Much research has been focussed on developing enzyme replacement therapy (ERT) and various ways to enhance it. Gene therapy is also an important area for attention.
Finding out about current trials
Your medical team
Medical teams at the Highly Specialised Centres for Pompe disease will know what trials they are running at any given time. When attending appointments and reviews it is useful to enquire and state a preference for being considered in the future. Your team will always be the first port of call for information about trials.
Internet alerts and general searches
Setting up an alert using key words and search terms will send regular emails at a frequency of your choosing. For those who like to read much medical detail about their condition this can be one way to stay informed. New trials are usually signaled by a press release.
Pharmaceutical Industry websites
AGSD-UK can give you details of those companies currently working in this field. Regularly check their press releases or sign up for their e-alerts information about trials.
Registries of clinical trials
There are several on-line registries of planned, current and past trials.
Read about the trials registries on the “Research agenda” tab.
Joining a clinical trial
For more information about joining a clinical trial, the NHS gives general advice on their website.
The information will be a little different for studies in very rare conditions; for example, there will usually be less than 100 patients studied and large payments will not be given, only travel and daily expenses will typically be covered.
When enrolling onto to a clinical study we would strongly advise you to take time to read the Informed Consent Form (ICF) very carefully before adding your signature.
More coming about the following subjects
- Legalities and commitments
- Reimbursement and travel
Experiences of joining a trial
Several people with Pompe disease have written about their experience of participating in in a clinical trial. Their accounts give a helpful insight into what is involved. You can download them here.
Download PDF of Story 1: Unexpected turns in my trial experience.
Download PDF of Story 2: Why I decided to join a trial.
Gemma Seyfang presented this account (story 2) of her trial experience at our 2018 conference.
