Clinical trials for Haemophilia

Overview
Depending on several factors, you may be eligible to participate in clinical trials studying new treatments for hemophilia. Eligibility may be based on specifics of your condition, such as which clotting factor you lack, the severity of your hemophilia, whether or not you have inhibitors, and previous medications you have tried. Other details such as your age or gender may also be important.

Clinical trials may test new drugs, new dosages or new combinations of existing drugs; or new combinations of treatments such as surgery and medication. Trials may also pair standard treatments with complementary practices such as a prescribed diet or meditation.

What does it involve?
Before you enroll in a clinical study, read the informed consent form very closely. Ask your doctor or the doctor in charge of the study to explain any portions you have trouble understanding. Pay close attention to descriptions of the potential risks and benefits associated with participation. Work with your medical team to decide whether a clinical trial is right for you.

Do not be afraid to ask questions; it is your right to know what will take place during the trial and what it could mean for you. Consider taking a family member or friend with you to meetings regarding a clinical trial to help take notes and ask questions. Find out what tests and treatments are involved in each step of the clinical trial. Enquire about the probability of receiving a placebo. Find out how long the trial will last. Ask about how the treatment being tested differs from the current standard treatments. Find out which costs are covered, and whether you or your insurance company will be expected to pay for the rest. You can ask whether you will still have access to the treatment after the study ends, in case the new treatment proves effective for you. You may ask to speak to someone who is already participating in the clinical trial about their experiences. You may also want to know how your private medical information will be protected during and after the trial.

You can withdraw from a clinical trial at any time.

Intended Outcomes
The goals of participating in a clinical trial are to contribute to medical knowledge about hemophilia and a chance to gain access to a new, experimental treatment.

Results
Depending on the clinical trial you are considering applying for, there may or may not already be results available from previous clinical trials. Ask the clinical research coordinator or a nurse or doctor participating in the trial for information about results.

Constraints
Although the new treatment may be more effective than the standard treatment against IBD for some people, it may not be more effective in your specific case.

The new treatment may cause unforeseen side effects. Although the doctors leading the clinical trial will educate you about known risks, it is impossible to know what might happen with a novel treatment.

Some insurance companies may not cover all costs associated with being in clinical trials. However, any remaining costs will usually be covered by the company or sponsor of the clinical trial. Be sure to ask about costs before enrolling. Find out which medical expenses will be paid by the trial, whether the sponsor will cover any care resulting from injury or damage you may receive as part of the trial, and whether long-term aftercare is included.

Some trials may be located far from your home and necessitate travel.