In recent years, medical science has made important breakthroughs in the treatment of rare kidney disease. Some new kidney drugs have been so successful in clinical trials that they have received accelerated approval by the U.S. Food and Drug Administration (FDA).

Clinical trials — also known as clinical studies or clinical research — are a main part of the research process for new treatments and are required before a drug or therapy can be approved by the FDA. In a clinical trial, volunteer participants help research scientists determine if a new therapy can effectively treat a health condition with minimal side effects.

If you’re living with a rare kidney disease and want to learn more about clinical trials, here are some facts you can discuss with your doctor.

1. New Treatments for Rare Kidney Disease Are Emerging Through Clinical Trials

A rare kidney disease is a kidney disease that affects fewer than 200,000 people in the U.S. There are about 150 rare kidney diseases, which are often aggressive and have a high risk of causing renal failure (kidney failure). Many rare kidney diseases do not have an FDA-approved treatment. However, the outlook for some rare kidney diseases is improving thanks to new research.

Budesonide (Tarpeyo) is a drug approved by the FDA to treat the rare kidney disease immunoglobulin A nephropathy (IgAN) — also known as IgA nephropathy or Berger’s disease.

Two other drugs, iptacopan (Fabhalta) and spartentan (Filspari), were also recently approved for treating IgAN. All three drugs received accelerated approval because they were found to be especially effective.

There are currently no FDA-approved drugs for treating rare kidney diseases like immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN) or complement 3 glomerulopathy (C3G). However, many promising studies are underway.

Complement inhibitor drugs are currently being studied for C3G. These drugs are designed to inhibit the complement system, a part of the immune system that, in people with C3G, damages the kidneys’ filters (glomeruli) and causes impaired kidney function.

Some drugs that have already been approved by the FDA for other conditions are being studied for C3G. For instance, danicopan (Voydeya) and pegcetacoplan (Empaveli) are approved for paroxysmal nocturnal hemoglobinuria, a rare blood disorder. The drug, iptacopan, which was approved for IgAN, is also being studied for C3G treatment.

A list of current clinical trials for rare kidney disease can be found at ClinicalTrials.gov, a database managed by the U.S. government. Be sure to talk to your doctor if you want more information on clinical trials.

2. Clinical Trials May Provide Access to New Treatments

In clinical trials, people with specific health conditions have a chance to receive investigational treatments — treatments under study — that are being evaluated for their effectiveness and safety. These new therapies aren’t yet available to the public, but they may turn out to be more effective than current treatment options.

Clinical trials are designed to safely test new medications, new combinations of existing medications, and new methods for taking existing medications. They can also investigate medical devices, such as dialysis machines, and procedures, such as kidney transplants. Additionally, clinical trials study how new treatments impact quality of life, explore therapies involving lifestyle changes, and address other areas of medical research.

However, there is no guarantee the treatment will work, and all medications carry the risk of side effects.

Importantly, some people who participate in a clinical trial may not receive the new treatment. Some participants may be given a placebo, sometimes called a sugar pill. A placebo looks like the treatment being studied but doesn’t have any active ingredients. To test the effectiveness of an investigational treatment, clinical trials sometimes assign a group of participants a placebo to compare results and see if the new drug really works. This is known as a placebo-controlled study.

Note that in some clinical trials, participants who initially receive a placebo may later receive the investigational treatment if the new medication shows very good results.

3. Clinical Trial Participants Are Told if They Might Receive a Placebo

In every clinical trial, participants must be informed if there is a chance they might receive a placebo instead of the treatment under study. This information might influence whether or not you decide to participate in a clinical study. In some cases, participants may be given an existing treatment in addition to a placebo, if their condition requires treatment.

To avoid bias, researchers randomly choose who will receive the investigational treatment and who will receive a placebo or the existing standard of care (the current accepted medical treatment practices). This type of study is known as a randomized trial. Many research studies are also double-blind (or double-masked). This means neither the participants nor the researchers know which treatment a participant is receiving until the trial has ended.

Some clinical trials are open-label studies in which both the researchers and participants know which drug is being tested.

4. Clinical Trials Are Designed To Protect Participants’ Safety

Clinical trials are strictly voluntary, and participants may leave a clinical study at any time for any reason. In the U.S., clinical trials are carefully regulated by the federal government to minimize risk. Before a clinical study of a drug starts, a treatment will undergo extensive preclinical studies, laboratory research, and animal testing. If preclinical research shows promising results that indicate a drug is safe and effective, the research study can proceed to a clinical trial with people.

Institutional Review Boards Oversee Clinical Trials

Most trials are reviewed by an institutional review board (IRB), which includes scientists, doctors, and other experts. The IRB closely reviews and monitors clinical studies to help ensure that potential benefits outweigh the risks. If a clinical study is found to be unsafe or if the investigational drug is not effective, the trial will be stopped.

Informed Consent

All participants must undergo an informed consent process prior to enrolling in a clinical study. During the informed consent process, researchers must explain how the trial will work, its potential risks, and any medical procedures that may be involved. You’ll be given time to ask questions and consult with your family, doctor, or any other advisers before deciding if you want to join the study. Throughout the study, you will receive updates and information.

Your doctor can help you determine the types of questions you should ask when considering participating in a clinical study, including whether there are any costs you may incur for travel or other expenses.

Phases of Clinical Trials

Clinical trials have phases to test a treatment’s effectiveness and potential risks. Researchers recruit participants for one phase at a time. If a phase is successful, the study will move on to the next phase.

• Phase 1 focuses on determining the highest dose of a new drug that can be administered safely. These studies generally involve small groups of participants who may be healthy or have the condition being studied.
• Phase 2 tests a drug’s effectiveness and monitors side effects. This phase usually includes a larger group of people.
• Phase 3 compares the new treatment to existing treatments to find out if it is safer and more effective. This phase may involve hundreds or thousands of participants, depending on the disease.
• Phase 4 evaluates a drug after it’s approved by the FDA and it is on the market. Sometimes called postmarket surveillance, this phase monitors long-term effects or side effects of the drug that may not have appeared in earlier phases.

Early trial phases may last several months to two years, while later phases can continue for four years or longer.

5. People Have Different Reasons for Participating in a Clinical Trial

Participating in a rare kidney disease clinical study is a personal decision and people have different reasons for participating. Some people participate because they may receive a new therapy that could potentially improve their condition and quality of life.

Some people enroll in clinical trials for financial reasons, particularly if they are uninsured or underinsured. The sponsors of clinical trials — universities, drug companies, foundations, or individuals — may cover the costs of the study. In some cases researchers may pay a fee to participants.

Others join clinical trials because they want to contribute to scientific research that may lead to new treatments for rare kidney disease. Some people are interested in the additional care, monitoring, and follow-up provided in clinical studies, along with the opportunity to gain fresh insights into their conditions by working with researchers.

6. Each Clinical Trial Has Requirements for Who Can Participate

Every clinical study for rare kidney disease has specific guidelines for who is eligible for the trial. Even if you’re not eligible for one clinical trial, another study may be open for you. Some of the factors that may determine whether or not you can participate in a clinical trial include:

• Age
• Sex
• Type of disease
• Stage of disease and kidney health
• Treatments used
• Overall health
• Other health conditions

Your healthcare providers and nephrology (kidney) care team can help you determine if a clinical trial might be right for you.

Find Your Support System

On MyKidneyDiseaseTeam, the social network for people living with rare kidney disease and their loved ones, members come together to ask questions, give advice, and share their stories with others who understand life with rare kidney disease.

Have you participated in a clinical trial? Have you discussed clinical trials with your doctor? Share your tips in the comments below, or post a question on your Activities page.