What Is a Clinical Trial | C3G | WeC3G.com
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What Is a Clinical Trial?

A clinical trial is a carefully designed research study of investigational drugs or therapies. These studies include observing or treating patients with the goal of advancing medical knowledge or developing new treatments.

These trials allow doctors and researchers to better understand how various diseases progress and how treatment or medications can be used to intervene.

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Why Are Clinical Trials Important?

While it’s important to understand what a clinical trial is, it’s equally meaningful to understand why they are so important. Before any new therapy or drug can be released, it must go through a series of studies to demonstrate that it is effective and safe for patients. This is why clinical trials are critical to developing new medications. Through the participation of healthy volunteers and patients, medical breakthroughs and innovations can be made possible.

When treating rare diseases, such as C3G, patient participation in clinical trials is critical. Due to the limited number of patients with a specific rare disease, it can be difficult to find enough volunteers to successfully complete a trial. This can limit the ability of researchers to develop new treatment options.

But, as more trials are performed, researchers and physicians develop a deeper understanding of the disease and potential treatment options. For the patients participating in these trials, that may mean earlier access to potentially life-saving investigational medication.

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How Do Trials Work?

Before trials begin, the research team puts together the protocol. This protocol is the framework for how the trial will be run and detail things like:

  • Study objectives: What the trial is testing for
  • Study design: What happens in each step of the trial
  • Patient criteria: What types of patients are eligible to participate
  • Intervention: What medication or treatment is being tested
  • Endpoint: What will be measured in the study

The protocol is intended to be as specific as possible to ensure accurate, reliable results.


Types of Trials

There are 2 main types of trials: observational and interventional.


In an observational study, researchers do not interfere with patients’ treatment or care plans. Instead, they observe the disease without active intervention to understand how it progresses and impacts patients’ lives.


In an interventional study, medicine or some other treatment is used to intervene in a patient’s care and the results are observed and studied by researchers. These studies are often sponsored by the pharmaceutical company that is developing the drug in question.

Interventional studies can be run in a number of ways. The study design may compare 1 drug to another already approved by regulatory authorities and available by prescription, or it may compare a drug with an inactive substance or sugar pill, called a placebo (pleh-SEE-bow).

In randomized, placebo-controlled studies, patients receive either the actual drug or an inactive placebo (a sugar pill). They are assigned to receive one or the other by chance (randomized). This type of study is often called single-blind because the patients don’t know which they are receiving. In double-blind studies, neither patients nor researchers know whether patients are receiving the actual drug or placebo. This is done to try to improve the ability of researchers to objectively evaluate whether the drug is working or not and to ensure that it is safe for patients.


Clinical Trial Phases

Studies for new drugs are typically classified into 4 stages, called phases. Each phase is designed to build on the knowledge and learnings from the previous phase.

  •  Phase 1


    Once a drug has been created and has gone through preliminary testing in animals and in a laboratory, it is able to move into a phase 1 trial. During phase 1, the new drug is tested on a small group of healthy volunteers.

    The goal of this phase is typically to understand how safe the drug is and what side effects it may cause. This includes examining how patients should take it, how much they should take, how the drug is processed in the body, and how the body eliminates the drug.


  •  Phase 2


    After the drug has been tested in phase 1, it can move on to phase 2. In this phase, the drug is tested in a small number of people with the specific disease.

    These types of trials can help researchers determine how the drug works in patients with the disease they are hoping to treat. The team of researchers will also evaluate the appropriate dosage and the potential side effects in patients with the disease.


  • Phase 3


    If the studies done in phase 2 are able to demonstrate that the drug may be effective in treating the targeted disease, the trial will progress to phase 3, where the treatment will be tested in a larger group of patients for a potentially longer period of time.

    During phase 3, researchers evaluate how effective the drug is over time. They continue to monitor the side effects and safety of the drug. At the end of phase 3, if the drug has been shown to be safe and effective, and can be manufactured appropriately, the government (for example the FDA in the United States or the EMA in the European Union) may approve it for use.



    Phase 4


    After a drug has been approved for use, phase 4 trials may be required to continue to learn more information about the drug and how it works to better inform doctors and patients.

    By monitoring patients’ ongoing progress, researchers and regulatory groups can help ensure that the drug continues to work and is safe for patients when used according to recommended usage guidelines.

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Could a Clinical Trial Be Right for Me?

As you continue your treatment journey, your doctor can help you identify potential clinical trials. Each trial will have its own set of rules for patients who are eligible to participate and those who are not, known as inclusion and exclusion criteria.

This set of requirements helps determine which patients are eligible to join the study. Although it unfortunately means that some patients will not be able to join a trial, these strict criteria help to provide more reliable results and ensure that the trial is specifically designed for the appropriate patients who are involved. This enables researchers to better understand how well a drug works, as well as whether it is safe for use.

Some of the patient selection guidelines may include things like:

  • Age
  • Sex
  • Disease severity
  • Previous treatments
  • Other medical conditions
  • Previous and/or current treatments


Along with the inclusion and exclusion criteria, you should also be mindful of the trial status. The status will inform you if the trial has begun and if it is still actively recruiting participants.

Not yet recruiting
Not yet recruiting The study has not yet started recruiting participants
Recruiting The study is actively looking for participants
Active but not recruiting
Active but not recruiting The study is ongoing but they are not looking for any new participants
Enrolling by invitation
Enrolling by invitation The study is actively looking for participants
Suspended The study has stopped early but might start again
Terminated The study ended early and will not resume
Completed The study has ended
Withdrawn The study stopped before enrolling any participants
Unknown No recruiting status has been verified

What to Expect

After speaking with your doctor, if you decide that a clinical trial is the appropriate next step in your treatment plan, you or your doctor can reach out to the study sponsor, who can direct you to your nearest trial location. The trial coordinator is the person responsible for all of the day-to-day trial operations, and he or she will be able to provide you with additional information about enrollment, as well as discuss your eligibility.

If you are deemed eligible after the screening process, there are several next steps that will likely take place.

First, since the trial location will likely not be at your doctor’s office, you’ll need to go to the trial site for an initial visit. At this appointment, you may meet the investigator. This person is the clinician (doctor) in charge of overseeing all aspects of the trial at that location. You will likely see him or her on occasion when you go back for follow-up appointments.

A doctor or nurse will review all of the informed consent forms with you. These are documents that describe the trial and the risks associated with participation. If you decide that you want to participate, you will need to sign these forms to indicate that you understand the expectations and risks of participating in the trial. The doctor or nurse will also go over your medical history, possibly perform a physical examination, and collect blood and urine samples to check that you meet the trial criteria. If the results of the visit show that you meet the eligibility criteria, you can officially begin the trial.

Your next visit will likely be your baseline visit. For example, in a trial for C3G, the baseline visit might include tests to measure how well your kidneys are working, as well as lab tests that will be followed over the course of the study. This allows the researchers to know where you started your journey and account for any changes throughout the trial.

The frequency of follow-up appointments will vary based on the specific trial. These could take place every few weeks or every few months depending on the study design. Before beginning a trial, make sure you know how often you will need to schedule appointments. By doing this, you can plan accordingly and make sure that you will be able to complete the required visits given your schedule and any required travel to the doctor’s office.

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What’s the Risk?

Any medical treatment or procedure has a certain level of risk, and clinical trials are no different. However, there are strict rules and regulations in place to help ensure patient safety during clinical trials.

Multiple teams of reviewers, such as an Institutional Review Board or an Ethics Committee, must approve all clinical trials. These groups work to review the study design and treatments to ensure that there is no unnecessary risk to the patients. There is also continued safety monitoring throughout the duration of the trial.

While patient safety is always at the forefront of clinical trial design, it is important to remember the potential risks and weigh them carefully when making your decision.

Some possible risks to consider:

  • Side effects of new medication
  • Effectiveness of new medication (may not be as effective as current treatment)
  • Risks associated with any tests performed during the study
  • Interruption of your schedule for study activities
  • Potential restrictions on other medications you can use during the course of the study

Withdrawing From a Trial

Patients are always able to withdraw from a trial at any time and for any reason. If you wish to withdraw from a clinical study, you must notify the study doctor that you no longer wish to participate. The study clinician or doctor can also stop your participation in the clinical study if he or she believes it is in your best interest. Should you want or need to stop participating in the clinical trial, you may want to talk to your doctor first. If after you talk to your doctor, you still decide that discontinuing is in your best interest, your doctor can help you take the proper steps to notify the research center and be removed from the study.

Once you have withdrawn, your regular doctor can help you determine the next steps in your treatment plan.

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What Else Should I Consider?

Now that you know more about how clinical trials work, you may be interested in joining one. As you begin looking into trials, there are several things to keep in mind.

Location: Where is the study being held and how often will I have to travel there?

Duration: How long is the study and how frequent will my follow-up visits be?

Commitment: Is there anything I must do to participate: biopsies, blood tests, etc?

Health: Do the benefits of participating outweigh the risks to my health?

Finances: Will there be travel expenses or lost income due to time off from work?

Lifestyle: What will change in my daily routine?

As you research and begin to answer these questions, rely on your doctor to help guide you through the decision-making process. He or she will be able to help you weigh the benefits and risks as they pertain to your specific situation.

When you begin these conversations with your doctor, consider:

  • Bringing a family member or friend with you for support
  • Making a list of any questions you have before your doctor visit so you don’t forget to ask
  • Bringing any materials or study information you’ve researched on your own
  • Bringing a notebook and pen with you to write down answers—a tape recorder or recording on your phone may be helpful to record answers, too

New investigational medications are currently being researched to treat C3G. See a list of ongoing trials, and talk to your doctor to determine if a trial could be right for you.