Blog 4 – Part II: What It Means to Be in a Clinical Trial – My Sickle Cell Research Xplained

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Blog 4 – Part II: What It Means to Be in a Clinical Trial

Curious as to what it means to be in a clinical trial?

Well, depending on the type of trial you’re in, it can look a little different.

If you’re in an observational trial, the study doctors are learning about a disease or looking for trends about something. They do this by looking over your medical charts and perhaps asking you a few questions about what you remember from the past. If a treatment is involved, it is not given by the study team, but rather by your personal physician who decides your treatment and care. So, it is non-interventional in that the study team doesn’t intervene in the decision about your treatment or care.

The other type of trial you’ve probably heard more about are interventional trials! In this case, the study doctors are changing  your care by giving you an intervention, such as a drug, device, or procedure that is being studied. This is referred to as the study treatment.

There are usually 2 groups in a trial. One group that gets the intervention (study treatment), and then there is another group that doesn’t get the intervention. That means they may get a placebo or the standard of care treatment for a disease.

 

A placebo is a medicine that has no active ingredients and has no known medical effects. It is used in clinical trials as something to compare the study treatment to.

Being in the placebo group of a trial may not mean you are receiving no treatment for your disease. For sickle cell disease trials, placebo is rarely used alone as a comparison to the study treatment; rather, patients may receive treatment given as standard of care, to ensure that patients with sickle cell disease don’t go untreated.

The standard of care treatment is the accepted standard treatment for a disease. By having some people get the intervention and some people not get it, the study doctors can better know if the intervention really works or doesn’t work. The placebo or the standard of care group helps the study doctors better know that their results are reliable.

 

Regardless of whether you’re in an observational or interventional trial, you may go to clinic visits, get tests done, and answer questions about how you’re doing. Each trial is a bit different, but in general, you may:

  • Attend clinic visits and discuss your care with the study doctor
  • Do bloodwork or other tests at several points in the trial
  • Do questionnaires to give the trial feedback on how you’re doing

Before the start of any trial, they’ll give you a detailed form called the “Informed Consent Form.” It tells you all about the trial and what’s expected of you, if you join. After you read it, you can ask any questions to your study doctor, so you fully understand the potential benefits and risks before signing and joining.

After this, you go through something called “Screening.” That’s to run through a checklist (ie, your medical history, blood test results, etc) to confirm that the trial is right for you. You see, every trial has a list of “eligibility criteria” which is a list of things that everyone in the trial needs to have to make sure that the right kind of person is recruited for the trial. That way, the study doctors will be able to see any true differences due to the study treatment more reliably.

If you pass “screening,” then you are ready to enter the trial! That means you’ll go on to either get the study treatment, or depending on the trial you’re in, a placebo and/or the already approved standard treatment, for your disease.

How do they decide who gets what? Well, some trials use what’s called “randomization.” It’s a process that randomly selects which group you’re in—sort of like drawing straws, while making sure the groups are still comparable in terms of age, gender, and other medical conditions. This makes sure there’s less bias in the way the trial is carried out. Regardless of which group you’re in, you’ll get the same medical care and you’re a part of the bigger research puzzle since doctors and scientists need enough people in the trial in order for their results to be trustworthy. That’s pretty key to being able to find future treatment options for people with sickle cell disease!

Trials can last anywhere from a few months to years. During that time, the study doctors track how you’re doing by checking in on you at the clinic visits and they may do some tests to check your health. At the end of it all, they compare your “before” and “after” results—to make their conclusions! Some trials will communicate results back to you at the completion of the trial, so that you can know how your participation may have contributed to conclusions about your disease! So be sure to ask if your particular trial will provide a summary to understand the results of the trial.

Be sure to check our website next month for a how-to guide on finding sickle cell disease clinical trials!

In the meantime, here are some links for extra learning:

What’s a placebo and what’s it mean to be in a placebo group of a trial?

Read More

How to get involved in sickle cell disease research:

Read More

What happens if I’m not eligible for a clinical trial?

Read More

See a real experience with clinical trials from someone living with sickle cell disease—Ada’s story

Read More

and Jewel’s story:

Read More

Say what? A recap of new words:

  1. Observational trial: A trial where conclusions are drawn by the study doctors from observing people and following them over time. If a treatment is involved, it is not given by the study doctors, but rather by a personal physician who decides treatment and care.
  2. Interventional trial: A trial where the study doctors are intervening in medical care by giving an intervention such as a drug, device, or procedure that is being studied.
  3. Placebo: a medicine that has no active ingredients and has no known medical effects. It is used in clinical trials as something to compare the study treatment to.
  4. Standard of care treatment: an accepted standard treatment for a disease.
  5. Informed consent form: a form that tells you all the details about a trial, like the design of the trial, what you’d have to do, the anticipated benefits, potential risks, and the voluntary nature of your participation. It represents your voluntary agreement to participate in research.
  6. Screening: a period where medical questions are asked and tests are done to see if you meet the criteria to be in the trial.
  7. Randomization: the way some trials decide what group you get put in in a trial. The process is random, sort of like drawing straws, while making sure the groups are still comparable in terms of age, gender, and other medical conditions.

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