After receiving a diagnosis for a rare condition like IgA nephropathy (IgAN) or C3G, the furthest thing from your mind, at least at first, is the idea of going through an experimental treatment or clinical trial. Instead, you’re probably focused on figuring out your treatment plan, how to deal with the symptoms of your condition and maybe even what you can expect from living with a kidney disease.
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But if you’re dealing with a relatively difficult or fast-growing form of kidney disease, your options for treatment may be limited. And understanding just how certain diseases and illnesses develop and spread is part of figuring out how to treat them. That’s where clinical trials and ongoing research come into play.
Nephrologist Ali Mehdi, MD, shares what to expect from a clinical trial, how to find one that’s currently under development and some things to consider before joining one.
Reasons to join a clinical trial
Clinical trials are designed in a variety of ways, but the core of many of these experiments is to determine how conditions develop and the effectiveness of various treatments. Think of how we’ve developed targeted therapies for rare cancers like cholangiocarcinoma (bile duct cancer) or how vaccines must go through clinical trials before they’re given to the public. Without the use of clinical trials, we’d never have a way to fully understand how these conditions work and we’d never have access to safe, ethically sourced treatments.
“For someone who has C3G, I can get maybe 50% into remission with the current medications I have,” says Dr. Mehdi. “And that’s when I pause and ask myself, ‘Are there any clinical trials happening that can give people better odds at beating this disease?’”
Before a medication makes it to market, it must go through several clinical trial phases in which the medication is tested for safety and effectiveness. Based on the results of those clinical trials, it must then be approved for use by the U.S. Food and Drug Administration (FDA) before it’s made publicly available. In essence, we don’t want to exactly throw everything at the wall and see what sticks without sound evidence.
“Clinical trials are very protocolized, meaning they have a lot of appointments and a fixed protocol,” explains Dr. Mehdi. “If you’re participating in a clinical trial, you do it because you want to do it, and you can stop at any time.”
Most clinical trials are randomized and double-blinded, meaning for the first part of the trial, you and the researchers won’t know if you’re getting the actual treatment that’s being studied or if you’re receiving a placebo instead.
Placebos can come in the form of sugar pills or saline and water injections. The point of using placebos is to compare the outcome of treatments against non-treatment options, and to ensure the team of researchers isn’t holding on to any biases during the clinical trial.
“Even if you get the placebo, you’re still getting what’s considered a standard form of care with some form of treatment. Otherwise, it would be unethical,” notes Dr. Mehdi. “If I refer someone to a trial, they will continue to see me because I will follow up with them to see what’s going on. If we see their kidney function is worsening, and the treatment isn’t working, we can just stop and move on to something else.”
You still may be given the new treatment that’s being studied, and the possibility of it working may be good considering the number of times it might have gone through other clinical trials. In some cases, even people who are given a placebo may experience a placebo effect, where their body responds positively despite not receiving any new treatment that’s being studied. But regardless of the outcome, taking part in a clinical trial can help push science forward to develop new and more effective ways to treat rare and challenging conditions.
If you have a particularly rare condition and/or other treatment options aren’t as effective as you’d hoped, joining a clinical trial is a personal choice that comes down to making sure you’re comfortable and committed to exploring new possibilities not yet available to the general public.
“There are many clinical trials ongoing that are really addressing the core of these diseases, at the cellular and biochemical level, to potentially offer more effective and safer options now that we didn’t have five or 10 years ago,” reports Dr. Mehdi.
How to find a clinical trial
You can join a clinical trial by speaking to your nephrologist or healthcare provider, who then puts in a referral to the research team working on the study. But if you’re curious, you can also search clinicaltrials.gov, which has a helpful tool to locate and find clinical trials that have been completed, as well as clinical trials that are currently recruiting new participants.
“Within nephrology, we have many clinical trials open and they’re multi-institution trials,” Dr. Mehdi says.
Risks of joining a clinical trial
Some people worry about the risk of not receiving the treatment that’s being studied and ending up with a placebo instead. Others may worry about how the clinical trial will work and whether or not it’ll have a lasting impact on their current condition.
But ultimately, the choice to join a clinical trial is up to you. If you have questions about how a clinical trial may work, or if you explore the clinical trials that are out there and would like more information, it’s always good to check in with your healthcare team to address some of the fears and concerns you may have.
“The key is to know you are consenting to a clinical trial,” encourages Dr. Mehdi, “and you can take away consent whenever you want.”