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Vtesse's Cyclodextrin Phase II/III Trial

Vtesse, a biotech firm dedicated to developing treatments for rare diseases, has initiated their Phase II/III clinical trial for cyclodextrin and is currently looking to enroll eligible patients. Learn more about the people behind Vtesse and about how the Vtesse Phase II/III trial could help make VTS-270 (cyclodextrin) available to families all over the world.

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Vtesse VTS-270 Trial (Phase II/III)

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About the Phase II/III Trial

Start Date

The Phase II/III began globally in September 2015.


The duration of the Phase II/III trial for each patient will be approximately 12 months.


The Phase II/III trial will be limited to 51 patients ages 4-21. For additional information on eligibility requirements, please contact Vtesse at carrie@vtessepharma.com.

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The Phase II/III trial will be global and multi-site. Vtesse plans to open up to 20 clinical trial sites globally. There are already 11 sites open in the USA, UK and Germany.


The Phase II/III trial will be available in the US and in some EU countries.

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Type of Treatment

The VTS-270 treatment featured in the Phase II/III trial will be bi-weekly intrathecal (IT), which gets VTS-270 to the brain.

Low Financial Cost

If you participate in the Phase II/III trial, Vtesse is financially responsible for your VTS-270 treatment during the trial. Vtesse will cover costs of travel, lodging, meals, etc. and can assist in coordination of travel.

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Controlled Trial

What a Controlled Trial Means

The Phase II/III trial will be a controlled study, meaning a percentage of patients, called the control group, will not receive VTS-270 during the trial. However, this control group will be fewer than half and perhaps as few as one third of the trial participants.


A controlled trial gives Vtesse the best shot at getting VTS-270 approved by regulatory agencies such as FDA and EMA. In a controlled trial, the resulting data from the trial is stronger, and thus more likely to demonstrate VTS-270's efficacy. Furthermore, a controlled study will allow the benefits/risks of VTS-270 to be more precisely understood.

After the Trial but Before Approval

When each patient completes their participation in the trial, Vtesse will offer enrollment in an Open Label Extension (OLE) until the approval of VTS-270.

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Just the First Step

Not only would a clinical trial mean getting closer to regulatory approval, it would also allow researchers to take a big first step toward refining VTS-270 and making it even more optimized for treating Niemann-Pick Type C.

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Contact Vtesse

Let Vtesse know that you want information about the Phase II/III trial for VTS-270 using the link to Vtesse's new clinical trial website to the right.

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About Vtesse

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Vtesse's Chief Executives

Ben Machielse, Drs

Ben is the President and Chief Executive Officer of Vtesse. He has over 25 years of experience in drug development, including participating in the development of the first publicly available vaccine for H1N1 (swine flu). He now brings his expertise to parents and doctors in the fight against NPC. Ben and his wife of over 30 years take great pride in their two children. He enjoys bicycling with his wife and cooking.

Ravi Venkataramani, PhD

Ravi is the Chief Business Officer of Vtesse. Although he was trained as a scientist, his extensive experience in corporate strategy, partnering, collaboration and implementation gives Vtesse the business know-how it needs to make things happen in the fight against NPC. He has been married for 12 years and has two sons.

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Vtesse's Commitment to the NPC Community

"Our promise is to think about the kids first, to think outside the box where we can, and to deliver on our promises."

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Vtesse's Motivation

Care for Children

The chance to help children with no other solutions motivates Ben to get approval for VTS-270.

Personal Involvement

While Ben has worked on many drug-development teams, he looks forward to helping the NPC community in a direct and personal way.


Ravi feels personal responsibility to use the experience he has in drug development to help these kids and families.

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Being a Parent


Ben recalls the nervousness and fear he felt when his daughter went through a simple heart surgery.


As the parent of a 2-year-old and a newborn, Ravi finds great pleasure in seeing the world through the eyes of his son.

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What To Do If You Can't Join The Trial: Compassionate Use

In some countries, parents can file for compassionate use (also called "expanded access" in the US and "named patient program" in the EU). This is when parents seek special permission from their nation's government to use an investigational drug that has not been approved yet. If you can't join the Phase II/III trial, compassionate use may be the option for you.

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Benefits of Compassionate Use


If you're approved for cyclodextrin under compassionate use laws, you will get cyclodextrin, with no risk of being in the control group of a trial.

Multiple Treatment Methods

Under compassionate use laws, you can choose multiple types of cyclodextrin treatment for your child. This includes intravenous infusion, which gets cyclodextrin to the lungs and liver.


Applying for compassionate use allows you a chance to get cyclodextrin if you don't meet the Phase II/III trial's eligibility requirements.

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Difficulties of Compassionate Use

Finding a Doctor

It can be very difficult to find a doctor who is willing to take on the extra responsibility of administering an investigational drug, and many doctors have never heard of NPC or cyclodextrin.

Drug Cost

In the US, Johnson and Johnson provides cyclodextrin for free. However, if you live outside the US and you are approved to use cyclodextrin under compassionate use, you will have to pay for the drug yourself.

Treatment Cost

Compassionate use treatment expenses may not be covered by your health insurance.

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File for Compassionate Use

If you choose to file for compassionate use, you can Download a sample American protocol here that you can use as a starting point to write your own protocol with your doctor. If you live outside the United States, you'll need to contact Roquette to get cyclodextrin.