There won’t be progress without research!
Since I am suffering from a disease that not many researches are interested in, I see it as my duty to participate in clinical studies that lead to a better understanding of Ehlers-Danlos syndrome and its comorbidities.
But what exactly does clinical study mean, and why should I think about participating?
A clinical study is used in research on human volunteers to test the effectiveness of drugs, diagnostic methods or therapies.
We distinguish between interventional and non-interventional studies (also called observational). The interventional studies usually include treatments with a specific drug that is to be tested.
The non-interventional studies include, for example, the observation of a patient group over a long period of time, or case studies about one particular patient’s case will be described.
In most countries, clinical studies are strictly regulated by law, and they are always voluntary!
An overview of international clinical trials can be found here:
Each study has certain inclusion criteria the volunteer has to meet, and some clearly defined exclusion criteria. If you fulfill all necessary criteria, you can apply for the study. The study investigator will then decide whether you will be allowed to participate or not.
If you are not completely sure that you fulfill all criteria, just contact the investigator for more information and any open questions.
Before you opt for a study it is very important that you know those studies are not designed to personally help you and your specific problem. You cannot expect any immediate benefit for yourself. Studies are designed to help a large group of people in the future (you are one of them) and make a difference for everyone, but they are not designed to offer the participants free health care or testing. Nevertheless, often you will have some benefits. But it should not and must not be expected.
You also should know that certain studies might have side effects. If you are not sure about whether to participate or not, please talk to a doctor you trust.
Travel expenses for studies are often paid by the investigator, sometimes there even is a small salary for participating. However, this depends on the study and has to be requested individually.
Every participation is voluntary and can be withdrawn at any time.
Before every study you have to give your written consent. You will be informed about the study process and about all possible side effects before you sign the paperwork.
My personal experiences:
My personal experiences with all the studies I so far participated in were only positive. I had the great opportunity to be part of a study at the National Institutes of Health in the US, and was given the chance to meet some great doctors of all disciplines there. Of course, the first priority was to find out more about the patient, but I still got a lot of new information and even written reports about my testing results, which I could submit to my specialists in Germany, and because of those my German physicians were able to order further testing and start new therapies.
Because of that experience I decided to take part in two other studies, one in Germany and one in Switzerland. However, those two did only consist of a blood sample and wearing a 24-hour-sleep-monitor.
My motivation to participate in a study was at first pretty selfish: With a rare mutation that no one really knows anything about, and for which no doctor could tell me how my disease might progress, I really wanted to learn more about it. That is why I finally decided to take part in a trial in which a skin biopsy was taken. I tend to keep away from clinical studies that involve invasive measures, but the risk of the skin biopsy seemed to be very small compared to the probable benefit.
Overall, I am surprised about the positive experiences I made, and how much I learned about my disease and my body because of those studies.
- Find a study that fits your profile.
- Read the study description very well.
- If you meet the criteria and carefully considered whether you want to participate or not, you can contact the study investigator and apply for the study via e-mail.
- You will then be informed whether you will be enrolled in the study or further testing is needed to determine if you are eligible.
- If you are finally enrolled, you will get lots of paper work to sign where you basically give your written consent to participate and in which you will be explained all possible risks of the upcoming tests or therapies. You should read those documents very carefully.
- You will then be given a date, all necessary documents for flights and accommodation, and will be invited to the location where your study takes place.
- Now the official study starts, tests are done…
- Later after your appointment you might get the report of your results.
- Maybe a follow-up appointment is scheduled.
Study participation – short overview
- You are helping to
make a difference for all people who suffer from your disease (and thus also for you);
possibly find a cure;
find better treatment options;
improve diagnostics for your disease.
- Sometimes you will get all the test results and learn more about your disease.
- You get to know specialists that are very familiar with your disease.
- You will receive new information about your disease.
- You have free access to highly innovative testing methods.
- You have access to new therapies.
- You will maybe be compensated for participating and your travel expenses. You will not have any financial burden related to the trial.
- Studies are relatively safe because they are strictly controlled by the country.
- Side effects do happen (drug tests, invasive surgery).
- Some studies do not share test results with you.
- In some studies you will have to pay your travel expenses yourself.
Other important terms:
- Controlled studies:
This means there is a control group consisting of people that are healthy; or in case of drug trials a group of people that receive a placebo instead of the drug.
This means coincidence will decide which study group someone belongs to.
- Double blind:
This means neither the participants nor the investigator will know which group the participant belongs to.
A placebo is something that looks like a medication but does not contain any form of drug.
International (most of the trials worldwide are registered here):
Australia / New Zealand: