiving PXT3003 or placebo. The placebo looks exactly like PXT3003 but does not contain any medicinally active drug.

You will attend the clinic every 3 months during the Treatment period (6 visits in total) and your study team will contact you by telephone at least twice between these visits.

Safety Follow-Up Period – 30 Days

This period includes a Safety Follow-Up visit, which will take place 30 days after the Treatment period ends. This will be the final visit in the study. The purpose of the Safety Follow-Up visit is to assess your ongoing health and well-being.

Study Medication Diary

At the beginning of the Treatment period, you will be provided with a Study Medication Diary. This will be an application on your smartphone, tablet, or computer. You need to complete the Study Medication Diary each day during the Treatment period and you will be required to bring it with you to each study visit so that your study doctors can check that you are taking the study medication correctly.

Am I Eligible?

Answer the following questions to check if you may be eligible for the PREMIER study, then click Submit.

Please note that clicking Submit does not mean that you agree to participate in the study.

1

Are you between 16 and 65 years of age?

2

Do you have a diagnosis of Charcot-Marie-Tooth Type 1A?

3

Have you previously participated in a trial with the study drug, PXT3003?

4

Are you living with somebody who is currently participating in a trial with the study drug, PXT3003?

5

Are you pregnant?

6

Do you have hypersensitivity or intolerance to baclofen, naltrexone, or sorbitol?

You may be eligible

Based on your answers, you are potentially eligible for the study. Please look at the map to find your nearest site and contact details. The site will be happy to answer your questions and discuss the next steps.

OK

You are not eligible.

Based on your answers, you are currently not eligible for the study. If your answers to any of these questions change in the future, please check back again to see if you are eligible. Thank you for your interest.

OK

FAQs

A clinical research study is a medical research study involving human subjects. It tests the use of a medical procedure, medical device, drug, or potential treatment, and is led by researchers and study doctors. Clinical studies provide valuable information, and may help researchers to understand a condition better and learn how best to treat it, or may lead to new or better treatments being made available.

A study drug must pass three phases of clinical testing before it is approved by a country’s regulatory authority and made available to the public. PREMIER is a Phase 3 study.

Phase 1: The study drug is given to a small group of healthy volunteers or patients. Researchers look to see how much is safe to take and how the human body responds when it receives the drug.

Phase 2: The study drug is given to a small group of people with the target disease or condition it is intended to treat. In this phase, researchers evaluate what dose is appropriate and begin to learn more about how safe the drug is and how well it works.

Phase 3: Researchers test the safety and effectiveness of the study drug in a larger group of subjects over a longer period of time. Sometimes, comparisons are made between the study drug and other medications that are already approved for the same purpose.

Phase 4: A Phase 4 study is conducted after a study drug has been approved for public use. The approved drug may be compared with other drugs on the market or researchers may monitor the drug’s long-term effectiveness and impact on patients’ quality of life.

Before you take part in a clinical study, it is important that you are aware of all aspects of the study, including what will happen at the study visits, how you will take the study drug, what is expected of you when you participate, any potential risks and benefits involved, and many other things. Before you agree to participate, the study doctor will meet with you to tell you everything you need to know. You will be given written information to read that you can discuss with your family and friends, and you will be given time to ask any questions you may have.

When you understand what is involved in the study and you decide to take part, you will be given an Informed Consent Form to sign, which documents that you voluntarily consent to taking part in the study. Even if you provide informed consent, you are free to leave the study at any time and for any reason.

Your participation in the PREMIER study will last for approximately 17 months. However, participation is voluntary and you are free to leave the study at any time.

All medications, both those that are approved and those undergoing clinical studies, may potentially cause side effects – some of these are known, but there could be others that have not previously been observed. Study procedures may also involve risks. You will be informed about all of the known potential side effects of the study drug and study procedures before you decide to take part in this clinical study. If any further risks are discovered while the study is ongoing, you will be informed immediately.

If your condition gets worse during the study, your study doctor will discuss additional or alternative treatment options with you, if necessary. These options may include: continuing with the study drug; withdrawing from the study; switching to a different type of treatment; or taking medication to relieve your symptoms.

Your health and well-being are of utmost importance during the study, and any decisions regarding the study drug or any alternative treatments will be made with that in mind.

If you are enrolled in the study, you will be randomly assigned by a computer to receive either PXT3003 or placebo. The placebo looks exactly like PXT3003 but does not contain any medicinally active drug. Placebos are commonly used in clinical studies to help understand if the drug being researched is effective. In this study, one in every two subjects will take placebo. Neither you nor the study doctor or nurses will know if you are receiving PXT3003 or placebo. In the case of an emergency, it is possible for the study doctor to obtain this information.

You are free to leave the study at any time, and this will not affect your future care in any way. There are a number of possible alternative treatment options available to you for your condition. Your doctor will continue to look after you and advise you about alternative treatment options.