Pancreatic cancer
Treatment studies at every stage, help with symptoms, and research into finding the disease sooner.
Read about Pancreatic cancerStudy area
Hemophilia and bleeding disorders
Treatment for bleeding disorders has changed a great deal, and studies now compare longer-acting medicines, injections under the skin and gene therapy.
About Hemophilia and bleeding disorders
In a bleeding disorder the blood does not clot as it should, because a clotting protein is missing, low or not working. Hemophilia A is a shortage of factor VIII and hemophilia B of factor IX. Von Willebrand disease is the most common bleeding disorder and affects women and men alike, often showing up as heavy periods, nosebleeds or bleeding after dental work.
Studies test replacement factors that last longer, medicines that are not factors at all and are injected under the skin, and gene therapy given once, followed by years of check-ups. Others look at joint health, at bleeding disorders in women, and at people who have developed inhibitors, the antibodies that stop factor working.
Research in this area
Kinds of study you will come across
Most studies in this area fall into one of these groups. A listing usually says which one in its first lines.
01
Preventing bleeds
Compare a new regular treatment with what a person uses now, counting bleeds over time.
02
Injections under the skin
Test medicines given less often and without a vein, for adults and sometimes children.
03
Gene therapy
A one-time infusion, then long follow-up with frequent blood tests at first.
04
Inhibitors and joints
Research for people with inhibitors, and on keeping knees, ankles and elbows healthy.
Be ready
Before your first call
What studies in this area often involve, and what a study team is likely to ask you for.
What visits tend to involve
- Blood tests of factor levels and for inhibitors
- A bleed diary, kept on paper or an app between visits
- Joint examinations and, for some studies, an ultrasound
- Training to give a study medicine at home
- Long follow-up for gene therapy, with visits spreading out over time
Have these to hand
- Your type of bleeding disorder and its severity, from your treatment center
- Whether you have ever had an inhibitor
- Your current treatment, its dose and how often you use it
- A rough count of bleeds you have had recently, if you keep a log
Your questions
Questions to ask the study team
Write them down before you call. A good team expects them, and the answers help you decide.
- Will the study replace my usual treatment, or add to it?
- What do I do if I have a bleed during the study?
- For gene therapy: how long is the follow-up, and what does it involve?
- Can my hemophilia treatment center stay involved?
Study areas
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Read about ALSUpdates on hemophilia and bleeding disorders
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