Vtesse's Cyclodextrin Phase II/III Trial
Vtesse VTS-270 Trial (Phase II/III)
About the Phase II/III Trial
The Phase II/III trial will be limited to 51 patients ages 4-21. For additional information on eligibility requirements, please contact Vtesse at email@example.com.
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.
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.
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.
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.
Care for Children
The chance to help children with no other solutions motivates Ben to get approval for VTS-270.
While Ben has worked on many drug-development teams, he looks forward to helping the NPC community in a direct and personal way.
What To Do If You Can't Join The Trial: Compassionate Use
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.
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.
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.