The world of Clinical Trials can be a mystery for many. The process can be cumbersome, and as we know, there are not always clinical trials going on in the wide range of rare diseases, making it sometimes frustrating and an emotional rollercoaster. Here are some pointers to get familiar with that will help you along your way towards finding the right clinical trial for you.

1. The most comprehensive source of clinical studies is clinicaltrials.gov

   All new studies for therapies seeking FDA approval are listed on the FDA website clinicaltrials.gov, and can be searched by the disease. 

2. Note the phase of the study

   These are the stages of study for a new medication which is part of the approval process with the FDA.

• Phase I studies are designed to study the safety of the medication. This may have only a few participants, and may be with healthy volunteers.

• Phase 2 studies are designed to see if the medication is effective. This is also a “proof” study, and the medication is given to people with the disease or condition.

• Phase 3 studies  have more participants to make sure the Phase 2 results are similar in a larger group of people with the condition or disease. It is usually the final phase before applying for approval  to the FDA.

• Phase IV studies after the medication is approved by the FDA, it continues to be studied over a longer period of time

3. Look at the type of study

• Biomarker studies are studies looking at risk factors, signs of disease, or severity of disease.

• Natural history studies: these studies show how the symptoms of a disease can change or progress over time.

• Interventional studies:  these are studies of medications, devices, or non-medical therapies that can be used to treat a condition or symptoms of that condition

  4. Check the inclusion/exclusion criteria which specifies the requirements to participate in the study

  1. The inclusion criteria are the qualities a person must have to participate such as your diagnosis, how long you’ve been diagnosed, your age, symptoms, what medication you’re taking and findings from your lab or blood tests (such as platelet or white blood cell levels, kidney and liver findings, genes or antibodies)

  2. Exclusion criteria if you have these factors, you probably will not be able to participate in the study. This can include: 

  1. Medications you might be taking, or a recent change in medication

  2. Other conditions you may have (such as heart disease, liver disease, etc.)

  3. Procedures or surgeries you may have had, like plasmapheresis or thymectomy

5. Ask about travel assistance.

If there is no study center location in your area, often the sponsor may reimburse you or pay for travel expenses or overnight stays in a hotel nearby. This is something you can ask the study team when you speak to them, or there may be an email address to contact if you are interested in learning more about the study.

6. You can get free access to a potential new treatment for your disease.

Although there is no guarantee that the medication in development will be effective for you, if  you do participate in a study and the medication seems to be working well for you, you may be able to continue receiving the medication free of cost for an extended period of time, until it’s approved by the FDA for use.  (Note that different studies can have different extended use provisions).

Deciding whether to participate is not always an easy decision, but doing so can offer the potential to help not just you, but other individuals with the same condition in the future. Not every clinical trial results in an approved medication, and many medications fail to meet their goals and are never approved.

7. Use Know Rare, which makes it easier to find a clinical study and talk to a researcher:

The Know Rare Study Finder provides an easy search tool for all the clinical studies listed on clinicaltrials.gov. By filling out a short survey just once, you can be matched to a clinical study for a potential treatment for your disease or condition.  

• If you choose to apply to a study offering virtual pre-screening visits, you can schedule a live virtual ‘visit’ with a member of the study’s research team who can review all you need to know about the study and see if you are eligible to participate in it.  

• If you don’t meet the requirements for one study, it matches you to any others that are listed for your disease, and it is updated constantly, so even if there’s no study for you today, there may be one for you tomorrow.

To find a study for you disease or condition, click here.